Chimerix (CMRX) Q4 2021 Earnings Call Transcript

Chimerix (CMRX -1.75%)
Q4 2021 Earnings Call
Mar 01, 2022, 8:30 a.m. ET

Contents:

• Prepared Remarks
• Questions and Answers
• Call Participants

Prepared Remarks:

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.

Michelle LaSpaluto -- Vice President of Strategic Planning and Investor Relations

Thank you. 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 investor 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 of Imipridones technology officer, Josh Allen. 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. 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 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.

Mike Sherman -- President and Chief Executive Officer

Thanks, Michelle. Good morning, everyone, and 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 TEMBEXA 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. The TEMBEXA 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 TEMBEXA's 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 robust resistance profile. As expected, BARDA's requests called for up to 1.7 million courses of treatment. 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 Oncoceutics, which brought to the organization some outstanding people and a pipeline of promising oncology assets. Compelling response data had already been reported for ONC201 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 revealed an even stronger dataset to support this drug as a treatment for H3 K27M-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. 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 internal consistency across the endpoints. The fact that many of the patients continued to receive ONC201 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. During 2021, we held a number of meetings with the FDA to discuss our preparations for a potential new drug application for ONC201 and have been executing on those deliverables in the meantime. This work includes important CMC and clean farm 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 frontline randomized trial design. We plan to initiate 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. We've also collaborated with the FDA on a natural disease history study.

Because the H3 K27M mutation is relative -- but 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 ONC201. You can imagine this won't be a large data set as awareness of ONC201 has been high and many patients sought out treatment with ONC201 through clinical trials or expanded access. We'll know more about the size of that dataset in the next couple of months as that information is collected and verified. Keep in mind, this is a retrospective study which will collect data from previously treated patients.

So the response analysis, we plan to subject this data to the same blinded independent central review process we use for the ONC201 efficacy analysis. We expect to be far enough along in each of these work streams and ongoing consultation with the FDA to provide guidance on our regulatory timelines by mid-year. Now, let me turn to our DSTAT program, the DASH phase 3 trial in front line 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.

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 steps development. One tailwind in that -- in that space in AML in particular, the momentum we anticipated supporting MRD as a potential [Inaudible] point 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. Lanier to discuss recent developments with CMX521.

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 antiviral library. As the world begins to 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, as this highly credible team of collaborators saw the results of initial experiments. This project became their highest priority, 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 -- Chief Science Officer

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. This was done by exciting collaboration with READDI, the rapidly emerging antiviral drug development initiative at the University of North Carolina Chapel Hill. READDI and UNC have deep expertise in this area and have been actively involved in efforts to develop other agents for the current pandemic, including data of the SARS-CoV-2 antiviral, which received emergency use authorization late last year.

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 SARS-CoV-2. CMX521 is particularly attractive because it was developed through a phase 1 healthy volunteer study for another indication. The build up to that study included standard [Inaudible] help and safety, GMP manufacturing, and all the other myriad studies needed to start human trials.

This previously completed were de-risks many of the common pitfalls of early drug development. The most important of which often involves safety, CMX521 has a very favorable safety profile in animals and humans. It's not genotoxic, cytotoxic or mitotoxic, and was well tolerated up to very high levels of 2.4 grams in a healthy volunteer phase 1 study. 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 a very compelling activity in a well-studied mouse model of COVID and in the mouse model, one day in the mouse model is roughly equivalent to a humans. Administration of inhaled aerosolized CMX521 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 placebo was administered at various times relative to SARS-CoV-2 infection to explore the potential for both prophylactic and therapeutic efficacy.

Prophylactic administration of CMX521 starting eight hours prior to infection to one dose eight hours prior to infection reduced average viral titers in the lungs on day four by more than 3.5 logs for about 99.99%, and preventing weight loss and clinical progression versus placebo. Treatment studies of CMX521 initiated at the time of infection, or 8 hours or 16 hours post-infection. Also significantly reduced viral load titer at day four. CMX521 treatment protected mice from 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, CMX521 initiated 16 hours post-infection, reduced average viral lung titer the amount of SARS-CoV-2 in the lungs by more than 2.5 logs, or more than 99%. Importantly, contemporary modeling methods to estimate the translation of these efficacious doses in mice to target clinical doses predicts the doses in humans should fall within a range that could be delivered with commonly used systems like battery-powered, portable nebulizer 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 antivirals in this model system and to evaluate efficacy in combination with other antivirals. 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 model.

Furthermore, we have kg of GMP drive ready for development, excellent patent life, and a simple potentially of short path to proof of concept in humans. While we focus on the advancement of our oncology pipeline and progress toward a TEMBEXA procurement agreement with BARDA, this collaboration with READDI allowed us to efficiently evaluate our antiviral library and CMX521 looks very promising. We have an upcoming [Inaudible] or presentation later this month at the International Conference on Antiviral Research in Seattle, where we'll discuss this data in greater detail. And with that, I'll turn the call over to Mike Andriole.

Mike Andriole -- Chief Financial and Business Officer

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 Oncoceutics in January of '21.

Importantly, we do not allow any further payments related to that transaction until U.S. 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.

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 TEMBEXA to BARDA, we could potentially use this credit facility to bridge the GAAP 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.  As Mike have 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 the potential revenue from the sale of TEMBEXA 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 ONC201 commercialization. As we have confirmation on the specifics of a potential BARDA procurement, we will provide more precise financial guidance. 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 dollars, or $0.19 per basic and diluted share in the fourth quarter of 2020.

The fourth quarter included a $20 million success milestone payment to the legacy Oncoceutics shareholders in relation to the achievement of a 20% overall response rate by RANO-HGG for ONC201. Revenues for the fourth quarter of '21 decrease to $46.000 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 is primarily related to the $20 million success payment for ONC201.

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. With that overview, I'll turn the call back to Mike for closing remarks. Mike?

Mike Sherman -- President and Chief Executive Officer

Thanks, Mike. We certainly have an exciting year ahead with catalysts in both the TEMBEXA and ONC201 programs. In addition now to CMX521, 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.

Questions & Answers:

Operator

[Operator instructions] Our first question comes from Maury Raycroft with Jefferies.

Maury Raycroft -- Jefferies -- Analyst

Hi, good morning. Congrats on the progress, and thanks for taking my questions. I was going to ask as barter 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 dialog with BARDA, particularly with our pricing premium versus [Inaudible]?

Mike Sherman -- President and Chief Executive Officer

I think typically you see these processes play out over 60 days 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 ends up being a small portion of the contract. We feel really good about our proposal for pricing and supported that analytically. And of course, we work with experts and former BARDA officials who can ensure that we're making that or 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.

Maury Raycroft -- Jefferies -- Analyst

Got it. 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 by '21?

Mike Andriole -- Chief Financial and Business Officer

Yeah. Maury, it's Mike Andriole. 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 CMX521 growing organically, ONC206 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 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 evaluate it.

Maury Raycroft -- Jefferies -- Analyst

Got it. OK. Thanks for taking my questions. I'll have back in the queue.

Operator

Our next question comes from Naureen Quibria with Maxim Group.

Naureen Quibria -- Maxim Group -- Analyst

Hi, good morning. Congrats on the progress, and thanks for taking my question. So I guess, I 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 triggers that [Inaudible] procurement? Is that part of the negotiation? Or how do you know, is it automatic? I guess that's what I meant when I asked.

Mike Sherman -- President and Chief Executive Officer

Yeah. That's part of the negotiation, is essentially a commitment on that first -- that first shipment quantities. So the quantity is -- this part of the negotiation along with the pricing. Future shipments are essentially identified.

We propose a way then to have an implied manufacturing schedule that prepares for those shipments over the course of -- as we've always said four to six years and of course, our our proposal is within that timeframe. Those are then subject to BARDA triggering those, those orders as they go. And we've done a lot of research on that. You can see other companies who operate in this space and have BARDA contracts, and they're pretty predictably and reliably executed over that timeframe, but that the initial shipment is part of the initial agreement.

So you would know that and essentially that would be committed upfront.

Naureen Quibria -- Maxim Group -- Analyst

OK. That's very helpful. And then, with regards to ONC201 filing, can you talk about the various components I think you have but that's needed for regulatory filing, what else remains apart from the natural history study data?

Mike Sherman -- President and Chief Executive Officer

So there are a few workstreams there that have been ongoing really since last year post-acquisition. They include [Inaudible] activities, CMC activities. And we're also gathering the data to support the what's a broader safety database than just the 50 that comprise the efficacy analysis. Recall that we've treated far more patients than the 50  with ONC201.

And in fact, the the central benefit for the drug goes far beyond the very narrow criteria that we're used to select patients that that criteria that was defined by the FDA was intended to isolate these single agents response data, and that's why that data sets narrow. That having been said the safety database, it 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.

And so the -- having data to support those that 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 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 in predicting the activity and outcomes in these patients. 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 an 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 design and that that dialog is underway.

Naureen Quibria -- Maxim Group -- Analyst

Well. That's actually very helpful. I was just going to ask about the complimentary file, but you just answered it. Thank you.

That's it for me.

Operator

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

Ed White -- H.C. Wainwright -- Analyst

Good morning. Thanks for taking my questions. So maybe just to continue on with the ONC201, you're going to discuss the -- with the FDA the first line randomized placebo controlled phase 3 trial. Do you need any of that safety data for this 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, length of time to completion, etc.? Thank you.

Mike Sherman -- President and Chief Executive Officer

Yeah. I think I can -- I'll answer that, and Allen or Josh, you want to add to it.  We do not expect that safety data from that trial would be required as part of the 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 then particularly given the safety profile we've seen on this drug, we think that will be sufficient.

Aside from describing this as a front line combination with radiation trial and randomize, I think it would be prudent to wait to have feedback from the FDA on things like endpoints and size, which would then determine the timeline to enroll that for a future update.

Josh Allen -- Chief Technology Officer, Imipridones

This is Josh -- I would just add that the safety of ONC201 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 CPD that's already leverage from prior experience. 

Ed White -- H.C. Wainwright -- Analyst

Thanks. And then a TEMBEXA question, just wanted to get your thoughts on demand outside the U.S.? With the ongoing war in the Ukraine, just wondering if you were getting any inquiries from outside the U.S. for other national stockpiles? And then, how should we be thinking about the manufacturing in the first delivery? Are you ready at this point? You had said you expect the the RPM first delivery in the second quarter, how much are you prepared to deliver right now? Could you deliver the full year worth in the quarter? Or should we expect to see it be spread out over the last three quarters of the year?

Mike Andriole -- Chief Financial and Business Officer

Mike, maybe I'll start with the first part of that question, and others can chime in as we get to the second, but the international opportunity it 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 stockpiling. In Canada, there have been other countermeasures that have successfully been stockpiled in Canada as it relates to Europe. HERA, the Health Emergency and Response Authority, is in the process of being organized and capitalized.

It's essentially the European equivalent of BARDA in the United States. I think that could be a centralized procurement mechanism for Europe over the lifecycle of TEMBEXA, 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, but we don't see that as probably a 2022 opportunity, but likely after that.

Mike Sherman -- President and Chief Executive Officer

And the second part of your question. We indeed have prepared and are in a position to ship roughly a fifth of that for 1.7 million. The question remains as to whether BARDA, how much of that they want to take on right away and the pricing associated with that. So that'll be one of the first key search terms, that's part of the negotiation. 

Ed White -- H.C. Wainwright -- Analyst

OK. Thanks, Mike, and perhaps just the last question on '521. 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 initial studies and when we can see the human data. 

Mike Sherman -- President and Chief Executive Officer

Yeah. 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 that's already been in humans and 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.

Ed White -- H.C. Wainwright -- Analyst

OK. Thanks, Mike, for taking my question.

Operator

Our next question comes from Soumit Roy with JonesResearch.

Soumit Roy -- JonesResearch -- Analyst

Hi, everyone. Thank you for taking the question and congrats on the progress. I wanted to ask a little bit if you can elaborate on the natural history study. 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 front line, second line treatment? And when should we expect that data to be available?

Mike Andriole -- Chief Financial and Business Officer

And maybe I'll have Allen can describe the nature of this study in both part A and then part B and we can come back to the timing.

Allen Melemed -- Chief Medical Officer

Sure. Thanks. This is Allen. We are looking at in two separate cohorts; The first cohort is we're looking at a population that exactly mirrors the data that we have for the ONC201 in the registration card.

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 a blinded, independent review to see what you would expect for not just response rates, but also duration response in a population. [Inaudible] the most representative of what you'd expect to see as an activity with some other age beside ONC201.

As you can imagine, with a small sample size, you have less understanding about prognostic variables such as [Inaudible] and survival because there are a lot of factors that could be aberrant. 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 of overall survival and progression free survival in a population that is not going to be blinded review. And 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 this specific population. And I'll pass it back to Mike, presenting questions on the timing.

Mike Sherman -- President and Chief Executive Officer

Yeah. And what's interesting is the FDA has really been engaged on all of 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 assess that that first cohort of patients, the one that matched 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'll do is essentially near our public communications of that data,  as we share with the FDA. So and expect to be able to give an update on that here and before before the middle of the year.

Soumit Roy -- JonesResearch -- Analyst

OK. OK. So the first tough talk with the FDA on the front setting trial, will that talk include the conversation about the NDA natural history filing? Or they're going to be two separate meetings?

Mike Sherman -- President and Chief Executive Officer

Yeah. We actually have had conversations with the FDA on natural disease history and that's actually ongoing. So it will it will touch both.

Soumit Roy -- JonesResearch -- Analyst

OK. Got it. And one last question is, you mentioned the progress on the MRD negative being a potential endpoint in AML, could you elaborate on that? Do we think that could be a go forward strategy for your aim of frontline AML trial?

Mike Sherman -- President and Chief Executive Officer

Yeah. It's something we've talked about before. When we acquired DSTAT, MRD was not really recognized as well -- and still isn't recognized formally as an endpoint or endpoint in AML. And yet, we had seen early data that suggests that 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. It would've allow us to essentially run a much smaller trial and faster to endpoints. They were not ready to agree 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 at least one other company is 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 then they would be open to that, as long as that trial hadn't or 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's always a lever that we can pull to potentially accelerate the timelines both smaller trial size than is currently contemplated, as well as a more more rapid time to an endpoint assessment. 

Allen Melemed -- Chief Medical Officer

And just to just to add in this. FDA has not approved a drug for a AML based [Inaudible], but I think there is becoming a potentially more openness to be 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 FDA.

Soumit Roy -- JonesResearch -- Analyst

Right. Really appreciate the color, and thanks again for taking the questions. Thank you.

Operator

Our next our next question comes from Troy Langford with Cowen.

Troy Langford -- Cowen and Company -- Analyst

Hi. Congrats on the progress, and thanks for taking our questions. I just have a couple of quick ones on ONC201 and then I have a follow up question. So first, ONC201, it looks like you'll need anything of an alignment from the FDA on the analysis of the natural history data in comparison with the ONC201 registration 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 history study, do you think you could file for approval?

Mike Andriole -- Chief Financial and Business Officer

Yeah. That's part of the discussion that will have is exactly what kind of analysis is expected, with the small data sets in both cases. I think there's 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 not durable as we see with ONC201. So part of these ongoing discussions are just clarification for 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 give you here before mid-year,  we'll have these discussions with the FDA have been pretty continuous. And so we've got a 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 middle of the year, we'll be able to give you a guidance on the timelines for a potential NDA submission.

Troy Langford -- Cowen and Company -- Analyst

OK. Great. And then, just one quick question on the pipeline. [Inaudible] has any update on the worker ONC206.

Do you think we still see data from this program possibly this year?

Mike Andriole -- Chief Financial and Business Officer

Maybe I'll let Josh give an update on that program. 

Josh Allen -- Chief Technology Officer, Imipridones

Sure. So just as a quick reminder, ONC206 proceeding through dose escalation currently in two advanced oncology trials really both focused on brain tumors. One of the sponsors study conducted at the NIH and adult recurrent CNS tumors. The other is the more recently launched trial in the pediatric brain tumor setting.

Both of those are aimed at efficiently and effectively proceeding up until a maximum tolerated dose in the preceding dose expansion cohorts where experience is to be gained in brain tumors that hold promise for this drug based on the clinical studies due to the nature of both of those, the timelines and outcomes of the study 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 guided 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 in dose escalation as would be expected.

Troy Langford -- Cowen and Company -- Analyst

OK. Great. Thanks.

Allen Melemed -- Chief Medical Officer

Troy, that surveillance, we're also continuing to that ONC212, which is we've had given guidance on that, but we're not just stopping on just one [Inaudible].

Troy Langford -- Cowen and Company -- Analyst

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

Operator

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

Mike Sherman -- President and Chief Executive Officer

Great. Thanks again, everyone, for your time this morning. Look forward to continue to update you in the coming months. Have a good day.

Operator

[Operator signoff]

Duration: 44 minutes

Call participants:

Michelle LaSpaluto -- Vice President of Strategic Planning and Investor Relations

Mike Sherman -- President and Chief Executive Officer

Randall Lanier -- Chief Science Officer

Mike Andriole -- Chief Financial and Business Officer

Maury Raycroft -- Jefferies -- Analyst

Naureen Quibria -- Maxim Group -- Analyst

Ed White -- H.C. Wainwright -- Analyst

Josh Allen -- Chief Technology Officer, Imipridones

Soumit Roy -- JonesResearch -- Analyst

Allen Melemed -- Chief Medical Officer

Troy Langford -- Cowen and Company -- Analyst

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