BioXcel Therapeutics, Inc. (BTAI) Earnings Call Transcript & Summary

[Audio Gap] and wish that next year, it will be in person. But it is what it is. We're thrilled to have in the very first session for today, BioXcel Therapeutics. And sitting with us here is CEO, Vimal Mehta; and we also have Will Kane, who is Head of Commercial. Guys, welcome.

Thank you, Geoff.

Welcome, Geoff.

All right. So we'll kick it off with just anything that you guys wanted to start off with prepared remark wise, we have a lot of questions here. We have some questions from investors as well. So, Vimal, over to you.

Thank you, Geoff. Good morning, everyone, and Geoff, thanks for hosting us today. Before I begin, I will give a very quick overview of BioXcel Therapeutics. We are a clinical stage biopharmaceutical company utilizing artificial intelligence platform to develop transformative medicines in neuroscience and immuno-oncology. During our discussion, we'll be making forward-looking statements, and joining me today on the call is, Will Kane, our Chief Commercial Officer. Our 2 most advanced clinical assets are BXCL501 and BXCL701. 501 is a proprietary orally available thin film for acute treatment of agitation resulting from multiple disease neuropsychiatric disorders like schizophrenia and bipolar and dementia, just to name a few. And BXCL701 is a orally available innate immunity activator, which we are combining with checkpoint inhibitor to turn cold tumors to hot, and we believe we have one of the most advanced innate immunity activator in the clinic right now. Just to give some few highlights on our lead program, BXCL501. Our NDA was accepted last month, so we are excited, and we are preparing for commercial readiness, which we will discuss today, and Will will outline what we have done to date and where we are heading. In addition, we got breakthrough therapy designation for dementia indication, agitation resulting from dementia. And we are very excited about that, and we are working with FDA to initiate a Phase III program in the second half of this year. In addition, we have already mentioned that we are planning to file our MAA application with the European regulator, and we expect to file that application in the second half of this year. So those are the kind of very high level key milestone that we have been able to achieve with 501. And just to give a little overview to get to the NDA, our journey from first-in-human to the NDA acceptance has been over 2.5 years. So we moved very rapidly. We have conducted so far, 7 clinical trials for BXCL501 program and dosed over 800 patients. So we have laid a really very strong foundation for the program to continue to build first NDA, which is we -- is in acceptance stage or filed and beyond that additional SNDS. In addition, our overall strategy around 501 is to continue to expand its potential with additional indication geographical expansion as we are doing in Europe as well as expanding in different medical setting. So that's -- those are the 3 key pillars for expanding our neuroscience franchise with 501. We believe we have laid a very robust foundation. And now we have demonstrated clearly the robust treatment effect in 3 different indications. So we will continue to build on that success. In -- for emerging pipeline, we continue to apply our AI platform to identify new agents, and our strategy is to work on the stress related symptoms and we are working on those symptoms, and we are identifying 502 and 503 that we would like to bring in the clinic in future, and we'll provide some updates on those candidates that are developing in our pipeline. For 701, as I indicated, we have 2 Phase II trials ongoing currently. One is in the cold tumors, like aggressive form of prostate cancer, adeno, CRPC and neuroendocrine prostate cancer as well as in the lukewarm or hot tumor checkpoints have gap like either they are refractory, those patients are refractory affected or they are treatment naive. So those are the 2 trials ongoing. We expect the data readout from those trials sometimes in the second half of this year. So that's the kind of overall strategy of the company, but we are moving these 2 programs forward, which were originated from our AI-based platform. And we continue to develop a sustainable pipeline outside this in our neuroscience area. So I will then -- with that brief summary, I will pass it back to Geoff.

Okay. Great. Thanks for that background, Vimal and that's helpful. Let's talk about the [indiscernible] approval, I guess, the most topical news of the day. Help us with kind of how you guys view that, the read-through from that, I would say, to your -- to 501 in dementia. I guess more broadly, though, looking to endpoints in neuroscience?

I think approval of the [indiscernible] was -- tells that this FDA is supportive and see the huge unmet need in this area, like in Alzheimer's patients and dementia. Our drug, which is for acute treatment of agitation resulting from Alzheimer's and dementia. And for all the companies who are working in this space, it could be good news, and it's very -- it seems that FDA supported because it underscores such a high unmet medical need. Particularly for us, we recently received a breakthrough therapy designation for our program, BXCL501, for acute treatment of agitation. And currently, we are having conversations with the FDA to develop a Phase III program. So that's what we are currently focusing on. Just to remind everybody that in our Phase Ib/II trial, which was a TRANQUILITY trial, we got like statistically significant responses with multiple doses, particularly 30 and 60 microgram. It was a dose-dependent response, and we had good safety profile in our trial. So we are building on all of that, and we had used, as Geoff asked, multiple endpoints to demonstrate the efficacy, particularly with 60 microgram dose. And going forward, what we are trying to assess is what is the trial design, size of the trial, safety database required and what is going to be the primary and secondary endpoint, we're getting an alignment on that with the FDA currently.

Got you. Okay. That's helpful. Yes, I mean, I guess, Vimal, is there a way to accelerate the dementia study? I know you mentioned fast track there are some options for that. But is it -- does it do anything to kind of your budget or your plan for the program in dementia, the FDA decision?

I think we feel very encouraged having 2 things approval in this space because Alzheimer's and dementia agitation, there has not been much innovation over the last 2 decades. There has been literally no drug that has ever been approved for acute treatment of agitation. And patients have need like once they get agitated, they need to be treated. Even the progression, even if it slows down the progression of the disease still if patients get agitated, they need to be treated. So we see a very large market opportunity for us. There are about 6 million patients who are suffering from Alzheimer's. In addition, globally, it's a much, much bigger population, and it's going to double by 2050 and almost double in U.S. by 2040. So it's a very large opportunity. 70% of the patient experience agitation. It could be like millions of episodes per year. And 501, we believe, can play an important role in that. So coming back to your specific question, how can we leverage. So we have a lot of interaction with the FDA on different indications. And as I mentioned, that we have already dosed 800 patients. So we leverage all that data to put a package together to come up with a optimal design for our Phase III study as well as endpoints and then translate that if upon successful of the Phase III that how can we get into the approval process. So that's what the company's focus is, and we feel that where we are and strong clinical foundation for 501, we laid a very strong path for us to move it forward in -- for acute treatment of agitation in dementia/Alzheimer's.

Got you. Okay. That's helpful. And good to see my colleague, Greg Harrison, also with us today. Yes. So let's switch gear to talk about the prelaunch prep. Will, help us out with kind of where you are with the launch preparations. What can be done now? And now that you have the filing and just help us with kind of the next -- the cadence of the next 6 months of investment and the milestones we should be thinking about.

Sure. So first, obviously, we're very excited about the NDA acceptance and a PDUFA date in January -- early January of 2022. I'm very confident that given that we're just over 6 months out, 6, 7 months out, we are in a great position with our launch prep we've been working on this for a while now since I joined a year ago and others have joined the team. Starting on the medical front, we have deployed our medical science liaisons as of March. We have 2 teams there. One is the clinical team, which interfaces with clinicians for scientific exchange, and they've been making great strides and introducing the company and also beginning to the dialogue around agitation, the unmet need, et cetera. And we also have the medical managed care team, which will focus more on pharmacy directors and hospitals and payers again, to enable scientific exchange and an understanding of the emerging clinical and economic value proposition that we think we can offer. On the commercial side, the leadership team is nearly in place with the Head of Marketing, market access and commercial ops, having been on board for months now. And we're advancing our recruiting efforts for the VP of Sales. And we're making great progress there. Our goal is to bring on our account management team this month actually. We have 2 members of that team joining. They will start interface with payers and pharmacy directors beginning in the third quarter. That will enable us to get more specific around the pricing elements that we'll have to address and then we're rapidly moving towards completing the design of our sales force size and structure. We've previously indicated we estimate about 75 hospital-based sales representatives and more than 1,500 targeted accounts, which will represent 75% to 80% of the initial value. So we're all in good place there. I'll also add that we continue to build presence in the broader medical community through our presence and presentations at medical conferences, the APA in May, the international society by pullet disorders in May as well. And we have a number of upcoming conferences for the second half of the year.

That's helpful, Will. Yes, I'll hand over to Greg to ask a couple, I mean, just with respect to awareness and things like that in the community. And I have a couple of follow-ups on OUS. Greg?

Sure. Thanks, Geoff. Yes. I guess just following on to your preparation for the launch. How would you characterize the level of awareness of 501 among physicians? And how strong is that? And what efforts do you need to make to ensure that the community is aware of the benefits of 501 relative to existing treatment?

So I would say that we have a building awareness of the company and also of our focus on stress related disorders, particularly agitation and the upcoming NDA review. That's being spearheaded, if you will, by the medical team up until the launch, that team can interface some medical to medical conversations. As I said, we'll also have a significant presence at upcoming medical meetings in the second half of this year, both psychiatry and emergency medicine focus as well. We'll also have a booth of these meetings where both commercial and medical can be present. To continue to advance our name recognition and an understanding of our mission to bring novel treatments for patients who suffer from agitation associated with their schizophrenia, bipolar disorder. We've also launched, as you know, an unbranded educational campaign called Partners in Calm. We launched that in sync with Mental Health Awareness Month in May and the APA Meeting. And that is actually proving to be a good move. We have multiple visits to that website. We're tracking the progress against that, but it's really helping to set a foundation for raising the level of awareness around agitation and the need to treat that agitation. So I think we have a steady cadence. Obviously, until we get to launch, and we have an approval and a package insert that we can promote off of the sales force will be in preparation mode, they'll be in training up to that point. But once we have an approval then we would be certainly ready to hit the streets and begin our launch execution.

Great. And then how do you make the economic argument for use of 501, given that it will likely be significantly more costly than some of the generic options. Just wanted to get your sense of what's the strategy there for communicating how maybe it could actually lower cost overall relative to having people in longer stays in the hospital, et cetera?

First, I'd say that in our market research with both payers and pharmacy directors, they readily acknowledge the unmet need. They also recognize that the current standard of care, if you will, has been in place for a very long time. I mean HALDOL was introduced in 1967. So to that point, it's been antipsychotics and benzodiazepines in multiple formulations, but the actual approach hasn't not really changed much. So they see opportunity for additional treatment options for these patients. They recognize, clearly, this is a generic category. They've managed their pharmacy budgets in that context because of the genericization of the classes et cetera, but they also recognize that innovation brings with it, obviously, a higher price. We're working on ultimately what that price will be for the long-term and also for the near-term in terms of our hospital initiated launch. But we believe we have an emerging value proposition that will ultimately underscore 501's clinical and economic value. A few things we've noted is that patients who present in emergency departments and are injected with antipsychotics and/or benzodiazepines many, many times or oversea dated, they take of a bed in that ER for quite a while that requires monitoring, et cetera. That's an opportunity cost for that hospital since the next patient that walks in with potentially another medical condition that bed is not available to them. And so pharmacy directors have actually raised that issue in our market research unprompted. So I think there is a recognition that as we start to evolve and advance our health economics work, that there are multiple intervention points that could offer resource utilization reductions or cost offsets. The bed issue, the bed occupancy issue being 1 that's already documented in the literature. The need of restraints adds time and complexity to the state and the ER. The impact on staff from a risk and safety perspective, needing to restrain and inject patients also has a high level of awareness in those institutions. So I think we have a solid platform for initial dialogue, and then we'll continue to advance our health economics work in order to start to put and quantify some potential cost offsets or resource benefits against that.

Great. That makes sense. And then could you give some color on whatever discussions you've had with payers? And what's their receptivity to reimbursing 501 and just the general feedback you've gotten from that?

Well, I'd say the nice thing is that in our interactions with both payers and pharmacy directors, there's a consistency. Again, payers underscore the unmet need. Payers like having multiple treatment options, particularly in mental health conditions, et cetera. They recognize the lack of innovation in this space over a long period of time. So 501's profile is very well received in the context of those discussions. They like the route of administration being orally dissolving in a film formulation. They like the rapid onset of action data they like the duration data, they like the tolerability data. So there's a lot of point -- there are lots of points of differentiation that we can obviously communicate once we have a label and we're on the market to press upon them the value of 501. They also anticipate that they will reimburse for it. Initially, the primary decision rests with the hospital, right? It's the hospital pharmacy and pharmacy and therapeutics committee that will make the decision on formulary adoption, et cetera. And then the payer will defer to the hospital. Hospital puts it on formulary, the payer will reimburse. And in this case, we know there's precedence for reimbursement of acute treatment of agitation, ADASUVE demonstrated that they received the C-code, which would enable the hospital to build for ASP plus 6% reimbursement. So there are -- there is not only the unmet need and the recognition of 501's favorable profile. But there are mechanisms already in place that we can leverage to access and engage from a reimbursement perspective.

Great. And then assuming approval, when the launch comes, what are your kind of expectations for the cadence of uptake? Is that a situation where you need to build up over time and really take that time to educate the market? Or if some of this work in advance maybe could lead to a faster initial launch?

Sure. So obviously, prior to launch, we'll continue to advance our campaign on an unbranded basis, if you will, on educating about the unmet needs in treating agitation in specific populations that we will -- that will be the basis of our launch. We'll also continue to engage on the differentiation front that the product will offer upon approval. Now hospitals are hospitals. They have their own processes and their own pace. As part of our ongoing efforts to characterize the target call panel, if you will, we'll be looking at a number of variables, not only volume and their rate of usage or their volume of usage of antipsychotics and benzodiazepines as a proxy of how they're actually treating agitation but we will also be looking at how their formulary processes evolve, which ones may be more efficient and how they will review new products and how the stocking programs will work in those hospitals. So our primary goals are obviously to accelerate formulary consideration. That will be driven by building a strong and broad cadre of local advocates, if you will, local opinion leaders who actually work in those hospitals who can make the initial request for a formulary review. At the same time, we'll be working to also engage those local advocates to request stocking of 501 in those hospitals to begin the process of trial and usage that can build momentum and also build experience within the institution that can accelerate the formulary cadence. So we'll continue that work. I mean, hospitals, as I said, have their own process, it can take 6 to 12 months, depending on the type of hospital, et cetera, and their process, on average, for this process to play out. But we'll look for opportunities in those hospitals that move quicker, and then we'll continue to persistently promote post-approval so that we can drive the necessary level of awareness and interest to accelerate the overall process.

This is Geoff, I wanted to follow up on one of Greg's questions. Maybe one for you, Will, and one for you, Vimal. Just when you think about the OUS market, particularly in Europe, maybe just compare and contrast, agitation and the standard there versus the U.S.? And then, Vimal, maybe at a higher level, how do you -- what goes into what informs the OUS commercial strategy vis-à-vis partner versus invest? I'm just trying to get a sense for the economics of it are going to be different. But what are the levers that you may pull to kind of maximize.

Sure, Geoff. I'll start talking about the marketplaces outside the United States, particularly in Europe. What is true actually in the European market as well as other ex U.S. markets is the unmet need is the same. The treatment approaches are the same, right? The standard of care doesn't really vary across geographic regions in the world. There is clearly a recognition that treatment options, new treatment options would be potentially valuable and would be considered. And remember, the population in Europe is larger than the population in the United States. And the rates of diagnosis schizophrenia, bipolar disorder are very similar. So there's a commonality and clearly an opportunity to leverage the data sets we have from the U.S. NDA to pursue an approval in Europe, and we're on track to file our MAA in the second half of this year. So turning it over to Vimal.

So Geoff, coming back to your question about the opioid withdrawal. The way we think about it is, in our trial, we achieved the primary endpoint, which was a safety and this was for the first time that we had done some sort of a sub chronic dosing of 501, where 2 doses were given over a 7 to 10-day period. And we did dose escalation, and we saw the safety profile was very reasonable and drug was tolerated well. So we have achieved a number of things in terms of the primary endpoint. The retention rate with a couple of doses, like 180 and 240, we started seeing that [indiscernible] retaining more patients, which is a very important factor in any treatment that is related to opioid withdrawal. So we have that background already established using our RELEASE trial. Now what we are trying to figure out is there are 2 markets, one is prescription market in opioid. And then there is a fentanyl. Fentanyl can be prescription or it can be with a street drug. So morphine can be laced with fentanyl. Fentanyl is very potent. It's almost 50 to 100x more potent than some of the other opioids. But in our trial, we realized that there were a lot of patients almost more than 85% who were on the fentanyl. And fentanyl does not clear from the system as fast like some of the other opioids. So it stays in the system much longer than even the morphine stabilization phase. So the trial design, what was designed is that as you're doing dose escalation, you are stabilizing with -- patients with morphine so that you have the same baseline, and then you can assess that efficacy using various doses. That did not work because morphine even at very high doses could not stabilize these patients who were fentanyl patients. So now where we are, we have 180, 240, a couple of doses that have been identified, they are safe and tolerable. And going forward, we are trying to understand how the market is evolving. And what the real opportunity is and can we develop 501 for both options, the prescription drug auction as well as fentanyl because that seems to be one of the major parts of the market where market is evolving over a period of time. So a trial of the nature of where we don't do any stabilization with morphine and directly take all-comers and use 501 to test the doses that we have already identified in release to see if we can get a separation on the 2 end point, the COWS and SOWS. Even though retention is an indicator that the efficacy signal, it's still COWS and SOWS are used for approval. So those are the things we are working with our advisers. To figure out how the market is going to evolve and what would make sense for this program to take it to the next level.

Got you. That's helpful. Beyond opioid withdrawal and what you have, not to say you guys don't have a lot going on in the program with 501. You've mentioned there are a number of other indications, PTSD, there's a number of neurological disease. What else would you say is something that's more in the exploratory stage that you could perhaps investigate in the next, say, 12, 18 months, for 501, just broadly for agitation?

So as you mentioned, we are already have working with our partner, Yale, to investigate PTSD. That trial is designed for patients, ultimately be able to take the drug home and so it can be tested. It's more or less like a trial, which will be initially done in institution at one patient and like have been tested and dose with 501. Then they will be given the 501 so that they can take it home. And that's our first entree, basically taking the 501 outside institutional setting into the home setting. And that will give us some data that will lay the foundation both for this drug that it can be moved to the home care setting as well as data to lay the foundation of PTSD. So we are excited about that because, as I mentioned, that we want to move the drug into different medical settings and keep expanding the indication that's inherent part of the strategy for 501. In addition, we see a large opportunity in depression. We have, in our bipolar study, a large number of patients, about 25% or so, which had bipolar and depression. And when we looked at the data, we saw that there was a calming effect in those patient population. So that's very encouraging. And our team is currently exploring what are the rationale to be able to move this drug into the depression space where patients have and share, they have agitation and where 501 can play a major role. So we are evaluating. It's an exploratory stage. Once we have more mature plan in our R&D Day, we will come out and present those plans as they mature. So as you indicated, Geoff, this product is a pipeline within a product. And the mechanism is very unique, is working through a central mechanism, by blocking the norepinephrine. And we continuously look at multiple areas, but in near term, I would say, PTSD and depression are the 2 areas we are exploring as beyond schizophrenia bipolar and dementia.

Got you. That's helpful. Think Greg maybe had a couple more in 501, but we also wanted to tackle 701, too. Greg?

For sure. Yes. Thanks, Geoff. So you touched on administration at home. Wanted to get your sense of what does that do to the market size and the economic opportunity there? How are you thinking about that relative to 501 being given in the hospital, it seems that could massively expand the usage?

Will, do you want to take it?

Sure. I'll speak to this, Geoff and Greg. So obviously, our ultimate goal is to develop and potentially deploy 501 across multiple treatment settings. Obviously, we're landing in the hospital setting with the initial indication of schizophrenia and bipolar disorder, but the work is ongoing to continue to understand what it will take in order for the drug to be used in other treatment settings. In Dimension, particularly, we've already obviously demonstrated application of the drug in assisted living facility setting, that's where the TRIM quality study was conducted. So that setting is very analogous, very similar, if you will, to minimally invasive care settings, which could be representative of the at-home setting. And so the goal clearly in dementia, is to be able to cover the full spectrum where more and more of the patients will be residing, and that would be in long-term care facilities and at home, right? As Vimal mentioned. Diagnosed population with Alzheimer's disease in the United States today, number is about 6 million, but the Alzheimer's association forecast that's going to double over the next 20 years. Agitation is very frequent. It's very prevalent in those patients up to 70% of patients can experience agitation. So that population will continue to grow as the population expands. There's obviously going to be a fixed number of beds in some of the long-term care facilities. And so at home administration, we believe, could be very valuable clinically and also very valuable from a caregiver perspective. As agitation oftentimes is what drives patients to nursing home admission or to repeat visits to emergency departments. So to get there, obviously, we'll need to continue the dialogue with the FDA to understand the data sets that they believe will be necessary in order to build that bridge to get to the community setting. As Vimal mentioned, we already have some information in hand. The dosing and the RELEASE trial, the opioid withdrawal symptom trial, 7 days, twice daily dosing, and then we'll learn more from the PTSD program ongoing at Yale with at home 28-day administration. While we're building that, we'll continue the dialogue with the FDA to further define and then align on the necessary steps.

Great. And then maybe we'll change gears now to your other asset in the clinic, 701. Is -- this is a program that is kind of emerging this year with the data readout previously. And wanted to get your sense of where do you see 701 eventually fitting into the treatment landscape? And then what could we see in the nearer term, data wise that could help de-risk the program and maybe lead to getting some more credit for it in valuation.

Thanks, Greg. You're right. Currently, all focus has been on 501, but now 701 is an asset we are very excited about. What we are excited about it is, it's an activator of innate immunity and it's systemic in nature. As you know, most of the innate immunity activators are injected. So it's quite differentiated. Not only that there is a big background that it has 700 patient data that we are building. And it was developed prior to the immunotherapy revolution. And we decided to combine it with immuno like checkpoint inhibitor because they are the backbone, and we had a lot of evidence preclinical that these agents can be synergistic, which we're going to now demonstrate in the clinic. In terms of the overall strategy has been what are the gaps for the checkpoint inhibitor and how can we fit those. So that's the large opportunity that we are targeting with 701. So first, we took the challenge of cold tumors. Like if you look at aggressive forms of the prostate cancer, adeno, CRPC and TNEPC, there is no drug approved in TNEPC. In adeno CRPC we are targeting the patient, which have been on a chemotherapy and 2 endogens receptor blockers. So these are late stage patients. That's what we are completing. In our trial, we already crossed the first stage of the Simon 2-stage, and we continue to recruit by the end of this year or in second half of this year, we plan to present the data from about 30-odd patients. So trial was 15, plus 13. So we expect that data will be in that order about. That will have established that a [ TPA ] inhibition results in like inflammasome activation, which in turn turns the cold tumors to hot and we will be able to show what impact it makes on the responses in real life. So that's exciting. We are running another arm of the study, which is in parallel, where we are looking at Neuroendocrine prostate cancer. And we see the new cancer, there's no current treatment approved for it, and it's a very, very high unmet medical lead. So we are conducting a similar trial like adeno-CRPC type of a trial, and we will announce our data from the neuroendocrine prostate cancer trial. The first stage, it's again a Simon 2-stage trial, the first stage in the second half of this year. So that's a company sponsored trial, and those are trials like Biotel is conducting. Then we have a partnership with MD Anderson, where we are looking at tumors or particularly lukewarm tumors, which are either refractory to checkpoint inhibitors or they're treatment naive, they have no markers. And response rates with checkpoint is not that high. So we are trying to increase and deepen the responses in those areas using 701. So that data will also set another pillar for proving how well 701 is performing. What we are trying to do is demonstrate that 701 is the activator of indemnity and whatever it inflames the tumor it results ultimately in clinical benefit to the patient. So using a cold tumor as well as refractory in treatment naive tumor. So that's the overall strategy and the plan and as it unfolds in next several months, we look forward to presenting that data.

Great. There's been some debate about whether an oncology asset needs to show single-agent activity or if you could just go straight into combos and have that be sufficient for a successful drug. What is your strategy with respect to that to date?

What we did is when our clinical team set up this plan, they already had checked what is a checkpoint, inhibitors activity in some of these cold tumors, which is under 5% or 10%. So we have set a bar, that if it can demonstrate 20% or so activity in any of these cold tumors that will be meaningful for the patient. And in our other strategy, patients are already on the checkpoint inhibitor, but they are refractory or they are naive so we are using that to also understand like what different 701 can make. Ultimately, you are right that it's good to have the impact of the individual agent. We have 700 patient data on 701, and we know that it was active as a single agent in melanoma. So we have a lot of evidence scientific, clinical and some of the biomarker evidence that we are collecting to build a case for 701 as orally available activator of immunity, which is systemic in nature. And one of the de-risking we have done already optimized 701 combining with KEYTRUDA. It took us about 12 to 18 months to figure out the step-up dosing. Now we feel we are there and now efficacy data will further de-risk can provide a human proof-of-concept for 701.

Vimal, just a follow-up to that. Before Greg gets into some of the strategy. I wanted to ask you on the -- on cold tumors, like that's one of the bigger questions in IO, right, is cold tumors. And then but the other piece, though, is sort of resensitizing a tumor to immunotherapy. Is that in the plan? How are you guys thinking about that? This would be like a refractory patient in lung or melanoma, one of the hot tumors and -- but a refractory one that see multiple checkpoints. Is there -- you think, from a mechanistic place, is there a basis for 701, maybe addressing that segment?

That certainly is the case, Geoff. That's a very relevant question. That refractory patients who are not responding to multiple checkpoint inhibitors that are like naive single or double. And you mentioned melanoma, that's perfectly a good scenario that if patients are not responding, what can you do to get the responses or deepen the responses in those patients. That's exactly the trial that is being conducted with MD Anderson. That it is focusing on -- there are 2 arms to it. One is checkpoint refractory patient and the second arm is treatment-naive patients. So we are testing in both arms and seeing what difference 701 can make because checkpoint inhibitor or KEYTRUDA is already the background in those patients.

Got you. Okay. Great. That's helpful. Greg?

So when you look beyond the initial 501 launch and into additional 501 indications and what you're doing with 701 and as well as the earlier platform, how do you kind of balance your efforts and priorities between developing the new commercial organization for the 501 launch. And on the other hand, continuing to innovate with the pipeline development?

That's a great question and is the evolution of the company. So it's very exciting that we started as an innovation company using our artificial intelligence platform to identify novel asset like 501 and 701. Now 501 has moved within a 2.5-year period to NDA acceptance. So now we are transitioning into the commercial organization. So it's an exciting step. But coming back to your question, how do we manage that. So we have an extreme laser focus in business unit. We have a neuroscience business unit and oncology unit. And we have leaders for each of the therapeutic area who are moving the clinical development for those agents. So by creating a capability and focus, we are managing that these 2 therapeutic areas. And in addition, we have an AI platform that continuously apply to come up with additional assay. That's the focus for our R&D team. Last year, we made an investment, and we had Will Kane join us as well as Reina Benabou to lead the medical. So that's the next piece we have added. We feel very good, and we believe that we are the only company that have all the capability from AI all the way to the commercial [ line ]. And going forward, we are making choices, and we are saying, we cannot be expert in every therapeutic area. So we have taken a choice of like focusing on the neuroscience, so our emerging pipeline, 502 to 503 will be in the neuroscience area, as I mentioned, we are focusing on stress-related symptoms. So it's an exciting phase, but at the same time, we had to put measures in place and bring the relevant team and the leaders to be able to manage that transition from an innovation to commercial.

Great. And then when could we hear about the next programs to come out of the AI platform? And you mentioned specific therapeutic areas, but just wanted to get an idea of what could be next.

So we plan to outline our pipeline in our R&D Day that we are planning to host sometime in 2021. Hopefully, that we will be able to do this face-to-face with situation changing with the COVID-19. That 502 and 503 will be, as I indicated, in stress-related symptoms. And not only the emerging pipeline is evolving. Our AI platform is evolving too. So we've been creating a more neuroscience focused platform to be able to meet our future need as the technologies are evolving, they are changing. We continue to augment. And I say that our next-generation of AI platform. So we are committed to the area. And we believe that we have a very unique platform that can continue to produce a very -- like a highly sustainable pipeline for BioXcel Therapeutics. Oncology, as I have mentioned in the past also, and I will reiterate, we will seek some strategic options, whatever that means, that will maximize the value for our stakeholders, once we have done the human proof of concept, and that's how we're thinking about the company creating a neuroscience focus.

Great. And then one final one. Kind of a broader level strategic question. And that's, how should we be looking at BioXcel 5 years from now? I mean, at that point, you won't be the 501 company anymore. But how do you see BioXcel in 5 years?

We will -- we strive to be 1 of the leaders in the neuroscience and neuropsychiatric space. That's where our current focus is. But over a period of time, we see a lot of opportunities for neurological disorders as well as some of the rare diseases. Neuro rare, in particularly, has a lot of unmet medical need, and we believe that our [ rare ] platform can make a difference. So we continue to evaluate initial focus will be on stress related axis for symptoms and then continue to evaluate if neuro rare is another exciting area. So that's how I will project where we will be over the 5 years, and that's how we see the strategy evolving.

Great. Well, thanks so much, Vimal and Will. We'll wrap up at this point. But thank you so much for joining us today for the great discussion, and hope to see you in actual MAPA next year.

Certainly looking forward to it. Thank you very much for hosting us today.

Thank you very much.

Bye.

Bye.