Q4 2021 Chimerix Inc Earnings Call

Operator: Good morning, ladies and gentlemen, and welcome to the Chimerix fourth quarter and year-end 2021 earnings conference call. I would now like to introduce your host for today's call, Michelle LaSpaluto, Vice President of Strategic Planning and Investor Relations at Chimerix. Please proceed. Thank you.

Good morning, ladies and gentlemen, and welcome to the <unk> fourth quarter and year end 2021 earnings conference call.

I would now like to introduce your host for today's call Michelle I'll spell that out Vice president of strategic planning and Investor Relations at <unk>. Please proceed.

Michelle LaSpaluto: Good morning, everyone, and welcome to the Chimerix fourth quarter and year-end 2021 financial and operating results conference call. This morning, we issued a press release on our fourth quarter operating results. You can access this press release in our investors section of the website. With me on today's call are President and Chief Executive Officer Mike Sherman, Chief Medical Officer Allen Melemed, Chief Financial and Business Officer Mike Andriole, Chief Science Officer Randall Lanier, and our Chief and Infrared-Owned Technology Officer Josh Allen.

Thank you good morning, everyone and welcome to the generics fourth quarter and year end 2021 financial and operating results Conference call. This morning, we issued a press release on our fourth quarter operating results.

You can access this press release in our investors section of the website with me on today's call are President and Chief Executive Officer, Mike Sherman, Chief Medical Officer, Alan Melamed, Chief Financial and business Officer, Mike Andriole, Chief Science Officer, Randall Lanier energy and effort, that's technology Officer, Josh Allen before we begin.

Michelle LaSpaluto: Before we begin, I would like to remind you that the statements made on today's call include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and are subject to risks and uncertainties and other factors that could cause actual results to differ materially from those referred to in the forward-looking statements.

I'd like to remind you that the statements made on today's call include forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995 and are subject to risks and uncertainties and other factors. These risks and uncertainties and other factors could cause actual results to differ materially from those referred to in the forward looking statements. Please refer to our filings with the SEC for a more.

Disclosure of these risks and uncertainties.

Michelle LaSpaluto: Please refer to our filings with the SEC for a more complete disclosure of these risks and uncertainties. At this time, I would like to turn the call over to our President and Chief Executive Officer, Mike Sherman. Thanks, Michelle. Good morning, everyone.

At this time I would like to turn the call over to our President and Chief Executive Officer, Mike Sherman.

Mike Sherman: Thanks for joining us. Reflecting on 2021, it was certainly a pivotal year for the company. As you know, the foundation of our strategy has been built around the Timbexa program, which will satisfy, of course, a critical need to protect the population from the threat of a smallpox outbreak. Public awareness of this threat has certainly increased in the last couple of years. The human health and economic consequences would be substantial.

Thanks, Michelle good morning, everyone and thanks for joining us not reflecting on 2021. It was certainly a pivotal year for the company as you know the foundation of our strategy has been built around the 10 Bucks a program, which will satisfy of course, a critical need to protect the pop.

<unk> from the threat of a smallpox outbreak.

Public awareness of this threat has certainly increased in the last couple of years, the human health and economic consequences would be substantial.

Mike Sherman: The Timbexa program is also strategically important to the company as it is expected to provide access to considerable non-dilutive capital to fund our oncology drug development. This potential long-term source of capital is particularly valuable and somewhat unique among our peers in this period of financial market uncertainty. With the FDA approval in June and then the BARDA request for proposal in December, this strategy is coming to fruition. The FDA's summary of Timbex's approval provided a very attractive and independent perspective on the value of this drug, easily administered in a crisis situation, indicated for all ages, and with a robust resistance profile. As expected, BARDA's request called for up to 1.7 million courses of treatment.

The <unk> program is also strategically important to the company as it is expected to provide access to considerable non dilutive capital to fund our oncology drug development.

This potential long term source of capital is particularly valuable in and somewhat unique among our peers in this period of financial market uncertainty.

Well the FDA approval in June and then the BARDA request for proposal in December This strategy is coming to fruition. The F. D. A summary of 10 boxes approval provided a very attractive and an independent perspective on the value of this drug easily administered in a crisis situation.

Indicated for all ages and with a real robust resistance profile.

As expected BARDA as request called for up to $1 7 million courses of treatment. The contract will cover procurement of Tim Baxter for the strategic national stockpile as well as the execution of post marketing approval commitments.

Mike Sherman: The contract will cover procurement of Tembexa for the strategic national stockpile, as well as the execution of post-marketing approval commitments. The most important elements of the negotiation include product pricing and initial order quantities, and we'll report on the outcome of the negotiation as it concludes. We remain well positioned financially ahead of the first shipment, which we are poised to trigger upon the signing of a contract. We started 2021 with the acquisition of Oncosutics, which brought to the organization some outstanding people and a pipeline of promising oncology assets.

The most important elements of the negotiation include product pricing and initial order quantities and we'll report on the outcome of the negotiation as it concludes we remain well positioned financially ahead of the first shipment, which we are poised to trigger upon the signing of a comp.

Correct.

We started 2021 with the acquisition of <unk>, which brought to the organization some outstanding people and a pipeline of promising oncology assets.

Mike Sherman: Compelling response data had already been reported for ONG-201 and glioma, although it had not been confirmed. And during the course of the year, we were able to confirm that response data through a rigorous regulatory quality blinded independent central review. And in the process, we revealed an even stronger data set to support this drug as a treatment for H3K27M mutant glioma. In particular, those responses were more durable than expected. Initial response of at least 50% tumor reduction was achieved at a median of just over eight months after the start of treatment and was followed by a median of an additional 11 months of durability.

Compelling response data had already been reported for arc 201 in glioma, although it had not been confirmed and during the course of the year, we were able to confirm that response data through a rigorous regulatory quality blinded independent Central review and in the process we reveal them.

Even stronger dataset to support this drug as a treatment for each three K twenty-seven out mutant glioma in particular those responses were more durable than expected initial response of at least 50% tumor reduction was achieved at a median of just over eight months. After the start of treatment and was followed by a meal.

The end of an additional 11 months of durability.

Mike Sherman: Importantly, it was apparent that these responses mattered as they were associated with performance status improvement, a reduction in steroid use, and longer survival. It's always reassuring when the data has this kind of internal consistency across endpoints. The fact that many of the patients continue to receive ONC-201 even after progression is probably the most compelling evidence for the safety profile of the drug, but it also is a sobering confirmation of the lack of treatment options for these patients.

Importantly, it was apparent that these responses mattered as they were associated with performance status improvement a reduction in steroid use and longer survival.

It's always reassuring when the data has this kind of internal consistency across end points.

The fact that many of the patients continue to receive all two O. One even after progression is probably the most compelling evidence for the safety profile of the drug but also is a sobering confirmation of the lack of treatment options for these patients.

Mike Sherman: During 2021, we held a number of meetings with the FDA to discuss our preparations for a potential new drug application for in 2019, and it's been executing on those deliverables in the meantime. This work includes important CMC and ClinPharm activities as well as the collection and validation of safety data from more than 200 patients. We're also engaged with the FDA in the review of a front line randomized trial design. We plan to initiate the trial in the second half of this year in this H3 K27M population. We want this trial to be up and running during FDA's review of a potential NDA.

During 2021 we held a number of meetings with the FDA to discuss our preparations for a potential new drug application for all two or one and have been executing on those deliverables in the meantime.

This work includes important CMC and Glenn farm activities as well as the collection and validation of safety data from more than 200 patients. We're also engaged with the FDA and the review of a frontline randomized trial design, we plan to initiate in the second half of this year in this age three K 27 a M.

Population.

We want this trial to be up and running during Fda's review of a potential NDA.

Mike Sherman: We've also collaborated with FDA on a natural disease history study. Because the H3K27M mutation is relatively newly discovered, we plan to submit data related to what the field and, frankly, the WHO have already recognized, the fact that it is rare to observe responses from current standards of care in this post-radiation setting. We've initiated this work at sites under a protocol that was submitted to the FDA, and we're actively gathering that data now for patients who meet the same criteria as were used for the 50 patient efficacy analysis. In this case, of course, these patients were not treated with ONC-201.

We are also collaborating with U F D. A on a natural disease history study.

Because of the Ace III K twenty-seven M mutation is relative relatively newly discovered we plan to submit data related to what the field and frankly the W. H O have already recognized the fact that it is rare to observe responses from current standards of care in this post radiation setting.

We've initiated this work in sites under a protocol, which was submitted to the FDA and we're actively gathering that data now for patients to meet the same criteria as was used for the 50 patient efficacy analysis.

In this case of course these patients were not treated with with all its all one you can imagine this won't be a large dataset as awareness of all 201 has been high in many patients thought out treatment with arc 201 through clinical trials or expanded access will know more about the size of that data set in the next couple.

Mike Sherman: You can imagine this won't be a large data set as awareness of ONC-201 has been high, and many patients have sought treatment with ONC-201 through clinical trials or expanded access. We'll know more about the size of that data set in the next couple of months as that information is collected and verified. Keep in mind, this is a retrospective study that will collect data from previously treated patients. For the response analysis, we plan to subject this data to the same blinded independent central review process we used for the ONC 201 efficacy analysis.

Months as that information is collected and verified.

Keep in mind. This is a retrospective study, which will collect data from previously treated patients.

For the response analysis, we plan to subject to this data to the same blinded independent Central review process, we used for the arc 201 efficacy analysis.

Mike Sherman: We expect to be far enough along in each of these workstreams and in ongoing consultation with the FDA to provide guidance on our regulatory timelines by mid-year. Now, let me turn to our D-STAT program, the DASH Phase 3 Trial, and Frontline AML. Enrollment in this study has proceeded more slowly than expected due to the ongoing hospital staffing shortages related to COVID-19 and the competitive environment for enrolling subjects in this population

We expect to be far enough along in each of these work streams and ongoing consultation with the F. D. A to provide guidance on a regulatory timelines by mid year.

Now, let me turn to our D Stat program the Dash phase three trial in frontline AML enrollment of this study has proceeded more slowly than expected due to the ongoing hospital staffing shortages related to COVID-19, and the competitive environment for enrolling subjects in this population.

Mike Sherman: As a result, we don't expect a complete enrollment of the first 80 patients by year end and are currently reviewing a number of options to accelerate D-STAT's development. One tailwind in that space, in AML in particular, the momentum we anticipated supporting MRD as a potential surrogate endpoint has materialized, and this may serve to help accelerate our longer-term development. With that, I'd like to hand the call over to Dr. Langford here to discuss recent developments with CMX 521.

As a result, we don't expect to complete enrollment of the first 80 patients by year end and are currently reviewing a number of options to accelerate these stats development.

One one tailwind in that they're not space in an email in particular the momentum we anticipated supporting M. D. As a potential surrogate endpoint has materialized and this may serve to help accelerate our longer term development.

With that I'd like to hand, the call over to see a doctor linear to discuss recent developments with C. M X five 'twenty one you've heard me mentioned previously that while our focus has shifted to oncology. We expected that we may be sitting on potential value with our legacy anti viral library as the world begins.

Mike Sherman: You've heard me mention previously that while our focus has shifted to oncology, we expected that we may be sitting on potential value with our legacy anti-viral library. As the world begins to focus on pandemic preparedness by identifying molecules that have the potential to fight entire families of viruses, the potential value of this library has been highlighted.

The focus on pandemic preparedness by identifying molecules that have potential against entire families of viruses the potential value of this library has been highlighted this collaboration with the University of North Carolina has really been phenomenal in helping us identify that value without losing focus on other projects.

Mike Sherman: This collaboration with the University of North Carolina has really been phenomenal in helping us identify that value without losing focus on other projects. As this highly credible team of collaborators saw the results of initial experiments, this project became their highest priority, and this subsequent work has progressed very quickly. Let me stop there and let Randall share what we've learned on one of the more mature assets from this library.

As this highly credible team of collaborators saw the results of initial experiments.

This project became their highest priority and and the subsequent work has progressed very quickly.

Let me stop there and let Randall share what we've learned on one of the more mature assets from this library Randall.

Randall Lanier: Thanks, Mike. We've recently generated some very promising pre-clinical efficacy data in an established animal model of COVID-19 with one of our proprietary drugs. This was done in exciting collaboration with Ready, the rapidly emerging antiviral drug development initiative at the University of North Carolina at Chapel Hill. [inaudible] REDI and UNC have deep expertise in this area and have been actively involved in efforts to develop other agents for the current pandemic, including that of the SARS-CoV-2 antiviral, which received emergency use authorization late last year.

Thanks, Mike.

We've recently generated some very promising preclinical efficacy data and an established animal model of COVID-19, with one of our proprietary drugs.

It was done my exciting collaboration with ready the rapidly emerging antiviral drug development initiative at the University of North Carolina Chapel Hill.

Already and you can see have deep expertise in this area.

Actively involved in efforts to develop other agents for the current pandemic, including down of the Sars Cov, two anti viral which received emergency use authorization late last year.

Randall Lanier: They are continuing to explore novel therapies with improved activity and resistance profiles to address anticipated variants of SARS-CoV-2. Our collaboration with them has enabled several key studies of CMX521, and this is a nucleoside analog from our chemical library, for Activity Against Artists Code 2.

They are continuing to explore novel therapies with improved activity in resistance profiles to address anticipated very much.

Sars Kobe too.

Our collaboration with them has enabled several key studies of <unk> five to one and this is a nucleoside analog from our chemical library.

Where activity against Sars Cov two.

Randall Lanier: CMX 521 is particularly attractive because it was developed through a Phase I healthy volunteer study for another indication. The build-up to that study included Sandra G. L. K. Safety, G. Intermanufacturing, and all the other merriot studies needed to start human trials. This previously completed work de-risks many of the common pitfalls of early drug development, the most important of which often involves safety. CMX 521 has a very favorable safety profile in animals and in humans.

Hemic by two one is particularly attractive because it was developed through a phase one healthy volunteer study for another indication.

The buildup to that study included standard safety GMP manufacturing and all the other myriad studies need.

At the start of human trials.

Previously completed work Derisk many of the common pitfalls of early drug development, the most important of which often involve safety.

In next 521 has a very favorable safety profile in animals and humans.

Randall Lanier: It's not genotoxic, not cytotoxic, or mitotoxic, and was well-tolerated up to very high levels of 2.4 grams, and it helped me volunteer for phase one studies. For these reasons, and the efficacy data I'll describe in a minute, the program can be moved into a human proof of concept study for COVID rather quickly. The completed non-clinical studies of SARS-CoV-2 demonstrate very compelling activity in a well-studied mouse model of COVID.

Genotoxic cytotoxic were minor concert and was well tolerated up to very high level two four grams.

The volunteer Phase one study.

For these reasons and the efficacy data I'll describe in a minute. The program. The program can be moved into a human proof of concept study for COVID-19 rather quickly.

The completed non clinical studies with Sars Covid, two demonstrated very compelling activity in a well studied mouse model of Covid and the mis modeled one day in the mouse model is roughly equivalent to humans.

Randall Lanier: And in this model, one day in the mouse model is roughly equivalent to a week in humans. Administration of inhaled aerosolized CMX 521 enables direct delivery to the lungs while minimizing systemic exposure, and we think this improves both efficacy and safety. In the mouse model, aerosolized CMX521, or placebo, was administered at various times relative to the SARS-CoV-2 infection, bringing forward the potential for both prophylactic and therapeutic applications.

Administration of inhaled Air salons to your next 521 enables direct delivery to the lungs, while minimizing systemic exposure and we think this improves for efficacy and safety.

And the mouse model Aerosolized 521, or placebo was administered at various times relative to Sars cov, two infection to explore the potential for both prophylactic and therapeutic catheter.

Randall Lanier: Prophylactic Administration of CMX 521 starting eight hours prior to infection and followed by one dose eight hours prior to infection reduced average viral titers in the lungs on day four by more than three and a half logs for about 99.99% and prevented weight loss and clinical progression versus placebo. [inaudible] Treatment Studies of CMX-521 initiated at the time of infection or at 8 or 16 hours post-infection also significantly reduced viral one-tider at day 4.

Prophylactic administration of <unk> five to one started eight hours prior to infection, one dose eight hours prior to infection.

Reduced average viral titers in the lungs on day, four by more than three and a half logs.

We're about 90, 999% and prevented weight loss and clinical progression versus placebo.

Treatment studies of <unk> five to one initiated at the time of infection or eight or 16 hours post infection also significantly reduce viral titer at the floor.

Randall Lanier: CMX 521 Treatment Protected Mice from Clinical Symptoms of Disease, significantly reducing the weight loss associated with the disease and decreasing lung pathology compared to placebo. Just as an example, CMX-521 initiated 16 hours post-infection would reduce the average viral lung tighter mass charge to be two in the lungs by more than two and a half logs or more than 99 percent.

The next five to one treatment protected mitral clinical symptoms of disease.

Significantly reducing the weight loss associated with the disease and decreasing the lung pathology compared to placebo.

Just as an example, <unk> X 521 initiated 16 hours post infection reduced average viral lung tighter Sars cov, two and the lungs by more than two and a half logs where more than 99%.

Randall Lanier: Importantly, contemporary modeling methods to estimate the translation of these efficacious doses and minds to target clinical notices predict that the doses in humans should fall within a range that could be delivered with commonly used systems like battery-powered portable nebulizers or dry powder inhalers. Additional studies are currently planned or underway to assess efficacy at lower doses and less frequent administration to compare with other anti-virals in this model system and to evaluate efficacy in combination with other anti-virals. Briefly, all the signs point to a proven virus-specific mechanism of action, broad variant coverage, a safety profile that looks clean enough for prophylactic use, and excellent efficacy in the animal body.

Importantly, contemporary modeling methods to estimate the translation of the efficacious doses in mind.

Clinical doses.

Predicts the doses in humans should fall within a range that could be delivered with commonly used system.

Battery powered portable nebulizer dry powder inhalers.

Additional studies are currently planned or underway to assess efficacy at lower doses and less frequent administration to <unk>.

Here with other Antivirals in this model system and to evaluate efficacy in combination with other anti virals.

Briefly all the signs point to a proven virus specific mechanism of action broad variant coverage a safety profile that looks cleaner now for prophylactic use and excellent efficacy in the animal model.

Randall Lanier: Furthermore, we have kilograms of G&P Drive ready for development, excellent patent life, and a simple potentially other short path to proof of concepts in humans. While we focused on the advancement of our oncology pipeline and progress toward a convex procurement agreement with BARDA, this collaboration with REDI allowed us to efficiently evaluate our antiviral library, and CMX521 looks very promising. We have an upcoming oral ratebreaker presentation later this month at the International Conference on Antiviral Research in Seattle, where we'll discuss this data in greater detail.

Furthermore, we have kilograms of GNP drive ready for development.

Pat in light and a simple potentially other short path to proof of concept in humans.

While we focus on the advancement of our oncology pipeline and progress toward a conducts a procurement agreement with BARDA. This collaboration with ready allowed us to efficiently evaluate or its environment library, and CMS 521 looks very promising.

We have an upcoming world late breaker presentation. Later this month at the International Conference on Antiviral research in Seattle, where we will discuss this later in greater detail.

Mike Andriole: And with that, I'll turn the call over to Mike Andriole, CEO of Chimerix Inc. Thanks Randall and good morning everyone. Earlier today, we issued a press release containing our financial results for the fourth quarter and full year 2021. Starting with our balance sheet at the end of December 2021, we had approximately $90 million in capital to fund operations.

And with that I'll turn the call over to Mike Andriole.

Mike Andriole: In January, we paid a $14 million note payable related to the acquisition of Oncosteutics on January 21. Importantly, we did not owe any further payments related to that transaction until US or EU approval of ONC201. Also, to provide additional financial flexibility, last month we entered into a $50 million revolving credit line with Silicon Valley Bank. We have not drawn down on this facility to date and have no obligation to use the facility.

Thanks, Randall and good morning, everyone.

Earlier today, we issued a press release containing our financial results for the fourth quarter and full year 2021.

Starting with our balance sheet at the end of December 2021, we had approximately $90 million in capital to fund operations in January we paid a $14 million note payable related to the acquisition of <unk> in January of 'twenty. One importantly, we do not owe any further payments related.

So that transaction until U S or EU approval of arc 201.

Also to provide additional financial flexibility last month, we entered into a $50 million revolving credit line with Silicon Valley Bank, we have not drawn down on this facility to date and have no obligation to use the facility.

Mike Andriole: We do see it as a tool to supplement our financial position by providing an alternative source of capital that can be utilized on an as-needed basis. For example, in advance of an anticipated or future shipment of Quebec's at Tabarda, we could potentially use this credit facility to bridge the gap between procurement orders. As we look forward to 2022, this year is expected to be a pivotal year for the company in many ways and certainly from a financial perspective as we expect our first commercial product sale of Tembexa to the U.S. Strategic National Stockpile to occur, likely during the second quarter.

We do see it as a tool to supplement our financial position by providing an alternative source of capital that can be utilized on an as needed basis.

For example in advance of an anticipated or future shipment of 10 Bucks with BARDA, we could potentially use this credit facility to bridge the gap between procurement orders.

As we look forward to 2022.

This year is expected to be a pivotal year for the company in many ways and certainly from a financial perspective, as we expect our first commercial product sale or come back to the U S strategic national stockpile to occur likely during the second quarter.

Mike Andriole: As Mike has mentioned, we have submitted to BARDA our response to their RFP, which details our proposal for price, quantities, and delivery schedule of 1.7 million treatment courses of Tembexa. We expect potential revenue from the sale of combats this year and in future years to enable us to fully invest in our oncology pipeline and build a U.S. sales and marketing organization for a potential onto a one commercialization. We expect potential revenue from the sale of combats this year and in future years to enable us to fully invest in our oncology pipeline and build a U.S. sales and marketing organization for potential. As we have confirmation on the specifics of a potential barter procurement, we will provide more precise financial guidance.

As mentioned, we have submitted to BARDA, our response to their RFP, which details our proposal for price quantities and delivery schedule of one 7 million treatment courses of <unk>.

We expect a potential revenue from the sale of come back to this year and in future years to enable us to fully invest in our oncology pipeline and build a U S sales and marketing organization for potential onto a one commercialization.

Because we have confirmation on the specifics of a potential BARDA procurement, we will provide more precise financial guidance.

Mike Andriole: Turning to our Statement of Operations, the company reported a net loss of $39.5 million, or $0.45 per basic and diluted share, for the fourth quarter of 2021 compared with a net loss of $11.7 million, or $0.19 per basic and diluted share, for the fourth quarter of 2020. The fourth quarter included a $20 million success milestone payment to the legacy Oncosteutic shareholders in relation to the achievement of a 20% overall response rate by Reino HCG for Onc 201. However, revenues for the fourth quarter of 21 decreased to $46,000 with the completion of the BARDA 2011 research and development contract.

Turning to our statement of operations.

Mike Andriole: That's compared to $1.1 million for the same period in 2020. Research and development expenses increased to $34.3 million for the fourth quarter of 2021, compared with $8.7 million for the same period in 2020. Again, this increase is primarily related to the $20 million success payment for OCT-201. General and administrative expenses also increased to $5.2 million for the fourth quarter of 2021, compared to $4.2 million for the same period in 2020.

Company reported a net loss of $39 $5 million were 45 cents per basic and diluted share for the fourth quarter of 2021, compared with a net loss of $11.7 million or <unk> 19 per basic and diluted share in the fourth quarter of 2020.

The fourth quarter included a $20 million success milestone payment to the legacy <unk> shareholders in relation to the achievement of 20% overall response rate by Rhino hcg for on 301.

Revenues for the fourth quarter of 'twenty, one decreased to $46000 with the completion of the BARDA 2011 research and development contract as compared to $1 $1 million for the same period in 2020.

Research and development expenses increased to $34 3 million for the fourth quarter of 2021, compared with $8 7 million for the same period. In 2020 again. This increase was primarily related to the $20 million success payment for onshore one general and administrative expenses also increased to $5 2 million for the fourth quarter of 2021 compared to $4 two.

For the same period in 2020 with that overview I'll turn the call back to Mark for closing remarks.

Mike Sherman: With that overview, I'll turn the call back to Mike for his closing remarks. Thanks, Mike. We certainly have an exciting year ahead with Catalyst and both the Timbex and Auto-01 programs, in addition now to CMX 521, which wasn't really on the radar even a few months ago. We look forward to building on this momentum during the year. With that, Elizabeth, I'm ready to open the call to questions. Ladies and gentlemen, if you'd like to ask a question at this time, please press the star, then the number one key on your touchtone telephone. To withdraw your question, press the pound key.

Thanks, Michael we certainly have an exciting year ahead with catalysts in both that come back then at 201 programs. In addition, now to CMS $5 21, which wasn't really on the radar even a few months ago. We look forward to building on this momentum during the year.

With that Elizabeth.

Ready to open the call to questions.

Ladies and gentlemen, if you'd like to ask a question at this time. Please press the star and the number one key on your Touchtone telephone.

To withdraw your question press the pound key.

Maurice Raycroft: Our first question comes from Maurice Raycroft with Jeffreys. Hi, good morning, congratulations on the progress, and thanks for taking my questions. I was going to ask about the BARDA process, just checking how competent you are that the negotiations could occur and finalize over the next couple of weeks or months. I guess if you can put any finer points on the timeline there. And what are your latest thoughts on the dialogue with BARDA, particularly with a pricing premium versus T-Pox? I think typically you see these processes play out over 60 to 90 days.

Our first question comes from Maury Raycroft with Jefferies.

Mike Sherman: I don't know that we have much more insight into that process. I would say that the elements of negotiation are pretty focused, as I mentioned. It's really pricing and delivery schedule; the rest is really about reimbursement for activities and ends up being a small portion of the contract. We feel really good about our proposal for pricing and support that analytically.

Hi, good morning, Congrats on the progress and thanks for taking my questions.

I was going to ask on the BARDA process, just checking how confident you are that the negotiations could occur and finalized.

Over the next couple of weeks or months I guess, if you can put any finer points on the timeline there and what are your latest thoughts on the on dialogue with BARDA, particularly with a pricing premium.

Versus T box.

I think typically you see these processes play out over a 60 to 90 days.

I don't know that we have much more insight into that process I would say that the elements of negotiation are pretty focused as I mentioned, it's really pricing and delivery schedule. The rest is is really about reimbursement for activities and ended up being a small portion.

The contract we feel really good about.

Our our proposal for for for pricing and supported that analytically.

Mike Sherman: And of course, we work with experts and former, you know, Barda officials who can ensure that we're making the that we're building that case in a way that is compelling to the reviewer. So, we'll know soon enough how they respond to that, but I feel very good about what was a very complex process. So, if I have a response to the RFP, OK.

And of course, we work with with experts in the former.

Oh.

BARDA officials, who can ensure that we're making that are building that case in a way that is compelling to the reviewers. So we'll know soon enough how they how they respond to that but I feel very good about what was a very comprehensive response to the RFP.

Mike Andriole: And also, once the BARDA contract is secured, are you saying whether you will actively pursue business development as a means to grow your pipeline? Or do you plan to focus internally with the 206, 212, and then the newer agent, 521? Yeah, Maurice, Mike Andriole, we do, as you know, continuously evaluate the external landscape for business development opportunities. That hasn't been said.

Got it.

Also once the BARDA contract is secured or are you, saying, whether you will actively pursue business development as a means to grow your pipeline or do you plan to focus internally on what the two O six to 12 and then the newer aging by two one.

Mike Andriole: The pipeline continues to grow, and in the case of CMX 5.21, growing organically on 206 and 212 are now organic programs that may well require additional investment as well. And so we'll continually evaluate the pipeline internal project opportunities in comparison to the external landscape. But I would say the bar to bring in additional external innovation right now is a little higher than it was, say, last year or the year before, but we do continuously evaluate. Got it. Okay, thanks for taking my questions. I'll have it back in the queue.

Maury, it's Mike again for real we do as you know continuously evaluate the external landscape for business development opportunities that haven't been said the pipeline continues to grow and in the case of <unk> hundred 21 growing organically.

Onto a six to 12 are now organic programs that that may well require additional investment as well and so we'll we'll continually evaluate.

The pipeline internal project opportunities and compare them to the external landscape, but I would say the BARDA bring in.

Additional external innovation right now is a little higher than it was say last year or the year before but we do continuously evaluated.

Got it okay. Thanks for taking my questions and I'll hop back in the queue.

Maurice Raycroft: Thanks, Maury. Our next question comes from Naureen Quibria with Maxim Group. Hi, good morning.

Thanks Martin.

Our next question comes from Noah <unk> with Maxim Group.

Naureen Quibria: Congratulations on the progress and thanks for taking my question. So I guess I also have a question on the bar to contract, and it's really rather very basic. You know, let's say moving forward, you already have a contract, prices are negotiated. What triggers that first procurement? Is that part of the negotiation or, you know, how do you know? Is it automatic?

Hi, good morning, Congrats on the progress and thanks for taking my question I guess.

Also have a question on the BARDA contract and it's really rather very basic.

Let's say moving forward you already have the contract prices are negotiated what triggered that.

The army is that part of the negotiation or adding now.

Is it automatic Vegas when it went.

Mike Sherman: I guess that's what happened. Yeah, that's part of the negotiation is essentially a commitment on that first shipment quantity. So the quantity is part of the negotiation along with the pricing. Future shipments are essentially identified, you know; we propose and have an implied manufacturing schedule that prepares them for those shipments over the course of, and we've always said four to six years, and of course, our proposal is within that timeframe. Those are then subject to BARDA triggering those orders as they go.

Yes, that's part of the negotiation is essentially.

Our commitment on that first.

At first shipment quantity. So the quantity is as part of the negotiation along with the pricing future shipments are essentially identified.

We propose a way and then have an implied manufacturing schedule that.

That prepares those.

For those shipments over the course of any we've always said four to six years and it and of course our proposal.

Within that timeframe. Those are then subject to BARDA triggering those.

Those those orders as they go and we've done a lot of research on that and you can see other.

Naureen Quibria: We've done a lot of research on that. You can see other companies who operate in the space and have BARDA contracts, and they're pretty predictably and reliably executed over that timeframe, but the initial shipment is part of the initial agreement. So you would know that, and essentially, that would be committed up front. Okay, that's very helpful. And then with regard to the one filing, you know, can you talk about the various components?

Companies, who operate in this space and have BARDA contracts, and they're pretty predictably and reliably execute it over over that timeframe, but the but the initial shipment is part of the initial agreement. So you would know that and essentially that would be committed upfront.

Mike Sherman: I think you have, but that's needed for regulatory filing, you know, what else remains apart from the natural history and history study data. Yeah, so there are a few work streams there that have been ongoing, really, since last year, post-acquisition. They include Quinn Farm activities, and CMC activities.

Okay. That's very helpful and then with regards to onto a one filing in.

Can you talk about the various components I think you have but you know that's needed.

They get literally filing you know what else remains apart from the natural history study data.

So there are few work streams there.

That had been ongoing really since since last.

Last year.

Post acquisition they include Clinton farm activities.

M C activities.

Mike Sherman: And we're also gathering data to support what's a broader safety database than just the 50 that comprised the efficacy analysis. Recall that we've treated far more patients than the 50 with ONC-201. And, in fact, the central benefit for the drug goes far beyond the very narrow criteria that were used to select patients. That criteria that was defined by the FDA was intended to isolate the single-agent response data.

And.

We're also gathering.

Data to support that.

What's the broader safety database than just the 50 that comprise the the efficacy analysis recall that we've treated.

Far more patients than the 50 with.

With <unk> 201 and in fact.

The the potential benefit for the drug goes far beyond the very narrow criteria that were used to select patients that criteria that was defined by the FDA was intended to isolate the single agents.

Response data and that's why that that datasets narrow that having been said the safety database it will be much broader too.

Mike Sherman: And that's why that data set is narrow. That having been said, the safety database will be much broader to support that submission. You mentioned the other element, natural disease history, which is underway. There's quite a bit of alignment among those in the field that this mutation, obviously, is a challenging one to treat, and yet it's relatively new.

To support that that submission you mentioned the other element.

Natural disease history, which which is underway.

There's there's.

Quite a quite a bit of alignment among among those in the field that this mutation.

Obviously, it is a challenging one to treat.

And yet it's relatively new and so the.

Naureen Quibria: And so having data to support that assumption is going to be helpful and supportive of the application. So we're gathering that both for patients that precisely meet the same criteria as in our efficacy analysis, as well as for even a broader population, which, frankly, will be helpful as we think about future development of the drug to identify which prognostic factors and demographic factors are important in predicting the activity and outcomes in these patients.

Having data to support those.

That that that assumption is is going to be helpful and supportive of the applications. So we're gathering that both for patients that precisely meet the same criteria as in our efficacy analysis as well as even a more broader population, which frankly will be helpful. As we.

Think about future development of the drug to identify which prognostic factors.

Demographic factors are important and predicting the activity and outcomes in these in these patients and then the final piece. We referenced is it's important to have alignment as youre going into a potential NDA.

Naureen Quibria: And then the final piece we referenced is it's important to have alignment as you're going into a potential NDA review on a confirmatory trial, which if we are able to secure a pool, you would want to have a confirmatory trial up and running. And so we are collaborating with the FDA to ensure we have alignment on that design and that dialogue. Well, that's actually very helpful. I was just going to ask where they come from and how they reach your child, but you just answered that. Thank you. That's it for me.

NDA review on a.

<unk> trial, which if we are able to secure a.

And accelerated approval you would want to have a confirmatory trial up and running and so we are collaborating with the FDA to ensure we have alignment on that on that design and that.

That dialogue is underway.

Okay.

That's actually very helpful and just anything that becomes Wilmington region, but you just answered it.

Thank you that's it from.

Ed White: Her next question comes from Ed White with H.C. Wainwright. Good morning. Thanks for taking my question. So maybe just to continue on with AHRQ 201. You're going to discuss the first-line randomized placebo-controlled phase three trial with the FDA. Do you need any of that safety data for the submission? I know you're gonna be running it concurrently with the submission. I'm just curious about that.

Our next question comes from Ed White with H C Wainwright.

Yeah.

Mike Sherman: And anything you can tell us about what your expectations are for that study, perhaps the size of the study, length of time to completion, et cetera. Thank you. Yeah, I think I'll answer that, and Allen or Joshua want to add to it, but I do not expect that safety data from that trial would be required as part of the NDA submission. We have identified the population of patients and number of patients that would be part of that submission in prior discussions with the FDA and have been, particularly given the safety profile we've seen on this drug.

Good morning, Thanks for taking my questions.

So maybe just to continue on with the two O one.

You're going to discuss the.

With the FDA.

First line randomized.

Placebo controlled phase III trial.

Do you need any of that safety data.

For the submission I know you're going to be running it concurrently with the submission I'm just curious about that and anything you can tell us about what your expectations are for that study, perhaps the size of the study.

The time to completion et cetera. Thank you.

Yeah, I think I can answer that and Alan or Josh you want to add to it but.

I do not we do not expect that safety data from that trial would be required as.

As part of the NDA submission, we have identified the population of patients number of patients that would be part of that submission in prior discussions with the FDA and they've been particularly given the safety profile. We've seen on this drug we think that will.

Mike Sherman: We think that will be sufficient, aside from describing this as a frontline combination with radiation trial and randomized. I think it would be prudent to wait to have feedback from the FDA on things like end points and size, which would then determine the timeline to enroll that for a future update. This is Josh.

Will be sufficient.

<unk> from <unk>.

Describing this as a frontline.

Combination with radiation trial randomized.

I think it would be prudent to wait to have feedback from the FDA on things like endpoints in size, which would then determine the timeline to to enroll that for future update.

Joshua Allen: I would just add that the safety of ARN2-01 has already been evaluated in this population in the pediatric setting, where a similar dose, as was used in monotherapy, was found to be appropriate for moving forward. So there is that safety data to already leverage from prior experience. Thanks, and then there is a Tim Bexford question.

This is Josh I would just add that I would just add that the.

The tier one has already been evaluated in this population in the pediatric setting.

We're at similar dose.

Used in monotherapy.

Was was found to be appropriate for moving forward. So there is that safety data to already leverage from prior experience.

Ed White: Just wanted to get your thoughts on demand outside the U.S. You know, with the ongoing war in the Ukraine, I'm just wondering if you're getting any inquiries from outside the U.S. for other national stockpiles. And then, how should we be thinking about manufacturing for the first delivery? Are you ready at this point? You had said you expected the RFP and first delivery in the second quarter. How much are you prepared to deliver right now?

Thanks.

And then attempt to extra question.

Just wanted to get your thoughts on.

Demand.

Outside.

The U S.

With the ongoing war, Ukraine, just wondering if youre getting any <unk>.

Inquiries from outside the U S for other national stockpiles.

And then how should we be thinking about the manufacturing and the first delivery are you ready at this point you.

You had said you expect the RFP and first delivery in the second quarter.

How much are you prepared to deliver right now.

Could you deliver the full year worth it in the quarter or should we expect it to see it be spread out over the over the.

The last three quarters of the year.

Mark maybe I'll start with the <unk>.

Mike Sherman: Could you deliver the full year's worth in the quarter, or should we expect to see it be spread out over the last three quarters of the year? Mike, maybe I'll start with the first part of that question, and others can come in as we get to the second, but the international opportunity is, as we have said, really all along: the case for pandemic preparedness has never been stronger than it is now. We'll start that work in Canada and explore registration there and potential stockpiles in Canada. There have been other countermeasures that have successfully been stockpiled in Canada.

First part of that question and others can chime in as well.

We get to the second but the.

The international opportunity.

As we have said.

Really all along be the case for pandemic preparedness has never been stronger than it is now we will start that work in Canada.

And explore registration there and potential stockpiling in Canada, there have been other countermeasures that have successfully been stockpiled.

Mike Sherman: As it relates to Europe, Hara, the health emergency and response authority, is in the process of being organized and capitalized to be essentially the European equivalent of Arda in the United States. I think that could be a centralized procurement mechanism for Europe over the life cycle of comeback, and yet in the near term, it's probably going to be member country specific. Where there could be opportunity, but we're continuing to explore and prioritize those opportunities and the... Geopolitical uncertainty in Eastern Europe is certainly a consideration for folks on this topic.

Canada is.

As it relates to Europe .

Kara the health Emergency response authority.

In the process of being organized and capitalized.

Essentially the European equivalent of.

BARDA in the United States, let's say that could be a centralized procurement mechanism for Europe over the lifecycle of cutbacks and yet in the nearer term, it's probably going to be member country specific.

Where there could be opportunity, but where.

We're continuing to work.

Explore and prioritize those opportunities.

And.

The GOP.

Mike Sherman: But we don't see that as probably a 2022 opportunity, but probably after that. And the second part of your question: we indeed have prepared for and are in a position to ship, you know, roughly a fifth of that full 1.7 million. The question remains as to whether, you know, BARDA, how much of that they want to take on right away and, and, and the pricing associated with that. So that'll be one of the first key terms that's part of the negotiation. Okay, thanks, Mike. And perhaps just the last question on 521.

To your political uncertainty in eastern Europe .

Certainly a consideration for folks on on this topic, but.

We don't see that as probably a 2022 opportunity but.

Likely after that.

And the second part of your question.

Deed.

We indeed have pre.

Prepared and are in a position to ship.

Roughly.

A fifth of that full $1 7 million.

<unk> remains as to whether BARDA, how much of that they want to take take on right away and and.

And the pricing associated with that so that that'll be one of the first key.

Sort of terms, that's part of the negotiation.

Ed White: You know, we saw the impressive data, and you reviewed it again. I'm just curious as to how we should be thinking about the timing for, you know, initial studies and when we can see human data. Yeah, I may I may just defer that question, perhaps to our next call, the data is pretty fresh, and there's actually data ongoing. I will say that, given the prior development, it's already been in humans. And, and, you know, really attractive safety profile; we think that we'd be able to move very quickly on that. But, but I think on a subsequent call, we'll give you a little bit more granularity on that. You know, we're looking at all strategic options for how we can move that one forward.

Okay. Thanks, Mike.

And perhaps just the last question on.

And $5 21.

We saw the impressive data and you reviewed it again I'm just curious as to how should we should be thinking about the.

Timing for <unk>.

Initial studies and when we can see.

The human data.

Yeah, I mean, I may just defer that question, perhaps to tilt to our next call. The data is pretty fresh and Theres actually data ongoing I will say that given the prior development that's already been in humans.

And and.

So really attractive safety profile, we think that we'd be able to move very quickly on that but.

I think in our in a subsequent call, we'll give you a little bit more granularity to that we're looking at all strategic options for how we move that one forward of course.

Mike Sherman: Okay, thanks Mike for taking my question. Our next question comes from Soumit Roy with Jones Research. Hi everyone, thank you for taking the question and congrats on the progress. Wanted to ask a little bit if you could elaborate on the natural history study. What are you looking at?

Okay. Thanks, Mike for taking my question.

Okay.

Our next question comes from Sumit Roy with Jones Research.

Hi, everyone. Thank you for taking my question and congrats on the progress.

Wanted to ask a little bit if you can elaborate on the natural history study.

Soumit Roy: If you can give us the size of the study you're doing, you're going to look at progression of the disease after frontline and second-line treatment. And when should we expect that data to be available? And maybe I'll, I'll have Allen describe the nature of the study in both sort of part A and then part B, and we can come back to the timing. Sure, thanks. This is Allen.

What are you looking at if you can give us the size of the study you're doing you're going to look at progression of the disease after frontline and second line treatment.

And when should we expect that data to be available.

And maybe I'll I'll have Ann.

Alan can describe the nature of the study and both sort of part a and then part B and we can come back to the timing.

Sure. Thanks This is Alan.

Allen Melemed: We are looking at it in two separate cohorts. I think the first cohort is we're looking at a population that exactly mirrors the data that we have for the OMB-201 in the registration cohort. So essentially, we're looking for a similar number of patients that we saw in the registration cohort. And in that analysis, again, it's a retrospective study. We're going to be doing it, blinded, independent review, to see what mood expects for not just response rate but duration response in the process.

We are looking at it in two separate cohorts.

Cohort as we're looking at a population that exactly mirrors the data that we have for the op two on in the registration cohort.

So essentially we're looking for a similar number of patients that we saw in the registration cohort and in that analysis and again its a retrospective study.

Going to be doing a blinded independent review to see what you would expect for not just response rate, but also duration of response in that population and that's going to be the most representative of what you would expect to see as an activity with some other agency side about 201.

Allen Melemed: I think that's going to be the most representative of what you'd expect to see as activity with some other agents besides OP201. As you can imagine, with a small sample size, you have less understanding about prognostic variables, such as PFS and survival, because there are a lot of factors that could be aberrant.

As you can imagine with a small sample size you have less understanding about prognostic variables, such as PFS and survival because there are a lot of factors.

Factors that could be.

Mike Sherman: Therefore, we're doing a second cohort, which is a much larger study, which will include more broader populations, regardless of their line and lineage, to really understand more about overall survival and progression of survival in the population. That is not going to be a blinded review, but we're going to be looking more to see general characteristics of the disease that can help us understand the prognostic and predictive variables better just in the... And I'll pass it back to Mike for any questions on it. Yeah, and what's interesting is the FDA has really been engaged in all of these programs and eager for data.

And therefore, we're doing a second cohort, which is a much larger study, which will include more broader populations regardless.

Lineage to really understand more of overall survival and progression free survival in that population that is not currently blinded review how about we're going to be looking more to see general characteristics of the disease that can help us understand their prognostic and predictive variables battery test in this specific population and all.

Pass it back to Mike are there any questions on the timing.

Mike Sherman: So it may be somewhat contingent on the pace with which they want to see this data. As it's generated, it's possible even that we assess that first cohort of patients, the one that matched the criteria used for the 50 patient cohort, in two steps so that we can share, you know, that with the FDA as we go. What we'll do is essentially mirror our public communications of that data as we share it with the FDA. So, and I expect to be able to give an update on that data. Data here and before before the middle of the year, so the first half.

Yes.

The what's interesting is the FDA has has really been engaged.

On all of these these programs and eager for data. So it may be somewhat contingent on the pace with which they want to see this data as it's generated it's possible even that we missed.

Assess that that first cohort.

Ah patients that are the ones that match the criteria used for the 50 patient cohort that we evaluate that in two steps so that we can share.

That with the FDA as we go what we will do is essentially mirror.

Our public communications of that data as well.

As we shared with the FDA, so and I expect to be able to give.

An update on that on that data here before it before the middle of the year.

Okay. Okay. So.

The first half talk with the FDA on the frontline setting trial.

Mike Sherman: Talk with the FDA about the frontline setting trial. Will that talk include the conversation about the NDA and the National History Filing, or are there going to be two separate talks? Yeah, we actually have had conversations with the FDA on natural disease history, and that's actually ongoing. So it's, it will, it will touch both. And one last question is: you mentioned the progress on the MRD negative being a potential end point in AML. Could you elaborate on that? Do we think that?

Will that talk include.

The conversation about the India, and the natural history of finding or they're going to be two separate meetings.

Yeah, we actually have had conversations with the FDA on natural disease history and that that is that's actually ongoing so.

It's it will it will touch both.

Okay got it and.

One last question is you mentioned the progress on the M already negative being a potential endpoint.

Any email could you elaborate on that do we think that could be a go forward strategy for euro him a frontline AML trial.

Soumit Roy: [inaudible] be a go-forward strategy for your front-line aim. Yeah, it's something we've talked about before. When we acquired DSTAT, MRD was not really recognized as, and it still isn't recognized formally as an endpoint or a certain endpoint in AML, and yet we had seen early data that suggested its power in terms of predicting both durability of responses, event-free survival, and overall survival. And so we incorporated that into our trial design, both as an early point assessment, as well as had conversations with the FDA about using it as a primary endpoint for the overall trial.

Yes, it's something we've we've talked about before when we when.

When we.

Quired D stat <unk>.

D was not really recognized as a well it still isn't recognized formerly as an endpoint or a surrogate endpoint in AML and yet.

We had seen early data that suggests that its power in terms of predicting both the durability of responses event free survival and overall survival and so we incorporated that into our trial design, both as an early point assessment as well as had conversations the EF.

P a about using it as a primary endpoint for the overall trial it would allow us to essentially run a much smaller trial and faster to two end points they were not ready to.

Soumit Roy: It would allow us to essentially run a much smaller trial and faster two endpoints. They were not ready to agree to that at the time, and so we used alternative endpoints, event-free survival, and overall survival for that trial, and yet they left the door open.

Agreed to that at the time and so we use alternative endpoints event free survival and overall survival for that trial and yet they left the door open and I think you've seen.

Mike Sherman: And I think you've seen at least one other company using MRD as a primary endpoint in that setting, and they've left the door open that if we're able to provide some additional data, in particular from the early cohort of this trial, that can support that, then they would be open to that as long as the portion of the trial that you use for submission hadn't been unblinded. So the reference there is that as that development continues, it may be that that's always a lever that we can pull to potentially accelerate the timelines, both smaller trial size than is currently contemplated, as well as more rapid time to an endpoint assessment.

At least one other company is using <unk> as a primary endpoint and in that setting and they've left the door open that if we're able to provide some additional data in particular from the early.

Cohort of this trial that can support that then they would be open to that as long as the trial hadn't or the portion of the trial that you use for submission hasn't been unblinded. So.

But the reference there is that.

As that development continues it may be that that's always a lever that we can pull to potentially accelerate the timelines. Both smaller trial size that is currently contemplated as well as more more rapid time to an endpoint assessment.

Hey, Thank you so much and just to just to add in this.

He has not approved a drug for AML. These M&A yet.

There is big.

Mike Sherman: Thank you for the conversation, becoming potentially more open to being looking at complete response rate and other measures. Again, we'll need to continue with this research and really show a clinically meaningful difference to have a conversation with them. Really appreciate the color, and thanks.

Becoming potentially more openness to looking at complete response rate and other measures again, we'll need to continue with this reset doesn't really show a clinically meaningful difference to have a conversation with FDA.

Alright.

Really appreciate the color and thanks again for taking the questions.

Thank you our next.

Soumit Roy: Thank you. Our next question comes from Troy Langford with Callum. Hi, congrats on the progress and thanks for taking our questions. I just have a couple quick ones on ONG-201 and then I have a follow-up. So first, on ONG-201, do you all expect that you will need any significant alignment from the FDA on the analysis of the natural history data in comparison with the ONG-201 registrational cohort? Or do you think you have a pretty good idea of what they want?

Our next question comes from Troy Lankford with Cowen.

Troy Langford: And then how quickly after the completion of the natural history study do you think you could file for approval? Yeah, that's part of the discussion that we'll have about exactly, you know, what kind of analysis is expected with small data sets in both cases. I think there are some limitations, obviously, on what you can do with that data and that having been said, we would expect to conclude from that data that again responses are rare. And if they do occur with current standards of care, that they're not, they're not durable, as we see with all 201.

Hi, Congrats on the progress and thanks for taking our questions I just have a couple of quick ones on October one and then I have a.

Follow up question.

So first on until one do you expect you'll need a seat now alignment from the FDA on the analysis of the natural history data in comparison with B until one registrational cohort or do you think you have a pretty good idea of what they want and then how quickly after the completion of the natural the natural history study do you think you could file for approval.

Yes, that's part of the discussion that we'll have is exactly what kind of analysis is expected with the with small datasets in both cases.

Yeah.

There's a there's some limitations obviously on what you can what you can do with that data.

As said.

We would expect.

Two to conclude from that data.

That debt again responses are rare and if they do occur.

With current standards of care that they're not that theyre not durable as we see with <unk>.

Mike Sherman: So part of these ongoing discussions is just clarification on how the FDA will be looking at that data. And as for the timeline to be able to submit, that's the update that will be given here before mid-year. These discussions, you know, with the FDA have been pretty continuous. And so we've got a nice open dialogue as we make progress and are able to share data as it comes from the work that we're doing on all of those work streams.

With onshore ones. So part of these ongoing discussions or just clarification for.

How the FDA.

It will be we'll be looking at that data.

And as for the timeline to be able to submit that's the update that will give you.

Here before mid year will have these discussions.

With the FDA had been pretty continuous and so we've got a.

Mike Sherman: And so by the middle of the year, we'll be able to give you guidance on the timelines for potential NDA submission. Okay, great. And then just one quick question on the pipeline. Can you provide any update on the work for ONC 206? Like, do you think we can still see data from this program possibly this year? Maybe Josh can give an update on that program. Sure, so just as a quick reminder, off to a six is proceeding through dose escalation currently and two advanced oncology trials, really both focused on brain tumors. One is a sponsored study conducted at the NIH on adult recurrent DNS tumors.

Nice open dialog as we make progress and are able to share.

Data as it comes from the work that we're doing on all of those work streams and so by by Middle of the year, we will be able to give you.

Guidance on the timelines for a potential NDA submission.

Okay, Great and then just one quick question on the pipeline keep her eyes any update on the worker onto a six like do you think we can still see data from this program, possibly this year.

Maybe Josh can give an update on that program.

Joshua Allen: The other is the more recently launched trial in the pediatric brain tumor setting. Both of those are being done efficiently and effectively proceeding up until the maximum tolerated dose and proceeding into dose expansion cohorts where experience is to be gained and brain tumors that hold promise for this drug based on clinical studies.

Sure. So just as a quick reminder, up to a six is proceeding through dose escalation correctly to advanced oncology trials really both focused on brain tumors.

One is the sponsored study conducted at the NIH and adult recurrent CNS tumors. The other is the more recently launched trial.

In the pediatric brain tumor setting.

Setting both of those are aimed at efficiently and effectively preceding up until a maximum tolerated dose in the preceding it.

<unk> cohorts.

Joshua Allen: Due to the nature of both of those studies, the timelines and outcomes of the studies are really dependent on the clinical data that emerges as they come. So at this point, we expect those studies to continue dose escalation. I think we've got it in the past that we expect that to play out throughout the course of the year, and once we get to the end of that, we'll be able to share that data. But in the meantime, you can just assume that we're continuing along, and dose escalation has to be expected.

Where experience is to be gained.

Brain tumors that hold promise for this drug based on the clinical studies due to the nature of both of those.

Timelines the outcomes of the study are really dependent on the clinical data that our merchants as they come. So at this point, we expect those studies to continue dose escalation I think we've guided in the past that we expect that.

Could play out throughout the course of the year and.

Once we get to the end of that we'll be able to share that data, but in the meantime.

Just.

Assume that we're continuing along in dose escalation as would be expected.

Okay, great. Thanks.

Troy. This is Alan just said, we're also continuing to evaluate 212.

Troy Langford: Okay. Great. Thanks. Troy, this is Allen.

Allen Melemed: We're also continuing to evaluate OX212, which is, we haven't given guidance on that, but we're not just stopping on just one admiprodont. That's really helpful. Thanks a lot for all the time. That concludes today's question and answer session. I'd like to turn the call back to Mike Sherman for closing remarks. Ray, thanks again, everyone, for your time this morning. I look forward to continuing to update you in the coming months. Have a good day. This concludes today's conference call. Thank you for participating. You may now disconnect. BF-WATCH TV 2021, The Ultimate Parody Site! BF-WATCH TV 2021

Which is we have given guidance on that but we're not just stopping on just one they didn't pronounce.

Pronounce.

Okay. That's really helpful. Thanks, a lot for all the color.

That concludes today's question and answer session I would like to turn the call back to Mike Sherman for closing remarks.

Yeah.

Great. Thanks again, everyone for your time. This morning look forward to continuing to update you in the coming months have a good day.

This concludes today's conference call. Thank you for participating you may now disconnect.

Okay.

[music].