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Orchard Therapeutics(NASDAQ:ORTX)Q12020 Earnings CallMay 9, 2020, 8:30 p.m. ET

Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Orchard Therapeutics First Quarter 2020 Investor Conference Call. [Operator Instructions]

I would now like to hand off the conference over to your speaker today, Renee Leck, Director of Investor Relations. Please go ahead, ma'am.

Renee T. Leck -- Director, Investor Relations

Thanks, Sonia. Good morning, everyone, and welcome to Orchard's First Quarter 2020 Investor Call. You can access the slides for today's call by going to the Investors section of our website, orchardtx.com.

Before we get started, I'd like to remind everyone that statements we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, and including those set forth in our annual 10-K filed with the SEC and any other filings we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

And with that, I'll turn the call over to our CEO, Bobby Gaspar.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thanks, Renee. Hello, everyone, and welcome. I'd like to start by first acknowledging the tremendous efforts of our organization and our partners in the healthcare field to ensure patients in need continue to receive care during this difficult time. Thank you, everyone. The last few weeks have been an important period for Orchard. Since taking on the leadership, Frank and I, together with the executive team, have thought very carefully about what the new Orchard can become, how we can ensure that Orchard can fulfill its true potential and what we need to do to make that happen.

When we think about our strategic vision as a company, it's really all based on the potential of the hematopoietic stem cell gene therapy platform, where it can take us and the benefit it can provide for many patient populations even beyond our current portfolio of ultra-rare diseases. We have taken some bold and decisive actions that we believe will allow Orchard to achieve long-term growth and focus the company on sustainable value creation. This vision is supported by a new strategic plan that we have developed and which is built around four pillars. Each of these forms a chapter in our remarks this morning.

First, operating efficiencies. We have made a series of important changes to our operations that will enable us to sharpen our focus and more efficiently execute our strategy, which I will detail in a moment. Second is our commercial build. We are focused on establishing the right model for the diagnosis and treatment of patients undergoing HSC gene therapy, and see the true value of this approach over a series of ultra-rare products. Third, one of the most exciting areas in gene therapy right now is the innovation taking place in manufacturing technologies that have the potential to deliver economies of scale. We want to be leaders and invest in this space, knowing that our near-term capacity needs are covered by our experienced CDMO network.

Finally, central to this strategy is prioritizing our portfolio to enable the expansion of Orchard's pipeline beyond ultra-rare to less-rare indications. We are disclosing two new research programs for the first time today, and these are a genetic subset of frontotemporal dementia or FTD, and a genetic subset of Crohn's disease. We believe that the biological and clinical validation that has already been shown in our ultra-rare indications allow us to expand with confidence to these larger indications.

Turning to the first chapter in our new strategic plan. We are focused on improving the operational efficiency throughout the organization. This started with an extensive evaluation over the past six weeks of each program in our portfolio using several criteria that are shown here on the left-hand side of slide five. We undertook an objective analysis that involved both financial metrics and strategic considerations in identifying those programs where there was high need for patients and high-value creation for shareholders. As you can imagine, these were difficult decisions given the potentially transformative nature of many of these programs. Each has value, and we intend to realize that in different ways and over different time horizons.

Today, however, we believe our resources are best focused on Metachromatic Leukodystrophy, Wiskott-Aldrich syndrome, the MPS programs and our research programs. This also means that we have a balanced portfolio with late, mid and early stage programs. The programs I haven't mentioned such as OTL-101 and ADA-SCID and the transfusion-dependent, beta-thalassemia program, OTL-300, will have a reduced investment moving forward. We will look for alternative ways to realize value with those programs, including through partnerships.

So slide six brings together a summary of the operational changes that we've announced today. We believe these changes were important and necessary to enable Orchard to execute its mission and objectives at the highest level by matching our attention and resources to a set of core imperatives for the business. As summarized here, we expect to realize cash savings of approximately $15 million from the prioritization of our portfolio. Another $60 million in savings results from the decision to consolidate our R&D teams to one site and defer the investment in the manufacturing facility. Finally, the more staged approach to the commercial build-out and 25% reduction to our existing workforce and future headcount planning will each yield another $25 million in savings.

All of these cash savings are expected to be realized over 2020 and 2021, and result in total expected savings of $125 million over that period. With the revised plan, we now have cash runway into 2022 and no near term need to finance. It's worth briefly mentioning that this $125 million savings is after making investments in the following key areas to support our new strategy, shown on slide seven. In commercial, diagnostic and screening initiatives, including no-charge testing programs to help identify patients with MLD and other neurodegenerative conditions in time for treatment. In manufacturing, the technology, process innovations and efficiencies to drive scalability.

In R&D, initiatives in less-rare diseases that have the potential to fuel the company's future growth in a substantial way. This wasn't just an exercise to reduce expenses, but important decision-making to ensure our capital is deployed in a disciplined manner, while building a pipeline that can leverage our success across all phases of our business.

Now let me turn the call over to Frank to discuss additional key elements of the new plan.

Frank Thomas -- President and Chief Operating Officer

Thanks, Bobby, and good morning, everyone. As you can tell from this morning's press release, we have carefully examined each aspect of our business. You heard that a moment ago from Bobby, with the way we are creating operational efficiencies, and I think you will see additional evidence in the next two sections as we summarize our latest thinking around commercial deployment and manufacturing. Starting with commercial. We understand the importance of developing a commercial model that will demonstrate our ability to execute and bring these therapies to the market successfully. This model and the infrastructure that we build will also be leveraged for any future product launches.

As you'll note from the bottom of slide nine, each rare disease has certain dynamics that will impact the launch trajectory and speed with which we can penetrate the market. In fact, we anticipate our first two potential launches in WAS and MLD having distinct but complementary launch curves, as you can see from the illustrative diagrams. Let me start with MLD on the left, where we expect to launch first in the EU, followed by the U.S. and then other countries around the world. We think an important inflection point on the revenue curve with MLD will come later when newborn screening is established, providing an opportunity for an acceleration in growth rate. Disease progression is a second important dynamic that will affect market penetration. Because MLD advances so rapidly, it will be important to diagnose patients early and get them treated.

For Wiskott-Aldrich syndrome, the dynamics are very different, and it's reflected in the shape of the curve on the right. Unlike MLD, this disease is slower progressing and more readily diagnosed. We believe that WAS will provide an opportunity to treat a number of prevalent patients from the outset and also give us additional long-term revenue stream. This program, the BLA and MAA filings are on track for 2021. Turning back to MLD for an update on the regulatory time line. We are on track to get a decision from the European Medicines Agency later this year, and if approved, launch in the EU in the first half of 2021.

In the U.S., we recently engaged with the FDA on our planned BLA submission of OTL-200 for the treatment of MLD. The FDA has provided written feedback on the sufficiency of the company's data package, including the clinical endpoint, the natural history comparator and the CMC data package. As a result of this feedback, we intend to file an IND later this year and also seek RMAT designation, both of which we believe will facilitate a more comprehensive dialogue to discuss the data more fully and resolve the open matters before submitting a BLA. We are committed to working closely with the agency, and we'll provide updated guidance on the new filing time lines for the BLA after further regulatory interaction.

On slide 10, you can see that we're tracking nicely for the launch of OTL-200 in the EU in the first half of 2021, if approved, with Germany being the first country where we expect to treat commercial patients. Many of the prelaunch activities are under way, and the team has been able to keep up momentum during the pandemic to work with key centers and progress with site qualifications. We intend to set up a network of treatment centers where MLD patients are often referred and who also have transplant expertise. These same centers can be leveraged in future launches, especially for programs in the neurometabolic franchise.

I previously mentioned the importance of diagnosis in MLD to identify patients at early stages of disease, and we are taking the necessary steps to achieve long-term success. Beyond typical disease awareness efforts, we are also looking at initiatives such as no-charge diagnostic testing with partners such as Invitae, and we are looking to facilitate newborn screening for MLD with funding of upcoming pilots in New York State and Italy that are designed to validate the assay and provide the data for wider implementation. Success in these key initiatives will support early MLD patient identification.

Coming up quickly behind MLD and the neurometabolic franchise, our two proof-of-concept programs in MPS disorders, where we have made recent progress even during this challenging period with COVID-19. For MPS-I, over the past year, we've shown promising preliminary proof-of-concept data with positive engraftment, biomarker correction and encouraging early clinical outcomes, and we are excited to announce our plan to begin a registrational trial next year, bringing this program one step closer to commercialization.

For MPS-IIIA, we announced late last month that the first patient was treated in a proof-of-concept trial at Royal Manchester Children's Hospital, with enrollment planned to continue this year and interim data to be released in 2021. You can see graphically on slide 12 how the aggregation of these commercial markets lead to sustainable revenue growth. In addition, the infrastructure build is designed to provide the necessary commercial capabilities to realize the potential of the portfolio. On this slide, we've included the incidence figures for MLD and the incidence and prevalence figures for WAS to help you understand each opportunity as we see it today.

Given the dynamics at play for MLD that I described on slide nine, we believe this opportunity should largely be tied to the incident patient population, which we believe ranges from 200 to 600 patients per year in countries where rare diseases are often reimbursed. We've taken a more conservative view than previously on the addressable patient and market opportunity in countries such as those in the Middle East and Turkey, where the literature has a wide range of differing incident figures. Also, over time, with improved disease awareness, there may be prevalent patients identified who also could benefit from therapy. Our commercial strategy has always been and continues to be based not only on one product, but rather the aggregation of multiple potential products launching off one HSC gene therapy platform and infrastructure.

Turning to manufacturing. We've also made some key changes to our approach in manufacturing and how we allocate capital in the short and mid-term. On slide 14, you'll see the main tenets of our new manufacturing strategy. First, in the near term, we plan to focus on innovative technologies to enable commercial scalability.

Second, to ensure the appropriate focus on those technologies, we've made a decision to consolidate R&D to a single site in London, which brings together our organization in a more efficient way. This will allow efforts made to improve our manufacturing processes to be quickly and easily shared and then scaled commercially to transfer to our third party manufacturers, all of whom are currently located in Europe. As part of this consolidation, we will close our California site, including the termination of the Fremont project and associated capital spend.

Third, we have strong relationships with CDMOs that will ensure supply of clinical and commercial product to satisfy near-term requirements. And longer term, we intend to identify a new site in the U.S. to eventually bring manufacturing capabilities in-house with a facility that is appropriately sized and fitted for future techniques and operations.

Slide 15 shows the three phases of our approach in manufacturing: invest, partner and build. Today, we are investing, and we'll continue to invest in technologies such as transduction enhancers, stable producer cell line and closed automated processing of the drug product. This will potentially reduce the amount of vector needed, drive down COGS and potentially change the way products are manufactured, making it less labor-intensive, less expensive and more consistent. In the near and mid-term, we will continue to rely on our manufacturing partners for the early planned launches in MLD and WAS. For example, MolMed has been with these programs since the beginning, and they've been a reliable commercial partner with Strimvelis.

In addition to our existing CDMO network, we have begun to search for a drug product partner in the U.S. to complete a tech transfer and serve the U.S. market, thereby reducing scheduling challenges and creating some redundancy. And finally, over time, we plan to build in-house manufacturing capabilities closer to when there is a need for additional capacity. This enables us to explore options that are more aligned with our business in terms of scale and timing.

And with that, I'll turn the call back over to Bobby.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thanks, Frank. In this section, I'm going to briefly highlight the potential of HSC gene therapy to correct not only blood lineage cells, but also how through natural mechanisms, specific cell types may allow correction of disease in specific organ systems and enable expansion of our portfolio into new research indications. As many of you know, and as shown on slide 17, through HSC gene therapy, we are able to insert a working copy of the gene permanently into the genome of HSCs, and these genetically modified cells can lead to multiple corrected cell types in the bloodstream, including immune cells, red blood cells and platelets.

In addition, HSCs can differentiate into cells of the monocyte macrophage lineage that naturally migrate into various organ systems, and thus gives us an opportunity to deliver genes and proteins directly to those organs, including the brain and the GI tract. Within the neurometabolic space, in particular, we have understood through our preclinical and clinical programs in MLD, MPS-I and MPS-IIIA how HSC gene therapy can deliver genes and proteins to the CNS to correct neurodegeneration. Here is an example of this natural mechanism at work in slide 18.

Data shows that there are a population of gene-modified HSCs that can naturally cross the blood-brain barrier, distribute throughout the brain, engraft as microglia and express enzyme that is taken up by neurons. We have seen this approach results in clinical benefits for patients with MLD, and we are also using the same approach for MPS-I and MPS-IIIA. Beyond this, we see that the HSC gene therapy approach could be used to deliver specific genes and proteins for other larger neurodegenerative conditions which have high unmet need.

One of the conditions we are disclosing today, and shown on slide 19, is a specific genetic subset of frontotemporal dementia, where the underlying pathogenesis has a number of parallels with the neurometabolic conditions that we are already addressing. This program involves a broad strategic alliance with Dr. Alessandra Biffi, Boston Children's Hospital and Padua University in Italy, to further explore the potential of ex vivo HSC gene therapy in neurometabolic and neurodegenerative conditions. In other organ systems, such as the GI tract, there are similar mechanisms at work which are illustrated on slide 20. Tissue resident macrophages in the gut wall are required to respond to bacterial invasion from the gut lumen and prevent infection. In certain disorders, such as X-linked chronic granulomatous disease or XCGD, defects in macrophage function results in an abnormal immune response and severe colitis.

Moving on to slide 21. We have already seen in our XCGD program the modification of HSCs and migration of gene-modified cells into the gut can lead to resolution of colitis through presumed reconstitution of the immune response. Certain subsets of Crohn's disease are also associated with mutations in genes that affect the response of macrophages to infection, and so our clinical observations that HSC gene therapy for XCGD suggest that the same approach may be applicable to this genetic subset of Crohn's disease. This preclinical work is ongoing in our Orchard research laboratories.

As we advance our work in FTD and Crohn's disease, and assuming we show preclinical proof-of-concept, these will become exciting opportunities for us to expand and address larger patient populations, either alone or in partnership. We believe we have truly just begun to explore the potential for HSC gene therapy in diseases such as these and others, and are excited to share more about the preclinical development of these programs later this year.

So to summarize our path forward on slide 22, the next 12 to 18 months offers many important milestones as we continue our evolution to a commercial stage company and advance our next wave of clinical stage therapies. We anticipate approval and launch of OTL-200 for MLD in the EU, additional regulatory filings in Wiskott-Aldrich syndrome and MLD, a new registrational study next year in MPS-I, multiple clinical data readouts from our neurometabolic franchise and further detail and progress on our research programs in FTD and Crohn's disease.

To wrap up our prepared remarks, we are confident that our new strategic plan and operational decisions announced today will set us on the right path to achieve long-term growth, build sustainable value and serve an even larger number of patients who could benefit from hematopoietic stem cell gene therapy.

Thank you very much. And now we'll use the rest of the time to answer your questions. So let's have the operator open up the line.

Operator

Thank you. [Operator Instructions] And our first question comes from Whitney Ijem from Guggenheim. Your line is now open. Please go ahead.

Whitney Ijem -- Guggenheim -- Analyst

Hey guys, thanks for the question. So first, just wondering, can you give us some more color maybe on the discussions you're having with the FDA in MLD? Kind of what are they looking for? And I guess is the IND just sort of a tool to get RMAT? Or is there additional kind of clinical work you plan you think you'll need to do?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Hi. Whitney, Bobby here. Thanks for that. In general, we can't go into all of the details, obviously, of the discussions with the FDA. But I think in the release and in the script, we've talked about the fact that they've commented on certain endpoints, the natural history, the CMC package, etc. Now I think I'd just like to say this is a and obviously, a very complex disease, a very ultra-rare population, we have extensive data set, and we have already filed with the EMA. Now for historical reasons, there hasn't been an IND in the U.S., and so we haven't had the opportunity to discuss that data in full with the FDA.

What I can say is that we do have an extensive body of data. We want to be able to talk to the FDA and have a comprehensive dialogue to be able to explain that full data set. We feel confident that we have the endpoints that they are looking for and the data that they are asking for. But we need to have that conversation with them in order to be explain to be able to do that fully. So that's why we're filing an IND filing, filing the RMAT, so we can have that dialogue. And once we can clarify those issues, then we can go ahead with submission of the BLA.

Whitney Ijem -- Guggenheim -- Analyst

Okay. Got it. And then just one quick follow-up on MLD. Can you remind us where you are with newborn screening, I guess, both in Europe and then in the U.S.?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Yes, sure. So newborn screening for MLD, I think, is an important, a very important issue, because, obviously, that means that we'll be able to get earlier diagnosis and have more patients be able to access therapy. So it's a very important part of our kind of diagnostic initiatives in this disease. What we have so far is that we have worked with a key scientist, where an assay has been developed, that's been published to show that there is an assay that we've done on a dry blood spot to understand the decrease in the enzyme activity and also the increase in the sulfur-type levels.

And that assay is now going to be put into pilots, and we are funding a pilot in New York State, and that will start later this year. And we're also looking at pilots in other states as well. We're also transferring that assay to Italy and that and we're funding a program in Tuscany and in Italy where that will be rolled out. And we're also looking for opportunities in other EU states as well. So I'd say, there are already two that are going to start, we are looking to fund other pilots as well.

And together, that data will allow us to validate the assay but also allow wider implementation of newborn screening, and also for nomination, for example, onto the WAS panel for implementation in states in the U.S. So I say there's a lot of work going on in order to make sure that happens.

Whitney Ijem -- Guggenheim -- Analyst

Great, thanks.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

And your next question comes from Esther Rajavelu from Oppenheimer. Your line is now open. Please go ahead.

Esther Rajavelu -- Oppenheimer -- Analyst

Hey guys. Congrats on all the changes. I guess, my first question again on MLD is I'm trying to understand the duration between EU approval and NBS. I don't know if that math or if that graph was drawn to scale, but it looks like it's almost a four-year lag from first approval to newborn screening. Can you help us understand the time line there?

Frank Thomas -- President and Chief Operating Officer

You mean between EU and U.S. or around newborn screening or both?

Esther Rajavelu -- Oppenheimer -- Analyst

Around newborn screening, generally, between EU approval and newborn screening.

Frank Thomas -- President and Chief Operating Officer

Yes, sure. As Bobby mentioned, there's a pretty active program planned around newborn screening that I think we will expect will come over time in order to even apply for the Ross Panel, there are certain requirements that need to be met in terms of the number of patients or a number of children that have to be screened, identifying the positive patients and then you can apply on the Ross Panel. And then from there, there's a process that you go through in the U.S., at least, on a state-by-state basis to get it added.

So I think there are a number of steps along the way. We haven't guided specifically on the time line, but I think there are other precedents out there that suggest that this could take years. Once we screen the once we apply for the Ross Panel to get sort of full reimbursement, but obviously, we'll focus on states initially after that approval that have the largest populations.

Esther Rajavelu -- Oppenheimer -- Analyst

And my Yes, go ahead.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Esther for I was just going to say for the EU, obviously, we're looking for approval for MLD later this year. As far as people screening in the EU is concerned, that's on a country-by-country basis, and sometimes it's even certain states. But I've worked on newborn screening for SCID, for example, in the EU. And now there are numerous countries in the EU that are screening for SCID with a number of pilots also in the pipeline as well. And so with that kind of experience, and we would be looking to kind of really facilitate that uptake in the EU and as in and in the U.S., as Frank has already mentioned.

Esther Rajavelu -- Oppenheimer -- Analyst

Understood. And then the decision to defer capex, is that related to some of the time lines for U.S. versus EU approvals and the newborn screening? Or what really kind of went into that delay, given you already have some cost into that facility?

Frank Thomas -- President and Chief Operating Officer

Yes. I can start, and Bobby can add on that again. I think, obviously, we continue to believe in-house manufacturing is an important capability that we're going to want to have over some period of time. It really comes down to sort of when is the need for that capacity and capability relative to the various programs we have. Working with the CDMOs that we have today, we know that we have capacity for the MLD and WAS launches and for a period beyond the launch. So there's not an imminent need to secure the capacity today, and we think that deferring it makes the most sense. We'll continue to work with CDMOs on those launches.

We will look at bringing on a U.S. supplier for drug product to be able to more easily service the U.S. market. And then longer term, look at, potentially, in-house manufacturing at a site and location that we think is more fitted to what the capacity needs will be. So I wouldn't say it's tied to any sort of launch time lines because the plan always was to utilize CDMOs for WAS and MLD. But certainly, as those launches roll out and demand grows, our capacity needs will grow and that will be the appropriate time, we think, to make the investment.

Esther Rajavelu -- Oppenheimer -- Analyst

Understood. Thank you very much.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

And your next question comes from Anupam Rama from JPMorgan. Your line is now open. Please go ahead.

Tessa Romero -- JPMorgan -- Analyst

Good morning, guys. This is Tessa on the call this morning for Anupam. You pointed out that updated interim data from the proof-of-concept trial for OTL-203 and MPS-I is expected at ASGCT upcoming here next week. Can you remind us of what will be the size and scope of data that we will see at the conference? And maybe can you just clarify if there is any other newly updated clinical data we should be thinking about for other programs at ASGCT?

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Okay, fine. Bobby here, and I'll take this question. On MPS-I, so just to remind you, the proof-of-concept study has enrolled all eight patients, so that's been fully recruited into. What we've shared with you previously is biochemical data showing the overexpression of IDUA activity, the decrease in the heparan and dermatan sulfate, the engraftment of gene-modified cells and some early clinical data on patients who have got beyond the one-year time frame after gene therapy. There was only previously one patient who had reached that time point.

So there's been further follow-up on those eight patients. We'll be able to show you longer-term engraftment of the gene-modified cells, more consistent overexpression of enzymatic activity, longer follow-up, decrease in GAG levels and also more clinical data on patients who have got to longer endpoints as well. So we'll be able to show data assay on clinical data on patients after longer follow-up. And this will be both on their cognitive outcome, but we also will have data on, for example, growth parameters as well, which is again a big issue in MPS-I. So that is for MPS-I.

We will also be sharing data on OTL-101 as well for ADA-SCID. There will be further follow-up on patients who have undergone treatment for transfusion-dependent beta-thalassemia, so longer follow-up on the patients who have been treated so far. So there's really quite, as well as other programs. So there's really quite an extensive body of data, and it just showcases the potential of Orchard's platform across a number of different diseases and how HSC gene therapy can correct the underlying defects in immune deficiencies, neurometabolic deficiencies and hemoglobin opportunities as well. And obviously, we'll give you more detail on those different abstracts next week.

Tessa Romero -- JPMorgan -- Analyst

Great, thank you.

Bobby Gaspar, M.D., Ph.D. -- Chief Executive Officer

Thank you.

Operator

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Orchard Therapeutics (ORTX) Q1 2020 Earnings Call Transcript - Motley Fool

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ENGLEWOOD, CO / ACCESSWIRE / May 7, 2020 / Aytu BioScience, Inc. (AYTU) (the "Company"), a specialty pharmaceutical company focused on commercializing novel products that address significant patient needs, announced today that the Company will present its operational results for the quarter ended March 31, 2020 on May 14, 2020, at 4:30 p.m. ET. The Company will review accomplishments from the quarter and provide an overview of its business and growth strategy.

Conference Call Information

1- 877-407-9124 (toll-free)1- 201-689-8584 (international)

The webcast will be accessible live and archived on Aytu BioScience's website, within the Investors section under Events & Presentations, at aytubio.com, for 90 days.

A replay of the call will be available for fourteen days. Access the replay by calling 1-877-481-4010 (toll-free) or 919-882-2331 (international) and using the replay access code 34718.

About Aytu BioScience, Inc.

Aytu BioScience, Inc. is a commercial-stage specialty pharmaceutical company focused on commercializing novel products that address significant patient needs. The Company currently markets a portfolio of prescription products addressing large primary care and pediatric markets. The primary care portfolio includes (i) Natesto, the only FDA-approved nasal formulation of testosterone for men with hypogonadism (low testosterone, or "Low T"), (ii) ZolpiMist, the only FDA-approved oral spray prescription sleep aid, and (iii) Tuzistra XR, the only FDA-approved 12-hour codeine-based antitussive syrup. The pediatric portfolio includes (i) AcipHex Sprinkle, a granule formulation of rabeprazole sodium, a commonly prescribed proton pump inhibitor; (ii) Cefaclor, a second-generation cephalosporin antibiotic suspension; (iii) Karbinal ER, an extended-release carbinoxamine (antihistamine) suspension indicated to treat numerous allergic conditions; and (iv) Poly-Vi-Flor and Tri-Vi-Flor, two complementary prescription fluoride-based supplement product lines containing combinations of fluoride and vitamins in various for infants and children with fluoride deficiency. Aytu recently acquired exclusive U.S. distribution rights to the COVID-19 IgG/IgM Rapid Test. This coronavirus test is a solid phase immunochromatographic assay used in the rapid, qualitative and differential detection of IgG and IgM antibodies to the 2019 Novel Coronavirus in human whole blood, serum or plasma.

Aytu recently acquired Innovus Pharmaceuticals, a specialty pharmaceutical company commercializing, licensing and developing safe and effective consumer healthcare products designed to improve men's and women's health and vitality. Innovus commercializes over thirty-five consumer health products competing in large healthcare categories including diabetes, men's health, sexual wellness and respiratory health. The Innovus product portfolio is commercialized through direct-to-consumer marketing channels utilizing the Company's proprietary Beyond Human marketing and sales platform.

Aytu's strategy is to continue building its portfolio of revenue-generating products, leveraging its focused commercial team and expertise to build leading brands within large therapeutic markets. For more information visit aytubio.com and visit innovuspharma.com to learn about the Company's consumer healthcare products.

Contact for Media and Investors:

James CarbonaraHayden IR(646) 755-7412james@haydenir.com

SOURCE: Aytu BioScience, Inc.

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Aytu BioScience to Report Fiscal Q3 FY 2020 Results and Business Update - Yahoo Finance

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Nasir Striggs, 7, who has sickle cell anemia, is home from the hospital after recovering from the coronavirus. (Courtesy: Deshannon Striggs)

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Maryland boy, 7, with sickle cell disease recovers from coronavirus that caused pneumonia in both lungs - News Info Park

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Protagonist Therapeutics, Inc. Common Stock(NASDAQ:PTGX)Q12020 Earnings CallMay 07, 2020, 5:00 p.m. ET

Operator

Good day, and welcome to the Protagonist Therapeutics PTG-300 development update Call. Please note that today's conference is being recorded. At this time, I would like to introduce Don Kalkofen, Protagonist's chief financial officer. Please go ahead.

Don Kalkofen -- Chief Financial Officer

Thank you, operator. Good afternoon, everyone. Please note that a replay of today's call will be available at the Investors section of our website at protagonist-inc.com. Before we begin, I'd like to remind you that today's discussion will include statements about the company's future expectations, plans and prospects that constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our quarterly and annual reports on forms 10-Q and 10-K, which are on file with the SEC. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so even if our view should change. With that, I will now turn the call over to Dinesh Patel, president and CEO, to provide you an update on the company's progress to date.

Dinesh Patel -- President and Chief Executive Officer

Thanks, Don. Good afternoon, everyone, and thank you all for joining us today. On today's call, we'll also hear from Samuel Saks, our chief medical officer; and Dr. Ronald Hoffman, Professor of Medicine in Hematology and Medical Oncology at the Mount Sinai Hospital in New York City.

Dr. Hoffman has been involved in the Phase II clinical study of PTG-300 in polycythemia vera, and he will provide a brief overview of the disease and his perspectives on PTG-300 and its potential benefits as the novel treatment for polycythemia vera or PV. Also present for the call today are David Liu, our chief scientific officer and head of R&D and Suneel Gupta, our chief development officer. We are pleased to have all these individuals available to address questions during the Q&A session of today's call.

So let's start with Slide No. 3. As you are well aware, Protagonist started the year 2020 with three clinical assets, all of which have been discovered through the use of our peptide technology platform in six different clinical proof-of-concept studies. The three assets fall in two broad categories: PTG-300 for various blood disorders, most of which are rare disease; and oral GI-restricted targeted peptide PTG-200 and PN-943 for inflammatory bowel disease, or IBD.

All of these assets have a multibillion-dollar potential in multiple indications. PTG-300 peptide mimetic of the natural hormone hepcidin that serves as a master regulator of iron homeostasis storage and distribution in the body was being pursued in Phase II open-label proof-of-concept studies in beta-thalassemia, or beta-thal; polycythemia vera, or PV; hereditary hemochromatosis, or HH; and an investigator-sponsored study in myelodysplastic syndrome, or MDS. As you may recall, a major objective for 2020 was to pick our first clinical indication for PTG-300 that we can progress toward a pivotal study in 2021. Today, we are pleased to announce initial but yet very robust clinical data from our polycythemia vera program.

And the selection and prioritization of polycythemia vera as the first indication for a pivotal study with 300. This decision is based on three criteria: strength and consistency of the clinical data, favorable regulatory path forward and the commercial opportunity. This is our major announcement today. And while most of our discussion will be around 300 and polycythemia vera, let me also take this opportunity to talk briefly about our oral gut-restricted IBD assets, largely in the context of corporate update and cash runway.

The enrollment rate of Phase II studies of these agents, namely the oral alpha-4-beta-7 integrin antagonist PN-943 for ulcerative colitis and the oral interleukin-23 receptor antagonist PTG-200 for Crohn's disease are understandably going to be influenced by the current COVID-19 situation. Therefore, while these studies are continuing, we believe it is appropriate to remove future guidance on the timelines for top line data from these studies. In addition to the decision to focus our efforts toward rapidly advancing 300 in polycythemia vera indication, we are continuing the ongoing proof-of-concept study of 300 in hereditary hemochromatosis as a potential second indication and are also deciding to now discontinue further development of 300 for beta-thalassemia as well as for myelodysplastic syndrome. On the financial side, we ended the first quarter of this year with about $117 million in cash and investments.

By factoring in the delay in enrollment and the associated clinical development costs, coupled with a reduction in operational needs and expenditures, we are now extending our cash runway by an additional six months through the middle of 2022. Now let's go to Slide No. 4, and let me turn to PTG-300, a major highlight of today's call and to provide rationale for why we are pursuing polycythemia vera as our first indication. First and foremost, it's the robust and consistent clinical responses we have seen in our ongoing Phase II PV trial.

Although small, the data set is very compelling and consistent. On six out of six, dose compliant patients treated for up to 28 weeks with 300 are phlebotomy free as of today. These are very noteworthy results. Keeping in mind that prior to entering our study, these subjects were receiving frequent phlebotomies in the weeks leading up to enrollment.

We are obviously very encouraged by these results, and the enrollment in the study continues with eight patients enrolled to date. And we have also decided to now expand the current study from 30 patients to 50 patients. The second factor is the regulatory path forward for the development of 300 in polycythemia vera indication. As you know, approved drugs for polycythemia vera fall into the orphan drug designation category by virtue of it being a rare disease in the U.S.

and Europe. And orphan status provide certain benefits to the drug developer, including seven years of market exclusivity upon FDA approval, prescription drug use of fee waivers and tax credits for qualified clinical trial. The third factor besides the data and the regulatory path is the very significant commercial opportunity that lies ahead of us based on the significant unmet need that exists today for these patients. And specifically, by virtue of PTG-300 being a novel mechanism-based, first-in-class non-cytoreductive natural hormone mimetic agent.

While PV is a rare disease, there are approximately 100,000 people in the U.S. living with this disease, and this number is unfortunately expected to grow as the population ages. The majority of PV patients are treated with phlebotomies, cytoreductive therapies or a combination of those treatments. Jakafi is the only FDA-approved cytoreductive therapy in PV and despite having a limited indication in patients who have had an inadequate response to current treatment, Jakafi is expected to reach almost $2 billion in sales in 2020, and the largest percentage growth is in the PV population, 19% year-over-year based on their most recently quarterly update.

PTG-300 has the potential to address a significant unmet need in this category where there is a lack of new cytoreductive agents in development. 300 offers the potential of keeping patients phlebotomy-free without causing iron deficiency. So to summarize, we are incredibly encouraged by our preliminary but strong and consistent data and, therefore, are electing to focus our resources on expedited development of 300 for polycythemia vera. To speak more about this, I would now like to introduce our chief medical officer, Dr.

Samuel Saks. Sam?

Sam Saks -- Chief Medical Officer

Thanks, Dinesh. As Dinesh mentioned, we are really encouraged by the results we have seen in the ongoing Phase II study of PTG-300 in patients with polycythemia vera. Dr. Hoffman is going to walk you through the study results in detail, but I wanted to highlight why I believe this data has prompted us to focus on PV as the first indication for 300.

The majority of PV patients today are treated with phlebotomy, or phlebotomy in combination with cytoreductive therapies to control erythrocytosis and keep the hematocrit levels below 45%, as indicated in many treatment guidelines. There is a large body of evidence that shows that managing hematocrit is challenging and regardless of best available therapy, they're a substantial portion of patient segments that have poorly controlled hematocrit and therefore, a higher mortality rate compared to age match controls. The treatment paradigm of PV allows patients to enter what I call the danger zone before they get the next treatment. So even those people who are considered well-controlled have increased hematocrit leading up to their phlebotomy.

Self-administered 300 may reduce doctor visits and the anxiety associated with the uncertainty of phlebotomy at that time. Phlebotomy is those of donated blood know, is associated with acute symptoms and difficulties. But the real problem in these patients is the potential for chronic symptoms related to iron deficiency in phlebotomized patients. PTG-300 offers the possibility of putting the control of the hematocrit in the patient's hands with a weekly self-injected mimetic of an endogenous hormone mimetic.

With a clear decision to focus PTG-300 development in PV, we are expanding the current study to include additional patients, and we'll be also hosting a scientific planning meeting with leaders in the field of myeloproliferative neoplasms. We will also be working with patient advocates to discuss pivotal and future studies in polycythemia vera. I will close by saying that while further follow-up and data from additional patients will be needed to confirm the continuity of the robust clinical responses observed to date, we believe this study provides a compelling rationale to initiate planning for a pivotal program in PV. In the near term, we are expanding the current study to include additional patients as we focus on these encouraging results.

Finally, I want to express how proud I am of the entire clinical development team of Protagonist and all the Protagonist employees that helped us to get where we are today with this program. With that, I would like to turn it over to Dr. Hoffman, who needs no introduction. He's a recognized expert in the field of myeloproliferative disorders and is Director of the Myeloproliferative Disease Program at The Icahn School of Medicine at Mount Sinai and an investigator in the 300 study.

Dr. Hoffman?

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

Yes. Thank you very much, Sam. Thanks for the opportunity to present this really exciting data. So if we can go to Slide 7, I can educate the audience on what polycythemia vera is so they can have a better understanding of the potential for 300 and put this in context.

So as you can see here, this slide is entitled "What is Polycythemia Vera?" Well, polycythemia vera is a member of a group of chronic hematologic malignancies termed myeloproliferative neoplasms. And these neoplasms are unusual in that patients frequently live several decades. And these neoplasms occur at the level of the hematopoietic stem cell. And unlike leukemia, they are characterized by increased production of mature blood cells.

In polycythemia vera, the cell that's most destructive to the patients are really the increased production of red blood cells. And that's the term erythrocytosis that you've heard previously and we'll hear subsequently. Now the interesting thing about this disease is that there are 100,000 cases of polycythemia vera in the United States. This disease occurs across races and age groups.

And the critical diagnostic warning sign is that of an elevated hematocrit, which is the hallmark of the disease. I think the importance of this drug is that although there's 100,000 cases of these patients, these patients, as I mentioned, live for several decades, so the usage of this drug would be quite prolonged over that period of time. The disease occurs primarily in individuals who are 50 to 70 years of age. There is, however, a higher incidence in young females who present with life-threatening thrombotic events.

The thrombotic events that occur in patients with polycythemia vera include strokes, as well as life-threatening thrombosis of arteries or veins within the venus within the abdominal cavity. There's a very high incidence of acute myocardial infarction, cerebral vascular incidents and strokes. The women that we've talked about, the young women, they are particularly prone to develop thrombosis within the abdominal cavity, especially the hepatic vein, and that leads to a life-threatening condition called Budd-Chiari syndrome. In its most severe forms, it requires liver transplantation.

Over the last 15 years, there's been tremendous understanding, new understanding of this group of myeloproliferative neoplasms. And they've been shown to be characterized by mutations in genes that are associated with myeloid blood cell production. So the myeloproliferative neoplasms are associated with three driver mutations: one involving JAK2, the other gene called calreticulin and the other MPL, which is a receptor for the hormone thrombopoietin. Virtually 99% of patients with polycythemia vera have a mutation in JAK2.

Most commonly, it is a mutation that occurs at exon 14, which is the JAK2V617F mutation. A smaller group of patients have a mutation in exon 12, and those are referred to as exon 12 patients, and they have a similar phenotype, again, elevated hematocrits, increased thrombosis and shorter lifespan due to these thrombotic events and severe erythrocytosis. Next slide, please. We're going to talk now about the diagnosis, symptoms and treatment of polycythemia vera, so that you can get an understanding of the role, the potential role of PTG-300.

The diagnosis of polycythemia vera is triggered by finding an elevated hematocrit serendipitously or by a thrombotic event, and that initiates the physician looking for the diagnosis. So for instance, a patient could present within an acute myocardial infarction and be found to have hematocrit. These warning signs, then prompt, the blood tests to be performed that look for the presence of the JAK2V617F mutation or the exon 12 mutation of JAK2V617F is not present. And sometimes, a bone marrow examination is required to perform histopathologic confirmation of the diagnosis.

This disease is not only complicated by thrombotic episodes, but it's also complicated by burdensome systemic symptoms, including fatigue, headache, visual symptoms, night sweats and itchiness. And this itchiness characteristically occurs on exposure to water and has termed aquagenic pruritus. This might seem like a trivial symptom but aquagenic pruritus can be so severe that patients are unable to shower for months or can lead to such stress that occasionally, we've unfortunately had patients who have succumbed to suicide. Thrombotic events is the greatest risk to patients with polycythemia vera.

These thrombotic events, as I mentioned, include heart attacks, strokes, deep vein thrombosis or pulmonary embolism or the Budd-Chiari syndrome. The cardinal feature of treatment and reduction of this thrombotic risk is to reduce the hematocrit below 45%. Again, the hematocrit is just the measure that we use of the number of red blood cells and the degree of erythrocytosis. And that assures that essentially the viscosity of the blood or the stickiness of the book is reduced, thereby reducing the incidence of thrombosis.

So normalization of the hematocrit using therapeutic phlebotomy is very common, and it is really the first treatment in newly diagnosed patients with polycythemia vera. Unfortunately, therapeutic phlebotomy leads to the removal of red blood cells, which contain iron and it leads to more severe iron deficiency contributing to many of the exacerbation, of many of the systemic symptoms that I referred to previously. And those patients that require frequent phlebotomies or at their advanced stage that is over 60 years of age or have thrombosis, a prior thrombosis, it is the recommendation of the standard of care that those patients all be treated with hydroxyurea, interferon or Jakafi. It is in this situation that 300 is especially attractive drug because it can be used to treat patients who are not requiring myelosuppressive agents or it could be a replacement for those myelosuppressive agents.

Again, the site of the reductive agents are hydroxy and interferon, which are used in combination with phlebotomy, with supplemental phlebotomy, or an alternative drug is Jakafi or ruxolitinib, which is the only U.S. FDA-approved product for phlebotomy. Each of these drugs, and I can go into that in detail during the question-and-answer period are associated with significant adverse effects and have a questionable surrounding their tolerability. Significant evidence, as I've mentioned, shows that controlling the hematocrit below 45% is the most important factor in minimizing thrombosis, cardiovascular events and death due to thrombosis.

Please let's go to the next slide. So we're going to talk a little bit about phlebotomy, which many of you have probably experienced when you donate blood. So most patients receive phlebotomy initially. Phlebotomy is basically the removal of a unit of blood to reduce the hematocrit below 45%.

So our target is to keep those patients always below 45% because it's been well demonstrated in the literature that, that reduces significantly the incidence of thrombosis. And the incidence of thrombosis has been shown to be reduced when the degree of time with an adequate of 45% is maximized. In addition, these would patients receive low-dose aspirin to paralyze their platelets and their or further augment this antithrombogenesis in patients with polycythemia vera. Patients who undergo phlebotomies will often report feeling tired or dizzy after the phlebotomy.

And especially in the elderly, this is a major factor. Many of these patients become hypertensive, require long stays in our outpatient facility or require fluid infusions because or going to congestive heart failure because of their poor fluid balance. Furthermore, regular phlebotomy results in iron deficiency that makes the augmentation of iron deficiency that may have debilitating symptoms. As I mentioned, particularly severe fatigue, weakness and cognitive impairments.

This cognitive impairment is a really big deal because many of the patients complain of having a fuzzy brain syndrome, and they cannot concentrate and do their jobs. In addition, some of the patients have craving for unusual substances such as ice or clay, which is termed pica. And then, unfortunately, some of the patients have a restless leg syndrome, which prevents them from going to sleep. Three or more phlebotomies in a year in those patients receiving hydroxyurea is associated with an inferior prognosis and a higher incidence of thrombotic events.

So you can also think that in that situation, even in a patient who is receiving hydroxyurea, their combination therapy with 300 could eliminate those frequent phlebotomies and further improve the treatment regimen for these patients. I think the most important part that you'll see about this drug are the real high-quality aspect of it is that we see these patients, let's say, a three or four-month intervals. At that three or four-month interval, we see that they have an elevated amount of hematocrit and that triggers us to do a phlebotomy. But in reality, we have no way to monitor them during the interval between their visits, and it's likely that for significant periods of time, they have an elevated hematocrit that's only detected at the time of their visit to our clinic.

So as you'll see, the really cool part about PTG-300 is that there's a sustained hematocrit control, which we've really never been able to achieve with any of the other agents or with phlebotomy alone. Let's go now on to Slide No. 10, which is PTG-300 and non-cytoreductive hepcidin hormone mimetic, its mechanism of action. So how does this drug really work? Well, hepcidin hormone, has been established recently in the last couple of decades, is playing a primary role in promoting the sequestration of iron and macrophages and decreasing iron availability for the production of red blood cells.

So macrophages are a type of white blood cell, and they are present not only in the bone marrow, but they're also present in all other body tissues. But the macrophages in the marrow are termed nursing cells because their activity is essentially to feed iron to red blood cells in the marrow and to alter to allow them to produce additional red cells. This process is disregulated by the JAK2 mutation leading to erythrocytosis. So PTG-300 is believed to limit the excess production red cells in polycythemia vera by essentially allowing that iron to remain in the macrophages.

Therefore, preventing its transfer into the erythroid precursors. In addition, it blocks the iron that's present in the macrophages in the tissues outside of the marrow. And therefore, we believe alleviates many of the systemic symptoms that are associated with polycythemia vera. Let's now go on to Slide 11, which is the Phase II study design.

This is a really interesting manner in which this study has been constructed. You can see there are three phases. Part one is the dose-finding period, which is over 28 days. And there are two components to that.

I believe effective dose-finding phase, where we essentially dose-escalate patients to meet the inadequate requirements. Then a 12 to 14-week period where we essentially have efficacy evaluation that is to maintain the patient's hematocrit below 45%. And then there's part two as a blinded withdrawal where the patients are randomized to receive either the fixed active dose or placebo dose for up to 12 weeks. And then I'm sure the patients will be happy about that, those that go on placebo, they will then allow them we will be allowed to put them on to an open-label extension phase up to 52 weeks where they will again be able to receive 300.

If the patient's hematocrits exceed 45%, they get supplemental phlebotomies. Let's go on to Slide No. 12. This is essentially the responses that we've seen in the seven patients that have been described, that have been evaluated.

The red triangles indicate the phlebotomies, and you can see a lot of them to the left of the solid line. And that's the 32 weeks prior to treatment. And you can see that each of these patients, and these were entry criteria, had at least three phlebotomies during that period. And then you can see this dotted line, and that's the time at which the doses the drug was started.

And the first thing that strikes you is that there are a lot of red triangles to the left and only one red triangle to the right, and that's one of our patients who missed one dose due to the unfortunately, due to the pandemic. And the numbers indicate the doses that were administered over that period of time. So for instance, you can see in patient 150201, there was a dose escalation and then de-escalation, which I'll discuss on the subsequent slide. So this slide really summarizes and probably is our most important slide.

And it basically shows that with the administration of this drug and careful titration that phlebotomy is almost virtually eliminated. Next slide. Let's focus on two patients. One is the top patient, which is the blue line.

And you can see that this patient, this is the individual who missed the dose because of the COVID infection. And you can see that he was started at 10 milligrams, then went up to 20 milligrams. And on 20 milligrams, he had a hematocrit 47.5, he was unable to come in at that time because of the COVID infection. So we increased his dose to 40 milligrams, his hematocrit dropped, but it was still above 45.

He came in, we phlebotomized him, and we've kept him now on 40 milligrams, and you would have seen on Wednesday, and he did not require a phlebotomy and his hematocrit was below 45. If you look at the bottom patient who is in bright red, you can see that, that patient started at time 0 on 10, then was increased to 20 and then on week seven got 40 milligrams and then on week 12 got 80 milligrams. And you can see that, that led to a hematocrit dropping to 37.5. We cut our dose to 40 milligrams, and you can see that over that period of time, she has remained phlebotomy-free.

We saw her on Wednesday, and she again remains below hematocrit of 45. So there's a dose adjustment that you can easily do. The drugs are being administered at home by the patients, giving them a lot of freedom and also empowering them to control their own disease. If we go to Slide 14.

This addresses how do we know that PTG-300 is really hitting its target. And what we're measuring here is serum ferritin. And if you can recall, ferritin is a molecule that reflects storage iron. And you can see these numbers in the vertical column, and if you're below 25, that indicates that one is iron-deficient.

And you can see it with the administration of the drug at subsequent doses over time, the ferritin doses, the ferritin levels increased in each of these patients except the bottom line where a patient received an extremely hematocrit control with an extremely low dose of 300. So these data indicate that the drug is acting on target. What about the adverse defense? Because this is a chronic disease, one must anticipate that patients will be treated over decades. This is a small sample set but you can see that we had no serious adverse events and the adverse events that were recorded were essentially just irritations, essentially at the injection site that slowly resolved and one of the patients had a bruise at a 10-milligram dose.

Let's go on now to Page 16, which is our summary. So just to start this off. I think phlebotomy has probably been used for almost 100 years. And for at least, since I've been in this field, which is from the 70s, there's been great debate about what is the optimal myelosuppressive agent.

There's great concern about chemotherapy being used in these patients, perhaps increasing the incidence of evolution to acute myeloid leukemia. We never really anticipated that a hormone therapy might be used for this type of disease. So I think this trial really represents a paradigm shift and it has great importance for patients with polycythemia vera. So I think we can easily conclude that the additional data demonstrates the potential of this drug to almost entirely avoid the need for bottoming the treatment of polycythemia vera.

All dose compliant patients were phlebotomy-free. Persistent control of hematocrit levels, I think that's really one of the most important issues is that the patients have a sustained control of their hematocrit, which we anticipate will lead to sustained alleviation of their systemic symptoms and elimination of thrombotic episodes. And again, as I tried to indicate to you these elevated hematocrit levels are associated with significant cardiovascular events such as heart attack and stroke. This drug also offers the possibility of weekly administration without the up and down excursions inherent in phlebotomy therapy or any of the toxicities associated with phlebotomy therapy that I discussed with you.

Also, this reduction in phlebotomy may allow sufficient iron to be available systemically to avoid symptoms that we know that are related to iron deficiency. So PTG-300 has the potential as the first non-cytoreductive therapy for PV. This drug is well tolerated and has a safety profile similar with results in prior studies. So as you can see from my presentation, I'm extremely enthusiastic about this drug and its future.

Thank you for your time.

Dinesh Patel -- President and Chief Executive Officer

Thank you, Dr. Hoffman. I would like to note that the results we have discussed today address the study data as of May 1, and the study continues to enroll. And our plan will be to submit to present additional data at upcoming medical meetings.

So at Protagonist, we are now working on four clinical programs with our three candidates, all discovered through our technology platform. Our priorities are: one, advancing PTG-300 in polycythemia vera as well as in hereditary hemochromatosis; two, advancing PN-943 to a Phase II study in ulcerative colitis; and three, working closely with our partner, Janssen, to continue Phase II development of PTG-200 in Crohn's disease. As a result of our ability to limit our focus with 300 to PV and HH, we have organized our efforts to extend our cash runway for an additional six months. We have taken steps to manage and lower our operating costs and align our resources around our current studies.

As a consequence, we now have adequate financial resources to fund planned operating and capital expenditures through the middle of 2022. Finally, we are mindful of the impact that the current coronavirus outbreak may have on our local operations and global activities. As we disclosed in our financial results released today, we are suspending guidance for PN-943 Phase II initiation and PTG-200 Phase II data, and we are actively working to minimize impact on timelines and move as quickly as conditions would permit. Our priority is maintaining the health and safety of our employees and those who are participating in our studies.

We will continue to monitor changing conditions carefully and provide updates as appropriate. With that, we would like to now open up the call for questions. Operator?

Operator

[Operator instructions] Our first question comes from the line of Chris from Nomura. Your line is now open.

Chris Marai -- Nomura Instinet -- Analyst

Hi. This is Chris Marai from Nomura Instinet. Maybe the first one is for the physician on the line. Thank you so much for the overview of PV.

I was curious how you look at a therapeutic like PTG-300 in terms of clinical practice on sort of an everyday basis as you see PV patients? And how do you think it may fit, assuming this profile remains consistent in sort of the treatment lines of PV, how you may see its usage, in particular relative to drugs like Jakafi? Thank you.

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

OK. I think that's a really terrific question. I think its use would be quite wide. I think virtually all patients with PV would be candidates for this drug.

As you can see, we have treated young patients and also older patients with this drug. So it's been well tolerated. I think the advantage, again, is that, especially with young patients, those individuals, we do not want to give life-long myelosuppressive therapy or interferon or Jakafi because of unanticipated potential for unanticipated adverse effects. So if they have an increased phlebotomy requirement, this would be an ideal treatment for those folks because basically, it would free them up from returning to the clinic as frequently as they do and requiring repeated phlebotomies.

When we risk-stratify these patients, those patients that go on the three treatments that I've discussed before, frequently, they still require phlebotomies, and it's been shown by European investigators, if you have additional phlebotomies during your administration of these drugs, that you are an increased risk of thrombosis. So in that setting, this drug could be an adjunctive agent that would eliminate the needs for phlebotomy. Alternatively, and I could see this developing quite quickly, patients are frequently unwilling to take any of these agents, especially hydroxyurea and interferon because of the potential of hydroxyurea and the concern about interferon essentially having systemic symptoms, problems with individuals about immune disease and also its effect to exacerbate depression in patients. So in those individuals, I could see and even in those high-risk patients, if we can control their hematocrits with this drug, which I think we can easily do, and we've demonstrated that.

That this drug would be a real competitor for any of those agents. So I think it could be essentially utilized potentially for the broad swath of patients with polycythemia vera. So I think and for long periods of time, that's the issue that I'd like to really emphasize to you. For instance, like young females who have Budd-Chiari syndrome, and we usually put those young females on myelosuppressive therapy and interferon for life-long periods of time, and they can live several decades.

They would far prefer being on a non-chemotherapy, non-biologic agent like interferon, it would really liberate them, especially from the adverse effects.

Chris Marai -- Nomura Instinet -- Analyst

OK. That's very helpful. And then I was wondering if we could touch upon the data for a minute. Dr.

Hoffman, could you elaborate on some of the effects of spleen size that you may have observed in the trial, if any? What you would hope to see there on an endpoint like that in a patient population like this? Thank you.

Ronald Hoffman -- M.D., Director of the Myeloproliferative Diseases Program

Yes. So in this situation, what you have to understand is this is not myelofibrosis. So in reality, most patients with or virtually all patients, I'll say most, the overwhelming majority of patients with patients with polycythemia that don't have symptomatic splenomegaly. So the endpoints that have been used in the past, let's say, for Jakafi for reduction of spleen size are of minimal importance.

So these patients, even though some of them have high-risk disease, really didn't have they had minimal splenomegaly at all, only 40% of patients with polycythemia vera have splenomegaly and only for our advanced disease it's symptomatic. So the sample size is too small. In animal models, when hepcidin mimetics were given, those are polycythemia vera animal models, the administration of a different hepcidin mimetic did lead to reduction in splenomegaly but we really were not able to assess this because none of these patients really had significant splenomegaly. So we await treating patients like that to see if the data in the mice is recapitulated in humans as it relates to spleen size.

Chris Marai -- Nomura Instinet -- Analyst

Very helpful. Thank you.

Operator

Our next question comes from the line of George Farmer of BMO. Your line is now open.

Gobind Singh -- BMO Capital Markets -- Analyst

Hi, everyone. This is Gobind Singh on for George. Congrats on making a decision and moving forward with those two indications. I guess we had two buckets of questions.

The first two, maybe for Dinesh. I know you guys were thinking about once-weekly dosing and possibly twice weekly dosing in beta-thal. I was wondering if maybe going forward, at least with PV, do you think you might be looking at twice weekly dosing at all? And I was wondering since we are moving past beta-thal and MDS, if you'd be willing to comment at all as to what kind of data we're seeing in those indications? And I'll have a follow-up for Dr. Hoffman, if that's OK.

Dinesh Patel -- President and Chief Executive Officer

Yes. Gobind, thanks for the question. And I will have Sam Saks, our CMO, answer the first question.

Sam Saks -- Chief Medical Officer

Sure. So with respect to the use of 300, we think that it's going to be used widely. You were asking me...

Dinesh Patel -- President and Chief Executive Officer

About the once-weekly.

See the rest here:
Protagonist Therapeutics, Inc. Common Stock (PTGX) Q1 2020 Earnings Call Transcript - The Motley Fool

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Founded in 2014, LetsGetChecked provides consumer customers with a range of at-home testing kits, such as ones for hormone levels, sexually transmitted infections, liver performance, nutritionand specifically for frontline healthcare workers COVID-19.

Customers receive their kit in the mail, with which they collect and return a personal sample for testing. One of the startup's lab partners analyzes the sample and returns it to a LetsGetChecked physician, who reviews the results and counsels customers over the phone, if necessary. For later reference, test results are saved in a LetsGetChecked patient app that interfaces with Apple Health, Fitbit, Garmin and other consumer-friendly health tools.

As of March, the company has also been offering a COVID-19 "twin-track" antibody and PCR test exclusive to providers working at the point of care. However, the company said it hopes to release a direct-to-consumer version of the testing kit down the road.

"The company's work has never been more important now that the world faces a global pandemic, forcing us all to re-evaluate how we obtain health and wellness services," Illumina's Naclerio said in a statement."LetsGetChecked is already having a profound impact on patient-led at-home healthcare, which is necessary now and will become increasingly common in the future."

WHAT IT'S FOR

The company said these funds will help build up its manufacturing, supply and testing capacity for COVID-19. It will be increasing activity at its California laboratorywhile boosting operations and partnerships across the U.S. and Europe.

"We have been developing our platform for more than five years and have helped hundreds of thousands of individuals by enabling access to high-quality testing and telehealth services in the home, across many health conditions," Peter Foley, CEO and founder of LetsGetChecked, said in a statement. "With the onset of COVID-19, we realigned resources with a view to helping with this global pandemic and are currently delivering tens of thousands of tests per day to critical and frontline healthcare workers"

MARKET SNAPSHOT

Consumer-facing home testing startups were already picking up momentum around the time of LetsGetChecked's $30 million Series B, with competitors like EverlyWell, healthy.io and Scanwell Health all announcing new rounds or services over the course of the last year. However, COVID-19 has ramped up demand across the industry and led to some confusion with the FDA on more than one occasion. But the window for these companies to take their COVID-19 tests directly to consumers appears to be reopening: It was just a few weeks ago that LabCorp's COVID-19 RT-PCR test became the first permitted for at-home sample collection.

ON THE RECORD

"HLM invests in companies that inherently drive adoption due to the need they serve, and companies that leverage important market trends," Tolle said in a statement. "In this case, LetsGetChecked is helping to improve consumer access to care, which is increasingly essential."

Original post:
LetsGetChecked locks in $71M Series C to expand COVID-19 testing capacity - MobiHealthNews

Recommendation and review posted by Bethany Smith

(MENAFN - GetNews)

Scottsdale, AZ - Getting medical help and attention does not have to be a stressful process. Essentially, a patient does not have to wait long hours to see a doctor for minor or major complaints and this is why the team at Desert Mobile Medical | Concierge Physicians has repackaged their medical services to offer patients the concierge and mobile medical attention that they deserve. At Desert Mobile Medical | Concierge Physicians, the doctors and primary healthcare providers come to patients wherever they may be, whether at home or work, to deliver the best quality of medical care services, all at an affordable price.

With the current global pandemic brought on by the novel coronavirus, Desert Mobile Medical | Concierge Physicians is working hard to ensure that the healthcare needs of its patients are met. This major concern has led to the announcement of offering the option for virtual appointments. With the capability to do virtual appointments, patients will be offered concierge services that are in line with the recommended social distancing and self-quarantine guidelines while still being able to dispense appropriate medications as necessary.

With Desert Mobile Medical | Concierge Physicians, patients can worry less about exposing themselves to more danger and health risks while waiting at the hospital or doctor's office for their appointments. The team of mobile medical doctors are always ready and on standby to attend to patients virtually or by visiting them in the comfort of their own home.

The concierge physicians at Desert Mobile Medical | Concierge Physicians are unique in the fact that they cut out the middle man (health insurance) so that they can spend more time with YOU, the patient. They have structured their model to be able to provide the highest quality of care while still saving their patients money, whether they have insurance or not. Over 70% of their patients have health insurance and have still found that Desert Mobile Medical provides better care at a lower cost.

Did you know that 90% of health issues can be solved with Direct Primary Care? Desert Mobile Medical | Concierge Physicians is Healthcare Built Around You' meaning they are convenient (since they go to your home), they are flexible (they typically accommodate for same day or next day appointments), and affordable (they take out the middleman, health insurance, to give you quality healthcare with no hidden costs or impossibly complicated invoices).

Overall one of the biggest benefits is saving on lost time. Not only the lost time of driving to the doctor's office and then waiting there for hours, but also because they give you the time needed so they can make sure you have the best care resulting in a more effective you.

Describing the personalized and unhurried care of a concierge doctor offered by the medical physicians at Desert Mobile Medical, a patient left a 5-star review for the online doctor saying, 'This is a fantastic service and I'm glad I found Dr Goel. He takes his time listening to your issue and explains what and why he is treating you the way he is. He doesn't have the typical just do as I say and all will be better' attitude. His focus on preventative care is unique, he prefers to prevent health problems from occurring rather than treat them after they happen. Being able to call, video chat or just message when you need him is the future of doctor appointments. He is quick to respond and does what he can to address your needs as quickly as he can.

Desert Mobile Medical | Concierge Physicians is currently accepting new patients, as they provide a full suite of the most important mobile doctor services to support your vibrant good health, even during this challenging time, by offering the option to visit your home or perform a virtual appointment.

The concierge physician will ensure that patients, during the pandemic, are well catered to. If a patient chooses to have a mobile doctor visit their home for their appointment, prior to their visit, the doctor will maintain COVID-19 safety procedures that will ensure the continued protection of patients from the global pandemic.

Desert Mobile Medical | Concierge Physicians offers services such as: hormone replacement therapy, mobile lab tests, vaccinations, in-home medical dispensing, mobile diagnostic imaging services, direct primary care services, as well as physical and massage therapy services.

Desert Mobile Medical | Concierge Physicians is headquartered at 9300 E Raintree Dr Suite 130 Suite 2, Scottsdale, AZ 85260. Contact their team via phone at (480) 427-0002 or send online inquiries via email to . For additional information regarding their services, visit their website.

Media Contact Company Name: Desert Mobile Medical Contact Person: Dr. Paresh Goel Email: Send Email Phone: (480) 427-0002 Address: 9300 E Raintree Dr Suite 130 Suite 2 City: Scottsdale State: AZ Country: United States Website: http://desertmobilemedical.com/

MENAFN0805202000703268ID1100137432

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Desert Mobile Medical | Concierge Physicians is Now Offering Virtual Appointments to Help Avoid the Spread of COVID-19 - MENAFN.COM

Recommendation and review posted by Bethany Smith

Working from home requires modifications to a traditional workday. The blending of responsibilities from two major areas of our lives, work and home, can be a considerable source of stress, especially during a pandemic.

This is a reality for me, and my husband is a clinical psychologist, so I asked him for some tips. Here is some advice from Dr. Eric W. Bravo on how to stay productive, reduce stress, and manage psychological well-being while working from home:

Cortisol, the main stress hormone in the brain and body, increases as aresult of stress. The blending of work and home obligations is certainly stressful. During a pandemic, everyones cortisol levels are elevated, even if they are not sick.

Chronic exposure to cortisol leads to fatigue, weight gain, sleep disturbance, and an overall negative mood. To reduce the brains exposure to heightened cortisol, several coping skills should be considered.

Maintaining a daily schedule is an important consideration in preventing depression, reducing stress, and increasing energy levels. In the morning, actively construct your day. Think of the tasks that you need to get accomplished.

Having an outline of your responsibilities will guide your actions through the day and leave you with a sense of accomplishment when you are ready to clockout. Think about how to use this time to your advantage. Ask yourself if it is possible to grow the business, make work more organized and efficient, or learn a new skill.

Be aware of limits your productivity and success caused by self-sabotaging. Vices are more accessible than ever. When working from home, it is easier to sleep in, nap during the day, and procrastinate. Hold yourself accountable to the same standard of discipline when you were going into the office.

Most importantly, wake up every day and dress for success. You may not need to wear a suit, but dress to be active. Be sure to wear proper shoes and leave the pajamas for sleeping only.

Changing clothesJanet Loehrke

The body and brain need routines from the most basic of sleeping and eating to other activities, such as work. We are programmed neurochemically to have basic daily cycles. When daily routines are disrupted, our neurological systems shift. Neurochemical imbalances can occur from a shift in daily cycles, leading to depression, anxiety, lethargy, and poor cognitive performance. The basics of a daily routine, including staying consistent with sleep/wake cycles, regular meals, exercise, and activity are essential.

Work-life boundaries are more important now than ever. Boundaries are the psychological barriers between our life at work and life at home. Create a separation between activities that are work-related and those that are not. As much as possible, have a dedicated workspace free from distractions, leisurely activities, and household responsibilities. The distance created by separating the space can let you focus on a productive workday and allow you to leave the office when needed.

To reduce packing on more stress, be careful to manage your expectations. It is common to be less productive when working from home. Develop a hierarchy of work tasks, target the most necessary first then work down your list. If you are unable to complete all of the work, be kind to yourself, take a break.

Think of coping skills as medication to treat an infirmity. We need to practice self-care to stay motivated, productive, and healthy. Immerse your mind and body in activities that are not related to being on duty. Meditation, exercise, cooking, reading, conversations with friends and support systems, and a good sweat all have an effect on lowering cortisol and increasing a positive mood. Furthermore, as important as it is to stay informed, limit your exposure to upsetting news.

Motivation and productivity take work, but, are attainable. The more that a person prepares for the day with a routine, the better off. Control the amount of time that you are not productive. It is fine to relax and watch a good movie, just have limits. Set boundaries for downtime. Getting in the habit of being sedentary will slow your mind, body, and energy. Conversely, consistent exercise and activity maintain and overall sense of motivation.

Hold yourself accountable by limiting the excuses to get work done. Chances are, these are the same rationalizations that are noticeable before working from home. Avoid working from your bed or bedroom. As comfortable as it may seem, a productive day requires being slightly uncomfortable. Our bedrooms have too many associations of relaxation. We do not want to contaminate the drive needed to complete our daily obligations. We also do not want to tarnish the mellow vibe of where we rest.

Engagement with online hangouts and meetings are crucial for perspective, social interaction, and coping with feelings of loneliness. Avoid isolation and make it a priority to reach out to your social network. Take it another step, change the dynamics, and reach out to a friend that you have not heard from in a while.

A new phenomenon, Zoom Fatigue, is leading to higher levels of lethargy and exhaustion during the workday. Video-conferencing requires much more focus and energy than in-person meetings. Paying attention to multiple people at once, figuring out eyecontact, and concerns about our digital appearance is draining. Interestingly, similar fatigue can also occur during social videoconferencing. In order to combat this fatigue, think about the necessity of using video. If a telephone call will suffice for work or social purposes, it is a much better option.

Practice the awareness of gratitude for your employment. Remind yourself of the reason that you do what you do. Think about your journey and accomplishments. Familiarize yourself with the mission of your job. In a time where unemployment rates are at an all-time high, you have employment to report to. Convince yourself that you have a purpose, arise, go forth, and conquer!

It can be difficult to work from home for long periods of time. By practicing these coping strategies, you can increase the likelihood of success, limit stress, and improve your mood. Keep in mind, we are not in control of what happens to us, but we are responsible for how we adapt and respond.

Editor's Note: The author and a source named in this article are married.

Read more here:
Working from home during the coronavirus pandemic: How to cope - USA TODAY

Recommendation and review posted by Bethany Smith

Deepak Chopra, Special to SFGate

By Deepak Chopra, MD, FACP, Rudolph E. Tanzi, PhD, Michelle Williams, ScD, Ryan Castle, William C. Bushell, PhD, Kimberly Brouwer, PhD, and Paul J. Mills, PhD

Although COVID-19 is very easily transmitted from person to person, the risk of subsequent hospitalization and death primarily affects people who are already at risk because of old age, infirmity and/or chronic diseases such as cancer, diabetes, autoimmune illness, obesity, and heart disease. All of these chronic illnesses are associated with measurable low-grade inflammation in the body. The chronic low-grade inflammation that develops with advanced age has become known as inflammaging. Most people with chronic illness unknowingly have low-grade inflammation. Recent research points to a second finding: these same disorders are often accompanied by persistent low-grade anxiety and depression.

All of this as a background increases the danger for a person when acute illness strikes. In addition to the elderly and chronically ill, COVID-19 is causing acute respiratory illness and stroke sometimes leading to death in seemingly otherwise healthy younger individuals. The transition from SARS-CoV-2 infection to diagnosed COVID-19 is typically accompanied by a cytokine storm. Cytokines are proteins that are major drivers of inflammation, and their rapid increase, or "storm is one of the bodys immune responses to acute threat.

In addition, studies have connected pro-inflammatory cytokines to the stress response; they regulate well-known stress hormones such as ACTH and cortisol. Three major systems are involved: the immune system, the central nervous system and the endocrine hormone system.

In the face of these connections, we are coming forward to suggest that complementary practicesdeep breathing, yoga, and meditationcan play an important role during this pandemic. These practices have been confirmed by hundreds of scientific studies to bring down over-activity of the autonomic nervous system, calm the mind from anxiety, reduce the stress response, regularize heartbeat, and lower blood pressure. Together, all of these diverse benefits are associated with reducing the invisible presence of chronic low-grade inflammation, especially if added to good sleep, exercise, and proper diet.

We dont fully understand how the immune response, linked to stress and inflammation, can turn lethal. As a response to cuts, wounds, invading pathogens, and other threats, prior to antibody formation, the body first responds with inflammation as a normal yet crucial healing function. But it has long been known that inflammation is paradoxical. Acute inflammation can over-react, harming or even killing the patient. (Instances of strokes and heart attacks among young COVID-19 patients might be linked to micro-cytokine storms in the brain and heart.)

The threat from low-grade chronic inflammation was not discovered until recently but seems to be widespread. It is unaccompanied by the swelling, burning, and redness of the skin that marks acute inflammation and therefore goes undetected by the patient or physician. Preventing and addressing chronic low-grade inflammation and its significant adverse consequences are urgent issues, even more urgent during a pandemic. There seems to be every reason to make the public aware how deep breathing, meditation, yoga, and other healthy lifestyle practices can help during this crisis and long afterwards.

Deepak Chopra MD, FACP, Clinical Professor of Family Medicine and Public Health at the University of California, San Diego

Rudolph E. Tanzi, PhD, Kennedy Professor of Neurology at Harvard Medical School/MGH

Michelle Williams, SM, ScD, Dean of the Faculty, Harvard T.H. Chan School of Public Health

Kimberly Brouwer, PhD, Professor and Chief, Department of Family Medicine and Public Health, Division of Global Health, Infectious Diseases Epidemiology, at the University of California, San Diego

Ryan Castle, Executive Director of the Chopra Library

William C. Bushell, PhD, medical anthropologist and research director of the Chopra Library

Paul J. Mills, PhD, Professor and Chief, Department of Family Medicine and Public Health, at the University of California, San Diego

Read more from the original source:
The Key to Handling Stress and COVID-19 - SFGate

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., May 5, 2020 /PRNewswire/ -- Akebia Therapeutics, Inc. (Nasdaq: AKBA), today announced positive top-line results from INNO2VATE, its global Phase 3 cardiovascular outcomes program evaluating the efficacy and safety of vadadustat, its investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), versus darbepoetin alfa for the treatment of anemia due to chronic kidney disease (CKD) in adult patients on dialysis. (Please refer to Akebia's INNO2VATE Data Announcement for the top-line data.) The Company also reported financial results for the first quarter ended March 31, 2020 and will host a conference call with slides today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time to discuss INNO2VATE top-line data, its first quarter financial results and recent business highlights.

"We are very excited about the positive top-line results from INNO2VATE and expect to build on this momentum with many potentially transformational near-term milestones," said John P. Butler, President and Chief Executive Officer of Akebia Therapeutics. "We believe we've developed an exciting path forward for vadadustat and although the COVID-19 environment remains unpredictable, we continue to execute and deliver solid progress against these initiatives. In collaboration with our partner, Mitsubishi Tanabe, we are advancing key pre-commercial activities in support of the first regulatory approval of vadadustat expected in Japan this year. We have also significantly advanced PRO2TECT, our global Phase 3 studies evaluating the safety and efficacy of vadadustat in adult patients not on dialysis with anemia due to CKD, and expect to report top-line data from these studies mid-year, as planned. In addition, we reinforced our intellectual property position for vadadustat, confirming the path for Akebia and our collaboration partner, Otsuka, to launch vadadustat in the U.K. and potentially the rest of Europe, upon approval."

"Akebia continues to provide and support our innovative therapies, which are critical to the care of adult patients with CKD, who are among the most at risk during this pandemic. To date, we have not experienced any significant adverse impact from COVID-19 and our fundamentals remain strong with a cash runway that extends well into 2021. However, these are unprecedented times and, due to COVID-19, we do not have clear visibility on how product demand and payer mix may be impacted. As a result, we continue to actively monitor and assess the potential impact of COVID-19 on our business and operations while continuing to support patients." Butler concluded, "We believe we have tremendous value-enhancing opportunities ahead and we remain focused on supporting patients and executing on our plans to position Akebia for long term growth."

Business Highlights

Today, the Company announced positive top-line results from INNO2VATE. Please refer to

for the top-line data.In April, the Patents Court of the United Kingdom issued a

in favor of Akebia and Otsuka Pharmaceutical Co. Ltd., which found that five of FibroGen, Inc.'s six HIF-related patents were invalid, and a sixth patent would not be infringed.In April, the Company announced that Myles Wolf, M.D., M.M.Sc., joined Akebia's Board of Directors. Dr. Wolf is a leading clinical nephrologist and physician-scientist in the fields of disordered mineral metabolism and cardiovascular disease in patients with CKD. Dr. Wolf serves as Chief of the Division of Nephrology and a Professor of Medicine at Duke University School of Medicine.In April, the Company settled Auryxia patent litigation with Teva Pharmaceuticals USA, Inc. (Teva) and its wholly owned, indirect subsidiary, Watson Laboratories, Inc. (Watson), resolving patent litigation brought in response to Abbreviated New Drug Application (ANDA) filings by Teva and Watson. Consistent with the Company's prior ANDA settlement with Par Pharmaceutical, Inc., the settlement allows Teva and Watson to market its generic version of Auryxia in the United States beginning on March 20, 2025 (subject to U.S. FDA approval), or earlier under certain circumstances customary for settlement agreements of this nature.In April, the Company entered into a new supply agreement with STA Pharmaceutical Hong Kong Limited, a subsidiary of Wuxi AppTec (WuXi), under which WuXi will manufacture vadadustat drug substance for commercial use. The WuXi supply agreement is the third commercial supply agreement the Company has entered into for vadadustat. The Company entered into a commercial supply agreement for vadadustat drug substance with Esteve Qumica, S.A. in April 2019, and a commercial supply agreement for vadadustat drug product with Patheon Inc. in March 2020.In April, the United States District Court for the District of Delaware dismissed a putative securities class action brought against the Company's wholly owned subsidiary, Keryx Biopharmaceuticals, Inc. (Keryx), and former members of Keryx's board of directors, relating to the Company's 2018 merger with Keryx in response to a motion to dismiss filed by Keryx and the former directors.In February, the Company entered into a letter agreement with Vifor (International) Ltd. (Vifor Pharma) relating to Vifor Pharma's agreement with a third party to purchase a Priority Review Voucher (PRV) issued by the U.S. Food and Drug Administration (FDA), subject to satisfaction of customary closing conditions. Pursuant to the letter agreement, Akebia paid Vifor Pharma $10 million. Vifor Pharma is obligated to reserve the PRV for the vadadustat NDA for the treatment of anemia due to CKD in dialysis-dependent and non-dialysis dependent patients until Akebia and Vifor Pharma agree on the financial and other terms under which it will assign the PRV to Akebia or make a mutual decision to sell the PRV. A PRV entitles the holder to priority review of an NDA or a Biologics License Application for a new drug, which reduces the target FDA review time to six months after official acceptance of the submission and could lead to expedited approval.

First Quarter Financial Results

Revenues: Total revenue increased 22 percent to $88.5 million for the first quarter of 2020 compared to $72.7 million for the first quarter of 2019.Collaboration revenue was $59.3 million for the first quarter of 2020 compared to $49.6 million in the first quarter of 2019, an increase of 20 percent.Net product revenue was $29.2 million for the first quarter of 2020 compared with $23.1 million in the first quarter of 2019, an increase of 26 percent.COGS: Cost of goods sold was $27.7 million for the first quarter of 2020, which includes non-cash charges, related to the application of purchase accounting as a result of the merger with Keryx, of $11.2 million for inventory step-up and $9.1 million for amortization of intangibles. Cost of goods sold was $31.3 million for the first quarter of 2019, and included non-cash merger-related charges of $14.6 million for inventory step-up and $9.1 million for amortization of intangibles.R&D Expenses: Research and development expenses were $81.2 million for the first quarter of 2020 compared to $82.4 million for the first quarter of 2019.SG&A Expenses: Selling, general and administrative expenses were $38.0 million for the first quarter of 2020 compared to $34.3 million for the first quarter of 2019.Net Loss: Net loss was $60.7 million for the first quarter of 2020 compared to $72.4 million for the first quarter of 2019.Cash Position: Cash, cash equivalents and available-for-sale securities as of March 31, 2020 were $115.4 million. The Company's cash runway extends well into 2021. The Company's cash runway, consistent with previous commentary, includes the receipt of a $15.0 million regulatory milestone from Mitsubishi Tanabe Pharma Corporation, Akebia's development and commercialization collaboration partner in Japan for vadadustat, assuming approval of vadadustat in Japan.

"Consistent with our prior first quarter periods, the decrease in the Company's cash, cash equivalents and available-for-sale securities from year-end 2019 was due to the timing of cash payments from our collaboration partner, Otsuka, for which $49.5 million was received in the current quarter rather than the first quarter of 2020. In addition, during the first quarter, we paid $10 million to Vifor Pharma in connection with Vifor Pharma's purchase of the PRV," stated Jason A. Amello, Chief Financial Officer of Akebia.

COVID-19 Response The current severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) (COVID-19), pandemic has presented a substantial public health and economic challenge around the world and is affecting Akebia's employees, patients, customers, collaboration partners, vendors, communities and business operations. On March 15, 2020, Akebia issued a press release detailing its response to the COVID-19 pandemic. Consistent with that statement, the Company continues to take action to safeguard its employees, patients and customers, ensure business continuity, and support supply of its innovative therapies which are critical to the care of adult CKD patients, who are among the most at risk during this pandemic. Akebia continues to actively monitor and assess the potential impact of the COVID-19 pandemic on its business and operations. Patients with any questions about accessing Akebia's marketed therapy may refer to AkebiaCares, the Company's patient access program, for further information.

Conference Call: Akebia will host a conference call today, Tuesday, May 5, 2020, at 8:30 a.m. Eastern Time to discuss its INNO2VATE top-line data announced earlier this morning and its first quarter financial results. To listen to the conference call, please dial (877) 458-0977 (domestic) or (484) 653-6724 (international) using conference ID number 8464788. The call will also be webcast LIVE with slides and can be accessed via the Investors section of the Company's website at http://ir.akebia.com.

A replay of the conference call and the slides will be available two hours after the completion of the call through May 11, 2020. To access the replay, dial (855) 859-2056 (domestic) or (404) 537-3406 (international) and reference conference ID number 8464788. An online archive of the conference call can be accessed via the Investors section of the Company's website at http://ir.akebia.com.

About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. The Company was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at http://www.akebia.com which does not form a part of this release.

About Vadadustat Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor currently in global Phase 3 development for the treatment of anemia due to CKD. Vadadustat is designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues. Vadadustat is an investigational therapy and is not approved by the U.S. Food and Drug Administration (FDA) or any regulatory authority.

About Anemia due to Chronic Kidney Disease (CKD) Anemia is a condition in which a person lacks enough healthy red blood cells to carry adequate oxygen to the body's tissues. It commonly occurs in people with CKD because their kidneys do not produce enough erythropoietin (EPO), a hormone that helps regulate production of red blood cells. Anemia due to CKD can have a profound impact on a person's quality of life as it can cause fatigue, dizziness, shortness of breath and cognitive dysfunction. Left untreated, anemia leads to deterioration in health and is associated with increased morbidity and mortality in people with CKD.

Forward-Looking Statements Statements in this press release regarding Akebia's strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding clinical trial data and results and the anticipated timing of the availability and reporting thereof; Akebia's momentum and potentially transformational near-term milestones; the safety and efficacy of vadadustat, the potential launch of vadadustat, the potential indications for and benefits of vadadustat, and market size, commercial potential, prevalence, and the growth in, and potential demand for, vadadustat; access to a Priority Review Voucher for vadadustat and the agreement relating thereto; potential and anticipated payments from our collaborators, including the timing thereof; expectations regarding financial position, including cash runway and the components thereof; and our intellectual property position. The terms "anticipate," "believe," "expect," "opportunity," "planned," "potential," "target," "will" and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators, vendors and customers operate; the risk that existing preclinical and clinical data may not be predictive of the results of ongoing or later clinical trials, including PRO2TECT; the risk that clinical trials may not be successful; manufacturing risks; risks associated with the Priority Review Voucher for vadadustat; risks associated with management and key personnel changes and transitional periods; the actual funding required to develop and commercialize our commercial product, vadadustat and other product candidates and operate the Company, and the actual expenses associated therewith; the actual costs incurred in the clinical studies of vadadustat and the availability of financing to cover such costs; the risk that clinical studies are discontinued or delayed for any reason, including for safety, tolerability, enrollment, manufacturing or economic reasons; market acceptance and coverage and reimbursement of our commercial product and vadadustat, if approved; the risks associated with potential generic entrants for our commercial product and vadadustat, if approved; early termination of any of Akebia's collaborations; Akebia's and its collaborators' ability to satisfy their obligations under Akebia's collaboration agreements; the timing and content of decisions made by regulatory authorities; the timing of any additional studies initiated for vadadustat; the actual time it takes to initiate and complete preclinical and clinical studies; the competitive landscape for our commercial product and vadadustat; the scope, timing, and outcome of any legal, regulatory and administrative proceedings; changes in the economic and financial conditions of the businesses of Akebia and its partners; the risk that we lose, or settle on less favorable terms, other ANDA litigation, or that other ANDA filers enter the market earlier than March 20, 2025, as well as any other potential settlements; and Akebia's ability to obtain, maintain and enforce patent and other intellectual property protection for our commercial product, vadadustat and any other product candidates. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Annual Report on Form 10-K for the year ended December 31, 2019 and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.

Investor Contact: Kristen K. Sheppard, Esq. Ir@akebia.com

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Akebia Announced Positive Top-Line Results from Global Phase 3 Program of Vadadustat for Treatment of Anemia Due to Chronic Kidney Disease in Adult...

Recommendation and review posted by Bethany Smith

In the early 1950s, psychiatrists began treating schizophrenia with a new drug called chlorpromazine. Seven decades later, the drug is still used as an anti-psychotic.

But now scientists have discovered that the drug, also known as Thorazine, can do something entirely different. It can stop the new coronavirus that causes Covid-19 from invading cells.

Driven by the pandemics spread, research teams have been screening thousands of drugs to see if they have this unexpected potential to fight the coronavirus. Theyve tested the drugs on dishes of cells, and a few dozen candidates have made the first cut.

Theyre startlingly diverse. Some, like chlorpromazine, have been used for years not for viral infections, but for conditions including cancer, allergies, arthritis, even irregular menstrual periods. Other drugs have not yet been approved by the Food and Drug Administration, but they have already proven safe in clinical trials. Their track records might help them get approved faster than a drug designed from scratch.

As researchers publish findings on these promising drugs, theyre starting tests on animals and people to see how well they perform. No one should try self-medicating with any of the drugs for Covid-19, the researchers warned, since they may have dangerous side effects and have yet to be proven effective in clinical trials.

Im going to be brutally honest with you: 95 to 98 percent of these are going to fail, said Sumit K. Chanda, a virologist at Sanford Burnham Prebys Medical Discovery Institute in La Jolla, Calif. But we only need one or two.

The strategy Dr. Chanda and other researchers are using is known as drug repurposing. It has a history that started decades before Covid-19 appeared. In 1987, for example, the cancer drug zidovudine became the first F.D.A.-approved drug against H.I.V.

The most obvious drugs to repurpose against the new coronavirus are those that work against other viruses. One high-profile antiviral being investigated is remdesivir, which Gilead Sciences previously tested unsuccessfully as an antiviral against Ebola.

But over the years, researchers have found some drugs that originally had nothing to do with viruses turn out to be good antivirals, too. Its just hard to tell in advance which ones have this hidden power.

We dont know a lot about why drugs do what they do, said Matthew Frieman, a virologist at the University of Maryland School of Medicine.

In 2012, another coronavirus disease known as MERS emerged in the Middle East. Dr. Frieman responded by starting a drug-repurposing study. He and his colleagues tested 290 F.D.A.-approved drugs and found that 27 of them blocked the MERS virus from infecting cells. They also proved effective against the related coronavirus that causes SARS.

Dr. Frieman and his colleagues have now tested those drugs against the new coronavirus, and made a preliminary report that 17 of them showed promise. Along with chlorpromazine, they include drugs for disorders as varied as Parkinsons disease and leukemia.

Recently, Dr. Chandas team in California began a mammoth search of their own for drugs to repurpose for Covid-19. They doused infected cells with 13,000 compounds and looked for ones that slowed down the virus. They then narrowed down these candidates by reducing their doses, in order to mimic the levels that would end up in a patients lungs.

On April 17, Dr. Chandas team reported in a preprint, which has not yet been peer-reviewed by a journal, that six drugs showed particular promise, including one for osteoporosis and one thats been investigated as treatment for arthritis.

Yet another team has been trying to find drugs that work against coronavirus and also to learn why they work.

The team, led by Nevan Krogan at the University of California, San Francisco, has focused on how the new coronavirus takes over our cells at the molecular level.

The researchers determined that the virus manipulates our cells by locking onto at least 332 of our own proteins. By manipulating those proteins, the virus gets our cells to make new viruses.

Dr. Krogans team found 69 drugs that target the same proteins in our cells the virus does. They published the list in a preprint last month, suggesting that some might prove effective against Covid-19.

The researchers shipped the compounds to the Icahn School of Medicine at Mount Sinai in New York and at the Pasteur Institute in Paris. Those labs tried them out on infected cells.

Brian Shoichet, a pharmaceutical chemist at U.C.S.F. who helped build the list, was keenly aware of how often drug repurposing fails.

I wasnt that hopeful at all, he said.

It turned out that most of the 69 candidates did fail. But both in Paris and New York, the researchers found that nine drugs drove the virus down.

The things were finding are 10 to a hundred times more potent than remdesivir, Dr. Krogan said. He and his colleagues published their findings Thursday in the journal Nature.

Strikingly, the drugs hit only two targets.

One group temporarily stops the creation of new proteins inside cells. This group includes molecules that are being tested as cancer drugs, such as ternatin-4 and Zotatifin.

Dr. Shoichet speculated that these compounds starve the virus of the proteins it needs to make new copies of itself. This attack may suddenly halt the viral production line.

Viruses are actually delicate beasts, he said.

The other compounds home in on a pair of proteins known as Sigma-1 and Sigma-2 receptors. These receptors are part of the cells communication network, helping the cell withstand stress in its environment.

Why does the new coronavirus need to manipulate Sigma receptors? We dont really know, Dr. Shoichet said.

One possibility is that the virus uses Sigma receptors to make a cell produce more of the oily molecules that form membranes for new viruses.

Among the substances that act on Sigma receptors and block the virus, the researchers found, are the hormone progesterone and the drugs clemastine and cloperastine, both used against allergies.

In addition, Dr. Krogan said that all of Dr. Friemans candidates, including chlorpromazine, target Sigma receptors. A third of Dr. Chandas candidates do too, he said.

The researchers also tested dextromethorphan, a Sigma-receptor-targeting drug in many brands of cough syrup. They were surprised to find that, at least in their cell samples, it actually made infections of this coronavirus worse.

In their paper, the researchers raised the possibility that Covid-19 patients may want to avoid dextromethorphan. Dr. Krogan emphasized that more study would be needed to see if it actually increases coronavirus infection in humans. But if it was me, he said, to be cautious, I would not be taking these cough syrups.

The anti-malaria drugs chloroquine and hydroxychloroquine act on the Sigma receptor. Dr. Krogans team found that they also fought the virus in cells. Those compounds were extolled by President Trump for weeks despite no firm evidence they actually helped cure Covid-19.

Dr. Frieman and Dr. Chanda also found that chloroquine-related drugs worked fairly well in slowing the virus in cell cultures. But Dr. Chanda found they didnt work as well as the six compounds at the top of his list.

Dr. Chanda expressed skepticism about the chloroquine drugs, noting their failure against other viruses.

Weve been down this road multiple times, he said. I would happy to be wrong about this.

Last week, the F.D.A. issued a warning against using hydroxychloroquine or chloroquine for Covid-19 outside the hospital setting or a clinical trial. Thats because the drug has a well-known risk for causing irregular heart rhythms.

In their new study, Dr. Krogan and his colleagues ran an experiment that might explain this risk at the molecular level.

They found that chloroquine and hydroxychloroquine bind not just to Sigma receptors, but to a heart protein called hERG, which helps control heartbeats.

I think its a rational argument, said Dr. Frieman, who was not involved in the Nature study. Chloroquine does a lot of things in the cell.

Dr. Krogan and his colleagues found that other compounds target Sigma proteins in a more promising way.

An experimental anticancer compound called PB28 is 20 times more potent than hydroxychloroquine against the coronavirus, for example. But its far less likely to grab onto the hERG protein.

Dr. Chanda said that PB28 in particular looks really fantastic.

Dr. Krogan said that studies are underway to test the drug in hamsters to see if that promise holds. Dr. Frieman and his colleagues are starting animal studies of their own, as well as testing drugs on a chip lined with human lung cells.

Timothy Sheahan, a virologist at the University of North Carolina who was not involved in the new studies cautioned that it will take more testing to make sure these promising drugs are safe to give to patients ravaged by Covid-19.

Cancer drugs, for example, can be like a sledgehammer to your body, he noted. Are you going to want to do that when someone is really sick?

In addition to animal tests and clinical trials, researchers are now planning to tweak the structure of these drugs to see if they can work even more effectively against the virus.

Originally posted here:
Old Drugs May Find a New Purpose: Fighting the Coronavirus - The New York Times

Recommendation and review posted by Bethany Smith

If we are looking for some entity to blame for the Coronavirus Pandemic, the obvious choice would be China. Scientists are fairly certain that is where the micro-organism began an assault on humanity.

The city of Wuhan is known as a center of bio-engineering experimentation. Whether or not the outbreak was an accident or part of, as some Russian military experts have claimed, a strategic effort to realign the political power structure on Earth, is a different question. If one wanted to put the question before a jury, as any prosecuting attorney would point out, China had the means, the opportunity, and the motive. Their economic light was dimming dramatically, and the recent surge of demands for democratic reforms, and the stiff tariffs imposed by Trump have shaken the Communist regime. With a population of nearly 1.4 billion, losing a few million citizens to disease would be a cheap price for them to pay to overcome Americas worldwide economic dominance.

What about Russia? Not a chance. They are super dependent on oil to prop up their weak economic infrastructure and they would never have contributed to crude prices falling into negative numbers. Europe? Not scientifically or politically motivated or capable.

That leaves the USA. The Chinese have suggested we started the outbreak, though they never outlined why we would want to destroy our own economy.

There is one group within the worldwide demographic that could qualify as beneficiaries of a complete economic and political realignment: That would be the post Baby Boomer generations of Millennials and X-Gens. We have surveys that claim most of them hate capitalism and economic inequality. If, as a theoretical political force, they wanted to clear the decks for their future, what would be the one thing that could redistribute economic wealth, restore the health of the environment, and expand the nanny state?

The answer is reducing or eliminating the drag that senior citizens put on civilization. Americas aging population is living longer, using more resources, drawing on retirement benefits and social services at higher rates than anyone ever predicted when statisticians did their amortization schedules back in the 70s and 80s. We old farts were supposed to have been gone by now. If there is any group that might see an economic upside to the pandemic it would be our kids and their kids.

Do I really think our descendants are anxious to kill us off? No, but the cause and effect are intriguing food for thought Do I really believe this Pandemic is manmade? Yes and no. I think science got burned playing with fire. In our never-ending search for the elixir of youth, science sometimes ventures into dangerous territory. So I think this epidemic was probably made worse by human experimentation, trying to understand more about how these molecular killers threaten our species. Were all living a nightmare resulting from good intentions gone horribly wrong.

Whether by accident or by design the Coovid19 pandemic is effectively culling the herd. Ranchers do this to improve the gene pool of their cattle, or to reduce their costs associated with illnesses. Mother Nature also has a way of draining the swamp just before it becomes too toxic, then replenishing it with freshwater and reviving the ecological balance.

Am I suggesting seniors are toxic and young people are willing to put us out to pasture? No, but the truth is life is terminal, and living in freedom is dangerous. No society can guarantee safety and security, under any political system. During this scary period, civilization should attempt to protect the most vulnerable, the aged, and the sick, and once again young people will bear the cost and lifestyle burden for them. Baby Boomers have made life extension our main goal, and now our offspring will have to pick up the tab.

Rick Elkin is an artist, author, and columnist. His most recent book, The Illusion of Knowledge: Why So Many Educated Americans Embrace Marxism, is available at most online booksellers. He resides in Escondido, California. You can follow him at RickElkin.com.

*Note: Opinions expressed by columnists and letter writers are those of the writers and not necessarily those of the newspaper.

Here is the original post:
Picking Up the Tab - times-advocate.com

Recommendation and review posted by Bethany Smith

New Delhi: Eight workers of NLC India Ltd were injured and hospitalised in a boiler blast at NLC India Limiteds thermal power station (TPS) II in Neyveli in Tamil Nadu on Thursday.

The power stations of the integrated mining-cum-power generating company are located at Neyveli in Cuddalore.

Here is all you need to know about at NLC India Limiteds thermal power station (TPS) II in Neyveli, the company where the boiler blast occurred.

Neyveli Thermal Power Station-I

The first unit of the 600 MW Neyveli Thermal Power Station-I was synchronized in May 1962 and the last unit was synchronized in September 1970

The 600 MW Neyveli Thermal Power Station-I consists of six units of 50 MW each and three units of 100 MW each.

The Power generated from Thermal Power Station-I is supplied to TANGEDCO, Tamilnadu which is the sole beneficiary.

Due to the aging of the equipments / high pressure parts, the Life extension programme has been approved by the government in March 1992 and was successfully completed in March 1999 thus extending the life by 15 years, the company profile reads.

This power station is being operated after conducting Residual Life Assessment (RLA) study. Government has sanctioned a 2x500 MW Power Project (Neyveli New Thermal Power Plant NNTPS) in June 2011 as replacement for existing TPS-I, it adds.

More:
NLC India TPS II, all about the company where boiler blast occurred in Neyveli - Zee News

Recommendation and review posted by Bethany Smith

When looking at treatment outcomes for small, hormone-receptor-positive, HER2-positive breast cancers that have not spread to the lymph nodes, a study suggests that women diagnosed with cancers 810 mm (about 0.31 inches to 0.39 inches) in size had better survival when they were treated with chemotherapy after surgery compared to women diagnosed with smaller cancers.

The research was published on April 9, 2020, by the journal JAMA Network Open. Read the abstract of Association of Survival With Chemoendocrine Therapy in Women With Small, Hormone Receptor-Positive, ERBB2-Positive, Node-Negative Breast Cancer.

The HER2 gene is also called the ERBB2 (Erb-B2 receptor tyrosine kinase 2) gene.

Research shows that very small, node-negative, hormone-receptor-positive, HER2-positive breast cancers have a 5-year recurrence risk the risk of the cancer coming back within 5 years that ranges from 5% to 25%, with or without treatments after surgery. Doctors call treatments given after surgery adjuvant treatments.

Because HER2-positive cancer is considered more aggressive than HER2-negative breast cancer, it is usually treated with chemotherapy after surgery to reduce recurrence risk. Still, it has been unclear whether the benefits of treating very small, node-negative, hormone-receptor-positive, HER2-positive breast cancers with chemotherapy after surgery outweighed the risks.

It wasnt clear what to do with these patients, who make up a really small population of breast cancer patients, Anurag Singh, M.D., of Roswell Park Comprehensive Cancer Center, one of the researchers who did the study, said in an interview.

We know that if you have an ERBB2 tumor, it's worse, and the idea has been they should get chemotherapy,he continued. You have an otherwise healthy 60-year-old, mammographically screened and detected patient. When you give them the information, they're saying, 'OK, I have all of these good things, but I've got a 2-mm tumor and you want to give me 6 months of chemo. Are you serious? I'm going to lose my hair, I'm going to have this, I'm going to have that. It seems like an awful long way to go.' We didn't really have good evidence for them.

The researchers looked at the records of 10,065 women diagnosed with node-negative, hormone-receptor-positive, HER2-positive breast cancer that was 10 mm or smaller in size between 2010 and 2015.

All of the women were treated with hormonal therapy after surgery:

From 2013 to 2015, anti-HER2 therapy such as Herceptin (chemical name: trastuzumab) was coded differently than chemotherapy in patient records; 15% of the women treated during this time had either chemotherapy or anti-HER2 therapy alone.

Follow-up time ranged from about 2 years to about 5 years.

The researchers compared the outcomes of women treated with hormonal therapy and chemotherapy to women treated with hormonal therapy alone. They also looked to see if the size of the cancer affected any benefits from chemotherapy.

The analysis showed that chemotherapy was associated with better overall survival for women diagnosed with cancers that were 810 mm in size. Women diagnosed with cancers that were smaller did not seem to get any survival benefits from chemotherapy.

To our knowledge, this is the first report to suggest that there is an association between improved survival and adjuvant chemoendocrine therapy specifically for HR-positive, ERBB2-positive tumors 8 mm to 10 mm compared with those smaller than 8 mm, the researchers wrote. It is evident that tumors 10 mm and smaller represent a heterogeneous group whose treatment should be tailored to improve the risk-to-benefit ratio of systemic therapy.

The results of this study offer helpful information about which women may benefit from chemotherapy after surgery for very small, hormone-receptor-positive, HER2-positive breast cancer that hasnt spread to the lymph nodes.

Still, the results are based on extremely small differences in cancer size millimeters and require expert pathology review of the cancer. The researchers also didnt know the exact chemotherapy regimens the women had.

Also, anti-HER2 medicines, such as Herceptin, are often given along with chemotherapy. In this study, for 2 years of the 5-year study, anti-HER2 therapy and chemotherapy were not coded differently. So, a number of women may have been receiving both anti-HER2 therapy and chemotherapy during that time, which may have affected the study results.

If youve been diagnosed with a very small, hormone-receptor-positive, HER2-positive breast cancer that hasnt spread to the lymph nodes and are considering treatments after surgery, it makes sense to talk about this study with your doctor. The results offer more information about outcomes and can help you decide on the best treatments for your unique situation.

For more information on chemotherapy, including types of medicines and side effects, visit the Breastcancer.org Chemotherapy pages.

Written by: Jamie DePolo, senior editor

Published on May 7, 2020 at 12:02 PM

Excerpt from:
Chemotherapy for Certain Small, Hormone-Receptor-Positive, HER2-Positive Breast Cancers Seems to Improve Survival - Breastcancer.org

Recommendation and review posted by Bethany Smith

Updated: May 8, 2020 3:18:58 pm

By Dr Uma Vaidyanathan

Thalassaemia is a disorder of the haemoglobin structure. A tetramer molecule present in the RBCs, haemoglobin is responsible for supplying oxygen in the body and is a rich source of protein in the blood. The human Hb/HbA has two sets of globin chains. These are a-globin chain and -globin chain. Four genes (two inherited from the mother and two from the father) regulate the production of a-globin chain, while only two genes (each inherited from father and mother) control the production of -globin chain.

The thalassaemia syndromes are characterised by a basic defect in the synthesis of one type of globin chains. As a result, there is insufficient Hb content in the resultant red cells, leading to decreased haemoglobin concentration, anaemia and need for multiple blood transfusions in severe cases.

Each year, more than 70,000 babies are born with thalassaemia worldwide and this defect is very often seen in the Indian subcontinent. Haemoglobin electrophoresis is the gold standard in diagnosing this condition. Patients with thalassemia traits do not require long-term monitoring. They usually do not have iron deficiency, so iron supplements are unlikely to improve their anemia.

Read| Pregnant during the COVID-19 crisis? Heres how to take care of mental health

Alpha thalassaemia

In alpha thalassaemia, the hemoglobin does not produce enough alpha protein. To make alpha-globin protein chains we need four genes, two on each chromosome 16. We get two from each parent. If one or more of these genes is missing, it will result in alpha thalassemia. The severity of thalassemia depends on how many genes are faulty, or mutated.

One faulty gene: The patient has no symptoms. A healthy person who has a child with symptoms of thalassemia is a carrier. This type is known as alpha thalassemia minima.

Read| Covid-19 and pregnancy: What expectant mothers need to know

Two faulty genes: The patient has mild anemia. It is known as alpha thalassemia minor.

Three faulty genes: The patient has hemoglobin H disease. This is a type of chronic anemia. In this case, the patient needs regular blood transfusions throughout their life.

Four faulty genes: Alpha thalassaemia major is the most severe form of alpha thalassemia. It is known to cause hydrops fetalis, a serious condition in which fluid accumulates in parts of the fetus body. A fetus with four mutated genes cannot produce normal hemoglobin and is unlikely to survive, even with blood transfusions.

Beta Thalassemia

Two globin genes are required to make beta-globin chains: one gene from each parent. Beta thalassaemia is caused when one or both genes are faulty.

Severity depends on how many genes are mutated.

One faulty gene: This is called beta thalassaemia minor.

Two faulty genes: There may be moderate or severe symptoms. This is known as thalassaemia major (earlier known as Colleys anemia).

Medical management of thalassaemia major depending on severity would be:

Blood transfusions

Chelation therapy removal of excess iron overload in body

Bone marrow transplant

Gene therapy

Management during Pregnancy can be divided as follows:

Periconceptional care

Screening and counseling pre-pregnancy: Screening can identify couples having 25 per cent risk or more of having a pregnancy with signi?cant haemoglobinopathy. If a pregnant woman is found to be a carrier of haemoglobinopathy, the partner needs to be screened as soon as possible. If a risk of the fetus having major haemoglobinopathy is detected, urgent expert counselling is provided to the couple so that they can make an informed choice regarding the prenatal diagnosis and the possible termination of pregnancy.

Folic acid supplementation: Beginning in the preconceptual period, at least three months prior to conception, folic acid in the dosage of 5 mg/day helps in preventing neural tube defects.

Some additional tests also might be needed. Doctor might ask the patient to take blood sugar and thyroid function tests. Patients could be asked for cardiovascular assessment. Ultrasound of liver and the gallbladder (and spleen, if present) should be performed. This helps in detecting gallstones and evidence of liver cirrhosis due to iron overload or transfusion-related viral hepatitis.

Antenatal care

Women with thalassaemia should be reviewed on a monthly basis until 28 weeks of gestation and fortnightly thereafter. Women with thalassaemia are best treated in a multidisciplinary team setting, including an obstetrician with expertise in managing high-risk pregnancies and a hematologist.

Ultrasound scanning

An early scan after 7 to 9 weeks of gestation is needed to determine viability as well as the presence of a multiple pregnancy. A detailed anomaly scan must also be done after 11-12 weeks and 18 to 20 weeks of gestation. Later, ultrasounds may be needed to assess foetal growth restriction.

Care during labour and delivery

Thalassaemia is not an indication for cesarean section. Patients may need blood transfusion in case of excessive blood loss during the delivery.

Postpartum care

Women with thalassemia are at high risk for venous thromboembolism due to the presence of abnormal red blood cells in the circulation.

Breastfeeding is safe and should be encouraged. In addition, there is no contraindication to the use of hormonal methods of contraception, such as the combined oral contraceptive pill, the progestogen-only pill, hormonal implants, and the Mirena intrauterine system in women with thalassaemia.

(The writer is Senior Consultant, Obstetrics and Gynecology Fortis Hospital, Shalimar Bagh.)

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Read more here:
World Thalassaemia Day: What does the condition mean for pregnancy? - The Indian Express

Recommendation and review posted by Bethany Smith

According to the World Health Organization (WHO), cardiovascular disease, specifically ischemic heart disease, is one of the leading causes of death worldwide. Cardiovascular diseases result in an estimated 17.9 million deaths each year. This is about 31% of all deaths worldwide (1). Medical researchers are continually working on ways to reduce those numbers, including the development of new technologies to combat premature deaths from cardiovascular diseases. This article will focus, in particular, on the value of induced pluripotent stem cells (iPSCs) in cardiac research.

iPSCs are a type of pluripotent stem cell. These are master cells that can differentiate into any cell or tissue the body needs. They are generated directly from somatic cells through ectopic expression of various transcription factors, such as

Theyve become key tools to model biological processes, particularly in cell types that are difficult to access from living donors. Many research laboratories are working to enhance reprogramming efficiency by testing different cocktails of transcription factors.

iPSCs have become essential in a number of different research fields, including cardiac research.

They are a valuable and advantageous technologic development for two main reasons:

Most people have heard of embryonic stem cells, which are one variation of pluripotent cells. Like iPSCs, they can be used to replace or restore tissues that have been damaged.

The problem is that embryonic stem cells are only found in preimplantation stage embryos (3). Whereas iPSCs are adult cells that have been genetically modified to work like embryonic stem cells. Thus, the term, inducedpluripotent stem cells.

The development of iPSCs was helpful because embryos are not needed. This reduces the controversy surrounding the creation and use of stem cells. Further, iPSCs from human donors are also more compatible with patients than animal iPSCs, making them even closer to their embryonic cousins.

The Japanese inventor of iPSCs, Professor Shinya Yamanaka earned a Nobel Prize in 2012 for the discovery that mature cells can be reprogrammed to become pluripotent. (4) The Prize was awarded to Dr. Yamanaka because of the significant medical and research implications this technology holds.

iPSCs hold a lot of promise for transplantation medicine. Further, they are highly useful in drug development and modeling of diseases.

iPSCs may become important in transplantation medicine because the tissues developed from them are a nearly identical match to the cell donors. This can potentially reduce the chances of rejection by the immune system (5).

In the future, and with enough research, it is highly possible that researchers may be able to perfect the iPSC technology so that it can efficiently reprogram cells and repair damaged tissues throughout the body.

iPSCs forgo the need for embryos and can be made to match specific patients. This makes them extremely useful in both research and medicine.

Every individual with damaged or diseased tissues could have their own pluripotent stem cells created to replace or repair them. Of course, more research is needed before that becomes a reality. To date, the use of iPSCs in therapeutic transplants has been very limited.

One of the most significant areas where iPSCs are currently being used is in cardiac research. With appropriate nutrients and inducers, iPSC can be programmed to differentiate into any cell type of the body, including cardiomyocyte. This heart-specific cell can then serve as a great model for therapeutic drug screening or assay development.

Another notable application of iPSCs in cardiac research is optical mapping technology. Optical mapping technology employs high-speed cameras and fluorescence microscopy to examines the etiology and therapy of cardiac arrhythmias in a patient-like environment. This is typically done by looking into electrical properties of multicellular cardiac preparations., e.g. action potential or calcium transient, at high spatiotemporal resolution (6).

Optical mapping technology can correctly record or acquire data from iPSCs. iPSCs are also useful in mimicking a patients cardiomyocytes with their specific behaviors, resulting in more reliable and quality data of cardiac diseases.

iPSCs are vital tools in cardiac research for the following reasons:

iPSCs are patient-specific because they are 100% genetically identical with their donors. This genomic make-up allows researchers to study patients pathology further and develop therapeutic agents for treating their cardiac diseases.

Induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), help researchers predict the cardiotoxicity of drugs like with widely used chemotherapy reagents (10). Predictions like this were close to impossible before iPSC technology entered the research game.

iPSCs really come into play with their ability to model diseases. Because iPSCs are genetic matches to their living donors, they are uniquely useful for the study of genetic cardiac diseases like monogenic disorders. iPSCs help researchers understand how disease genotypes at the genetic level manifest as phenotypes at the cellular level (5).

Long QT syndrome, a condition that affects the repolarization of a patients heart after a heartbeat, is a notable example of iPSC-based disease modeling (7). This syndrome has been successfully modeled using iPSCs and is an excellent model for other promising target diseases (7).

Long QT syndrome is not the only disease that has been modeled by iPSCs. Other cardiac diseases like Barth syndrome-associated cardiomyopathy and drug-induced kidney glomerular injuries have been modeled as well (8).

The advent of iPSC technology has created a wealth of new opportunities and applications in cardiovascular research and treatments. In the near future, researchers hope that iPSC-derived therapies will be an option for thousands, if not millions of patients worldwide.

More from this author: The Promising Future of Nanomedicine and Nanoparticles

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What is the Value of iPSC Technology in Cardiac... - The Doctor Weighs In

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The U.S. Food and Drug Administration (FDA) announced on Tuesday that it has approved dapagliflozin, also known under the brand name Farxiga, for the treatment of heart failure in adults with reduced ejection fraction. The drug can potentially reduce the risk of cardiovascular death and hospitalization for heart failure.

AstraZenecas Farxiga is now the first in its drug class of sodium-glucose co-transporter 2 (SGLT2) inhibitors to be approved to treat adults with the New York Heart Associations functional class II-IV heart failure with reduced ejection fraction. AstraZeneca was granted with the approval of Farxiga related to heart failure by the FDA.

In a clinical trial, Farxiga appeared to improve survival and reduce the need for hospitalization in adults with heart failure and reduced ejection fraction.

To determine the efficacy of the drug, researchers looked at the number of instances of cardiovascular death, hospitalization for heart failure and urgent heart failure visits. Some trial participants were given a once-daily dose of 10mg of Farxiga, while others were given a placebo. After approximately 18 months, those who were given Farxiga had fewer cardiovascular deaths, hospitalizations for heart failure and urgent heart failure visits compared to their counterparts.

Heart failure is a serious health condition that contributes to one in eight deaths in the U.S. and impacts nearly 6.5 million Americans, said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDAs Center for Drug Evaluation and Research. This approval provides patients with heart failure with reduced ejection fraction an additional treatment option that can improve survival and reduce the need for hospitalization.

Farxiga can cause side effects including dehydration, urinary tract infections and genetical yeast infections. It can also potentially result in serious cases of necrotizing fasciitis of the perineum in people with diabetes and low blood sugar when combined with insulin.

On Tuesday, BioCardia, Inc. also announced positive preclinical data supporting its new drug application for anti-inflammatory cell therapy for heart failure. BioCardias allogenic neurokinin 1 receptor positive mesenchymal stem cell (NK1R+ MSC) therapy appeared to improve heart function in a study. NK1R+ MSC is being marketed under the name CardiALLO.

Researchers looked at 26 animals treated with both low dose and high dose CardiALLO in their study. Echocardiographic measures of cardiac ejection fraction, fractional shortening and cardiac outflow all notably improved in the animals.

In light of these positive data on our allogenic NK1R+ MSC therapy, we expect to meet our internal timeline to complete our submission to the FDA for our first indication for CardiALLO, and potentially receive IND acceptance by the end of the second quarter, said BioCardia Chief Scientific Officer Ian McNiece, PhD. The MSCs that were studied are subtypes of MSC that we have delivered previously in our co-sponsored trials, which we believe have enhanced potency over MSC generated from unselected bone marrow cells. We look forward to seeing additional data from this animal study that are currently being analyzed, including histology and pathology of the heart and lungs.

BioCardia also intends to submit an IND for the use of NK1R+ MSC delivered via intravenous infusion for the treatment of Acute Respiratory Distress Syndrome caused by COVID-19.

Approximately 6.5 million adults in the U.S. are living with heart failure, according to the Centers for Disease Control and Protection. In 2017, it was a contributing cause of death in one out of eight people.

Read the original:
FDA Approves AstraZeneca's Farxiga for Heart Failure in Adults with Reduced Ejection Fraction - BioSpace

Recommendation and review posted by Bethany Smith

(MENAFN - iCrowdNewsWire) May 8, 2020

According to the new market research report " Cell Therapy Technologies Market by Product (Consumables, Equipment, Software), Cell Type (Human Stem & Differentiated, Animal), Process Stages (Cell Processing, Distribution, Handling, QC), End User, and Region - Global Forecast to 2023, , published by MarketsandMarkets, The global cell therapy technologies market is projected to reach USD 19.9 billion by 2023 from USD 10.2 billion in 2018, at a CAGR of 14.4% during the forecast period.

Browse in-depth TOC on 'Cell Therapy Technologies Market" 75 - Table30 Figures116 Pages

Download PDF Brochure: https://www.marketsandmarkets.com/pdfdownloadNew.asp?id=213334978

Rising government investments for cell-based research, the increasing number of GMP-certified production facilities, and the large number of oncology-oriented cell-based therapy clinical trials are the key factors driving the growth of this market. China, India, Japan, Korea, and Brazil are emerging markets for cell therapy instruments. These markets boast comparatively lenient standards and government regulations as opposed to developed markets in North America and the EU, and thus offer significant growth potential for providers. However, the high cost of cell-based research and the low success rate is expected to restrain market growth to some extent during the forecast period.

Consumables are expected to account for the largest cell therapy technologies market share in 2018 : By product, the cell therapy technologies market is segmented into consumables, equipment, and systems & software. The consumables segment is expected to account for the largest share of the market in 2018. Factors such as increasing investments by companies to develop advanced products as well as government initiatives for enhancing cell-based research are contributing to the growth of the cell therapy consumables market.

Cell processing segment to witness the highest growth during the forecast period :

Based on process, the cell therapy technologies market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control. The cell processing segment is expected to account for the largest market share in 2018 and is projected to witness the highest CAGR during the forecasted period.

Human cells segment accounts for the large share of the cell therapy instruments market, by cell type :

Based on cell type, the market is segmented into human cells and animal cells. In 2018, the human cells segment is expected to account for the largest share of the cell therapy technologies market. The rising adoption of human cells over animal cells for cell therapeutics research, technological advancements, and the rising incidence of diseases such as cancer and cardiac abnormalities are the key factors driving the growth of this segment.

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North America to dominate the cell therapy technologies market during the forecast period : The market is segmented into four major regions, namely, North America, Europe, Asia Pacific, and the Rest of the World (RoW). North America is expected to dominate the market in 2018 owing to the high burden of chronic diseases and increasing R & D activities in the pharmaceutical and biotechnology industries. The Asia Pacific region is expected to register the highest CAGR during the forecast period.

The major players in the western blotting market are Beckman Coulter (US), Becton, Dickinson and Company (US), GE Healthcare (US), Lonza (Switzerland), Merck KGaA (Germany), Miltenyi Biotec (Germany), STEMCELL Technologies, Inc. (Canada), Terumo BCT (US), and Thermo Fisher Scientific (US).

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Cell Therapy Technologies Market to Receive Overwhelming Hike in Revenues by 2023 - MENAFN.COM

Recommendation and review posted by Bethany Smith

Pratteln, Switzerland, May06, 2020 Santhera Pharmaceuticals (SIX: SANN) announces the signing of two agreements with Rutgers, The State University of New Jersey as part of its program to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2MD or MDC1A). Under the agreements, Santhera gains rights to intellectual property developed at Rutgers on certain gene constructs that will be further studied under a collaboration agreement.

Santhera has entered into a license agreement with Rutgers, The State University of New Jersey and a collaboration with Prof. Peter Yurchenco, a pioneer in a novel gene therapy approach for the treatment of LAMA2MD. These agreements complement the ongoing collaboration of Santhera with Prof. Markus Regg from the Biozentrum of the University of Basel [1]. Previous collaborative work by Prof. Regg and Prof. Yurchenco has established the potential of this approach in animal models.

The novel gene therapy strategy developed by these leading experts uses two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen [2-5]. In animal models for LAMA2MD, this approach has led to restoration of muscle fiber basement membranes, recovery of muscle force and size, increased overall body weight and markedly prolonged survival thus demonstrating strong evidence for disease modifying potential [2].

The coordinated work of both collaborations will further advance Santheras effort to bring this innovative gene therapy approach to patients with LAMA2MD.

Gene replacement is a promising therapeutic option for the treatment of LAMA2MD, said Peter D. Yurchenco, MD, PhD, Professor at Rutgers Robert Wood Johnson Medical School, USA. We have been working on continuously optimizing linker proteins engineered from extracellular matrix proteins which will aid in advancing such gene therapy approach towards clinical use.

Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University, added Kristina Sjblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera. This will add value to our gene therapy program for LAMA2MD and complements the work already under way with the Biozentrum at the University of Basel, which was awarded a grant by Innosuisse in 2019. Both of our collaboration partners have pioneered this field and will work closely with Santhera, clinical experts and the patient community to establish the best way to bring this approach to clinical use.

About LAMA2MD (CMD Type 1A or MDC1A) and Emerging Therapy ApproachesCongenital muscular dystrophies (CMDs) are inherited neuromuscular diseases characterized by early-onset weakness and hypotonia alongside associated dystrophic findings in muscle biopsy. Progressive muscle weakness, joint contractures and respiratory insufficiency characterize most CMDs. Laminins are proteins of the extracellular matrix that help maintain muscle fiber stability by binding to other proteins. LAMA2-related muscular dystrophy (LAMA2MD, also called MDC1A), is one of the most common forms of CMD. It is caused by mutations in the LAMA2 gene encoding the alpha2 subunit of laminin-211. Most LAMA2MD patients show complete absence of laminin-alpha 2, are hypotonic (floppy) at birth, fail to ambulate, and succumb to respiratory complications.

Previous work has demonstrated that two linker proteins, engineered with domains derived from the extracellular matrix proteins agrin, laminin and nidogen, could compensate for the lack of laminin-alpha2 and restore the muscle basement membrane [2-5]. Through simultaneous expression of artificial linkers (SEAL), this gene therapy approach aims to overcome the genetic defect by substituting laminin-alpha2 deficiency with small linker proteins containing necessary binding domains to re-establish muscle fiber integrity. In a transgenic mouse model, the linker expression increased the lifespan of LAMA2-deficient mice 5-fold to a median of 81 weeks compared to 15.5 weeks in the disease model without the therapeutic linker expression [2]. Recently, it was demonstrated that such linker constructs could be applied by standard adeno-associated virus (AAV) vectors [6, 7]. First results using the AAV technology have been presented by Prof Regg [8].

References [1] Santhera press release on gene collaboration with Biozentrum Basel (May 21, 2019), accessible here

[2] Reinhard et al. (2017). Sci Transl Med 9, eaal4649[3] Moll et al. (2001). Nature 413, 302-307.

[4] Meinen et al. (2007) J. Cell Biol. 176, 979-993.[5] McKee et al. (2017) J. Clin. Invest. 127, 1075-1089.[6] Qiao et al. (2018) Mol Ther Methods Clin Dev 9, 47-56.

[7] Qiao et al. (2005) Proc. Natl. Acad. Sci. U. S. A. 102, 11999-12004.[8] Reinhard, J. et al. (2019) Neuromuscular Disorders, Volume 29, S164

About Rutgers, The State University of New JerseyRutgers, The State University of New Jersey, is a leading national research university and the state of New Jerseys preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth-oldest higher education institution in the United States. More than 71,000 students and 23,000 faculty and staff learn, work and serve the public at Rutgers University-New Brunswick, Rutgers University-Newark, Rutgers University-Camden, and Rutgers Biomedical and Health Sciences.

About Santhera Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to license vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases, as well as omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit http://www.santhera.com.

Raxone and Puldysa are trademarks of Santhera Pharmaceuticals.

For further information please contact: public-relations@santhera.com orEva Kalias, Head External CommunicationsPhone: +41 79 875 27 80eva.kalias@santhera.com

Disclaimer / Forward-looking statements This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.# # #

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Santhera Signs Agreements in Gene Therapy Research for Congenital Muscular Dystrophy with Rutgers University - GlobeNewswire

Recommendation and review posted by Bethany Smith

For Immediate Release: May 08, 2020

Today, the U.S. Food and Drug Administration approved Retevmo (selpercatinib) capsules to treat three types of tumors non-small cell lung cancer, medullary thyroid cancer and other types of thyroid cancers in patients whose tumors have an alteration (mutation or fusion) in a specific gene (RET or rearranged during transfection). Retevmo is the first therapy approved specifically for cancer patients with the RET gene alterations.

Innovations in gene-specific therapies continue to advance the practice of medicine at a rapid pace and offer options to patients who previously had few, said Richard Pazdur, M.D., director of the FDAs Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDAs Center for Drug Evaluation and Research. The FDA is committed to reviewing treatments like Retevmo that are targeted to specific subsets of patients with cancer.

Specifically, the cancers that Retevmo is approved to treat include:

Retevmo is a kinase inhibitor, meaning it blocks a type of enzyme (kinase) and helps prevent the cancer cells from growing. Before beginning treatment, the identification of a RET gene alteration must be determined using laboratory testing.

The FDA approved Retevmo on the results of a clinical trial involving patients with each of the three types of tumors. During the clinical trial, patients received 160 mg Retevmo orally twice daily until disease progression or unacceptable toxicity. The major efficacy outcome measures were overall response rate (ORR), which reflects the percentage of patients that had a certain amount of tumor shrinkage, and duration of response (DOR).

Efficacy for NSCLC was evaluated in 105 adult patients with RET fusion-positive NSCLC who were previously treated with platinum chemotherapy. The ORR for the 105 patients was 64%. For 81% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 39 patients with RET fusion-positive NSCLC who had never undergone treatment. The ORR for these patients was 84%. For 58% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for MTC in adults and pediatric patients was evaluated in those 12 years of age and older with RET-mutant MTC. The study enrolled 143 patients with advanced or metastatic RET-mutant MTC who had been previously treated with cabozantinib, vandetanib or both (types of chemotherapy), and patients with advanced or metastatic RET-mutant MTC who had not received prior treatment with cabozantinib or vandetanib. The ORR for the 55 previously treated patients was 69%. For 76% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in 88 patients who had not been previously treated with an approved therapy for MTC. The ORR for these patients was 73%. For 61% of patients who had a response to the treatment, their response lasted at least six months.

Efficacy for RET fusion-positive thyroid cancer was evaluated in adults and pediatric patients 12 years of age and older. The study enrolled 19 patients with RET fusion-positive thyroid cancer who were radioactive iodine-refractory (RAI, if an appropriate treatment option) and had received another prior systemic treatment, and eight patients with RET fusion-positive thyroid cancer who were RAI-refractory and had not received any additional therapy. The ORR for the 19 previously treated patients was 79%. For 87% of patients who had a response to the treatment, their response lasted at least six months. Efficacy was also evaluated in eight patients who had not received therapy other than RAI. The ORR for these patients was 100%. For 75% of patients who had a response to the treatment, their response lasted at least six months.

The most common side effects with Retevmo were increased aspartate aminotransferase (AST) and alanine aminotransferase (ALT) enzymes in the liver, increased blood sugar, decreased white blood cell count, decreased albumin in the blood, decreased calcium in the blood, dry mouth, diarrhea, increased creatinine (which can measure kidney function), increased alkaline phosphatase (an enzyme found in the liver and bones), hypertension, fatigue, swelling in the body or limbs, low blood platelet count, increased cholesterol, rash, constipation and decreased sodium in the blood.

Retevmo can cause serious side effects including hepatotoxicity (liver damage or injury), elevated blood pressure, QT prolongation (the heart muscle takes longer than normal to recharge between beats), bleeding and allergic reactions. If a patient experiences hepatotoxicity, Retevmo should be withheld, dose reduced or permanently discontinued. Patients undergoing surgery should tell their doctor as drugs similar to Retevmo have caused problems with wound healing.

Retevmo may cause harm to a developing fetus or a newborn baby. Health care professionals should advise pregnant women of this risk and should advise both females of reproductive potential and males patients with female partners of reproductive potential to use effective contraception during treatment with Retevmo and for one week after the last dose. Additionally, women should not breastfeed while on Retevmo.

Retevmo was approved under the Accelerated Approval pathway, which provides for the approval of drugs that treat serious or life-threatening diseases and generally provide a meaningful advantage over existing treatments. The FDA also granted this application Priority Review and Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Additionally, Retevmo received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Retevmo to Loxo Oncology, Inc., a subsidiary of Eli Lilly and Company.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Approves First Therapy for Patients with Lung and Thyroid Cancers with a Certain Genetic Mutation or Fusion - FDA.gov

Recommendation and review posted by Bethany Smith

It works like a very fine "molecular knob" able to modulate the electrical activity of the neurons of our cerebral cortex, crucial to the functioning of our brain. Its name is Foxg1, it is a gene, and its unprecedented role is the protagonist of the discovery just published on the journal Cerebral Cortex. Foxg1 was already known for being a "master gene" able to coordinate the action of hundreds of other genes necessary for the development of our anterior central nervous system. As this new study reports, the "excitability" of neurons, namely their ability to respond to stimuli, communicating between each other and carrying out all their tasks, also depends on this gene. To discover this, the researchers developed and studied animal and cellular models in which Foxg1 has an artificially altered activity: a lack of activity, as it happens in patients affected by a rare variant of Rett Syndrome, which leads to clinical manifestations of the autistic realm; or an excessive action, as in a specific variant of the West Syndrome, with neurological symptoms such as serious epilepsy and severe cognitive impairment. As deduced by the scientists in the research, the flaw in the "knob" lies in an altered electrical activity in the brain with important consequences for the entire system, similar to what happens in the two syndromes mentioned.

Shedding light on this mechanism, say the researchers, allows to understand more deeply the functioning of our central nervous system in sickness and in health, a fundamental step to assess possible future therapeutic interventions for these pathologies. What has just been published is the latest in a series of three studies on the Foxg1 gene, recently published by the researchers of SISSA on Cerebral Cortex. It is the result of a project begun more than five years ago, which saw the team of Professor Antonello Mallamaci of SISSA in the front line with researchers of the University of Trento and the Neuroscience Institute of Pisa, with the support of the Telethon Foundation, of the Fondation Jerome Lejeune and of the FOXG1 Research Foundation.

The many abilities of the "master gene"

"We knew that this gene is important for the development of the anterior central nervous system" explains the Professor Antonello Mallamaci of SISSA, who has coordinated the research. "In previous studies we had already highlighted how it was involved in the development of particular brain cells, the astrocytes, as well as the neuronal dendrites, which are part of the nerve cells that transport the incoming electrical signal to the cell. The fact that it had mutated in patients affected by specific variants of the Rett and West Syndromes in which we see, respectively, an insufficient and excessive activity of this gene, made us explore the possibility that its role was also another. And, from what has emerged, it would appear that way".

The research findings

According to the study, the activation of the electrical activity of Foxg1 follows a positive circuit. Professor Mallamaci explains: "If the gene is very active there is increased electrical activity in the cerebral cortex. In addition, the neurons, when active, tend to make it work even harder. One process, in short, feeds the other. Obviously, in normal conditions, the system is slowed down at a certain point. "If, however, the gene functions abnormally, or it is found in a number of copies other than two, as it happens in the two syndromes above, the point of balance changes and the electrical activity is altered. All this, in addition to making us understand the mechanisms of the pathology, tells us that Foxg1 functions precisely as a key regulator of the electrical activity in the cerebral cortex".

The next step, explains the professor, will be to understand the role of the mediating genes, namely of some of the many genes whose action is regulated by the master gene Foxg1. This analysis is important to understand in more detail how this gene works under normal and pathological conditions.

How the master gene produces the pathological effects, when and how to intervene

Understanding the molecular mechanisms that Foxg1 controls is also important to study what could be the targets on which to intervene for possible therapeutic approaches. "Given that finding a therapy for these illnesses is very difficult, working so in depth you might find, for example, that most problems are caused precisely by some of the "operators" that Foxg1 regulates. And that we should therefore focus our attention on these goals, rather than on the master gene, maybe using drugs that already exist and have been seen to be useful in remedying those specific flaws". In the case of a future approach that would instead correct the anomalies of the FOXG1 gene with the gene therapy, explains Professor Mallamaci, "it is necessary to understand when to intervene, namely from what moment on the pathological effects due to the mutation of this gene become irreversible. To replace the flawed copy with the correct one, it is necessary to intervene before that moment, which might suppose you would have to make a prenatal gene diagnosis and treatment". "The next steps we will take", concludes Professor Mallamaci "will be directed precisely in the direction of a deeper understanding of all these aspects".

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Like a molecular knob: That is how a gene controls the electrical activity of the brain - Science Codex

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This is an excerpt fromSecond Opinion, aweeklyroundup of eclectic and under-the-radar health and medical science news emailed to subscribers every Saturday morning.If you haven't subscribed yet, you can do that byclicking here.

As researchers around the world race under immense pressure to develop a COVID-19 vaccine, a controversial approach could potentially help get them there faster but it's incredibly risky.

The process is known as a human challenge study, and it involves intentionally infecting willing volunteers with the coronavirus that causes COVID-19 in order to test the effectiveness of potential vaccines and treatments against it.

"These are very powerful studies that could make a difference, especially since we don't know a lot about the novel coronavirus," said Seema Shah, a medical ethicist at Northwestern University and Lurie Children's Hospital of Chicago.

"They could clarify what's happening in infections with people who are not symptomatic and people who have more severe disease even if they don't have underlying conditions that put them at higher risk."

Shah said COVID-19 human challenge studies could also help identify people who have developed immunity to the coronavirus, while also helping to narrow down the growing list of potential vaccines and treatments for patients.

"If there were a couple of vaccine candidates that had gotten through safety testing and there was a question about which one of those vaccines was more likely to work, a human challenge study could be a quick way to pick the best vaccine of the candidates," she said.

"It could also be useful to study whether the vaccine itself causes different kinds of harm."

The WHOsays any potential vaccine is still at least a year away, but human challenge trials could accelerate theprocess because of the time they save in the clinical trial phase.

Typically, researchers inject thousands of study participants with a vaccine or placebo and wait for symptoms to develop an approach that can take months or years after vaccine development.

Human challenge studies instead vaccinatea small group of people and then intentionally infectthem with the virus, saving critical research time, especially during a global pandemic.

But despite the potential benefits of a controlled human challenge study on COVID-19 patients, experts say the controversial approach is an ethical minefield that could have disastrous consequences if not handled carefully.

Human challenge studies typically recruit young, healthy volunteers in an effort to keep the risk of severe medical complications low.

Thousands of potential volunteers have already pledged to participate in human challenge trials on a website called 1DaySooner, but no such studies are yet underway.

Yet given what we know and don't know about the different ways in which COVID-19 attacks the human body even in young, healthy people, how do we effectively inform participants who may be unknowingly putting themselves at severe risk?

"We know that younger people tend to tolerate COVID-19 as an illness better, but what would worry me about that is there's a lot that is still unknown," said Kerry Bowman, a bioethicist and professor of global health at the University of Toronto.

He said the potential for COVID-19 patients of all ages to face long-term health implications and even death from a virus we still know so little aboutcalls into question how truly informed participants could be on the risks of a human challenge study.

"Do you truly have an informed decision?" he said. "You have consent, but is itreally well-informed? Do people fully understand? Because if we don't understand the virus itself, I wonder about the quality of informed consent that you can ask of people."

In a new paper published in the journal Science Thursday, Shah and a team of international researchers outline an ethical framework for how human challenge studies could be effectively used to combat COVID-19.

The researchers supportdeveloping a "challenge strain" of the coronavirus a stabilizedversion of the one thatis circulating worldwide to potentially infect participants, but stopped short of advocating for the work to proceed.

"The pandemic has affected just about everyone in the world in various ways, so the potential amount of social value is unprecedented here," Shah said.

"That's why our group concluded it's really important to give challenge studies a hard look and potentially invest in laying the groundwork for doing them.

"But then make that judgment call about whether and how to do them at a later date when they're ready to go."

The World Health Organization released specific criteria this week outlining its recommended approach to conducting human challenge studies, without advocating for or against them.

Among those recommendations is a need for "strong scientific justification" for the studies, ensuring that the potential benefits outweigh risksand that the selection of participants should be done with "rigorous" informed consent.

"The overarching, really important one is to minimize risk to participants as much as possible," said bioethicist Dr. Ross Upshur, of the University of Toronto's Dalla Lana School of Public Health, who helped work on the WHO guidelines.

"You need to make sure that people understand what's being proposed:what they're going to be doing;how they're going to be managed in this situation;how their safety and their well-being is going to be protected.

"But it's also incumbent on the researchers to outline all of the uncertainties,because we may not be able to actually quantify some of those risks."

Timothy Caulfield, a Canada Research Chair in health law and policy at the University of Alberta who has researched human challenge studies, said those ethical dilemmas have historically plagued this approach.

"I understand the desire to use human challenge trials, especially in this context, because people are desperate to get a vaccine quickly, not just for clinical reasons, but also for economic and social reasons," he said.

"So the pressure is intense, but the exact reason that we have research ethics guidelines is to protect research participants."

Caulfield said the damage that could be done if a human challenge trial were to go awry would be devastating.

He points to the ethics scandal involvingJesse Gelsinger, a teenager who died in a clinical trial for gene therapy in 1999, as an example of a failed human study that set the research ethics field back immensely.

Gelsingerhad a genetic disease called ornithine transcarbamylase (OTC) that he controlled through diet and medication, but after enrolling in the trialhe was injected with an experimental therapy and died a few days later.

"Just imagine the impact that it could have on vaccine research, especially in this environment where there's so much uncertainty," Caulfieldsaid.

"If it doesn't go well and if we cut corners on research ethics standards, it could end up backfiring and being really problematic."

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Could intentionally infecting volunteers with COVID-19 help find a cure sooner? - CBC.ca

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Global Gene Therapy for Inherited Genetic Disorder Market statistical report provides a wide-ranging research on the key players and in-depth insights which includes the competitiveness of the trending players. This Market research report that evaluates its current value, size, performance and statistics. The report is an important dynamic of the market and gives an idea of the types, the process, and value chain that has been included in the report.

The geographical sector of the global Gene Therapy for Inherited Genetic Disorder Market comprises the leading regions in the market during the forecast tenure. The competitive landscape section of the statistical report presents information on major key players in the global market. On the basis of product profile, introductions, SWOT analysis and contact information, these key players are selected.

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BioMarin Pharmaceutical Inc., bluebird bio Inc., Novartis AG, Orchard Therapeutics Plc, and Spark Therapeutics.

Key questions answered in this report:

Geographically, regions such as North America, Europe, Asia-Pacific (APAC), Middle East & Africa and Latin America can be segmented on the basis of the global Gene Therapy for Inherited Genetic Disorder Market. Market drivers, restraints, and opportunities have been evaluated to explain the anticipated nature of investments and its impact on the global market in terms of future prospects. The report provides qualitative as well as quantitative researched data of the global Market.

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In the last sections of the report, the recent innovations along with its impact on innovative growth that is expected to be introduced by the significant players form a core part of the report. This study is done by considering the demographics, regional trends, product demand evaluation in a forward-looking perspective on different factors driving or restraining market growth.

Table of Contents

Global Gene Therapy for Inherited Genetic Disorder Market Research Report

Chapter 1 Gene Therapy for Inherited Genetic Disorder Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

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How COVID-19 Impacting On Gene Therapy for Inherited Genetic Disorder Market Trends, Drivers, Strategies, Segmentation Application with Top Key...

Recommendation and review posted by Bethany Smith

BURLINGTON, Mass., May 05, 2020 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) will present positive data from two studies of FX201, an investigational gene therapy for osteoarthritis (OA), at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place virtually May 12-15, 2020. The abstracts were published in the May supplement of Molecular Therapy.

The data from the rodent model served as a basis for the FX201 IND and reinforce our belief that FX201 holds the potential to become a transformative therapy for OA, saidMichael Clayman, M.D., President and Chief Executive Officer of Flexion. Further, we are delighted to be presenting manufacturing studies at ASGCT which demonstrate a capable Good Manufacturing Process (GMP) to support the production of FX201 for our clinical trials.

Establishing the Efficacy, Safety, and Biodistribution of FX201, a Helper-Dependent Adenoviral Gene Therapy for the Treatment of Osteoarthritis, in a Rat Model (Abstract 747)

Flexion assessed the efficacy of HDAd-rat-IL-1Ra, the rat surrogate of FX201, in a rodent surgical model of OA. Efficacy was assessed after a single intra-articular administration one week post-surgery via histopathology at Week 12 using a semi-quantitative microscopic grading system (Osteoarthritis Research Society International [OARSI] score) for OA-related cartilage, synovium, and bone changes. In addition, two Good Laboratory Practice (GLP) studies were performed in a rodent model of OA to evaluate the safety and biodistribution of FX201 and the rat surrogate (a helper-dependent adenovirus vector with a transgene encoding rat variant of IL-1Ra) administered four weeks post-surgery. Key study findings include:

Development of a Highly Productive and Reproducible Manufacturing Process for FX201, a Novel Helper-Dependent Adenovirus-Based Gene Therapy for Osteoarthritis (Abstract 1273)

Using a fit-for-purpose manufacturing process suitable for early development, Flexion successfully produced four batches of drug substance, which will enable GLP toxicology, pharmacology, and GMP clinical studies. Key findings include:

About FX201FX201 is a locally administered gene therapy product candidate which utilizes a helper-dependent adenovirus (HDAd) vector, designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Inflammation is a known cause of pain, and chronic inflammation is thought to play a major role in the progression of osteoarthritis (OA). By persistently suppressing inflammation, Flexion believes FX201 holds the potential to both reduce OA pain and modify the disease.

About Osteoarthritis (OA) of the KneeOA, also known as degenerative joint disease, affects more than 30 million Americans and accounts for more than $185 billion in annual expenditures. In 2017, approximately 15 million Americans were diagnosed with OA of the knee and the average age of physician-diagnosed knee OA has fallen by 16 years, from 72 in the 1990s to 56 in the 2010s. The prevalence of OA is expected to continue to increase as a result of aging, obesity and sports injuries. Each year, approximately five million OA patients receive either a corticosteroid (immediate-release or extended-release) or hyaluronic acid intra-articular injection to manage their knee pain.

About Flexion TherapeuticsFlexion Therapeutics(Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form of arthritis. The company's core values are focus, ingenuity, tenacity, transparency and fun. Visitflexiontherapeutics.com.

Forward-Looking Statements This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; expected increases in the rate of individuals with OA of the knee; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, risks related to clinical trials, including potential delays, safety issues or negative results; risks related to key employees, markets, economic conditions, and health care reform; and other risks and uncertainties described in our filings with theSecurities and Exchange Commission(SEC), including under the heading "Risk Factors" in our Annual Report on Form 10-Kfiled with theSEConMarch 12, 2020and subsequent filings with theSEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contacts:

Scott YoungVice President, Corporate Communications & Investor RelationsFlexion Therapeutics, Inc.T: 781-305-7194syoung@flexiontherapeutics.com

Julie DownsAssociate Director, Corporate Communications & Investor Relations Flexion Therapeutics, Inc.T: 781-305-7137jdowns@flexiontherapeutics.com

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Flexion Therapeutics Announces Virtual Poster Presentations for FX201, an Intra-Articular Gene Therapy Candidate for the Treatment of Osteoarthritis,...

Recommendation and review posted by Bethany Smith

SDMR International recently published a report on the Global Viral Vectors and Plasmid DNA Manufacturing Market, which offers an in-depth overview of the factors that are impacting the state and progress of the worldwide business. The research report incorporates all the vital data that will help you updated with the latest market trends and get ahead in the competition in the various market segments and the leading geographies studied in the report. The study gives valuable insights into the future progress of the market and all essential aspects of the Viral Vectors and Plasmid DNA Manufacturing market for the forecast period from 2020 to 2026.

The Viral Vectors and Plasmid DNA Manufacturing Market research report gives the latest market information, highlighting the product range and services offered in the market. The report provides key facts and figures relating to the market status, size, share, and growth aspects of the Viral Vectors and Plasmid DNA Manufacturing industry. The research comprises of elaborate profiles of the leading companies operating in the global sector by taking into account their gross revenue, total sales, market share, and competitive landscape.

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Scope of the Report:The all-encompassing research weighs up on various aspects including but not limited to important industry definition, product applications, and product types. The pro-active approach towards analysis of investment feasibility, significant return on investment, supply chain management, import and export status, consumption volume and end-use offers more value to the overall statistics on the Industrial Antifungal Agents market. All factors that help business owners identify the next leg for growth are presented through self-explanatory resources such as charts, tables, and graphic images.

Prominent players profiled in the study:BioRelianceCobra BiologicsOxford BioMedicaUniQureFinVectorMolMedMassBiologicsRichter-HelmFUJIFILM Diosynth BiotechnologiesLonzaAldevronEurogentec

Market Synopsis:

The report includes details about important products, revenue, production, and the business of top industry players. The report helps the companies to understand the threats and challenges in front of the businesses. This is a well-established, and precisely formulated report acknowledges major Viral Vectors and Plasmid DNA Manufacturing industry vendors, key regions, demand & supply, applications, technology, revenue cost, and challenges. According to the report, it will be expected to gain a healthy CAGR by 2020-2026. Additionally, it focuses on manufacturing analysis including the raw materials, cost structure, process, operations, and manufacturing cost strategies. Sub-segments are the report covered along with the clear definition, sales, value, market share, volume, market competition landscape, SWOT, and development plans during the forecast period.

In market segmentation by Types of the Viral Vectors and Plasmid DNA Manufacturing , the report covers the following uses-Plasmid DNAViral Vectors

In market segmentation by applications of the Viral Vectors and Plasmid DNA Manufacturing , the report covers the following uses-CancersInherited DisordersViral InfectionsOthers

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Take a look at some of the important sections of the report

Market Overview: Readers are informed about the scope of the global Viral Vectors and Plasmid DNA Manufacturing market and different products offered therein. The section also gives a glimpse of all of the segments studied in the report with their consumption and production growth rate comparisons. In addition, it provides statistics related to market size, revenue, and production.

Production Market Share by Region: Apart from the production share of regional markets analyzed in the report, readers are informed about their gross margin, price, revenue, and production growth rate here.

Company Profiles and Key Figures:In this section, the authors of the report include the company profiling of leading players operating in the global Viral Vectors and Plasmid DNA Manufacturing market. There are various factors considered for assessing the players studied in the report: markets served, production sites, price, gross margin, revenue, production, product application, product specification, and product introduction.

Manufacturing Cost Analysis: Here, readers are provided with detailed manufacturing process analysis, industrial chain analysis, manufacturing cost structure analysis, and raw materials analysis. Under raw materials analysis, the report includes details about key suppliers of raw materials, price trend of raw materials, and important raw materials.

For further information on the Viral Vectors and Plasmid DNA Manufacturing market report, visit: https://www.supplydemandmarketresearch.com/global-viral-vectors-and-plasmid-dna-manufacturing-market-828740

Countries mapped in the study:

Argentina, Australia, Austria, Belgium, Brazil, Canada, Chile, China, Colombia, Japan, United States, Mexico, South Africa, Nigeria, Tunisia, Morocco, Germany, United Kingdom (UK), the Netherlands, Spain, Italy, Turkey, Russia, France, Poland, Israel, United Arab Emirates, Qatar, Saudi Arabia, Taiwan, South Korea, Singapore, India, and New Zealand.

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We have a strong network of high powered and experienced global consultants who have about 10+ years of experience in the specific industry to deliver quality research and analysis. Having such an experienced network, our services not only cater to the client who wants the basic reference of market numbers and related high growth areas in the demand side, but also we provide detailed and granular information using which the client can definitely plan the strategies with respect to both supply and demand side.

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Covid-19 impact on Viral Vectors and Plasmid DNA Manufacturing Market Research Report Analysis and Forecast till 2026 | Cell and Gene Therapy...

Recommendation and review posted by Bethany Smith

Shares of Orchard Therapeutics fell more than 13% in premarket trading after the company announced a new strategic plan that includes the shifting of its clinical focus, the shuttering of a proposed manufacturing facility in California and the termination of 25% of company staff.

On Thursday, London-based Orchard announced the new plan that will shift the companys clinical priorities away from some of its long-time focuses, including a stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) and other ultra-rare conditions. Instead, Orchard said it will now focus its development priorities on more common conditions that include Crohns disease, a type of dementia and some neurometabolic disorders. Among those are the development of a treatment for metachromatic leukodystrophy (MLD), a rare and life-threatening inherited metabolic disease. Another priority will be its treatment for Wiskott-Aldrich syndrome (WAS), a life-threatening immune disorder. Orchard will also continue to develop its treatments for mucopolysaccharidosis types I and IIIa.

The shifts in Orchards plans were announced a couple of months following a management change. In March, company founder and Chief Scientific Officer Bobby Gaspar took over the helm of the company from Mark Rothera, who led Orchard since 2017.

I feel privileged to lead Orchard as we embark on this new chapter, which is rooted in fulfilling the powerful possibilities for HSC gene therapies beyond ultra-rare diseases, Gaspar said in a statement. Moving forward, we are focusing on advancing therapies for high need and high-value diseases, and our work in neurometabolic disorders is a clear example of this. Were also excited to announce new research programs which we believe will demonstrate the breadth of the HSC platform approach.

Orchard said it intends to adjust its manufacturing plans to focus on the expected regulatory approvals of OTL-200, the MLD treatment and its WAS drug, OTL-103. The company hopes to obtain approval in the European Union for OTL-200 for the treatment of MLD in the second half of 2020 and launch in the first half of 2021. Orchard is also preparing a Biologics License Application for its WAS treatment in both the U.S. and EU in 2021.

Orchard said it also intends to stop its plans to build a gene therapy manufacturing facility in Fremont, Calif. That project was first announced in 2018.

The culling of one-fourth of its employees is expected to save the company approximately $125 million through the end of 2021. That will extend the companys existing cash runway into 2022, Orchard said. Cash, cash equivalents and investments as of March 31, were $263.9 million compared to $325.0 million as of Dec. 31, 2019.

Frank Thomas, Orchards chief operations officer, called the strategic shift and layoffs necessary steps for the company to achieve its objectives by matching our attention and resources to a set of core imperatives for the business.

I believe that these are necessary steps, especially in light of the current environment in which we are operating, with focused investments in areas such as commercial and manufacturing operations supporting the needs we have now without a near-term dependence on the capital markets, Thomas said in a statement.

See the rest here:
Orchard Therapeutics Cuts 25% of Staff as it Shifts Clinical Focus - BioSpace

Recommendation and review posted by Bethany Smith