Q2 2020 Onconova Therapeutics Inc Earnings Call

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Good afternoon, and welcome to Onconova Therapeutics, corporate update and Twoq 2020 financial results Conference call. At this time all participants are in listen only mode. After the speakers presentation. There will be a question answer session. So the question during the session you will need to press star one on your telephone.

As a reminder, this call may be recorded at this time I would like to turn the call with the Abbvie Ohler Senior Vice President corporate development and General Counsel.

Thank you operator, good afternoon, and welcome talking open second quarter 2020, corporate update and financial results Conference call earlier. This afternoon, we issued a press release outlining our financial results and business progress during the quarter. If you have not seen this press release it is available on the investor.

Relations page of our website at Www Dot Onconova dotcom on today's call Dr. Shi for us.

President and CEO will discuss the company's recent highlights and anticipated clinical and business milestones. After Steve completes his opening remarks, Mark Guaran, Our Chief Financial Officer, who will review second quarter financial results. Following March report, we will move to the Q in a portion of the call.

Which will be joined by Dr. brick Woodman, our Chief Medical Officer, Lastly people come back with some final comments, you know view of our upcoming milestones before we begin I remind everyone that statements made today. During this conference call will include forward looking statement under the Safe Harbor provisions of private Securities litigation.

And reform Act of 1995, which involve risks and uncertainties that can cause actual results could differ materially.

Forward looking statements speak only as of the date there 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 sensors circumstances. Please see the forward looking statements. This claim in the press releases keep this afternoon.

The risk factors in the company's current and future filings with the FCC with that you just now my pleasure to turn the call over to Steve.

Thank you obviously good afternoon, everyone and thank you joining todays call.

First I.

That was the coal read pandemic.

Continues to evolve in the U.S. and were board.

We wish all safety good health to you and your loved ones.

We remain committed to executing our goals.

Supplier and beyond.

We remain focused on inspired David or read out.

And we'll speak more about the coal good pandemic shortly.

Okay no.

Had a productive second quarter.

Exited the quarter carrying a significant momentum.

During the second half of Twentytwenty.

As previously indicated our pivotal phase three inspired trial recently achieved the required number.

Survival events to allow for data analysis.

And we anticipate topline data read out by the end of September.

Oh really has made access to hospitals and clinics more difficult. So data verification keys dates stretch your survival and the most recent clinical encounters I'm more difficult to verify.

But verify we must and reach out.

Following topline data read out we expect to present more detailed data at a major medical meeting later this year.

The meeting to be identified based on timelines.

Note most if not all major major medical meetings I now virtual.

To shorten the timelines.

Our anticipated new drug application war and the submission to the F.D.A.

We have already begun M.D.A. preparation prior to date or read out.

Real working all show with regulatory consultancy experts on our own D.A. documents for the U.S. Ftn, both the clinical and manufacturing modules of the M.D.A.

As well as on the marketing authorization application war and a document for the European Medicines Agency.

We anticipate that this initial work.

Put us in a position to expedite our health authority applications mean data becomes available.

As part of this process, our clinical team on the Rick wouldn't use leadership.

There's preparing for a pediatric investigational plan or pick.

An important component of the M. anyway.

We're also advancing our plans to be ready for commercialization.

Including the recent announcement.

Yeah that should have a commercial expert.

Ms Terry Shoemaker to our board of directors.

He was instrumental in this successful commercialization of age decided there.

The most commonly used hypomethylating agent in the world So high risk Mds.

As you recall inspired isn't an open label randomized control.

Two national study designed to determine he advocacy.

Safety and Tolerability, a single agent intravenous rigosertib.

In the treatment of patients the second mine high risk Mds.

Patients in this study are less than 82 years of age and you have progressed art relapse will fail to respond to previous treatment with the standard of care I Hypomethylating agent.

Also calls in Hmm.

The study randomize patients to receive either Regals theater with best support of Cat.

For the physician's choice of therapy with best support of Cat.

The primary endpoint of this study.

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.

The overall survival of the very high risk Mds sub.

As defined by the receiver revised international prognostic, scoring system.

[noise] should intravenous rigosertib demonstrate a survival advantage.

Pad to physicians choice of therapy on the aspire trial in a statistically significant matter.

We believe we go through to could be a major events for high risk second line Mds patients with a novel mechanism of action.

Hopefully broader and additional novel indications will follow.

Yeah, no progress within spire.

I like the caught up with the initiation of an investigator initiated phase one two trial oral rigosertib plus the immune checkpoint inhibitor novolen bat in advanced metastatic K Ras positive long at no cost.

No.

Over half of non small cell lung cancers are classified as lung adenocarcinoma.

And I'll disease, the largest subset.

They tell you asked mutation as the predominant genetic driver.

Given the utility in most of all cancer setting.

Checkpoint inhibitors are among the world's top selling pharmaceutical products and continue to obtain sta approval for broader indications.

And our view this makes our novel combination approach, which we go certain it potentially meaningful option to pursue in lung cancer.

We hope this will offer patients who have progressed, our first line therapy, a second lie approach.

We're also initiating studies in other Ras pass grade <unk>, driven cancers as part of our investigator initiated development program.

I can Nova has also recently featured pipeline developments in multiple venues.

The European Hematology Association Universe. Your conference in June we announce updated aggregated baseline genomic data ha may failure patients screened and entered into the inspire trial.

Briefly the presentation shows that RASK pathway mutations in these higher risk Mds patients were observed more frequently inpatient you progressed on nature may therapy as defined by the international working groups definition of progression.

Compared to those who failed to respond to HMH therapy.

In fact in this study patients you mutations of the RASK pathway had a higher likelihood of progression on Hypomethylating agents, then those with T. P 53 mutations they will recognize genomic abnormality predicting for progression.

In a number of cancers.

We believe this is informative to the potential role Ralph targeting agents, such as we go circuit and to our knowledge. This mutational analysis is among the largest such databases to be collected.

Patients on the Rigosertib and physicians choice arms will have repeat analysis of their genome its status performed as part of the inspire trial.

Following the close of the second quarter Onconova also announced a publication a phase one data.

The combination of all Rigosertib, plus a society and higher risk Mds and AML in the journal leukemia research.

A key strategy.

Bridging into Cheapens up Mds.

Yeah identification of safe and effective combination.

Particularly those involving oral agents.

We anticipate meeting with the FDA in conjunction with the pivotal data read out from the inspire trial.

Alignment with the agency on a registration trial for the combination.

No recourse or them plus is decided D. H a may naive high risk Mds.

We look forward to these interactions with the agency.

In the journal molecular shall.

We also announced the publication.

Additional preclinical data supporting Regal sorters mechanism of action as a last targeted anti cancer therapy.

Oh no rubber leads this data sheds light on the ability rigosertib to modulate the rat pathway.

We also disclosed that we've embarked on Rudy.

<unk> clinical work exploring Reger service potential included 19.

The back to actually this is as follows.

These preclinical studies follow a presentation at ash in 29 team.

Suggesting the ability reger assertive to up regulate viral defense pathways such as interferon.

It is hypothesize that the immune system may play a role in the past the Genesis of Mds and immune modulation has been studied in Mds.

More recent preclinical studies conducted Ludovico assertive demonstrated impressive inhibition.

Sars pools to replication in zero cells when compared to controls.

And provide the continue with optimism that further research in humans infected with Sars codes to is warranted.

In late July I can know right now said have applied to the National Institute of allergy and infectious diseases or and I Air I'd.

And also to bother Biomed advanced research and development authority to see funding and could participate in therapeutic trials under the NIH umbrella to conduct human studies, which Meego circuit.

We caution that our work in coal bed methane team is very early.

And the need for therapeutics and effective vaccines is evolving rapidly as the pandemic continues in various geography freeze across the globe.

Hence.

We cannot predict what the outcome of our efforts will be.

We hope to provide greater clarity sometime during the second half of this year.

In addition to the U.S., we have their rights to Rigosertib in Europe, and China, and all the key markets around the world.

The yard Reger circuit.

Well when one threethree hundred it is our first in class inhibitor of CDK foolish snacks and arc five.

We believe Oh anyone to three 300 has the potential to treat numerous cancers, including refractory metastatic breast cancer with CDK for six inhibitors are already commercially available.

CDK inhibitors have emerged as promising products and Tom pounds targeting very large cancer indications such as hormone receptor positive metastatic breast cancer.

Due to its unique targeting of arc five as well as CDK four and six rebuild the Oh in 123 300.

Overcome many of the existing planets limitations potentially making it suitable for certain cashes that may not be responsible responses sorry to the current generation CDK for six inhibitors.

If successful we believe this product candidate could you address this very large market opportunity.

We maintain global rights. So all in 123 300 outside of China.

Our apartment partner in China for this compound is harmonic biopharmaceuticals.

Oh, that's funded the Chinese I NZ, enabling studies.

The Chinese I energy was approved in January 2020 by the Chinese health authorities.

We anticipate a phase one study may begin in China in the second half of Twentytwenty.

We also intact for the Chinese I N D, enabling studies to comply with Sta regulations for the U.S., we plan to file a U.S. R&D in the fourth quarter of 2020.

And now I liked it turn the call over to Mark gearing, our Chief Financial Officer for a discussion of our financial results for second quarter 2020.

Mark.

Thanks, Steve and good afternoon, everyone.

First as a reminder, early this month talking over it's either a letter from NASDAQ stock market, stating that it had regain compliance with the minimum bid price requirement of the NASDAQ listing rule 555 year old way too.

Because its common stock at a minimum closing price at at least one dollar per share for a minimum of 10 consecutive business days.

Our cash and cash equivalents as of June Thirtyth, 2020 totaled 27.2 million compared to 22.7 million as of December 31st 29 team.

Common stock warrant exercises since our financing transactions in November and December 2019 have added 9.8 million to our balance sheet since January 1st 2020.

And as of August 12, 2020, we have 183.568 million 267 common shares outstanding.

Additionally, the almost oh, the warrants outstanding as of June Thirtyth 2020.

Over 80% of them are in the money as of August 12.

Based on our current projections, we expect that our cash and cash equivalents will be sufficient to fund ongoing trials in operation into the fourth quarter of 2021.

Our net loss was 7.4 million for the quarter ended June Thirtyth 2020, compared to 3.6 million for the comparable period and 29 team.

Research and development expenses were 4.8 million for the quarter ended June Thirtyth 2020 at 3.9 million for the comparable period and 29 King.

General and administrative expenses were 2.6 million for the quarter ended June Thirtyth 20, 21.8 million for the comparable period and 29 King.

Our operating cash burn in the second quarter of 2020 with approximately 5.4 million.

It's complete my financial review I'll now turn the call back speak.

Thank you so much mark with that we'd like to open the call for questions.

After the questions.

Finances, I'll finish with some closing comments.

Operator, please open the queuing a session.

As a reminder class for question you want me to press Star one on your telephone Silicon try your question for the banking. Please standby will become father culinary roster.

Our first question comes on the line of gel thinking that's from H.C. Wainwright. Your line is now from.

Hey, guys. Good afternoon. Thanks for taking the question and hope you are all doing well first I'd like to ask about your initial comments about the data verification process for the study what do you think are the real rate limiting steps now either covered related or unrelated and then secondly with that.

I think this is more of a what if question do you have any visibility now with regard to any potential patients lost due to the due to cope with that might impact the statistics and have you had any discussions with the FDA about that since they have.

Put out a recent public guidance a couple of months ago about being a little more amenable to adjusting statistical plans due to cobot. Thanks a lot.

Joe Thanks for that question and I'll ask Rick can you. Please provide the answers.

Thank you Stephen Thank you Joe.

So all along the biggest challenge that we observed with our study related to pull that was access to document since at the sites.

And this is not necessarily physically being able to get to sites, but even having remote monitoring opportunities.

We have been able to overcome.

Almost all of those.

Difficulties and challenges.

Now we were still in database lock and so we are continuing to clean data and monitor and source data verified.

But to date, it's gone very well and I think it's in part.

The understanding by the sites regarding where we are in the lifecycle of the study in the importance of this data to the outcome of the study into this disease.

We have not lost any patients due to cold, but at this time.

The the comment is that challenge we see is that a patients are have symptoms suggestive of Cove. It and then undergo testing and then report us.

The results negative or positive.

Got it. Thank you very much for that and if things continue it continually get more exciting for you guys coming into the data I guess I'll ask the question at this point again and I know I do at this a lot about what you feel its outstanding with regard to be I'm oral study.

Head of your upcoming FDA meeting.

Ah well I think the primary challenges are related to the unique adaptive design, we're proposing to the FDA.

And that will require some discussion between us and the agency.

I think that the timing of inspire yeah if positive.

It could very much support their interest in an adaptive study design, then has and expedient execution and conduct with oral rigosertib.

Got it guys. Thanks, a lot and good luck for this a major thing coming up for you guys real exciting.

Thank you Jackie Joe.

Thank you Sir our next question comes from the line of knowing Cambria from Maxim Group. Your line is now from.

Hi, Stephen themed events in the quarter. So I guess I am I wouldn't start up with that it came at study that's going on right. Now obviously, so I was wondering you. It's an open label study you have any idea will have any updates this year on that and then.

What kind of response they would he will give you some confidence in terms of you know you feel there's activity in this in my tight you know I know, it's a long ways away, but I'm curious about that.

So thank you now you just going to point out that this is the phase one study and I'll ask Rick Gil offer the more details that we may have.

Thank you Steve So we're in the early phases of enrollment to assist study.

I think obviously with these studies are data can become available sooner than anticipated or conversely take much longer than anticipated depending upon.

Dosing and development deal teas.

Because this is a phase one studies, Steve mentioned safety.

Deal team determination.

And determination of a recommended phase two dose for the combination is the priority.

I think like all phase one studies.

You know we are always are excited about responses, but that is not what is going to determine.

The success of the study or the ability to proceed with other studies it will be dosing and safety.

That's helpful. Recently on and then in terms of you know I'm moving oil or looking at well make a scenario in cold. It on and you mentioned that either Piper brand I was just wondering what the turnaround time for that and.

Hey, when will you know anything and then in terms of the economics is this just disappears.

Would it be sufficient for Iranian England settings, or actually for a clinical trial.

So I'll take that banking. So you mentioned origo surgeon look why we believe legal assertive should or shouldn't be studied.

Oh good disease is due course, we have oral and intravenous rigosertib. So as you know there's different levels of disease with covert inceptions some relatively mild.

And some tantamount to pulmonary salad.

So based on the inhibition.

Our size can move to.

And so culture systems as you mentioned we've applied.

For funding through the NIH federal mechanism.

Striding coded disease.

Moving in the disease ideally it would be with oral rigosertib to see if it would prevent progression to pulmonary insufficiency. The patients who have already reached unfortunately, the stage of pulmonary insufficiency, requiring rents a little tardy support.

But that would be a group of patients who could be studied with intravenous rigosertib.

Because the global pandemic is changing rapidly it is unclear to us how NIH and the federal funding agencies will make decisions regarding both therapeutics and vaccines.

We believe as long as the pandemic continues to rage, there is a great needs the development.

Of additional applications Therapeutics gosh, we believe based on the preclinical studies or we would like.

To participate in any clinical trials at 88, NIH may be considering therapeutics.

Clearly the NIH.

Appropriately focused on the development the vaccines and gosh, it's unclear to us where NIH stands on the issuing question of the role therapeutics during independent Mick and the role of vaccines any information we have going forward.

Third we will be happy to share that with the investment community.

Yeah, Yeah. That's helpful. That's coming up over to be Frank study like this out and they are paying taxes.

[laughter].

Thank you. Our next question comes in a line of a cooler than the air from Noble capital. Your line is now open.

[noise] [noise] Hello, gentlemen, thank you very much for taking my question I. Congratulations on the progress made into quite Eric I get a few questions and outside of it as Neil leading excitedly about as for the topline inspired data could you provide some color on the Indiana <unk> and <unk>.

[noise] application timeline and commercial efforts following the topline data readout.

So I'll take Turner, who are in fact you.

We anticipate filing the NDA in the first half of 2021.

And we've already begun.

A pre commercial Tony.

Well that per barrel that as I mentioned, we have a commercial expert and she is very unique being in Mds expert who also rose.

The leader of the commercialization surveys deciding when we began initial epidemiology and other studies to have a better understanding of the commercial value.

The second line Ivy League or certain product. So the summary is we anticipate filing and the a first half of 2021.

Okay. That's helpful think Oh.

My second question will be under all of the legal settlement as well. So they usually expect SDN, we need to conclude or have the discussions me to conclude.

For the product and puts a cold and I mean, I didn't fit into the clinic literally be declined nine or 10, GE that you think that.

Well happen.

So again I'll ask Rick to answer your question regarding regulatory guidelines for the oral combination study and what we anticipate and our plans Rick.

Thank you our plans are to have a type C meeting with the FDA calling.

You know inspire topline analysis.

Typically that would be late fourth quarter.

And from that will proceed once we get their feedback on our adaptive phase two phase three design.

And move forward, then with the as quickly as possible.

With opening the study and it's rolling patients.

We think that an oral compound coupled with a positive inspire outcome.

Would generate a lot of interest by physicians and investigators for participating.

In an oral combination study.

Thank you right Oh, we for my next question, So Mike I'm I kind of curious piece the expected any increase in there a actually any expenses in the fourth quarter or is it going to be more into 22 anyone timeline.

Hi, Thanks for the question I think.

We forgot that our cash burn per quarter will still be in the range of five to 6 million in the near term.

As we talked about last quarter. The composition of that bar has shifted from primarily clinical to now include regulatory spending for our anticipated N. da and also the long lead time items for pre commercialization activities I suspect and project that that shift will continue but I do not expect that will have any space.

Turning on a commercial precommercialization activities in earnest until.

Later in the fourth quarter early 2021.

All right. That's they take it may have nights for taking my question.

Thank you Sir our next question comes the line of Yale Jen from Labor Law and company. Your line is now open.

Good afternoon, and thanks for taking the questions and Ah Congrats on the development that Ah you got to be waiting for it very exciting or a month and half whether data and off. My first question is that are in terms of the topline data read out what it is at the.

Financial analysis, so even you beat the best for the.

The I.E. Birkin off the lead out would you still are analyzing for that I read patient outcome and.

I would do recall that the concurrently with the offline or you will recall that much later at all.

[noise] <unk> I'll take that Yeltsin that what you're asking about the sequential analysis and the way. The statistical analysis plan was written if we had a positive pivotal data on the intent to treat population, there's actually no need to look a just a very high.

Risk that doesn't mean, we can't do it but from an eight sta perspective, if we meet the statistical hurdle they intend to treat that's the primary endpoint to now be today.

So.

Community and the Sta going so.

Okay do have a second we do have a second option. If you intend to treat population does not mean statistical hurdle than the FDA has given us permission because of the tremendous unmet medical need for the very high risk Mds population to look at them separately.

In a sequential fashion and then share that information with yesterday in this setting of the intent to treat not meeting its statistical hurdle.

So I hope that answers your question yeah.

Sure I mean acuity I'm thinking about that I read even you'll have a rich so that did that Ah.

Positive outcome for the intend to treat is that the probably four or maybe even just academic reasons or other.

Inflammation reason to to do that analysis for the.

That makes it that.

And yes, we have a number of we have a number of secondary endpoints and we are well call. It an academic groups and such questions like time to leukemia transformation.

Misconceptions.

Responses in Iraq sub three ever a whole variety of analysis as an academic group that at Onconova, we will be interested in looking at and no debt. The very high risk is part of that more generalized analysis that you're asking about.

Okay, Great. That's very helpful and you mentioned above that depend on the timing you might you could presented at a major medical conferences or if we get that could be as would that be possible thing, you'll get up but frankly, a place holder there before you actually.

The full day that that proved to be presented a later on.

Greg could you mention our plans for Ashton, possibly other conferences as well please.

Yes. Thank you Steve so in terms of the upcoming Ash meeting.

We are plan is to utilize the mechanism of a late breaking abstract.

And in that particular mechanism.

Sponsors have the opportunity is submitted an abstract.

Typically in the middle of October.

And that abstract often those abstracts, our phase three studies with novel, New therapies advances in variety of hematologic diseases.

And so given that this is a randomized controlled study in the population. It is it's the study were to have a positive the primary endpoint.

We are optimistic that asked should be very interested and considering this is a late breaking abstract.

And we'd move forward with that approach at this time.

Okay. That's very helpful and maybe a two quick ones. The first one is in terms of oral.

Rigosertib.

You mentioned about adaptive study design I know you still need Oh, it by end, but would you briefly describe that means that the studies.

Yes, so if I understood. Your question. It was just briefly describe the study design.

The adaptive Ah Ah yes.

So the it has two parts first part is a phase two study.

With three arms, one arm being asked decided dean has to control and then the other two arms being investigational arms with two different doses of Rigosertib.

And one dose is 11 20 per day and the other is 840 per day and divided doses.

And the there would be an interim analysis at the end of phase II to pick up.

So to speak the most efficacious and safe.

We go search a dose.

Patients would then continue in the phase three.

To be enroll to that.

Rigosertib arm, which was determined by an idmc to be the choice.

Preferred choice for moving into the phase three we'd continue in the phase three with the control arm evasive siding.

And the primary end points would be a.

Complete response partial response and both the end of the phase two and ended the phase three.

This particular design has a lot of advantages for us.

Two of which I'm sure. Most importantly, the time to execution of it a sequential phase two and and phase three is not nearly as long. It isn't this adaptive design to the adaptive design is faster and it requires fewer patients and so both of those are clearly advance.

And teaches that were part of our decision to use this particular study design.

For all Rigosertib.

[noise] could you remind us in terms of a phase two in phase three.

Each one how many patients you were intended at this moment.

Yeah. So the total study would be 475 patients.

Right now the sample sizes are for the phase to be around 225 patients and in the phase Threeb around 250 patients now these are somewhat artificial numbers.

Because obviously.

We need we haven't had chance to discuss this these numbers with the FDA.

But this this study design has been published last year as a poster at ash.

And there are many of the details are listed in that poster.

And the rationale for the study.

Okay, Great and maybe the last question here is that in terms of your filings abuse agent will be in the first half of next year should we assume that the rolling submission and the you might be able to submit for some of the empty so little bit earlier, and then ultimately the clinic.

Great I and others.

So so the answer is yes, yes, we've got our estimate clearly want assured me timelines as much as possible that will participate in a rolling submission.

Thank you.

Okay. Thank God I appreciate it congrats.

Thank you that's all the time, we have for questions at this time I'd like to turn the call back over to the speakers for closing remarks.

Sure. Thank you all for participating on todays update call.

Well, obviously very excited we hope you are as well.

About the important progress we've made during the quarter.

Important milestones, we look for in the near and medium term include and in no particular ordering.

First.

Topline data from the aspire trial sometime before the end of the current quarter.

Followed by presentation at a major medical conference later this year.

To meeting with the FDA following the inspire readout.

Alignment on a registrational trial with oral Rigosertib, plus eight plus any deciding.

Hmm naive higher risk Mds as Rick described to us.

Three.

Can you.

Spansion Rigosertib investigator initiated study program into additional tumor types.

For us I am the submission.

Oh in 123 300 during the fourth quarter of Twentytwenty, followed by clinical trial initiation.

Five determining next steps encoded 19 research.

We truly appreciate your continued interest in the programs at Onconova.

Should you have any additional questions.

Please feel free to contract any of us.

Thank you.

Operator, you may now end the call.

Ladies and gentlemen, thank you for your participation on today's conference call. This concludes today's event you may now disconnect.

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