Newtown Apr 2, 2020 (Thomson StreetEvents) -- Edited Transcript of Onconova Therapeutics Inc earnings conference call or presentation Tuesday, March 24, 2020 at 8:30:00pm GMT

* Abraham N. Oler

Onconova Therapeutics, Inc. - VP of Corporate Development & General Counsel

Onconova Therapeutics, Inc. - CFO

Onconova Therapeutics, Inc. - Chief Medical Officer and Senior VP of Research & Development

* Steven M. Fruchtman

Onconova Therapeutics, Inc. - CEO, President & Director

Laidlaw & Company (UK) Ltd., Research Division - MD of Healthcare Research & Senior Biotechnology Analyst

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

Good afternoon, and welcome to Onconova Therapeutics Corporate Update and Full Year 2019 Financial Results Conference Call. (Operator Instructions) As a reminder, this call may be recorded.

At this time, I would like to turn the call over to Avi Oler, Senior Vice President of Corporate Development and General Counsel.

Abraham N. Oler, Onconova Therapeutics, Inc. - VP of Corporate Development & General Counsel [2]

Thank you, operator. Good afternoon, and welcome to Onconova's fourth quarter and full year 2019 corporate update and financial results conference call. Earlier this afternoon, we issued a press release outlining our financial results and business progress during the year. If you have not seen this press release, it is available on the Investor Relations page of our website at http://www.onconova.com.

On today's call, Dr. Steve Fruchtman, President and CEO, will discuss the company's recent highlights and anticipated clinical and business milestones. After Steve completes his opening remarks, Mark Guerin, our CFO, will review our 2019 financial results. Following Mark's report, we will move to the Q&A portion of the call, which will be joined by Dr. Rick Woodman, our Chief Medical Officer. Lastly, Steve will come back with some final brief comments and a review of upcoming milestones.

Before we begin, I'll remind everyone that statements made today during this conference call will include forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially. Forward-looking statements speak only as of the date they are made as the underlying facts and circumstances may change. Except as required by law, Onconova disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances. Please see the forward-looking statements disclaimer in the press release issued this afternoon and the risk factors in the company's current and future filings with the SEC.

With that, I'll turn the call over to Steve.

Thank you, Avi. Good afternoon, everyone, and thank you for joining today's call. I would like to apologize for the delay in initiating the call. There were some technical factors from the operating system that really was out of our control. I also hope all of you are safe and practicing the guidelines as per our CDC. As you know, this is a very serious pandemic. So obviously, please take good care.

2019 and early this year represented a period of significant advancement in Onconova, highlighted by our recently completed enrollment of the pivotal Phase III INSPIRE trial in higher-risk myelodysplastic syndromes. With planned INSPIRE enrollment now complete, we await the reporting of 288 survival events before releasing top line survival data. As disclosed earlier this afternoon, we expect top line survival data to be available during the second half of 2020.

Survival events INSPIRE trial are occurring at a slower rate than anticipated from historical published data. The reporting of this data set is expected in the second half of this year. This is our best estimate at this time, however, the impact of the evolving situation with COVID-19 is not known.

As you recall, the INSPIRE trial is an open-label, randomized, controlled international study designed to determine the efficacy, safety and tolerability of single agent intravenous rigosertib in the treatment of patients with second-line higher-risk MDS. Patients in this study are less than 82 years of age and have progressed on, relapsed or failed to respond to previous treatment of methylating agent therapy. The study randomizes patients to receive either rigosertib with best supportive care or the physician's choice of therapy with best supportive care. The primary endpoint of this study is overall survival of all randomized patients in the intent-to-treat population.

There is a second opportunity for an FDA approval, which is the sequential analysis of the overall survival of the very high-risk subgroup as defined by the revised International Prognostic Scoring System. Should rigosertib prolong survival in the INSPIRE trial in a statistically significant manner, we believe rigosertib could be the first new treatment for higher-risk MDS in more than 15 years.

In addition to the INSPIRE trial, we are preparing for a pivotal Phase II/III combination trial of oral rigosertib and azacitidine for the treatment of adult patients, which [aid the risk] HMA-naive, high-risk MDS. We received feedback from the FDA in 2019 and are preparing a Phase II/III protocol. We anticipate this study will begin later this year in conjunction with the data readout from the INSPIRE study.

At the American Society of Hematology Annual Meeting in December 2019, Onconova presented a number of abstracts highlighting our development programs for both intravenous and oral rigosertib. The genomic data from the INSPIRE trial identifies the most common mutations in high-risk MDS following azacitidine failure, including those of the RAS pathway that are targeted by rigosertib. We believe the Phase II data of oral rigosertib in combination with azacitidine forms a foundation of a future adaptive trial in HMA-naive, high-risk MDS patients. We appreciate the recognition by ASH expert reviewers designate this data to be of such value that it was given an oral presentation at ASH.

As presented at ASH, a Phase II combination of oral rigosertib and azacitidine showed an overall response rate of 90%, 9-0, and a complete response rate of 34%. Complete response or CR, by definition, signifies the patient has a normal-appearing bone marrow and the bone marrow produces a normal peripheral blood count. Thus, these patients who are typically transfusion-dependent are rendered transfusion independent, which clearly offers great clinical benefit. The median duration of response is 12.2 months. The company believes this data support the design of a planned Phase II/III adaptive trial in HMA-naive high-risk MDS patients.

We have also made important progress with additional pipeline programs as well. Beyond rigosertib's focus in MDS, we are pleased about our other pipeline progress. And in particular, the progress of our plans to study rigosertib in RAS-driven cancers, including a study in KRAS-mutated lung adenocarcinoma. We anticipate the first patient to be entered onto the trial once the COVID-19 environment improves sufficiently. While a checkpoint inhibitors represent a significant advancement in the standard of care in treating lung cancer, tremendous unmet medical need remains. In our view, making our novel combination approach, which now will target RAS, of great interest to pursue.

ON123300 is our investigational first-in-class dual inhibitor of CDK4/6 and ARK5, which we believe has the potential to treat numerous cancers, including refractory metastatic breast cancer where CDK4/6 inhibitors have helped authority approval. As a reminder, we entered into a license agreement with HanX Biopharmaceuticals for ON123300 in December of 2017, under which HanX will provide all funding required for Chinese IND-enabling studies performed for Chinese health authority IND approval. We and HanX also intend for these studies to comply with FDA standards. The IND was approved in January of 2020 by the Chinese health authority. The manufacturer for ON123300 has been identified already and qualified. We plan to file a U.S. IND to ON123300 in the fourth quarter of 2020 after obtaining the required manufacturing data for the aspirant.

For those who are not familiar with the field, CDK inhibitors have emerged as promising compounds targeting very large indications such as hormone receptor positive metastatic breast cancer. The current generation of commercially-approved CDK inhibitors has limitations. Due to its unique targeting of ARK5 as well as CDK4 and 6, we believe ON123300 has the potential to overcome many of these limitations, making our drug candidate potentially suitable for certain cancers that may not be responsive to the current generation of CDK4/6 inhibitors. If successful, we believe ON123300 could address this very large market opportunity with a potentially better therapeutic. We maintain global rights to ON123300 outside of China.

The fourth quarter of 2019 and early 2020 was productive on the business development front as well. We executed a license agreement for rigosertib for Canada with Knight Therapeutics, we executed a license agreement for rigosertib for Australia and New Zealand with Specialised Therapeutics, we reacquired the rights to rigosertib in Greater China from HanX Biopharmaceuticals and we entered into a pre-approval access collaboration with Inceptua Medicines for rigosertib in select countries outside of the United States.

As a reminder, Onconova retains the rights in the United States, Europe and China for rigosertib, and we look forward to further business development opportunities.

And now I'd like to turn the call over to Mark Guerin, our Chief Financial Officer, for a discussion of our financial results for fourth quarter 2019. Mark, please?

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Mark Patrick Guerin, Onconova Therapeutics, Inc. - CFO [4]

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Thanks, Steve, and good afternoon, everyone. Cash and cash equivalents as of December 31, 2019, totaled $22.7 million compared to $17 million as of December 31, 2018. Besides the $9 million of net proceeds from the financing we closed in early 2020, common stock warrant exercises since December 31, 2019, have added $5.7 million to the company's cash balance, resulting in a cash balance at February 29, 2020, of approximately $32.6 million. Based on our current projections, we expect that our cash and cash equivalents will be sufficient to fund ongoing trials and operations into the third quarter of 2021.

Our net loss was $21.5 million for the year ended December 31, 2019, compared to $20.4 million for the comparable period in 2018. Research and development expenses were $15.5 million for the year ended December 31, 2019, and $16.9 million for the comparable period in 2018.

General and administrative expenses were $8.3 million for the year ended December 31, 2019 and $7.6 million for the comparable period in 2018. We continue to manage our resources carefully while maintaining our primary focus on completing the INSPIRE trial.

This completes my financial review. I'll now turn the call back to Steve.

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Steven M. Fruchtman, Onconova Therapeutics, Inc. - CEO, President & Director [5]

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Thank you so much, Mark. With that, we'd like to open the call for questions. After the Q&A, I'll finish with some closing comments.

Because of COVID-19, the Onconova office is closed. So to make this as expeditious as possible, when a question is asked, I will -- we're all in our home offices, so I will ask our internal expert to answer your question.

So operator, please go forward.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Dr. Joe Pantginis with H.C. Wainwright.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [2]

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Hope you're all well and hope you all stay well. A few questions, if you don't mind. First, on INSPIRE. Steve, I want to merge 2 of your prepared comments. Obviously, as you said, that nothing is new in 15 years, especially in the advanced population. And then you had your comment that based on observations for unblinded data, it appears to have a slower rate of events versus historical controls. So I'd like to approach that from a devil's advocate perspective. Obviously, all of us want to hope that it's due to the presence of rigosertib. So I guess from a devil's advocate perspective, what do you believe could also be impacting that? Obviously, beyond the placebo group or the control group acting better than expected, but no changes in therapeutic regimens, et cetera. So I'm just curious what your thoughts might be.

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Steven M. Fruchtman, Onconova Therapeutics, Inc. - CEO, President & Director [3]

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So Joe, thank you for that question. I will ask our expert devil's advocate, Dr. Rick Woodman, to answer your question. Rick?

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Richard Charles Woodman, Onconova Therapeutics, Inc. - Chief Medical Officer and Senior VP of Research & Development [4]

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Thank you, Steve, for that illustrious introduction. So thank you for the question, Joe. I think we have to keep in mind that the historical data regarding survival was collected well over 12 years ago and was not really collected in a randomized controlled trial. I think one of the potential factors that could be contributing to the event rate not being what we historically anticipated is improved supportive care that's occurred since that time the original azacitidine studies were done and survival was captured. I think that at this point in time, we do not see any impact of COVID on the survival events. But obviously, this is something we're monitoring through our safety reporting process, and we will continue to go forward with that, looking very carefully on a regular basis at the event rates.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [5]

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Very, very helpful. And then I'll put 2 in here at the same time. With regard to the news flow around INSPIRE, based on your goal of hitting a medical conference later this year, I guess, presumably ASH, if the data -- or if you hit the events ahead of time, would you anticipate just putting out a more simplistic press release that the -- maybe the primary had been met and then hold the data until ASH? And then secondly, based on the relatively late addition of additional geographies enrolling sites like in Brazil, are you looking at any potential over enrollment?

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Steven M. Fruchtman, Onconova Therapeutics, Inc. - CEO, President & Director [6]

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So I'll take the first one first, Joe, if I may. So regarding release of information, if -- it looks like it's aligned and ideally, we will present the survival data at ASH. Clearly, if we have the data prior to ASH, we will be in discussions with ASH to inform them of our need based on SEC guidance that the data needs to be released to the Street, and we will do that in a timely fashion via a press release of some sort. And at the same time, make sure we inform ASH that the data will be released and yet ASH will permit us to present the data at that medical conference. And I think you had a second question, Joe, that I'll ask Rick to take.

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Richard Charles Woodman, Onconova Therapeutics, Inc. - Chief Medical Officer and Senior VP of Research & Development [7]

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Yes. Thank you, Joe. Your question was regarding over enrollment in INSPIRE now that we've reached 360. The current industry practice for sponsors is that patients who have consented to the study prior to achieving planned enrollment be given the opportunity to be screened and considered the study for participation. We are doing that. We are obviously following closely health authority guidelines such as the FDA's as well as national guidelines. None of those guidelines have yet said that over enrollment with screened patients is not possible. So I anticipate there may be a few patients that would still come into the study.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [8]

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Got it. And my last question. Obviously, you had some BD developments as well with regard to China rights to rigosertib from HanX, but you're obviously still dealing with that company for 123300, which is a mouthful. You got to get a better name. But the -- just curious how things might be going and potentially relicensing in China and beyond.

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Steven M. Fruchtman, Onconova Therapeutics, Inc. - CEO, President & Director [9]

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Thanks, Joe. Avi, can you take that, please?

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Abraham N. Oler, Onconova Therapeutics, Inc. - VP of Corporate Development & General Counsel [10]

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Sure. Thanks for the question, Joe. And you're right. We have an existing program with HanX Biopharmaceuticals, but the program with rigosertib is now -- China rights are available. So that means the largest markets in the world outside of Japan and in China, Europe and the United States, Onconova maintains all those rights. And now we've licensed rights in the fourth quarter, as Steve mentioned, to partners in Canada and in Australia and New Zealand. Those territories very much remain available. And as we complete enrollment here and as we approach data, we believe it's going to be an exciting time for Onconova on various fronts, including business development.

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Operator [11]

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Our next question comes from Dr. Ahu Demir with NOBLE Capital.

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Ahu Demir, NOBLE Capital Markets, Inc., Research Division - Biotechnology Research Analyst [12]

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Nice to hear everyone's voice, and I hope you're safe and you all stay safe. So my first question, first of all, congratulations. We needed some good news. Congrats on the completion of enrollment. So since you're expecting data in the second half, do we expect any genomic, any other types of data that will be press released or presented at the conference prior to the top line data?

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Steven M. Fruchtman, Onconova Therapeutics, Inc. - CEO, President & Director [13]

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Rick, please?

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Richard Charles Woodman, Onconova Therapeutics, Inc. - Chief Medical Officer and Senior VP of Research & Development [14]

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Read more:
Edited Transcript of ONTX earnings conference call or presentation 24-Mar-20 8:30pm GMT - Yahoo Finance

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