Archive for the ‘Gene Therapy Research’ Category

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them or one of the couple of Americans who arrived later got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

We dont have too much time, Andre Kalil, the trials lead investigator, told Endpoints News. Everythings moving really fast.

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Biogen touts new evidence from the gene therapy company it wagered $800M on - Endpoints News

Alongside resolutions, the new year tends to come with biopharma acquisitions. The first two months of 2018 brought four deals worth $1 billion or more. The same was true in 2019, which barely got off the ground before a mammoth bid for Celgene was announced.

The early days of 2020, by contrast, have notched just one billion-dollar deal.

Analysts and consultants are mostly waving off this period of lighter activity. "We don't think a lack of deals over a couple of months is really anything notable," said Phil Nadeau of Cowen & Co. "It's probably just a bit of a dry period for no reason at all."

And yet, the slow start could seem oddly timed. It doesn't fit with the early and splashy dealmaking seen in recent years, nor does it align with some investment bank expectations that drug companies might try to ink deals sooner in the year to avoid possible disruptions from the U.S. presidential election.

But perhaps most glaring: the downswing comes as many large biotech and pharmaceutical companies are seemingly in need of M&A.

Two of the biggest biotechs, Gilead and Biogen, are under pressure to acquire because they haven't convinced investors that their experimental drugs can make up for problems on the commercial side of the business.

Among the pharmas, Sanofi, Eli Lilly and GlaxoSmithKline are trying to play catch up in the industry's hottest research area, oncology, and have shown a willingness to speed up that process through buyouts. Swiss drug giants Roche and Novartis, meanwhile, paid hefty premiums for footholds in the rapidly evolving gene therapy field.

Even Vertex and Merck & Co., which are considered to be in strong financial and competitive positions, turned to M&A several times last year to seed themselves for future growth.

"It's clear that, given the growth rates among the large companies in the industry, they have to keep filling their pipeline through acquisition, partnership or in-licensing," Nadeau said.

*Transaction value classified by acquiring company type. 2019 data through Nov. 30 of last year

Image Source: Jacob Bell / BioPharma Dive, data from EY

With buyers still on the lookout, industry followers predict 2020 will deliver a healthy level of M&A in spite of the early drought.

What they don't foresee, however, is this year being defined by a mega-deal like the last two were. Instead, bolt-on deals in the $5 billion to $10 billion range will be a "sweet spot" for large pharma acquirers, according to PwC.

Companies hold ample resources to support such deals too, as EY estimates the life sciences industry has more than $1 trillion at its disposal for deals. Notably, that's after a record-setting 2019 in which the industry spent north of $350 billion on M&A.

"There was so much moving and shaking that happened last year. And for me, I just feel like the underlying activity is still there. It just hasn't reached the surface yet," said Glenn Hunzinger, U.S. Pharmaceutical and Life Sciences Deals Leader at PwC.

If and when the activity surfaces, it's likely to target rare disease treatments, cell and gene therapy, and cancer drugs, with the latter proving particularly attractive. A recent Jefferies analysis found two-thirds of small- to mid-sized biotechs that got a cancer drug approved since 2010 were later acquired.

Many potential buyers, though, are in more comfortable financial positions now than they were even a couple years ago, and may not be as open to a less-than-perfect fit when considering a deal.

"Growth has returned to the large cap space, partially through acquisitions, and free cash flow across a number of large-cap companies has never been higher," said Kennen MacKay, an analyst at RBC Capital Markets. These companies now "have the luxury of time on their side, and they can be a little bit more selective."

That's especially true with regard to pricing, according to MacKay, who said the current lull in M&A may have something to do with a rebound in biotech stocks. The XBI, an exchange traded fund of about 130 biotechs, is up about 20% since hitting a relative low point in October. The rise also comes after multiple years in which cancer and gene therapy-focused biotechs have sold for high double-digit and sometimes triple-digit premiums.

"I think there's real reluctance to buy when small-, mid-cap stock prices are really running up," MacKay said.

Nadeau from Cowen, conversely, sees valuation as less of a barrier.

"Occasionally you do hear business development executives or former management teams talk about how valuation has gotten rich and they don't see attractive targets at a right price," he said. "But on the other hand, when you get these business development folks in private, offline, they'll admit that valuations are very flexible too. And if there's a strategic deal that a company wants to do, they can make any valuation work."

On his end, Nadeau isn't reading too much into 2020's slow M&A start. He guesses it's either due to happenstance, or because some of the deals that might have been inked this quarter were tied up early. Between October and December there were at least nine biopharma acquisitions, including five valued at more than $2 billion.

Peter Behner, global transactions leader for EY's Health Science & Wellness business, also doesn't envision the quiet period will last much longer. Whether in pharmaceuticals or other industries, companies don't just go dormant after a productive year of dealmaking, he said.

"I don't have a great explanation," Behner added. "At the same time, I don't have much doubt that the year should be a solid year."

Read the original here:
Pharmacquired: Where are all the deals? - BioPharma Dive

Princeton Feb 28, 2020 (Thomson StreetEvents) -- Edited Transcript of IVERIC bio Inc earnings conference call or presentation Thursday, February 27, 2020 at 1:00:00pm GMT

* David F. Carroll

IVERIC bio, Inc. - Senior VP, CFO & Treasurer

* Glenn P. Sblendorio

IVERIC bio, Inc. - CEO, President & Director

IVERIC bio, Inc. - VP of IR & Corporate Communications

* Kourous A. Rezaei

IVERIC bio, Inc. - Senior VP & Chief Medical Officer

Good day, and welcome to IVERIC bio Fourth Quarter and Year-End 2019 Results Conference Call. Today's conference is being recorded.

At this time, I would like to turn the conference over to Kathy Galante. Please go ahead.

Kathy Galante, IVERIC bio, Inc. - VP of IR & Corporate Communications [2]

Good morning, and welcome to IVERIC bio's conference call. Representing IVERIC bio today are Mr. Glenn Sblendorio, Chief Executive Officer and President; Dr. David Guyer, Executive Chairman; Dr. Kourous Rezaei, Chief Medical Officer; Mr. Dave Carroll, Chief Financial Officer; and Mr. Keith Westby, Chief Operating Officer.

I would like to remind you that today we will be making statements relating to IVERIC bio's future expectations regarding operational, financial and research and development matters, including statements regarding our expectations to use our previously announced clinical trial of Zimura for the treatment of geographic atrophy as a pivotal trial; our development strategy for Zimura, including our plans and expectations for our second pivotal clinical trial evaluating Zimura for the treatment of geographic atrophy; our hypothesis regarding complement inhibition as a mechanism of action for the treatment of geographic atrophy; our projected use of cash and cash balances; the timing, the progress and results of clinical trials and other research and development activities; the potential utility and development potential for our product candidates; the size of the potential market indications our product candidates are intended to treat; and the potential for our business development strategy.

These statements constitute forward-looking statements for the purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. These statements cover many events and matters that are subject to various risks that could cause actual results to differ materially from those expressed in any forward-looking statement including risks related to initiation and the progress of research and development programs and the clinical trials; availability of data from these programs; reliance on university collaborators and other third parties; establishment of manufacturing capabilities; expectations for regulatory matters; need for additional financing and negotiation and consummation of business development transactions and other risks. I refer you to our SEC filings and in particular to the Risk Factors section in our quarterly report on Form 10-Q filed on November 12, 2019, for a detailed description of the risk factors affecting our business.

In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we disclaim any obligation to do so as required by law.

I would now like to turn the call over to Glenn.

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]

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Thanks, Kathy, and good morning, everyone. As always, we appreciate you joining our call this morning. We made tremendous progress last year, as we continue to build a diversified portfolio in retinal diseases that include both therapeutics and gene therapy, setting the stage for IVERIC bio to be a leader in developing transformative therapies to treat retinal diseases.

At the end of October last year, we achieved a major milestone with positive Zimura clinical trial date in geographic atrophy or GA, secondary to age-related macular degeneration or AMD. These positive results position IVERIC bio as a late-stage clinical company, with Zimura as a potential treatment option for geographic atrophy, secondary to dry AMD, a potential multibillion dollar market, for which there are currently no FDA- or EMA-approved treatments available. I'd like to review some of the characteristics and results of this trial with you.

As reported in October 2019, individual administration of both Zimura 2-milligram and 4-milligram dose met the prespecified primary efficacy endpoint reached statistical significance in our international, multicenter, randomized, double-masked, sham-controlled clinical trial for GA secondary to dry, showing a 27% reduction in GA growth over a 12-month period in both the 2-milligram and 4-milligram treatment groups.

Based on our recent FDA guidance, we believe that this reduction in growth is not only statistically significant, but also clinically meaningful.

Zimura was well tolerated in this trial. And based on the potentially superior safety profile of Zimura observed to date, we believe that Zimura may potentially differentiate itself in the field of complement inhibitors for the treatment of GA secondary to dry AMD in elderly patients that we are seeking to treat.

Based on the prespecified screening trial design, robust masking, detailed statistical analysis with a prespecified statistical analysis plan and an independent and masked imaging analysis performed by one of the leading imaging centers in the world for retinal imaging, we believe that this clinical trial may serve as 1 of 2 pivotal trials typically required for marketing approval.

We are leveraging our deep expertise and efficient execution in retinal drug development, with the goal of bringing Zimura to patients with GA secondary to dry as soon as possible. We are in the process of initiating the second Zimura pivotal trial called ISEE2008 and are planning to enroll the first patient next month. As we initiate ISEE2008, we will seek to leverage our global network of top clinical trial sites for retinal diseases to assist with our plan to enroll approximately 400 patients. Kourous will review the design of ISEE2008 in more detail in a moment.

The results from OPH2003 Zimura pivotal trial have already been presented or planned to be presented at several prestigious retinal meetings around the world, including the Macula Society in San Diego; Angiogenesis, Exudation, and Degeneration Meeting in Miami; the Association for Research in Vision and Ophthalmology, the ARVO meeting in Baltimore; the American Society of Retinal Specialists, ASRS, that's going to be held in Seattle; the International Symposium on Ocular Pharmacology and Therapeutics in Spain; the Retina 2020: New Trends conference in Italy; and the Asia-Pacific Vitreo-retina Society meeting in China.

The safety and efficacy of Zimura is being assessed in an ongoing Phase IIb randomized, double-masked, sham-controlled screening trial in patients with autosomal recessive Stargardt disease. This is an orphan inherited retinal degeneration disease. Although autosomal recessive Stargardt disease is a monogenic retinal disease caused by genetic mutations to the ABCA4G, complement activation may play a role in this rare disease. In 2019, we completed the enrollment of 95 patients in this trial, and the trial is on track with initial top line data expected during the second half of 2020.

Although bringing Zimura to patients is our top priority, we continue to advance our gene therapy portfolio in orphan inherited retinal diseases.

Natural history studies and IND-enabling activities for IC-100, which is intended to treat with rhodopsin-Mediated adRP; and IC-200, which is intended to treat BEST1-related retinal diseases are both on track, and we expect to identify the lead minigene construct for LCA10 by midyear.

Over the past several months, we hosted 3 research and development symposium with panelists that include top retinal specialists from the U.S. and around the world, gene therapy scientists and KOLs, providing their insight, ensuring their expertise and feedback in regard to our Zimura and gene therapy programs.

We hope that these R&D symposium provided a deep understanding of our programs and want to thank all the investors and analysts who attended these meetings or listened to the corresponding webcast. These symposiums are currently posted on our website, and we'd like to invite you to participate. For those that did not participate live can listen to them online.

In November, we had the privilege of announcing Dr. Guangping Gao as our Chief Strategist, Gene Therapy at IVERIC bio. Guangping has deep gene therapy expertise with over 30 years of scientific research experience in AAV vectors and gene-based treatments.

As one of the world's leading gene therapy experts, Guangping's highly distinguished career includes his major contributions to the development of the adeno-associated virus gene delivery technology. Guangping is currently President of the American Society of Gene and Cell Therapy. He's also a Director of the Horae Gene Therapy Center, Co-Director of the Research Institute for Rare Diseases, and Professor of Microbiology and Physiological Systems at the University of Massachusetts Medical School.

Our goal is to combine Guangping's deep expertise in gene therapy and advisory capacity together with our expertise in drug development for retinal diseases to shape IVERIC bio's gene therapy strategy in the coming years.

We're also happy to welcome Dr. Abraham Scaria to the position of Chief Scientific Officer. He joined us in October. Abraham leads the company's research and preclinical gene therapy activities. His extensive experience includes positions at Genzyme, Sanofi, and most recently, Casebia Therapeutics, leading multiple ocular gene therapy programs. Extremely happy and pleased to be working with both Guangping and Abraham.

On the financial front, we continue to build our financial position and completed a successful follow-on public offering of our common stock and prefunded warrants, resulting in net proceeds for the company of approximately $42.6 million in December 2019. This transaction resulted in the company finishing 2019 with approximately $126 million in cash and cash equivalents. With the addition of this capital, we expect that we will have sufficient cash and cash equivalents to fund our operations and capital expenditures as currently planned into the beginning of 2022.

We are currently -- we are continuing to explore all options for future development and potential commercialization of Zimura, including plans for potential partnering.

We are pleased about the excitement generated by Zimura data and the progress in our gene therapy programs. We are excited about Zimura's potential to positively impact and transform the lives of the many patients with GA, who currently do not have any treatment options available to them and are potentially confronted with irreversible bilateral vision loss as well as the potential to bring our gene therapy to patients with orphan inherited retinal disease, who also do not have any treatment options available to them.

I'll now turn the call over to Kourous.

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Kourous A. Rezaei, IVERIC bio, Inc. - Senior VP & Chief Medical Officer [4]

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Thank you, Glenn, and good morning, everyone. Age-related macular degeneration is characteristically a disease of the elderly and is the leading cause of visual loss in individuals of 50 years of age or older in developed countries.

In the United States, it is estimated that approximately 11 million individuals are affected with AMD, with a reported global prevalence of approximately 170 million individuals. Because of increasing life expectancies in developed and developing countries, the elderly sector of the general population is expected to continue to increase in the coming decades. While 1 in 8 Americans was considered to be elderly in 1994, it is expected that the 1 in 5 will fall into this category by 2030.

As AMD progresses with age, it generally progresses to either the nanovascular or the atrophic dry form of AMD or the neurovascular form of the disease or wet AMD. In the dry atrophic form, the loss of photoreceptors, these are the cells that perceive light; RPE cells, these are the cells that support the photoreceptors; and associated choriocapillaries, which is the blood supply, leads to the formation of the geographic atrophy.

Currently, approximately 1.5 million individuals are reported to have geographic atrophy in the United States, with an incidence of approximately 159,000 patients per year, which is even slightly higher than what is reported for annual incidents for wet AMD.

Furthermore, development or progression of geographic atrophy over time is the common cause of vision loss in patients diagnosed with the wet form of AMD. For generally treated with anti-VEGF therapy, indicating that in many patients, regardless of whether they have the dry or wet form of AMD, the final anatomic outcome leading to loss of vision is geographic atrophy. Currently, there are no FDA- or EMA-approved treatment for GA, which leads to bilateral irreversible loss of vision in this large group of patients.

As Glenn pointed out, we are excited about the results of our first pivotal trial for Zimura in geographic atrophy, secondary to AMD, indicating that Zimura was well tolerated over 12 months and was able to slow down the growth of GA over the 12-month period in a statistically significant degree. Following these results, we immediately started a process to initiate our second pivotal trial in GA, ISEE2008, with the goal of enrolling our first patient next month. And we believe that we are on track.

ISEE2008 is an international, randomized, double-masked, sham-controlled, multicenter pivotal clinical trial evaluating the safety and efficacy of Zimura 2-milligram in patients with geographic atrophies secondary to dry AMD. As you may recall, in the first pivotal clinical trial, both Zimura 2-milligram and Zimura 4-milligram cohorts demonstrated a statistically significant reduction in the mean rate of GA growth over 12 months when compared to the corresponding sham-controlled cohorts with a similar 27% reduction. And since Zimura 2-milligram is administered as a single intravitreal injection, whereas the Zimura 4-milligram requires 2 intravitreal injections, we selected the Zimura 2-milligram dose for evaluation in the ISEE2008 clinical trial.

Our understanding from the FDA is that regarding the Zimura 2-milligram dose, for marketing approval purposes, from a safety perspective, at least 300 patients need to be treated with monthly Zimura 2-milligram or at a higher dose for a duration of at least 12 months, with a portion of these patients treated for 24 months. Therefore, we are planning to enroll approximately 400 patients in the ISEE2008 trial. These patients will be randomized 1:1 into 2 cohorts, the first cohort receiving monthly administration of Zimura 2-milligram for 12 months and a second cohort receiving monthly administration of sham.

The prespecified primary efficacy endpoint will be the same as our first pivotal trial and is the mean rate of change in geographic atrophy growth over 12 months measured by fundus autofluorescence at 3 time points: baseline, month 6 and month 12.

If we receive positive 12-month data from ISEE2008, we plan to file for marketing approval for Zimura for the treatment of GA with the FDA and EMA. At month 12, we plan to re-randomize patients in the Zimura 2-milligram arm to receive either monthly or every other month administration of Zimura 2-milligram. All patients will initially receive monthly administrations of sham, will continue to receive monthly administrations of sham. We intend to treat and follow our patients for 24 months.

The inclusion and exclusion criteria for ISEE2008 are similar to our first Zimura pivotal trial. However, in the first trial, when patients were reported to have developed choroidal neovascularization or CNV in the study eye during the trial, they got excluded from further participation in the study, because we were concerned that fundus autofluorescence images could not be reliably evaluated in the presence of CNV in the study eye. After discussions with our independent reading center who have reviewed the images from our ongoing Zimura GA trial, we believe that many of these images could potentially be reliably assessed by fundus autofluorescence.

Therefore, in the upcoming ISEE2008 trial, we are planning to keep patients who develop CNV in the study eye in the trial. And the measurement of these patient GA size will be included in the primary efficacy analysis as well as their fundus autofluorescence images can be reliably assessed by the masked reading center.

As Glenn mentioned earlier, Zimura met its prespecified primary endpoint in the first pivotal trial by reducing the rate of GA growth in patients with dry AMD in an international, multicenter, randomized, double-masked, sham-controlled clinical trial. The reduction in the mean rate of GA growth over 12 months was 27.38%, with a P-value of 0.0072 for the Zimura 2-milligram group as compared to the corresponding sham-controlled group and 27.81% with a P-value of 0.0051 for the Zimura 4-milligram group as compared to the corresponding sham-controlled group.

These data for both those groups were statistically significant. And based on the recent FDA guidance, we consider them to be clinically meaningful. Most importantly, based on our preliminary review of the safety data today, Zimura was generally well tolerated over 12 months of administration. Over this 12-month period, there were no investigator-related -- investigator-reported Zimura-related adverse events, no cases of Zimura-related interocular inflammation, no ocular serious adverse events, no cases of Zimura-related increase in intraocular pressure, no cases of end of endophthalmitis and no discontinuations attributed by investigator to Zimura.

Further, the investigator-reported rate of choroidal neovascular membrane incidents appears to be lower than what has been reported in literature for complement inhibition in GA. Based on recent literature, this may potentially be to Zimura blocking the complement cascade downstream at the level of C5 and not blocking the cleavage of C3. We believe that potentially superior safety profile of Zimura today could potentially differentiate Zimura in the field of complement inhibitors for the treatment of GA in these elderly patients.

The design of our first pivotal clinical trial for Zimura in patients with GA, secondary to dry AMD, possessed a few important characteristics that differentiated from other Phase IIb trials done in the field and we believe supported to qualify as a pivotal trial. I would like to go over some of these important differentiating factors.

This trial was designed as a Phase IIb screening trial, which means that it's designed and conducted as a Phase III trial with all necessary Phase III requirements, but with a smaller number of patients. The prespecified design of a screening trial provided if the study drug is efficacious enough to reach statistical significance with a smaller sample size, then trial could qualify as a pivotal clinical trial. As reported earlier, the reduction of GA growth reached statistical significance for both the Zimura 2-milligram dose and the 4-milligram doses, with compared to the corresponding sham-controlled arms at month 12, fulfilling this important criteria.

To minimize bias in this trial, patients, evaluating physicians, IVERIC bio as a sponsor and the independent reading center were all masked to the treatment that the individual patients were receiving throughout the trial.

A prespecified statistical analysis plan or SAP was used for statistical analysis. Based on the prespecified criteria in this SAP, a dose of Zimura would be statistically significantly more effective than the sham-control is the strength of evidence met the standard requirement of a 0.0125 one-sided false-positive error rate, incorporating an adjustment for multiplicity arising from comparing each dose with a sham control. Further, robust prespecified density analysis was planned and performed, which indicated the analysis results were robust to missing data.

For the assessment of the primary endpoint, the images were evaluated by a leading independent masked reading center. The reviewers were completely masked, and its visit -- and each visit was evaluated independently.

As indicated earlier, our first Zimura pivotal trial is still ongoing and patients continue to be treated and followed until they reach the month 18 time point. Patients, evaluating physicians, the independent reading center and we, as a sponsor, continues to be masked regarding the 2 main groups, in which each individual patient was randomized and expect to remain masked until the patients reach month 18. It is important to point out that this trial was not designed to assess statistical significance between individual cohorts at month 18 and any month 18 results will be descriptive only. We expect to report month 18 data by the end of second quarter.

To conclude, GA is a significant cause of bilateral, irreversible and severe loss of functional vision with a major impact on the quality of life and independence of our elderly patients. Although anti-VEGF therapy is available for treatment of wet AMD, no FDA- or EMA-approved treatments are currently available for geographic atrophy.

Further, development or progression of geographic atrophy over time is a common cause of vision loss in patient diagnosed with wet AMD or being treated with anti-VEGF therapy, indicating that regardless of whether patients have the dry or the wet form of AMD, the final anatomic outcome leading to loss of vision in many patients is geographic atrophy.

The absence of treatment options for geographic atrophy represents an area of urgent unmet medical need and a major public health concern for the expanding elderly population. We look forward to keeping you updated regarding the progress of our Zimura program and to potentially help these patients.

I would now like to turn the call over to Dave. Dave?

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David F. Carroll, IVERIC bio, Inc. - Senior VP, CFO & Treasurer [5]

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Thank you, Kourous, and good morning, everyone. I'd like to highlight a few items from our press release of this morning and provide some guidance on our expected year-end 2020 cash balance and cash runway.

For the quarter, our net loss totaled $17.5 million or $0.39 per share compared to a net income of $104.1 million or $2.62 per share for Q4 2018, as Q4 2018 reflects $125 million gain on extinguishment of a royalty purchase liability payable to Novo Holdings.

Our net loss for 2019 totaled $58.9 million or $1.39 per share compared to a net income of $63.1 million or $1.70 per share for 2018, as 2018 reflects the impact of the aforementioned gain on extinguishment of a royalty purchase liability.

Turning to our expected year-end cash balance and cash runway. As we previously announced, our cash balance at December 31 was approximately $126 million. We now estimate our year-end 2020 cash balance will range between $60 million and $70 million. We also estimate that our available cash will be sufficient to fund our operations and capital expenditures as currently planned into the beginning of 2022.

These estimates are based on our current business plan, which includes the initiation of our ISEE2008 trial and the continuation of our other R&D programs. Of course, these estimates don't reflect any additional expenses resulting from the potential in-licensing or acquisition of additional product candidates or technologies or any associated development the company may pursue.

I'll now turn the call back over to Glenn. Thank you for your time.

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [6]

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Well, thank you, everybody, for listening this morning. And we're focused on execution over the next few months. The first, obviously, is to get patients into our second pivotal trial next month. And obviously, we're looking forward to the 18-month data that will come in the second quarter. So it's a focus on execution, working with our collaborators to move these programs forward.

So thanks again. Thanks for your continued support. And operator, would you please open the line for some questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) We'll now take our first question, it comes from Ken Cacciatore of Cowen and Company.

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Kenneth Charles Cacciatore, Cowen and Company, LLC, Research Division - MD & Senior Research Analyst [2]

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Just congratulations on all the progress you've been making. Just a few questions here. First, just wondering if you've actually been thinking about or entered into any ex U.S. partnering discussions and trying to think of nondilutive ways you may be looking to bolster your cash position? And then secondly, I know it's difficult to do, and you're just going to get going here on enrollment. But can you give us some thoughts or ways of putting perspective around the timing to complete enrollment for the Phase III program? And then lastly, Apellis is clearly running a program as well, and we understand they're doing 2 studies versus your 1, given you already have a pivotal completed. But can you talk about any subtleties and differences between the 2 programs that you'd like to share?

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]

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Yes. Thank you, Ken, for those 3 questions. I'll take the first 2. On the partnering, we had a very good JPMorgan, and we had an opportunity to talk with a number of potential partners. We're going to continue to speak to those partners. What I was encouraged by was sort of the strength of the data, obviously, attracts some people that have interest. So as you know, these are -- and we've been saying that we're committed to exploring partnering. And we'll keep you updated over the coming months if those discussions progress. Obviously, I don't want to put any time lines or commitments on that. But your thought about ways to further strengthen the company, not only in terms of collaboration, but also nondilutive financing is something we're thinking about as well.

On the topic of enrollment, let us get started first. Obviously, those are always very competitive questions as to enrollment. I'll just focus -- in my summary, I talked about our focus on execution. I think you'll see the same intensity once we get the trial up and going. And as we move forward, if possible, we'll update the community. But right now, that's something we want to hold a little close. You know that we can do this based on our past track record, so stay tuned on that too. And I'm sorry for not being more specific. Kourous, do you want to take the third question about differences in the trial?

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Kourous A. Rezaei, IVERIC bio, Inc. - Senior VP & Chief Medical Officer [4]

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Originally posted here:
Edited Transcript of OPHT earnings conference call or presentation 27-Feb-20 1:00pm GMT - Yahoo Finance

Wall Street has been sharply attuned to coronavirus news. In fact, the extreme volatility in the market brought 24 trading halts on Thursday.

Several biotech or pharmaceutical-related stocks were halted and reopened several times for volatility in the regular trading session. Here is a look at those stocks and each company's connection to the coronavirus.

In partnership with Italys Takis Biotech, Applied DNA Sciences Inc (NASDAQ: APDN) is pursuing a linear DNA vaccine for COVID-19.

Allied Healthcare Products Inc (NASDAQ: AHPI) manufactures respiratory devices, emergency medical supplies and mass casualty ventilation products designed for pandemics.

With schools closed for quarantines, China Online Education Group - ADR (NYSE: COE) is taking over Chinas English tutoring services.

Oncology pharmaceutical Cleveland BioLabs, Inc. (NASDAQ: CBLI) develops immune-receptor activators for vaccines targeting various types of infection. Its vaccine adjuvant is currently in Phase 1 trials.

Utah medtech company Co-Diagnostics Inc (NASDAQ: CODX) is developing a test for the new coronavirus strain. Chinese officials and fearful travel companies demonstrate high demand for accurate diagnostics, as fever has proven an unreliable indicator of disease.

Global consulting firm CRA International, Inc. (NASDAQ: CRAI) is likely reacting less to coronavirus fears than to a quarterly earnings miss.

Tech developer Digimarc Corp (NASDAQ: DMRC) is similarly responding to an earnings miss.

EQT Corporation (NYSE: EQT), a natural gas producer, beat fourth-quarter earnings estimates and announced a deal with Equitrans.

Equitrans Midstream Corp (NYSE: ETRN) announced a deal with EQT, a stock buyback and a restructuring into a C-Corp.

Genetic Technologies Limited (NASDAQ: GENE), which develops and commercializes genetic risk assessment technology, commonly partners with medical companies and research organizations on R&D.

Biotech company Genprex Inc (NASDAQ: GNPX) touts a proprietary gene technology platform but has made no public entrance into coronavirus therapies.

Greenland Technologies Holding Corp (NASDAQ: GTEC) manufactures machine transmission products in China.

Biotech Ibio Inc (NYSE: IBIO) has partnered with CC-Pharming to develop a coronavirus vaccine using tobacco-related plants.

Jiayin Group Inc - ADR (NASDAQ: JFIN) is an online finance marketplace that facilitates consumer lending in China. Its remote offering may see increased uptake amid local quarantines.

LHC Group, Inc. (NASDAQ: LHCG) beat bottom-line estimates but missed the top line in its fourth-quarter earnings report.

Liberty Tripadvisor Holdings Inc (NASDAQ: LTRPB) holds an online travel site whose forum has seen some activity by fearful travelers.

IT services company Mastech Digital Inc (NYSE: MHH) offers clients data management, engineering and science.

Moderna Inc (NASDAQ: MRNA) received new funding from vaccine alliance CEPI to accelerate its work on a coronavirus vaccine, and it has joined forces with the Coalition for Epidemic Preparedness Innovations on a vaccine approach.

Diabetic therapy manufacturer Oramed Pharmaceuticals, Inc. (NASDAQ: ORMP) announced a public offering to raise funds for its oral insulin trial.

Qurate Retail Inc (NASDAQ: QRTEA), which curates product collections on Zulily, Frontgate and other online marketplaces, missed fourth-quarter earnings estimates and attributed the miss partly to Chinese exposure.

Smart home manufacturer Resideo Technologies Inc (NYSE: REZI) beat fourth-quarter earnings forecasts.

SigmaTron International (NASDAQ: SGMA) provides electronics manufacturing services through facilities in four countries, including China.

Vaxart Inc (NASDAQ: VXRT) has initiated a coronavirus vaccine program using its proprietary oral-delivery platform.

The portfolio of Vir Biotechnology Inc (NASDAQ: VIR) includes an antibody for another coronavirus strain, but management is testing to determine efficacy on the Wuhan strain.

"We have a library of multiple fully-human mAbs that bind and neutralize coronaviruses such as SARS and MERS,"Chief Scientific Officer Herbert 'Skip'Virgin said in a press release. "...Some of these mAbs are able to neutralize zoonotic coronaviruses, and we believe may have the potential to treat and prevent Wuhan coronavirus. We are also exploring the isolation of new mAbs specific for this virus."

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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Diagnosis: Volatility Every Coronavirus-Related Stock That Was Halted For Trading Today - Benzinga

The Center for Gene Therapy at Nationwide Childrens Hospital is working to develop childrens gene therapy treatments. Officials say the gene therapy research and clinical trials there are starting to attract companies to central Ohio.

Nationwide Childrens Hospital is in the forefront of curing several genetic childhood diseases, transforming Columbus into a major medical hub, several gene therapy experts say.

The hospital's Center for Gene Therapy at the Abigail Wexner Research Institute is working to develop treatments for children, which is attracting patients and companies to Ohio, according to officials at Nationwide Childrens and JobsOhio, the state's economic development organization.

The illnesses that were making use of in gene therapy are devastating illnesses, said Dr. Kevin Flanigan, the director of Nationwide Childrens Center for Gene Therapy. These are ones we know that children would be significantly impaired for life or die because of the disease.

Gene therapy involves altering the genes inside the patient's cells in an effort to treat or stop disease. It gives doctors the chance to treat many previously untreatable rare and genetic diseases.

Gene therapy is currently available primarily in a research setting, with only four gene therapy products approved by the U.S. Food and Drug Administration for sale in the United States. One of the four, Zolgensma, started as a clinical trial for spinal muscular atrophy at Nationwide Childrens in 2014.

The hospital is working on a handful of gene therapy treatments for various childhood diseases that affect muscle, motor or mental functions, Flanigan said.

Gene therapy presents a tremendous opportunity for our medical system, and Columbus has been a huge part of that growth thanks to the work being done at Nationwide Childrens Hospital, Edith Pfister, chairwoman of the American Society of Gene & Cell Therapys communications committee, said in an email.

The FDA approved Zolgensma, a one-time treatment that intravenously delivers the gene that is missing in children with spinal muscular atrophy, on May 24.

SMA is a progressive childhood neuromuscular disease that is caused by a mutation in a single gene that attacks nerve cells. It causes major physical limitations including the inability to breathe, swallow, talk or sit up. Children born with SMA typically die or need permanent breathing assistance by the time they turn 2 years old.

Donovan Weisgarber was diagnosed with SMA type 1 at Nationwide Childrens in November 2015 when he was 5 weeks old. His parents, Matt and Laura Weisgarber, decided to participate in a clinical trial at the hospital and Donovan received Zolgensma.

Before the treatment, Donovan was unable to swallow and had difficulty breathing. Today, the 4-year-old has doubled his life expectancy and is able to talk, sit up, roll over and hold his head up on his own. He also attends the Early Childhood Education and Family Center on Johnstown Road on the East Side, which offers services from the Franklin County Board of Developmental Disabilities.

(Gene therapy) has given us an opportunity that we otherwise wouldnt have to love Donovan and experience him, said Matt Weisgarber, 33, of the Northeast Side.

A lot of people hear Ohio and think flyover state, but now Columbus is going to be a hub of the most groundbreaking science known to mankind and thats a really cool thing, he said.

Boston Childrens Hospital and Childrens Hospital of Philadelphia also have impressive gene therapy centers, but Columbus sets itself apart from those East Coast cities, said Severina Kraner, JobsOhios health care director.

The cost to operate, manufacture and live in Ohio is cheaper than Boston and Philadelphia, putting Ohio in a position to win cell and gene therapy companies, she said.

People are being priced out of these coastal cities, Kraner said.

One of the companies who has committed to building in Columbus is Sarepta Therapeutics, a Massachusetts-based biopharmaceutical company. Sarepta signed an agreement with Nationwide Childrens in May 2019, giving the company the licensing to a gene therapy treatment that came out of hospital research for limb-girdle muscular dystrophies, a group of diseases that cause weakness and wasting of the muscles in the arms and legs.

Sarepta is scheduled to open an 85,000-square-foot Gene Therapy Center of Excellence near Nationwide Childrens Hospital in the fall to do early research for all the companys gene therapy programs. A team of about 30 employees from Sarepta is currently working at a facility at Easton Town Center.

The region has every ingredient needed for a thriving gene therapy cluster: a strong academic foundation, world-renowned research hospitals, and, now, industry investment, Louise Rodino-Klapac, Sareptas senior vice president of gene therapy, said in an email. All of these contribute to creating a pipeline of talented people who will accelerate scientific advances that help patients.

Nationwide Childrens recently also announced it will be expanding its gene therapy research by creating Andelyn BioSciences, a new for-profit subsidiary that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.

Were hoping, and we have a vision, that Andelyn can help capitalize a biotechnology hub in central Ohio focused on developing and advancing gene therapies, said Dr. Dennis Durbin, Nationwide Childrens chief science officer.

Andelyn BioSciences will launch this summer and operate out of the Abigail Wexner Research Institute, 575 Children's Crossroad. Nationwide Children's is trying to secure a permanent location for Andelyn and is looking at land on Ohio State Universitys West Campus.

Gene therapy treatment, however, comes at a high price.

The manufacturer set the price of Zolgensma at more than $2.1 million. Insurers can pay $425,000 a year for five years for one treatment.

Insurance companies are used to regular installment payments, but the single-dose nature of gene therapies are adding a level of uncertainty to health insurance structures, Pfister said in an email. A one-time administration gene therapy costs less overall, but it occurs in one upfront payment.

Pfister said she is hopeful the cost of gene therapy will go down.

Currently, most of the FDA-approved gene and cell therapies are tailored for the specific patient, but theres an incredible amount of research going into standardizing the components and delivery mechanisms behind gene therapy, Pfister said in an email.

Dr. Jerry Mendell helped usher in the era of gene therapy at Nationwide Childrens when he came to the hospital in 2004.

Nationwide Childrens first gene therapy trial was in 2006 for duchenne muscular dystrophy, a rare, inherited, degenerative muscle disorder that almost exclusively affects boys.

Things have really changed significantly in the gene therapy world because of the contributions weve made here, and its been a very gratifying experience, said Mendell, the principal investigator in Nationwide Childrens Center for Gene Therapy.

mhenry@dispatch.com

@megankhenry

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Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch

DUBLIN--(BUSINESS WIRE)--The "Cancer Therapies - Market Analysis, Trends, and Forecasts" report has been added to ResearchAndMarkets.com's offering.

The global market for Cancer Therapies is projected to reach US$220.5 billion by 2025, driven by the rise in cancer prevalence to epidemic proportions and the still ongoing search to find effective treatments for the disease. Despite decades of research costing billions of dollars, a cure for cancer still remains elusive. This fact when juxtaposed with the epidemic spread of cancer will result in the disease emerging into the single most difficult to tackle public healthcare burden in the coming years. The pressure is therefore intensifying to research and develop newer and more effective therapies and treatment options. The reason why cancer is complex is due to its ability to continuously evolve and undergo molecular, genetic changes that affect behavior and response of tumor cells. Cancer cells evolve myriad ways to sabotage, stymie and trick the immune system preventing it from recognizing cancer cells, making the disease more resilient, aggressive and deadly. This has profound implications for the progression of the disease despite interventional therapies. Also, there are over 100 types of known cancer types. In addition the genetic diversity of tumors especially intra-tumor genetic heterogeneity makes finding a cure a challenge which the medical community continues to grapple with. Although the Cancer Genome Atlas (TCGA) has increased understanding of the diversity of cancer types, the disease continues to elude a cure while continuing to stretch the boundaries of medical science and understanding. Significant research is still required to understand the vast diversity of tumor gene expression, mutations and drug sensitivities.

Against the backdrop of tumor diversity, the universal 'one size fits all' therapy which is the current standard of care is primitive. Therapies like chemotherapy and radiation, although help increase survival rates are beset with side effects as they act as sledgehammers that destroy even healthy dividing cells at the cellular level. There is an urgent need for developing newer ways to target cancer's diversity and evolution. While a cure for cancer is unlikely, targeted therapies will witness huge gains for their better prognosis. Targeted therapies revolve around identifying major pathways responsible for the disease and its progression and administering specific drugs targeting these pathways. Targeted therapies have lower side effects and are more effective than conventional therapies. However, targeted therapy increases the risk of emergence of treatment-resistant phenotypes. As an antidote to this problem is the interest shed on combined therapy targeting, stem cell transplants, molecular targeted therapy, and nanotechnology. Will these emerging therapies offer new paradigms in cancer treatment in the future, is however a question which only time will answer. Nevertheless, new advancements being made infuse optimism.

For instance, scientists are close to identifying the key molecule involved in cancer's mix and match diversity and evolution. Dubbed as DHX8, the protein influences the fundamental process in a cell called 'alternative splicing'. Aberrations in alternative splicing are linked to cancer's progression and drug resistance. Drugs targeting the DHX8 Gene can likely help us find the elusive chink in cancer's biological armor allowing us to finally steal a march over this complex disease. An exciting future currently awaits cancer therapies through 2025 despite all the challenges involved. The United States and Europe represent large markets worldwide with a combined share of 68% of the market. China ranks as the fastest growing market with a CAGR of 11.2% over the analysis period supported by the massive strides taken by the country in developing affordable next-generation therapies. Aggressive reforms in drug regulations and approval mechanisms have helped China emerge into the second largest pharmaceutical industry worldwide.

Key Topics Covered:

I. Introduction, Methodology & Report Scope

II. Executive Summary

III. Market Analysis

IV. Competition

For more information about this report visit https://www.researchandmarkets.com/r/r0jl83

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Global Cancer Therapies Market Forecast to Reach US$220.5 billion by 2025 - ResearchAndMarkets.com - Business Wire

A new Profession Intelligence Report released by Stats and Reports with the title Global Gene Therapy for Ovarian Cancer Market can grow into the most important market in the world that has played an important role in making progressive impacts on the global economy. Global Gene Therapy for Ovarian Cancer Market Report presents a dynamic vision to conclude and research market size, market hope and competitive environment. The study is derived from primary and secondary statistical data and consists of qualitative and numerical analysis. The main company in this survey isTakara Bio, VBL Therapeutics, CELSION, Targovax.

Free Sample Report @:www.statsandreports.com/request-sample/298945-global-gene-therapy-for-ovarian-cancer-market-size-status-and-forecast-2019-2025

Preliminary Data:Get raw market data and contrast from wide front. Data is constantly filtered so that only validated and authenticated sources are considered. The data is also collected from many reputable paid databases and many reports in our repository. A comprehensive understanding of the market is essential to understanding and facilitating the complete value chain. We collect data from raw material suppliers, distributors, and buyers.

Sample Table: Global Gene Therapy for Ovarian Cancer Market Size By Regions (USD Million) (2014-2025)

Furthermore, the years considered for the study are as follows:Historical year 2014-2018Base year 2019Forecast period** 2019 to 2025[** unless otherwise stated]

Research Methodology:The market engineering process uses a top-down and bottom-up approach and several data triangulation methods to evaluate and validate the size of the entire market and other dependent sub-markets listed in this report. Numerous qualitative and quantitative analyzes have been conducted in the market engineering process to list key information / insights. The major players in the market were identified through the second survey and the market rankings were determined through the first and second surveys.

Exclusive Discount Offer on Quick Purchase@www.statsandreports.com/check-discount/298945-global-gene-therapy-for-ovarian-cancer-market-size-status-and-forecast-2019-2025

Crucial Research:During the first survey, we interviewed various key sources of supply and demand to obtain qualitative and quantitative information related to this report. Key supply sources include key industry participants, subject matter specialists from key companies, and consultants from several major companies and organizations active in the digital signage market.

Minor Research:The second study was conducted to obtain key information on the supply chain of the industry, the markets currency chain, pools of major companies, and market segmentation, with the lowest level, geographical market, and technology-oriented perspectives. Secondary data was collected and analyzed to reach the total market size, which was verified by the first survey.

Key Segments Studied in the Global Gene Therapy for Ovarian Cancer Market

What are the Major applications of the Gene Therapy for Ovarian Cancer Market?Applications cover in these Reports Is:Ovarian Cancer (unspecified), Recurrent Ovarian Epithelial Cancer, Platinum-Resistant Ovarian Cancer

What are the Types of the Gene Therapy for Ovarian Cancer Market?Types Cover in this Research :Intravenous, Intratumoral, Intraperitoneal

Who are the main competitors in the market and what are their priorities, strategies, and developments?Lists of Competitors in Research Is: Takara Bio, VBL Therapeutics, CELSION, Targovax

Read Full TOC of Gene Therapy for Ovarian Cancer Research Study at @www.statsandreports.com/report/298945-global-gene-therapy-for-ovarian-cancer-market-size-status-and-forecast-2019-2025

All percent shares, breaks, and classifications were determined using the secondary sources and confirmed through the primary sources. All parameters that may affect the market covered in this study have been extensively reviewed, researched through basic investigations, and analyzed to obtain final quantitative and qualitative data. This has been the study of key quantitative and qualitative insights through interviews with industry experts, including CEOs, vice presidents, directors and marketing executives, as well as annual and financial reports from top market participants.

Years considered for the study are:Historical year 2014-2018Disreputable year 2019Estimate period** 2019 to 2025 [** unless otherwise stated]

Essentials of Table of Content:

1 Report Overview1.1 Research Scope1.2 Key Market Segments1.3 Target Player1.4 Market Analysis by Type1.5 Market by Application1.6 Learning Objectives1.7 years considered

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2 Global Growth Trends2.1 Global Gene Therapy for Ovarian Cancer Market Size2.2 Trends of Gene Therapy for Ovarian Cancer Growth by Region2.3 Corporate trends

3 Gene Therapy for Ovarian Cancer Market shares by key players3.1 Global Gene Therapy for Ovarian Cancer Market Size by Manufacturer3.2 Global Gene Therapy for Ovarian Cancer Key players Provide headquarters and local3.3 Major Players Products / Solutions / Services3.4 Enter the Barriers in the Gene Therapy for Ovarian Cancer Market3.5 Mergers, acquisitions and expansion plans

4 Market By-products4.1 Global Gene Therapy for Ovarian Cancer Sales by Product4.2 Global Gene Therapy for Ovarian Cancer by Product Revenue4.3 Global Gene Therapy for Ovarian Cancer

Note: Regional Breakdown & Sectional purchase Available We provide Pie chats Best Customize Reports As per Requirements.

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Stats and Reports is a global market research and consulting service provider specialized in offering wide range of business solutions to their clients including market research reports, primary and secondary research, demand forecasting services, focus group analysis and other services. We understand that how data is important in todays competitive environment and thus, we have collaborated with industrys leading research providers who works continuously to meet the ever-growing demand for market research reports throughout the year.

Contact:Stats and ReportsSatish K. (Global Sales Manager)Mangalam Chamber, Office No 16, Paud RoadSankalp Society, Kothrud, Pune, Maharashtra 411038Phone: +1 650-646-3808Email: [emailprotected]Web: https://www.statsandreports.comFollow Us on: LinkedIN| Twitter|

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Gene Therapy for Ovarian Cancer Market Research & Clinical Advancements by 2025 | Takara Bio, VBL Therapeutics, CELSION - Instant Tech News

There is no question that shopping can become an addiction, a serious problem that can put your financial house at risk. But did you know that saving money can also become an addiction? Whether we become addicted to spending or to saving appears to boil down to how our brains operate.

Something happens when we feel pleasure, whether that pleasure is a result of spending money or saving money: Our brains release dopamine. This "happiness hormone" is triggered when we get a reward. For one person the reward may be something new they have purchased, while another will enjoy the same feeling when they save money or make a new investment.

Image source: Getty Images

And here's where addiction comes in: The better we feel each time we spend or save, the more likely we are to reach for that same "high" again.

So what do you do if you're a spender who would rather be a saver? Here are five steps you can take:

In order to train your brain to save money, you must first train it to stop spending impulsively. Unless you are going out to pick up items you need to live (like groceries), leave your debit card, credit cards, and checkbook at home. Take only as much cash as you're willing to spend. For example, if a friend asks you to go shopping, take enough cash to buy a coffee and go to lunch. Even if you walk into a store brimming with amazing sales, you won't miss out on anything important if you didn't need it to begin with.

Cancel catalogs and flyers being sent from your favorite stores. Unsubscribe from emails and disable one-click ordering on your computer. Create stumbling blocks to spending -- what behavioral economists refer to as "friction." Often, slowing yourself down by seconds offers enough time to rethink what would have been an impulse purchase.

A survey by Ally Bank found that saving money has a bigger effect on happiness than the amount a person earns. Of those surveyed, 84% said that having money in the bank contributed to their overall sense of well-being and was more important than having a job they enjoy, getting regular exercise, or eating healthy foods.

Money can't buy happiness, but it can benefit your mental health by decreasing stress and increasing confidence. Having money put away offers choices. You can invest, travel, go back to school, retire earlier, take up a new hobby, or simply quit a job that is sucking you dry and find one that better matches your talents.

Just knowing these facts may not be enough to alter your brain, but knowledge can become your touchstone. Any time you're tempted to spend needlessly, ask yourself this question: Would I rather have this thing right now, or do I want to feel more secure and confident in the future?

The idea is to reframe the way you think about shopping. You can give your money to a company and allow the business to get rich, or you can keep that money and allow it to work for you.

What you're aiming for is that surge of dopamine. Finding the same satisfaction in saving as you once found in spending takes repeated application. Here are some ways you can get practice:

Learn as much as you can about investing and how it can change your life. Fill your mind with all the reasons you deserve to be financially secure. The more you read, the more anxious you will be to invest in your future.

Once you take the step beyond simply saving and find out how to invest your money, you'll discover that the stock market offers higher rewards in the long term, although there are no guarantees. Check out these online stock brokers that give you access to plenty of research as well as low (or no) commissions and fees.

You didn't become addicted to shopping overnight and you won't become addicted to saving overnight. Each positive emotion you experience, each rush of dopamine, makes it a little easier to do the right thing the next time. The goal is to practice until you learn to crave the feeling of satisfaction that results from socking money away in your savings account.

Over the long term, there's been no better way to grow your wealth than investing in the stock market. But using the wrong broker could make a big dent in your investing returns. Our experts have ranked and reviewed thetop online stock brokers- simplyclick hereto see the results and learn how to take advantage of the free trades and cash bonuses that our top-rated brokers are offering.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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How to Turn Saving Money Into an Addiction - Nasdaq

A leading market research firm Facts & Factors (FnF) added a market research report on Cell and Gene Therapy Consumables Market By Product Type (Kits & Buffers, Diagnostic Assay, Culture Medium, and Cryopreservation Media) and By Application/ Therapeutics (Cardiovascular, Urology, Dermatology, Critical Care, Respiratory, Endocrine & Metabolic, Neuroscience, Hematology & Oncology, Obstetrics, Immunology, and Gastroenterology): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027 that includes 110+ pages research report with TOC in its research database.

The report gives a granular investigation of the different factors and patterns affecting the development direction of the Cell and Gene Therapy Consumables market. It incorporates in-depth data relating to the overarching progression of the market and displays refined development forecasts for the market in light of solid information. An evaluation of the effect of government strategies and holistic on the market processes is likewise included to give an all-encompassing outline of the Cell and Gene Therapy Consumables market's future viewpoint.

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Our Free sample report provides a brief introduction to the research report overview, TOC, list of tables and figures, an overview of major market players and key regions included.

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The majority of the information is portrayed in the form of graphical representation with precisely calculated figures. The performance of the associate key players, vendors, and suppliers are additionally distinguished in the report. The reports also emphasize on the restraints and drivers keenly that are derived from the prudent perceptive of our experts. The characteristics present in the report are assessed with an inherent and quantifiable stance to improve understanding of the Cell and Gene Therapy Consumables market.

Major Company Profiles Covered in This Report:

Amgen Inc., ATLANTA BIOLOGICALS, bluebird bio, Inc., Cook, Dendreon Pharmaceuticals, LLC, Fibrocell Science, Inc., General Electric, Kolon TissueGene, Inc., Orchard Therapeutics plc., Pfizer, Inc., PromoCell GmbH, RENOVA THERAPEUTICS, Sibiono GeneTech Co. Ltd., Spark Therapeutics, Inc., Vericel, Helixmith Co., Ltd., and Vitrolife

(To Know Full List of Market Players Request Free sample Pages)

The reports also explain the point-to-point comprehension of some critical parameters such as profit and loss figures, logistics & distribution channels, item value, production capacity, and many others. The report showcases end-to-end parameters such as application, innovation, product development, and diverse frameworks and procedures. It also focuses on a variety of modifications made to alleviate the process of functioning.

A meticulously collated Cell and Gene Therapy Consumables market share report on the basis of a primary and secondary source is presented in a more expressive and articulated format, which enables the clients to set up a full-fledged plan for the forecast period.

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This report gives a detailed survey of the market by examining changing aggressive elements of the Cell and Gene Therapy Consumables market. It offers a forward-looking point of view on various elements driving or limiting business sector development. It gives a forecast based on how the market is anticipated to develop. It helps in understanding the key product sectors and their future. It gives pin point analysis of changing rivalry flow and keeps you in front of contenders.

It helps in settling on educated business choices by having complete bits of knowledge of the market size of Cell and Gene Therapy Consumables and by making inside and out the investigation of market sectors. It gives unmistakable designs and exemplified SWOT analysis of the main sectors of the Cell and Gene Therapy Consumables market.

The research report for the Cell and Gene Therapy Consumables market incorporates a worldwide viewpoint. Imperative areas all over the globe are secured and the patterns, advancements, drivers, restrictions, and difficulties affecting the development of the Cell and Gene Therapy Consumables industry over these vital regions are considered.

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Global Cell and Gene Therapy Consumables Market:Regional Segment Analysis

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Cell and Gene Therapy Consumables Market Size, Top key players, and Forecasts 2026, Market Status - Northwest Trail

Ducommun (DCO) came out with quarterly earnings of $0.80 per share, beating the Zacks Consensus Estimate of $0.64 per share. This compares to earnings of $0.06 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 25%. A quarter ago, it was expected that this aerospace industry supplier would post earnings of $0.61 per share when it actually produced earnings of $0.70, delivering a surprise of 14.75%.

Over the last four quarters, the company has surpassed consensus EPS estimates four times.

Ducommun, which belongs to the Zacks Aerospace - Defense Equipment industry, posted revenues of $186.93 million for the quarter ended December 2019, surpassing the Zacks Consensus Estimate by 0.32%. This compares to year-ago revenues of $164.18 million. The company has topped consensus revenue estimates four times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

Ducommun shares have lost about 17.5% since the beginning of the year versus the S&P 500's gain of 4.8%.

What's Next for Ducommun?

While Ducommun has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for Ducommun was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #5 (Strong Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is $0.08 on $155.44 million in revenues for the coming quarter and $2.15 on $717.73 million in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Aerospace - Defense Equipment is currently in the bottom 16% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

To read this article on Zacks.com click here.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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Ducommun (DCO) Q4 Earnings and Revenues Top Estimates - Nasdaq

February 20, 2020 -The Institute for Gene Therapies (IGT), a new advocacy foundation focused on policy issues surrounding gene therapies, recently launched with the goal of modernizing US regulatory and reimbursement framework for gene therapies.

According to IGT, the US health system currently spends about 85 cents of every healthcare dollar managing the symptoms of chronic diseases over a patients lifetime. With gene therapies, providers could alter patients non-functioning genes or replace absent ones, leading to long-lasting effects and potentially reshaping the way thousands of diseases are treated.

While traditional biologic and pharmaceutical medicines help manage the symptoms of disease over time, gene therapies target the cause of disease at the DNA level, creating lasting changes in the body. Some gene therapies are also designed to be one-time treatments that offer lifelong benefits.

IGT will work to educate stakeholders across the healthcare system about the potential for gene therapies to treat and cure common and rare chronic diseases, and advocate for policies that help ensure patients who need gene therapies can benefit from them.

Existing regulatory and reimbursement structures were established and adjusted over time to support pharmaceutical and biologic medicines, IGT noted, and need refining to accommodate the potential of gene therapies.

READ MORE: Data Mining Techniques Could Improve Cancer Gene Therapies

Many crippling conditions like Charcot-Marie-Tooth (CMT), which I was diagnosed with before the age of two take hold at a very young age, cut lives far too short or cause ongoing daily suffering, said Susan Ruediger, CEO of the CMT Research Foundation (CMTRF) and member of the IGT Patient Advocacy Advisory Council.

Like so many diseases, CMT currently has no cure. I am proud to stand with other leading patient advocates, members of the research community and companies that are developing gene therapies to help ensure patients can fully realize the benefits of these giant leaps toward treatments and cures.

The FDA has already approved four gene therapy products, and researchers are studying hundreds more in clinical trials for rare and common diseases, including many types of cancer, neuromuscular diseases, blood disorders and infectious diseases, and other conditions.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, FDA Commissioner Stephen M. Hahn, MD, said in a statement.

We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

READ MORE: Genetic Sequencing Study Identifies 102 Genes Associated with Autism

The FDA has also released six final guidance documents on gene therapy manufacturing and clinical development of products. These documents incorporate input from stakeholders across the healthcare industry and make important strides in designing a modern structure for gene therapy development.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

IGT will being together experts across the healthcare community, including corporate leaders, patient advocacy groups, and academic and scientific stakeholders, to ensure health policies reflect the latest advancements in gene therapies. The institute will also work to remove barriers that limit patient access to these therapies and promote sustainable, long-term solutions.

Experts will learn more about the value gene therapies bring to patients, their families, and the healthcare system as a whole. IGT will help advance chronic disease treatments and get to the root cause of some of the most debilitating, expensive conditions that affect patients throughout their lives.

READ MORE: 77% of Americans Are Optimistic About Genetics Research, Potential

The incredible scientific advancements in this space present unique opportunities to directly improve and save the lives of patients suffering from debilitating diseases, said IGT Chairman, and former Congressman Erik Paulsen.

This is not some far-off future patients are already benefiting from the first FDA-approved gene therapies. But we need policy to move faster toward this new reality where we can treat the causes of many diseases. The Institute for Gene Therapies and our members believe unique regulatory and reimbursement structures need to be established, novel development pathways need to be embraced and new value-based arrangements need to be tested.

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New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com

Dive Brief:

Though analysts continue to debate the particulars of a valrox launch, they generally expect the therapy to take a significant chunk of the hemophilia market. Cantor Fitzgerald found through a survey that doctors seem quite willing to prescribe valrox within a few years of its launch. SVB Leerink also saw patient and prescriber enthusiasm in its own research.

"We believe the hemophilia market may be less 'sticky' and more poised for disruption by next generation agents than is generally assumed based on historical behavior," SVB Leerink analysts wrote in an investor note last February.

For BioMarin, a market open for disruption could translate to big sales. According to Jefferies, consensus estimates on Wall Street put valrox sales at $44 million this year, growing to $190 million in 2021 and more than $1.3 billion in 2025. Last May, Jefferies said valrox accounted for 16% the value it modeled for BioMarin's stock price.

On the other end, valroxcould threaten leaders in the hemophilia drug space. Roche's blockbuster drug Hemlibra, which comes with convenient dosing and a high list price, looks particularly vulnerable. Survey data from Citi Research cited by BioMarin showed U.S. hematologists said they would switch close to 40% of their Hemlibra-treated patients to a gene therapy within three years of valrox's launch.

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BioMarin inches closer to a gene therapy first - BioPharma Dive

John Hopkins University will use Orgenesis point-of-care processing technology for cell and gene therapy research in a collaboration announced this week.

The US research University has licensed access to Orgenesiss point-of-care (PoC) platform which as the name suggests is used to develop and process cell therapies in the clinic

Orgenesis CEO Vered Caplan said, Our POCare platform is designed to provide unique cell and gene therapy solutions in a cost effective, high quality and scalable manner, using closed systems and other advanced cell processing technologies at the point of care.

Image: Tingtingou/creativecommons

Caplan added the aim is to support John Hopkins Universitys growing development and processing needs in order to advance and accelerate cell and gene based clinical therapeutic research.

News of the collaboration comes weeks after Orgenesis sold Masthercell, its third-party cell and gene therapy business, to Catalent for $315 million (285 million).

At the time Orgenesis said it expects to use the net proceeds from the sale of Masthercell to grow its point-of-care cell therapy business

Caplan said, We decided it was the right time to sell Masthercell to maximize value for our shareholders, and focus our efforts around our POCare solutions, which we believe represent a major paradigm shift and will play a major role in the future of the cell and gene therapy market.

She explained that by helping healthcare providers switch from costly, centralized manufacturing models to a localized point-of-care model it can reduce costs and accelerate cell therapy development.

The point of care platform is designed to collect, process and supply cells within the patient care setting for various therapeutic treatments.

The aim is to reduce cost and complexity of supplying cell and gene therapies, as well as elevate quality standards by integrating automated processing.

John Hopkins University is the third institution to license use of the technology.

In January, Orgenesis announced the University of California, Davis will use the platform to develop, commercialize and supply cell and gene products and therapies.

Prior to that Orgenesis joint venture with Theracell signed an agreement with Greece-based Hygeia Group covering use of the platform at three hospitals owned by the latter organization.

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Orgenesis teams with John Hopkins' in PoC deal - Bioprocess Insider - BioProcess Insider

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What Challenges Gene Therapy Technologies May See in Next 5 Years: Bluebird bio, Adaptimmune, GlaxoSmithKline, Merck - Nyse Nasdaq Live

PITTSBURGH, Feb. 21, 2020 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (NASDAQ: KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from skin diseases, today announced that Krish S. Krishnan, chairman and chief executive officer, will present at the Cowen 40th Annual Healthcare Conference in Boston being held March 2-4, 2020 at the Boston Marriott Copley Place.

Details for the presentation are as follows:

A webcast of the presentation will be available for 90 days and can be found on the Krystal Biotech website at: http://ir.krystalbio.com/events-and-presentations/events.

AboutKrystal BiotechKrystal Biotech, Inc.(NASDAQ:KRYS) is a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases. For more information, please visithttp://www.krystalbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for Krystal, including but not limited to statements about the development of Krystals product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of bercolagene telserpavec (B-VEC) and KB105, the clinical utility of B-VEC and KB105 and Krystals plans for filing of regulatory approvals and efforts to bring B-VEC and KB105 to market, the market opportunity for and the potential market acceptance of B-VEC and KB105, plans to pursue research and development of other product candidates, the sufficiency of Krystals existing cash resources and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including B-VEC and KB105, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with theU.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

CONTACTS:

Investors:Ashley R. RobinsonLifeSci Advisorsarr@lifesciadvisors.com

Media:Darren Opland, PhDLifeSci Communicationsdarren@lifescicomms.com

Source: Krystal Biotech, Inc.

Excerpt from:
Krystal Biotech to Present at the Cowen 40th Annual Healthcare Conference - BioSpace

NEW YORK, Feb. 21, 2020 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium") announced today findings from the SIERRA trial that were presented at the 2020 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR (TCT) in Orlando, FL. Dr. Boglarka Gyurkocza, the principal investigator from Memorial Sloan Kettering Cancer Center, revealed that there were key differences in side effects reported in patients treated in the Iomab-B and control arms of the study with rates of febrile neutropenia, sepsis and mucositis being markedly lower in the Iomab-B arm.

The oral presentation also featured updated results from the fifty percent enrollment mid-point analysis including BMT access rates, engraftment and 100-day non-relapse transplant related mortality (TRM). After accounting for these factors, the results showed that, on an intent-to-treat or ITT basis, 78 percent of patients in the Iomab-B arm are potentially evaluable for the primary endpoint compared to 13 percent in the control arm. In addition, an important and recent protocol amendment was highlighted; the approximately 30 percent of patients who are expected to fail induction therapy with venetoclax plus hypomethylating agents1 are now eligible for enrollment in the SIERRA trial. The change is expected to increase the addressable patient population of the study given that this combination is now recommended as part of the NCCN or National Comprehensive Cancer Network guidelines, is widely used and expected to become the treatment of choice.

Key Interim Results:

The data tables that follow summarize key findings that were presented as part of the TCT proceedings. As highlighted in the table below, patients receiving Iomab-B showed lower rates of key adverse events relevant in the BMT setting in compared to patients randomized to receive physician's choice of salvage chemotherapy on the control arm. For example, patients receiving Iomab-B had a much lower incidence of sepsis of 3 percent compared to the 42 percent incidence in the control arm.

Adverse Event* N (%)

Randomized to Iomab-B andreceived BMT (N=31)

Randomized to Control Armand received BMT (N=7)

Febrile Neutropenia

8 (25.8)

3 (42.8)

Sepsis/Septic Shock

1 (3.2)

3 (42.8)

Stomatitis (mucositis)

3 (9.7)

2 (28.6)

Pneumonia/Lung Infection

4 (12.9)

1 (14.3)

Hypertension

61 (19.4)

1 (14.3)

Decreased Appetite

5 (16.1)

0 (0.0)

Device related infection

4 (12.9)

1 (14.3)

Hypophosphatemia

2 (6.5)

1 (14.3)

* All adverse events reported irrespective of attribution to protocol-directed procedures

1) 5 patients had hypertension considered unrelated to Iomab-B and 1 patient had hypertension possibly related to Iomab-B

"This detailed safety data from SIERRA is highly encouraging, particularly in this patient population, as neutropenia and sepsis are hallmark toxicities associated with chemotherapy-based conditioning regimens that next to relapse are leading causes of morbidity and mortality post-transplant," said Dr. Vijay Reddy, Vice President, Clinical Development and Head of BMT at Actinium. "Chemotherapy-based conditioning damages normal organs, such as the gastrointestinal tract, which allows gut bacteria to cause serious infections. The data showing less mucositis, febrile neutropenia, and sepsis are consistent with the targeted nature of Iomab-B, since a lack of damage to the gastrointestinal track would lead to a reduction in these adverse events. BMT is the only curable treatment option for older patients with active, relapsed or refractory AML. Yet these patients face restricted access and suboptimal outcomes due to reliance on chemotherapy-based conditioning regimens and perceptions in the hematologist community around safety and eligibility for BMT. With the SIERRA trial, our goals are to eliminate these barriers leading to more patients receiving BMT and with better patient outcomes. With this additional safety data in hand, we have even greater confidence in our ability to change the perceptions around BMT and are excited to update the transplant and hematology communities on Iomab-B's potential to positively impact patients through improved access to BMT and better outcomes."

The presentation highlighted that 100 percent (31/31) of patients receiving a therapeutic dose of Iomab-B achieved successful BMT engraftment with only a 6 percent (2/31) TRM rate compared to the control arm where 18 percent (7/38) achieved engraftment with a 29 percent (2/7) TRM rate. At the 100-day post BMT time point, on an ITT basis, there were 29 patients from the Iomab-B study arm potentially evaluable for the primary endpoint of durable Complete Remission (dCR) at 180 days compared to 5 patients in the control arm. By this measure, 78 percent of patients in the Iomab-B arm are potentially eligible for the dCR primary endpoint compared to 13 percent of patients in the control arm. The mid-point analysis and data presented at TCT can be viewed here.

Detailed engraftment data is presented in the table below:

BMT Feasibility andOutcome Data

Randomized toStudy Arm (N=37)

Randomized to Control Arm (N=38)

ReceivedTherapeutic Doseof Iomab-B andreceived BMT(N=31)1

Achieve CR andreceived standardBMT (N=7)

Did not Achieve CR(N=31/38)2

Crossed over from toIomab-B and receivedBMT (N=20)

BMT Engraftment Rate (%, N)

100% (31/31)

18% (7/38)

100% (20/20)

Median Bone Marrow Blasts% at randomization

(%, range)

29% (5-88)

26% (5-97)

At crossover: 31%

(6-87)

At randomization: 35%(5-75)

Median Days to BMT postrandomization (days, range)

30 (23-50)

67 (51-86)

64 (44-161)

Median Days to AbsoluteNeutrophil Count Engraftment(days, range)

15 (9-22)3

18 (13-82)4

14 (10-37)5

Median Day to PlateletEngraftment (days, range)

20 (4-39)3

22 (9-35)4

19 (13-38)5

100-day non-relapsetransplant related mortality(%, N)

6% (2/31)

29% (2/7)

10% (2/20)6

1) No therapeutic dose (6) due to: declining Karnofsky Performance Scale (PFS) (3), Infusion reaction (1), unfavorablebiodistribution (1), post-randomization eligibility (1)

2) Ineligible for crossover (9) due to: hospice care/progression (4), declined/ineligible for BMT (2), died pre-crossover (3),eligible for crossover (2) did not receive Iomab-B due to declining status

3) Absolute Neutrophil Count engraftment data not available (1), platelet engraftment data not available (4)

4) ANC and platelet engraftment data not available (1), engraftment failure (1)

5) ANC engraftment data not available (1) out of 20, platelet engraftment data not available (3)

6) 1 patient at 161 days had delayed transplant due to infection and respiratory failure, received Iomab-B and BMT when stable

Dr. Mark Berger, Actinium's Chief Medical Officer, stated, "As we approach critical enrollment milestones in the SIERRA trial, our focus turns to bringing Iomab-B to as many patients as possible that can benefit from this product candidate and as expeditiously as possible. Consequently, we made an important amendment to the SIERRA protocol to expand the potential patient pool by including in the eligibility criteria patients who fail induction therapy with venetoclax plus hypomethylating agents. As targeted agents such as venetoclax and others have gained approval, the acute myeloid leukemia treatment landscape has evolved with a significant percentage of patients being treated with these agents in frontline and relapsed settings. In fact, as of mid-2019, venetoclax plus hypomethylating agents have been included as part of the AML National Comprehensive Cancer Network guidelines and medical practice is embracing these regimens widely. However, these regimens are not curative, nor do they eliminate the need for BMT. Indeed, approximately thirty percent of patients fail to achieve a remission after two cycles of induction therapy with venetoclax and most patients ultimately relapse with a median duration of response of less than one year. This amendment has already had a positive impact on the trial that we expect to continue through the remaining portion of enrollment. Most importantly, if Iomab-B gains approval, this amendment will support its use for the significant and growing number of patients receiving and failing venetoclax as induction therapy instead of traditional 7+3 induction chemotherapy. We look forward to continuing to provide key updates as SIERRA reaches key milestones and completes enrollment in 2020."

Sources: 1) DiNardo et al. Venetoclax combined with decitabine or azacitidine in treatment-nave, elderly patients with acute myeloid leukemia. Blood 2019 133(1): 7-17 https://doi.org/10.1182/blood-2018-08-868752

About the SIERRA TrialThe SIERRA trial (Study ofIomab-B inElderlyRelapse/RefractoryAcute Myeloid Leukemia) is the only randomized Phase 3 trial that offers BMT (Bone Marrow Transplant) as an option for older patients with active, relapsed or refractory AML or acute myeloid leukemia. BMT is the only potentially curative treatment option for older patients with active relapsed or refractory AML and there is no standard of care for this indication other than salvage therapies. The SIERRA trial is a 150-patient, multicenter randomized trial that studying Iomab-B compared to physician's choice of salvage chemotherapy. The primary endpoint of the SIERRA trial is durable Complete Remission of 180 days and the secondary endpoint is 1-year overall survival. Iomab-B is an ARC or Antibody Radiation-Conjugate comprised of the anti-CD45 antibody apamistamab and the radioisotope I-131 (Iodine-131). The 20 active SIERRA trial sites in the U.S. and Canada represent many of the leading bone marrow transplant centers by volume. For more information, visit http://www.sierratrial.com.

About Transplantation & Cellular Therapy Meetings (TCT)

TCT, formerly known as the BMT Tandem Meetings, are the combined annual meetings of the American Society for Blood and Marrow Transplantation (ASBMT) and the Center for International Blood & Marrow Transplant Research (CIBMTR).Each year the conference brings together several thousand investigators, clinicians, researchers, nurses and other allied health professionals from over 500 transplant centers from over 50 countries around a full scientific program focused on bone marrow transplant and cellular therapies.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3Study ofIomab-B inElderlyRelapsed orRefractoryAcute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over fifty percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. I-131 apamistamab will also be studied as a targeted conditioning agent in a Phase 1/2 anti-HIV stem cell gene therapy with UC Davis and is expected to be studied with a CAR-T therapy in 2020. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 100 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website: https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Investors:Hans VitzthumLifeSci Advisors, LLCHans@LifeSciAdvisors.com(617) 535-7743

Media:Alisa Steinberg, Director, IR & Corp Commsasteinberg@actiniumpharma.com(646) 237-4087

View original content to download multimedia:http://www.prnewswire.com/news-releases/actinium-presents-positive-findings-from-mid-point-analysis-of-pivotal-sierra-trial-of-iomab-b-at-2020-transplantation--cellular-therapy-meetings-of-astct-and-cibmtr-tct-301009012.html

SOURCE Actinium Pharmaceuticals, Inc.

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Actinium Presents Positive Findings from Mid-Point Analysis of Pivotal SIERRA Trial of Iomab-B at 2020 Transplantation & Cellular Therapy Meetings...

TARRYTOWN, NY In 2015, as the Ebola epidemic raged through swaths of West Africa, Kristen Pascals roommates sat her down on their couch and staged an intervention.

Are you sure this is what you want to be doing with your life? she recalls them asking her.

Pascal, a research associate for Regeneron, had been coming home at 2 am and leaving at 6 am. At one point, she didnt see her roommate for a week. For months, that was life in Christos Kyratsous lab as the pair led a company-wide race to develop the first drug that could effectively treat Ebola before the outbreak ended. For Pascal, that was worth it.

Im ok, I dont have Ebola, Pascal told them. I see that death toll rising and I cant not do something about it.

Last August, Regeneron learned they had succeeded: In a large trial across West Africa, their drug, REGN-EB3, was vastly more effective than the standard treatments. It was surprise news for the company, coming just 10 months into a trial they thought would take several years and a major victory in the global fight against a deadly virus that killed over 2,000 in 2019 and can carry a mortality rate of up to 90%.

For Kyratsous and Pascal, though, it brought only fleeting reprieve. Just four months after the NIH informed them REGN-EB3 worked, Kyratsous was back in his office reading the New York Times for updates on a new outbreak on another continent, and wondering alongside Pascal and senior management whether it was time to pull the trigger again.

In late January, as the death toll swelled and the first confirmed cases outside China broke double digits, they made a decision. Soon they were back on the phone with the multiple government agencies and their coronavirus partners at the University of Marylands Level 3 bio lab. The question was simple: Can Kyratsous and his team use a process honed over two previous outbreaks, and create a treatment before the newest epidemic ends? Or worse, if, as world health experts fear, it doesnt vanish but becomes a recurrent virus like the flu?

Christos likes things immediately, Matt Frieman, Regenerons coronavirus collaborator at the University of Maryland, told Endpoints. Thats what makes us good collaborators: We push each other to develop things faster and faster.

Click on the image to see the full-sized version

The first time Regeneron tried to respond to a global outbreak, it was something of a systems test, Kyratsous explains from his office at Regenerons Tarrytown headquarters. Kyratsous, newly promoted, has crammed it with photos of his family, sketches of viral vectors and a shark he drew for his 3-year-old son. He speaks rapidly an idiosyncrasy his press person says has only been aggravated this afternoon by the contents of his Regeneron Infectious Diseases-minted espresso glass and he gesticulates with similar fluidity, tumbling through antibodies, MERS, the novel coronavirus, Ebola-infected monkeys.

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Merck loses its chief digital officer, spotlighting talent hunt for the hottest title in Big Pharma - Endpoints News

WALTHAM Feb 21, 2020 (Thomson StreetEvents) -- Edited Transcript of Repligen Corp earnings conference call or presentation Thursday, February 20, 2020 at 1:30:00pm GMT

* Anthony J. Hunt

Repligen Corporation - CEO, President, Director & Member of Scientific Advisory Board

* Jon K. Snodgres

* Sondra S. Newman

Stifel, Nicolaus & Company, Incorporated, Research Division - MD & Senior Analyst

H.C. Wainwright & Co, LLC, Research Division - Equity Research Associate

* Jacob K. Johnson

William Blair & Company L.L.C., Research Division - Partner & Healthcare Services Analyst

* Joseph P. Munda

Janney Montgomery Scott LLC, Research Division - MD, Head of Healthcare Research & Senior Equity Research Analyst

SVB Leerink LLC, Research Division - MD of Life Science Tools & Diagnostics and Senior Research Analyst

Good day, ladies and gentlemen, and welcome to the Repligen Corporation's Year-End and Fourth Quarter of 2019 Earnings Conference Call. My name is Rocco, and I will be your coordinator. (Operator Instructions) Please note, today's event is being recorded.

I would now like to turn the call over to your host for today's call, Sondra Newman, Global Head of Investor Relations for Repligen. Please go ahead.

Sondra S. Newman, Repligen Corporation - Senior Director of IR [2]

Great. Good morning, everyone. Thanks for joining our call today. On this call, we'll cover our financial results and business highlights for Repligen's fourth fiscal quarter and full year 2019, and we'll provide financial guidance for the year 2020. Our President and CEO, Tony Hunt, will cover business updates and our CFO, Jon Snodgres, will cover our financial results and guidance.

As a reminder, the forward-looking statements that we make during this call, including those regarding our business goals and expectations for financial performance of the company are subject to risks and uncertainties that may cause actual events or results to differ.

Additional information concerning risk factors is included in our annual report on Form 10-K, the current report on Form 8-K, which we filed today and other filings that we make with the Securities and Exchange Commission. Today's comments reflect our current views, which could change as a result of new information, future events or otherwise. And the company does not obligate or commit itself to update forward-looking statements, except as required by law.

During this call, we are providing non-GAAP results and guidance. Reconciliations of GAAP to non-GAAP financial measures are included in the press release that we issued this morning, which is posted to Repligen's website and on sec.gov. The non-GAAP figures in today's report include revenue growth at constant currency, gross profit and gross margin, operating expenses, including R&D and SG&A, operating income and operating margin, income tax expense, net income and earnings per share as well as EBITDA and adjusted EBITDA. These adjusted financial measures should not be viewed as an alternative to GAAP measures, but are intended to better enable investors to benchmark Repligen's current results against historical performance and the performance of peers.

Now I'll turn the call over to Tony Hunt.

--------------------------------------------------------------------------------

Anthony J. Hunt, Repligen Corporation - CEO, President, Director & Member of Scientific Advisory Board [3]

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Thank you, Sondra. Good morning, everyone, and welcome to our 2019 year-end update. We are delighted with the way we finished off the year with 21% organic growth in the fourth quarter, 33% organic growth for the full year and overall 2019 growth for the company, coming in at 39%. All 3 franchises accelerated in 2019 as we benefited from customers scaling up on mAb processes and new customers implementing our technologies, especially in the area of gene therapy, where we now have greater than 50 significant customers today and just as many smaller customers.

Throughout the year, our team continued to execute on our long-term growth strategy with M&A and R&D remaining at the core of our success.

In 2019, we completed our acquisition of C Technologies, establishing an important new vertical for the company in process analytics.

Our R&D team delivered TFDF filtration technology, a game-changing technology and harvest clarification, an area that has seen little innovation over the last 10 to 20 years. Supporting our growth strategy and operational investments in 2019, we completed a series of financings that leave us with over $500 million in cash at year-end and positions us well for the future.

Looking back at the year, there were 5 key drivers of growth. First, as I mentioned, we saw strong adoption of gene therapy accounts where we help to deliver yield and efficiency improvements in viral vector manufacturing. Gene therapy represented approximately 15% of our overall 2019 revenue with filtration products accounting for 60% and chromatography products accounting for 35% of sales into these accounts. We saw a fairly even split between CDMO and gene therapy developers at 52% and 48% of revenue, respectively. And with the strong base of core customers and differentiated technology, we are really well positioned for growth in gene therapy. Second, we saw increased adoption of our XCell ATF products in both N-1 seed train, a traditional perfusion applications. The expansion of ATF applications into fed-batch processes is a key growth driver for us as it is the catalyst for sustained long-term growth north of 20%.

Third, we saw significant uptick of our TFF systems portfolio where we put more focus on tying systems and consumables into targeted upstream and downstream applications. We made the decision 12 months ago to focus on building out systems for our TFF portfolio. And not only has this team delivered on more traditional UF/DF applications, but they've also delivered on the development and launch of TFDF technology and fed-batch harvest clarification.

Fourth, we saw accelerated adoption of our OPUS prepacked columns as CDMOs and pharma customers continue to scale and expand. Combined with new demand for OPUS and gene therapy, we delivered approximately 1,400 comps to our customer base in 2019, up from 700 comps in 2018.

To ensure our ability to stay ahead of demand, we made significant investments in 2019 and here again in 2020 to expand our capacity and resources at OPUS production. And finally, just the overall strength of the biologics market, where in the U.S. alone, 10 new mAbs and 2 gene therapy drugs were approved in 2019. With a rich pipeline of over 1,000 biological drug candidates, the expectations are high for strong growth in the years ahead.

Before jumping into the quarter, I also wanted to highlight some of our key accomplishments in 2019. Starting with C Technologies, which was our first acquisition in process analytics. During our 7 months of C Tech ownership in 2019, we focused our efforts on commercial expansion, R&D acceleration and building out the financial team and implementing public company processes at C Tech.

We successfully built our commercial team with 10 dedicated reps, we focused R&D efforts on accelerating next-gen FlowVPE, and we completed the build-out of the finance team. For the year, C Tech products contributed close to $16.4 million in revenue, right in line with our $16 million to $17 million projection. We look forward to seeing the impact of our new commercial team in 2020, where we expect C Technologies to generate approximately $32 million in revenue.

Moving now to R&D. 2019 was a year where we made significant progress. A key goal for us entering the year was to develop and launch TFDF technology. We did a very successful technical launch of the product in September, and then our systems team followed up with the development of benched-up and production scale systems, which will be launched here in Q1. We also made progress with our next-gen ATF controller, which is scheduled to launch in late Q1, early Q2. In addition, key products like SIUS gamma and next-gen ligands are reaching their final stages of product development and will soon be available in the marketplace. From a capacity and infrastructure standpoint, 2019 was a huge year for us. We increased our office capacity fivefold and have dedicated programs in place to expand again in 2020. Our operations team also expanded and centralized manufacturing of XCell ATF in Marlborough and completed the Phase I implementation of SAP. Financially, we were able to raise close to $500 million net through a series of equity and convertible debt financings, again, putting us in a strong position for future M&A.

So moving now to our Q4 results and full year 2019 performance. As reported today, we had a great quarter with $69.5 million in sales. The story of the quarter was our organic growth performance against difficult comps. Each of our proteins, filtration and chromatography franchises performed well and together delivered 20% plus organic growth in the quarter and 20%, 30% and 40% organic growth, respectively, for the year.

In filtration, our XCell ATF product line had a record year. Our customers scaled up into late stage trials, and we saw increased use of ATF and N-1 seed train applications, reflecting a broader application of the technology into fed-batch processes. Single-use ATF continued to perform well in 2019, with revenue growth of 45%. Our SIUS flat sheet TFF cassette business also had a very strong quarter and year, up over 25%. The story continues to be around new accounts and key wins in gene therapy applications. Our hollow fiber portfolio also had a good quarter in Europe, 30% with the robust demand for single-use flow paths, hollow fiber modules and KrosFlo systems. We expect that our hollow fiber business will have another strong year in 2020. And our overall filtration franchise will grow approximately 25%.

Moving to chromatography for our OPUS business finished up over 30% for the quarter and 50% for the year. The story in the quarter was the continued adoption of our prepacked column technology in CDMOs and gene therapy accounts, which now account for 20% of our OPUS revenue. In parallel, we were able to improve our lead times significantly by bringing 5 new production suites online in December. We also saw accelerating adoption to focus ATF columns, as customers put our technology into late-stage and commercial processes.

We expect continued momentum for OPUS in 2020 as our customers scale and expand. We expect our OPUS franchise to grow at or above 20%, with overall chromatography at 15% in 2020.

Our OEM proteins business performed well in Q4 and was up 20% for the full year, led by strengthened growth factors with 50% growth from ligands. As mentioned, throughout 2019, we expect GE to transition to in-house ligand manufacturing here in 2020, creating a $12 million to $13 million or 4% to 5% headwind for the company. We expect that NGL-Impact A ligand will continue to gain traction in the marketplace as customers implement this technology in early-stage clinical trials. So overall, we expect the company to grow at 14% to 18% in 2020, with organic growth in the 10% to 14% range.

We expect H2 to be stronger than H1 as some of the larger scale-up projects is in the second half of 2020. Unlike other companies, it will take a few more months to understand what the long-term impact might be on overall company performance from the coronavirus in China in fiscal year 2020. For quarter 1, there may be some shift in revenue into Q2 based on customer timing.

As we move into 2020, our strategic priorities will center on the following: new product launches with a focus on TFDF and ATF controllers, expanding our market presence in harvest clarification applications with TFDF technology, further expanding our market presence in gene therapy through our filtration and chromatography franchises; broadening the customer base and applications for C Technologies; implementing capacity expansion and operating margin improvement programs; and finally, evaluating M&A opportunities to supplement our organic growth.

In summary, we believe we are well positioned to gain further share by processing. We believe that the blueprint we've put in place over the last 5 years around building out a world-class commercial team, bringing disruptive technologies to market and supplementing our technology base with select M&As will be the catalyst for growth over the coming years. Also, based on the investments we've made in R&D over the last 3 years, we expect 2020 to be a milestone year for us in terms of product launches.

Before concluding, I wish to recognize our employees around the globe for their commitment and leadership in 2019. I also want to thank our loyal shareholders and customers for their parts in Repligen's success and we look forward to another strong year for the company and bioprocessing.

With that, I'll turn the call over to Jon for a more detailed financial report.

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Jon K. Snodgres, Repligen Corporation - CFO & Secretary [4]

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Thank you, Tony, and good morning, everyone. Today, we are reporting our financial results for the fourth quarter and full year 2019, as well as providing our financial guidance for the year 2020. Unless otherwise mentioned, all financial measures discussed reflect non-GAAP measures. As you've seen in our press release this morning, we delivered strong financial performance for both the fourth quarter and full year 2019. We had another strong quarter with revenues of $69.5 million, representing 34% reported growth and 21% organic growth. For full year 2019, we reported record revenue of $270.2 million. The year-over-year increase of $76 million represents 39% overall growth, including 2 points of foreign currency headwind and 33% organic growth, $16 million or 8% of our revenue growth was attributed to our June 2019 acquisition of C Technologies.

Operationally, we continue to prioritize key areas of investments in the company. In 2019, 7% of revenue supported R&D programs that continue to produce disruptive technologies, like our new TFDF platform that we launched in the second half of the year. We also made important capital investments in our manufacturing sites, with a significant portion of our spend dedicated to increasing overall production capacity and expanding and upgrading our IT systems and infrastructure.

Now to provide more insights into our overall financial performance. As Tony mentioned earlier, our direct-to-customer franchises continue to perform well. Our direct products represented 79% of the company's total revenue in the fourth quarter and 76% for the full year compared to 72% for full year 2018. On a regional basis, for the full year, pro forma direct product revenue growth was fairly consistent across regions, with approximately 30% growth in North America, Europe and Asia. For the full year 2019, Asia represented 16% of direct revenue, while Europe and North America accounted for 28% and 56%, respectively.

Now moving to our income statement. Fourth quarter adjusted gross profit was $39.8 million, representing an increase of $11.3 million or 40% over the fourth quarter of 2018. Our adjusted gross margin was 57.2% for the fourth quarter of 2019 compared to 54.8% for the same period in 2018. The 240 basis point improvement was driven by favorable OPUS column to resin mix in our chromatography franchise and by increased demand for growth factor products in our proteins franchise. Full year 2019 adjusted gross profit of $154.1 million reflects an increase of $45.9 million or 42% compared to the full year of 2018. Adjusted gross margin was 57% for the full year 2019 compared to 55.8% for 2018, with the 120 basis point increase, driven by overall sales volume leverage in our factories, our new C Technologies product line and favorable product mix, partially offset by investments in capacity and operations.

With respect to operating expenses. Adjusted research and development costs for the fourth quarter of 2019 were $4.9 million compared to $3 million for the fourth quarter of 2018. For the full year 2019, adjusted R&D expenses were $18.8 million compared to $15.7 million in 2018. Key drivers of the year-over-year increase were the timing of our C Technologies acquisition, investments in our next-generation filtration technologies, as well as continued investments in our ligand programs.

Overall, R&D expenses finished the year at 7% of revenue.

Adjusted SG&A for the fourth quarter of 2019 was $22.2 million compared to $14.4 million for the fourth quarter of 2018. Full year adjusted SG&A was $71.8 million in 2019 compared to $53.1 million in 2018. The year-over-year increase in adjusted SG&A was related to the timing of our C Technologies acquisition and the build-out of our process analytics commercial team, as well as expansions and enhancements of our facilities, IT systems and commercial team.

Now moving to adjusted earnings and EPS. In the fourth quarter of 2019, our adjusted operating income was $12.7 million, a 15% increase compared to $11.1 million reported in the fourth quarter of 2018. Our adjusted operating margin was 18.3% compared to 21.3% for the fourth quarter of 2018. Our operating costs during the fourth quarter of 2019 included approximately $1 million of nonrecurring expenses, mostly in IT and recruiting. For the full year of 2019, our adjusted operating income was $63.5 million, a 61% increase compared to $39.4 million for the full year of 2018.

Our 2019 full year adjusted operating margin was 23.5%, a 320 basis point improvement compared to 20.3% for the 2018 period, reflecting a strong year of growth and operational execution in our business.

Adjusted net income for the fourth quarter of 2019 was $10.8 million, an increase of 21% compared to $8.9 million in the same period in 2018. Full year 2019 adjusted net income was $52.5 million, an increase of 74% compared to $30.1 million for the full year 2018.

Adjusted EPS for the fourth quarter of 2019 increased to $0.20 per fully diluted share from $0.19 for the fourth quarter of 2018.

For the full year of 2019, adjusted EPS increased to $1.07, up 62% from $0.66 in 2018.

Our cash and cash equivalents, which are GAAP metrics, totaled $528.4 million at December 31, 2019. For full year 2019, we generated free cash flow of $44.1 million, inclusive of $67.2 million of operating cash flow, less $23.2 million of capital investments primarily related to our facility and capacity expansion projects and IT systems investments.

Now moving to 2020 full year guidance. Our GAAP to non-GAAP reconciliations for our 2020 financial guidance are included in the reconciliation tables in today's earnings press release. As previously mentioned, unless otherwise noted, all 2020 financial guidance discussed will be non-GAAP. Please also keep in mind that our 2020 guidance may be impacted by fluctuations in foreign exchange rates beyond our current projection of a net 0 impact on full year sales and does not include the potential impact of any new acquisitions that the company may pursue.

Today, we are setting our 2020 full year revenue guidance, a GAAP metric at $309 million to $319 million, reflecting growth in the range of 14% to 18% as reported and 10% to 14% on an organic basis. Our adjusted gross margin guidance for 2020 is 55% to 56%, which reflects the impacts of expected headwinds from lighter GE volumes and investments in our facilities, capacity, IT systems and staffing to support expected strength and long-term market demand and overall growth. Adjusted operating income is expected to be in the range of $70 million to $74 million, with adjusted operating margins in the range of 22% to 23% of revenue for the year.

We are expecting 2020 adjusted income tax expense of approximately 23% of adjusted pretax income, which anticipates impacts from strong international revenue growth in higher tax rate countries in Europe and Asia.

We are expecting full year 2020 adjusted net income in the range of $57 million to $60 million for the year, and adjusted EPS in the range of $1.07 to $1.12 per fully diluted share. Please note that our 2020 adjusted EPS guidance reflects a $0.09 dilution impact due to share count increases, primarily related to our 2019 financing activities.

Our guidance reflects an estimated 53.4 million fully diluted shares outstanding for the full year, an increase of approximately 4 million. Adjusted EBITDA is now expected to be in the range of $80 million to $84 million for the full year 2020, with depreciation and intangible amortization expense is expected to be approximately $10.5 million and $15.5 million, respectively.

The company again expects to invest an estimated $20 million to $22 million in 2020 for capital expenditures as we proceed with our build-out of our OPUS manufacturing facility in Breda, planned capacity expansions in our Massachusetts and California facilities and with continued investments in SAP.

We expect 2020 year-end cash and cash equivalents, a GAAP metric to be in the range of $580 million to $590 million, with our CapEx investments being fully funded by cash generation from our operations. As you can see, we've executed on another strong year of performance in 2019, including making substantial investments in the company as part of our 2-year plan to prepare us for continued long-term growth.

This completes our financial report, and I will now turn the call back to the operator to open the lines for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Today's first question comes from Dan Arias of Stifel.

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Daniel Anthony Arias, Stifel, Nicolaus & Company, Incorporated, Research Division - MD & Senior Analyst [2]

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Maybe just to start on guidance. 32% organic is a pretty big year. So obviously, you have a tough comp there. When you look at the momentum that you're exiting 2019 with though, can you just talk about the approach to the 2020 outlook in terms of being conservative or not conservative? And then how you feel about just the shape of the business and the drivers that you have relative to this time last year when you guided to, I believe, 13% to 17% organic growth. So less than half of what you ended up doing?

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Anthony J. Hunt, Repligen Corporation - CEO, President, Director & Member of Scientific Advisory Board [3]

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Yes. Thanks, Dan. Yes, I think in general, we finished off the year. We had good momentum through the second half of the year. I think as we looked at 2020 guidance, I think it was important for us to really gauge what was going on in each of the businesses, right, in each of the divisions. So within chromatography and filtration, we feel pretty good about it. Our OPUS business came off a massive year last year, up over 50%. And our filtration product line also had a really, really big year. So we've got really difficult comps in 2020. We expect that our filtration franchise will grow 25%. We think that our OPUS franchise will grow 20% plus. Our proteins business is probably the main headwind that we have and so we know we have a 4% to 5% headwind going into 2020. So if you take our 10% to 14% guidance. And we didn't have that headwind, we would really be doing 14 -- 15% to 19%. So we think, actually, the guidance we're putting out there right now is very realistic. While we feel really good about our businesses, really good about our products, we think this is the right guidance for us.

To your second part of your question about last year where we guided mid-teens, and we came in at 30%. I think the piece that really surprised us last year was the proteins business, right? And we had guided down 5%, and it came in really, really strong. So if you take that out of the equation, it would really drop down the overall organic growth pretty significantly. So I think that was a big factor. I think the other factor last year is that no one really in the industry predicted how fast the gene therapy market was going to grow. And I think we all kind of missed that a little bit. And so that's kind of the explanation for last year.

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Daniel Anthony Arias, Stifel, Nicolaus & Company, Incorporated, Research Division - MD & Senior Analyst [4]

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Yes. Okay. That's actually my second question. Just on gene therapy, is there some color you can give on just how you feel about the expansion and the evolution of that market in terms of new activity and the scale of our project work. And then I guess, along those lines, I mean, one of the things that it sounded like you were trying to stay grounded on last year was this idea that a lot of things went right in 2019 in that field. And that, that always doesn't happen, Phase I don't always go to Phase II, et cetera. So I guess the question is that sitting 2 months into the new year, how are you looking at things relative to the way that you would have hoped they would have been 6 to 12 months ago?

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Anthony J. Hunt, Repligen Corporation - CEO, President, Director & Member of Scientific Advisory Board [5]

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Yes. I think when you look at how we finished in Q4, the gene therapy customer base was again strong for us in terms of shipments, in terms of orders as we -- it's difficult for us to see much beyond the first half of the year. We know, as I said earlier in my prepared remarks that we know there are some significant scale-up programs that are happening in the second half of the year. But in general, we haven't seen any slowdown in gene therapy, and we're expecting gene therapy will drive -- will grow about 30% for us in 2020.

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Daniel Anthony Arias, Stifel, Nicolaus & Company, Incorporated, Research Division - MD & Senior Analyst [6]

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Okay. It sounds like you've given the 3 questions here, so I'll take the opportunity. Maybe just on TFDF, that sounds like it's one of the more meaningful products in the portfolio these days. So what is the thought on just the industry coming around to the dual filtration benefit that you get there. My sense is that, that might take 1 year or 2 for that to ramp and be a material contributor. Is that the way that you're thinking about it? And then what does the runway look like if you wanted to look beyond just this year and into 2021 and 2022?

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Anthony J. Hunt, Repligen Corporation - CEO, President, Director & Member of Scientific Advisory Board [7]

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Yes, I think you have it spot on. The -- it's going to take a couple of years for TFDF to take off, but we've got a significant number of trials lined up here in Q1. We expect it will generate $1 million, $2 million in revenue for us this year. But every year, going forward, we expect that revenue will double. And so it's not one of those product lines that we expect to grow at 20%, 25%, 30%. We expect that over the next few years, this is a technology that's going to scale quickly. But obviously, we've to get through the trials, we've got to prove the technology. We've done that so far, but on a limited basis, and I think we're very confident that the technology is going to be a key technology for us. That said, I wouldn't underestimate the rest of the product launches that we're bringing to market this year. So we really are bringing innovation in ATF. We're bringing out the next-generation FlowVPE technology in the second half of the year. We've got new ligands coming through. We've got next-generation, SIUS gamma. Really feel this year is a key year for us in terms of product launches that will set us up really well for 2021 and beyond.

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Continued here:
Edited Transcript of RGEN earnings conference call or presentation 20-Feb-20 1:30pm GMT - Yahoo Finance

Presented positive clinical and pre-clinical data from its MarzAA and DalcA hemophilia programs

Announced a global license and collaboration agreement with Biogen to develop and commercialize pegylated CB 2782 for Dry AMD

SOUTH SAN FRANCISCO, Calif., Feb. 20, 2020 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc. (CBIO), today announced its operating and financial results for the fourth quarter and full-year ending December 31, 2019 and provided a corporate update.

We made exceptional progress in both our subcutaneously-dosed (SQ) MarzAA (FVIIa) and SQ DalcA (FIX) programs this past year. Earlier this month at EAHAD, positive data was presented in an oral presentation from the Companys Phase 2b SQ DalcA trial in subjects with hemophilia B clearly demonstrating efficacy and safety. We also presented data from MarzAA and Factor IX gene therapy programs in three posters, said Nassim Usman, Ph.D., president and chief executive officer of Catalyst. Catalysts therapies have demonstrated the potential to effectively treat hemophilia subcutaneously in a $3.4 billion market.

Dr. Usman continued, "Additionally, our February 2020 financing combined with our current cash provides funding through several major milestones for our lead Phase 3 ready MarzAA and Phase 2b DalcA product candidates, as well as our FIX gene therapy and complement inhibitor programs."

Recent Milestones:

Expected Milestones

Fourth Quarter and Full-year 2019 Results and Financial Highlights

About Catalyst Biosciences

Catalyst is a clinical-stage biopharmaceutical company focused on addressing unmet needs in rare diseases and systemic complement mediated disorders. Our protease engineering platform includes development programs in hemophilia and a research program on subcutaneous (SQ) systemic complement inhibitors. Our engineered coagulation factors are designed to overcome the significant limitations of current IV treatment options, facilitate prophylaxis, and ultimately deliver substantially better outcomes for patients using SQ dosing. Our lead asset, MarzAA has completed Phase 2 development having met its primary endpoint of significantly reducing the annualized bleed rate (ABR) in individuals with hemophilia A or B with inhibitors. Our second hemophilia asset, DalcA is completing a Phase 2b clinical trial and is being developed for the treatment of hemophilia B. We also have a global license and collaboration agreement with Biogen for the development and commercialization of pegylated CB 2782 for the potential treatment of geographic atrophy associated dry age-related macular degeneration.

For more information, please visit http://www.catalystbiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential uses and benefits of MarzAA and DalcA to effectively and therapeutically treat hemophilia subcutaneously, the potential market opportunity for MarzAA and DalcA, plans to start a Phase 3 trial of MarzAA in second half of 2020 and report final data in the second quarter of 2020 from a MarzAA Phase 1 pharmacokinetic and pharmacodynamic study to support future SQ treatment of bleed studies, to announce final Phase 2b trial data for DalcA in the second quarter of 2020, plans to announce primate data for Factor IX gene therapy in the second quarter of 2020, and potential future milestone and royalty payments from Biogen. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that trials and studies may be delayed and may not have satisfactory outcomes, that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of DalcA, or MarzAA, including the generation of antibodies, which has been observed in patients previously treated with DalcA, the risk that costs required to develop or manufacture the Companys products will be higher than anticipated, the risk that Biogen will terminate our agreement with them, competition and other risks described in the Risk Factors section of the Companys quarterly report filed with the Securities and Exchange Commission on November 7, 2019, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.

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Contact:

Ana KaporCatalyst Biosciences, Inc.investors@catbio.com

Catalyst Biosciences, Inc.Consolidated Balance Sheets(In thousands, except shares and per share amounts)

Catalyst Biosciences, Inc.Consolidated Statements of Operations(In thousands, except share and per share amounts)

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Catalyst Biosciences Reports Fourth Quarter and Full-Year 2019 Operating & Financial Results and Provides a Corporate Update - Yahoo Finance

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Cell and Gene Therapy Consumables Market 2019-2026 by Top Key Players, Industry Size, Share, Demand, Revenue Overview Report - Instant Tech News

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The key players influencing the market are:

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Gene Therapy Market report also provide a in-depth understanding of the cutting-edge competitive analysis of the emerging market trends along with the drivers, restraints, and opportunities in the market to offer worthwhile insights and current scenario for making right decision. The report covers the prominent players in the market with detailed SWOT analysis, financial overview, and key developments of last three years. Moreover, the report also offers a 360 outlook of the market through the competitive landscape of the global industry player and helps the companies to garner Gene Therapy Market revenue by understanding the strategic growth approaches.

Gene Therapy Market is a combination of qualitative as well as quantitative analysis which can be broken down into 40% and 60% respectively. Market estimation and forecasts are presented in the report for the overall global market from 2020 2027, considering 2020 as the base year and 2020 2027 forecast period. Global estimation is further broken down by segments and geographies such as North America, Europe, Asia-Pacific, Middle East & Africa and South America covering major 16 countries across the mentioned regions. The qualitative contents for geographical analysis will cover market trends in each region and country which includes highlights of the key players operating in the respective region/country, PEST analysis of each region which includes political, economic, social and technological factors influencing the growth of the market.

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Gene Therapy Market Growth, Trends and Demands Research Report and Forecast 2027 - Instant Tech News

Viral Vector and Plasmid DNA Manufacturing Market with Insights and Key Business Factors

Reports Monitor offers its latest report on the [Global Viral Vector and Plasmid DNA Manufacturing Market size and CAGR between 2020 and 2025.] report that includes comprehensive analysis on a range of subjects such as segmentation, competition, market dynamics and regional expansion. The report offers highly detailed competitive analysis of theGlobal Viral Vector and Plasmid DNA Manufacturing Market, where the business and industry growth of leading companies are thoroughly evaluated on the basis of production, recent developments, technology, geographical footprint, and various other factors. This will help players to prepare themselves well for any unforeseen situations in the industry competition and give a tough competition to other players in the global Viral Vector and Plasmid DNA Manufacturing Market.

TheMajor Manufacturers Covered in this Report:Merck, uniQure, The Cell and Gene Therapy Catapult, Waisman Biomanufacturing, Addgene, Creative Biogene, Novasep, Cobra Biologics, Aldevron, , and more.

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Scope of the ReportThe research report provides key information on the supply chain of the industry, the markets currency chain, pools of major companies, and market segmentation, geographical market, and technology-oriented perspectives.The authors of the report have also provided qualitative and quantitative analyses of several microeconomic and macroeconomic factors impacting the global Viral Vector and Plasmid DNA Manufacturing market. In addition, the research study helps to understand the changes in the industry supply chain, manufacturing process and cost, sales scenarios, and dynamics of the global Viral Vector and Plasmid DNA Manufacturing market.

Market segment by Type, the product can be split intoAdenovirusRetrovirusPlasmid DNAOthers

Market segment by Application, the product can be split intoBiopharmaceutical CompaniesResearch Institutes

On the basis of geography, the market is segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa.

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Key Benefits for Viral Vector and Plasmid DNA Manufacturing Market:A.In-depth analysis of the market is conducted by constructing market estimations for the key market segments between 2020 and 2025. The report provides an extensive analysis of the current and emerging Viral Vector and Plasmid DNA Manufacturing market trends and dynamics.B. Key market players within the market are profiled in this report and their strategies are analyzed thoroughly, which helps to understand the competitive outlook of the industry.D.Extensive analysis of the market is conducted by following key product positioning and monitoring of the top manufacturers within the market framework.E. A comprehensive analysis of all the regions (North America, Europe, Asia-Pacific, South America, The Middle East and Africa)

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Further, the Viral Vector and Plasmid DNA Manufacturing industry research report determines the Marketing Analysis, Regional Market Analysis, International Trade Analysis. The market Traders or Distributors with Contact Information by Region and Supply Chain Analysis. That is followed by various business strategies, the report contains essential outcome help could boost the interest level of the individuals in the market.

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Viral Vector and Plasmid DNA Manufacturing Market 2020 Regional and Growth Opportunity by Top Players: Merck, uniQure, The Cell and Gene Therapy...

Audentes Therapeutics, an Astellas company, announced it is building a gene therapy manufacturing plant in Sanford, North Carolina.

The company is investing $109 million in a new 135,000-square-feet facility, with the initial phase to take place over about 18 months. It is planned to go into operation in 2021. It is expected to create more than 200 new jobs in Lee County, North Carolina. Hiring is expected to start this year.

Our investment in large-scale manufacturing has always been a cornerstone of our strategy to develop and ultimately deliver our important genetic medicines to patients as rapidly as possible, said Natalie Holles, president and chief executive officer of Audentes. This new facility in Sanford will support the next phase of our growth as we establish a robust, global supply chain and expand our therapeutic and geographic scope as a part of the Astellas group of companies. We are excited to join the vibrant biopharmaceutical research and manufacturing community that the state of North Carolina has established.

Audentes is headquartered in San Francisco and focuses on gene therapy. It was acquired by Tokyo-based Astellas Pharma in January.

No specifics were given about what the site will manufacture. In October 2019, Audentes announced positive data from ASPIRO, the clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). AT132 is an AAV8 vector that contains a functional copy of the MTM1 gene. XLMTM is a serious, life-threatening, rare neuromuscular disease marked by extreme muscle weakness, respiratory failure and early death.

The company indicates it hopes to submit a Biologics License Application (BLA) for AT132 to the U.S. Food and Drug Administration (FDA) later this year.

An announcement ceremony was held in the industrial shell building the company is buying in the Central Carolina Enterprise Park. It is about 45 miles southwest of Raleigh.

Gov. Roy Cooper stated, With our powerhouse research centers and highly skilled workforce, biotech pioneers recognize North Carolinas role as a leader in the life sciences. Lee County is a perfect fit for Audentes as they seek to become a global leader in genetic medicines.

The employees at the new factory are expected to earn an average salary of $83,900, which is a little over twice the Lee County average of $41,800. If Audentes hits hiring milestones, it will qualify for a state Job Development Investment Grant worth up to $3.7 million.

The county and the city of Sanford are also offering $5.7 million incentives, which includes almost $400,000 in training support from the North Carolina Community College System.

Audentes chose the location over California, Massachusetts and Colorado.

In every interaction, I was impressed with Audentes patient-centric approach to developing their AAV-based gene therapy to transform the lives of affected patients and families, said Laura Rowley, NCBiotechs director of life science economic development. She led the Centers outreach activity with Audentes. Their decision to grow in North Carolina reflects the Research Triangle regions specialized training capabilities and strengths in gene therapy and biomanufacturing. The passion and focus of the Audentes team makes me confident that they will be an outstanding addition to North Carolinas gene therapy community.

Audentes is acting as the Center of Excellence for Astellas newly founded Genetic Regulation Primary Focus.

Audentes Therapeutics is joining one of the nations top life science clusters, said Anthony M. Copeland, North Carolinas Commerce Secretary. North Carolina has the largest biomanufacturing workforce in the nation and a growing concentration of gene therapy scientists, researchers and workers.

The site of the new plant is quite close to Pfizers new gene therapy campus, which is under construction. That $600 million research and manufacturing facility has a 230-acre campus in Sanford and will employ 340 people.

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Audentes to Build $109 Million Gene Therapy Factory in North Carolina - BioSpace

Image caption Matthew Wood hopes the gene therapy will help him keep his remaining vision

A new gene therapy has been used to treat patients with a rare inherited eye disorder which causes blindness.

It's hoped the NHS treatment will halt sight loss and even improve vision.

Matthew Wood, 48, one of the first patients to receive the injection, told the BBC: "I value the remaining sight I have so if I can hold on to that it would be a big thing for me."

The treatment costs around 600,000 but NHS England has agreed a discounted price with the manufacturer Novartis.

Luxturna (voretigene neparvovec), has been approved by The National Institute for Health and Care Excellence (NICE), which estimates that just under 90 people in England will be eligible for the treatment.

The gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents. The gene is important for providing the pigment that light sensitive cells need to absorb light. Initially this affects night vision but eventually, as the cells die, it can lead to complete blindness.

An injection is made into the back of the eye - this delivers working copies of the RPE65 gene. These are contained inside a harmless virus, which enables them to penetrate the retinal cells. Once inside the nucleus, the gene provides the instructions to make the RPE65 protein, which is essential for healthy vision.

Matthew Wood started losing his sight as a child, and is now registered blind. However, he does have some peripheral vision and can detect large objects and bright lights. He told the BBC: "Since I was a child I was continually told there was no treatment for this condition, so it's amazing to receive this gene therapy."

Mr Wood, from London, had his right eye treated during an hour-long operation at the John Radcliffe Hospital in Oxford.

His left eye will be injected in a few weeks. The surgery was carried out by Prof Robert MacLaren, who has pioneered research into gene therapies for preventing blindness.

He told the BBC: "This is very exciting - this is the first approved NHS gene therapy for an eye disease, but there are opportunities to use gene therapy to treat other diseases in future, not only in the eye."

The treatment is only suitable for patients who have some remaining vision. It should bring the biggest benefits to children with RPE65 retinal dystrophy, as it could halt sight loss before permanent damage is done.

It is not known how long the benefits of the treatment will last, but it's thought it could be several decades.

Jake Ternent, 23, from Durham, had his gene therapy at Moorfields Eye Hospital in London.

Like Matthew Wood, he is registered blind, but has some limited sight. He told the BBC: "I hope the treatment could improve my night vision, and possibly even my day vision, which would be incredible. I feel lucky and privileged to get this on the NHS."

Prof James Bainbridge - from Moorfields Eye Hospital - who treated Jake, told the BBC: "To be at the point now where we are able to offer this treatment on the NHS, is truly remarkable. This is the first example of what's anticipated to be a whole new generation of treatments."

It will take a month or two before Matthew and Jake know what changes the gene therapy has made to their vision. But even if it simply prevents further sight loss, both say they will be delighted.

Professor Stephen Powis, NHS medical director, said: "Loss of vision can have a devastating effect, particularly for children and young people, but this truly life-changing treatment offers hope to people with this rare and distressing condition."

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Gene therapy to halt rare form of sight loss - BBC News

Industry leaders, patient advocates, researchers unite to maximize the incredible potential of transformative gene therapies

WASHINGTON--(BUSINESS WIRE)-- The Institute for Gene Therapies (IGT) launched today with a focus on advocating for a modernize the U.S. regulatory and reimbursement framework so that gene therapies can deliver their significant potential to patients. IGT will educate stakeholders across the healthcare community about the transformational nature of gene therapies and advocate for policies that help ensure patients who need them can benefit from them.

Gene therapy is poised to change human health as we know it. By altering non-functioning genes or replacing absent ones, gene therapies have the potential to reshape the way thousands of diseases are treated with long-lasting effects for patients. The first of these transformative therapies have already been approved by the U.S. Food and Drug Administration (FDA) and hundreds more are currently being studied in clinical trials for rare and common diseases, including many types of cancer, neuromuscular diseases, blood disorders and infectious diseases, among others.

Many crippling conditions like Charcot-Marie-Tooth, which I was diagnosed with before the age of two take hold at a very young age, cut lives far too short or cause ongoing daily suffering, said Susan Ruediger, CEO of the CMT Research Foundation (CMTRF) and member of the IGT Patient Advocacy Advisory Council. Like so many diseases, CMT currently has no cure. I am proud to stand with other leading patient advocates, members of the research community and companies that are developing gene therapies to help ensure patients can fully realize the benefits of these giant leaps toward treatments and cures.

Gene therapies are fundamentally different from traditional pharmaceutical and biologic medicines in that they target the cause of the disease at the DNA level to create a change in the body. Further, some gene therapies are designed to be one-time treatments that offer life-long benefits. Today, the vast majority of medicines help manage the symptoms of disease over time rather than address or halt diseases at their root. The U.S. healthcare system from the drug approval process to the way treatments are paid for reflects this reality. The existing regulatory and reimbursement structures, which were established and adjusted over time to accommodate pharmaceutical and biologic medicines, need revisiting in light of gene therapies and their significant potential.

The incredible scientific advancements in this space present unique opportunities to directly improve and save the lives of patients suffering from debilitating diseases, said IGT Chairman, and former Congressman Erik Paulsen. This is not some far-off future patients are already benefiting from the first FDA-approved gene therapies. But we need policy to move faster toward this new reality where we can treat the causes of many diseases. The Institute for Gene Therapies and our members believe unique regulatory and reimbursement structures need to be established, novel development pathways need to be embraced and new value-based arrangements need to be tested.

As part of IGTs effort, experts from across the healthcare system will work together to ensure health policies reflect the latest medical advances, remove barriers that hinder patient access to gene therapies and advocate for sustainable, long-term solutions. IGT will work to ensure a greater understanding about the value gene therapies bring to patients, families, the healthcare system and our society so that gene therapies can achieve their full potential.

About the Institute for Gene Therapies

The Institute for Gene Therapies (IGT) works with stakeholders across the healthcare system to advocate for a modernized regulatory and reimbursement framework that encourages the development of transformative gene therapies and promotes patient access. Members of our advisory councils include Johnson & Johnson, PTC Therapeutics, Sarepta Therapeutics, Spark Therapeutics, Patient Advocate Foundation, Cure SMA, CMT Research Foundation, American Autoimmune Related Diseases Association (AARDA), Khrystal Davis, Founder of Texas Rare Alliance, Jenn McNary, Founder of One Rare, Seth Rotberg, Co-Founder and Head of Strategy & Engagement of Our Odyssey, Rolf Benirschke, Patient Advocate for Crohns disease, ulcerative colitis, colorectal and bladder cancer, Friedreichs Ataxia Research Alliance, and Foundation Fighting Blindness. For more information, visit gene-therapies.org and follow us on Twitter @gene_therapies

View source version on businesswire.com: https://www.businesswire.com/news/home/20200219005526/en/

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New Institute Launched to Ensure the US Healthcare System Is Ready for Gene Therapies - BioSpace