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

All right. Good afternoon, everyone. Thanks for joining us at the 2022 Barclays Global Healthcare Conference. My name is Justin Burns. I'm an associate on the U.S. Biopharmaceuticals team, and it's my pleasure to introduce Dr. Vimal Mehta and BioXcel Therapeutics.

Thank you, Justin. Thank you to our hosts at Barclays for the opportunity to present today, and good afternoon to all of you. I will be making some forward-looking statements, and my goal today will be to introduce the company as well as provide you update that -- exciting things that are happening around our business for 2022. So as you know, BioXcel Therapeutics is developing transformative medicines using AI-based approach, artificial intelligence-based approaches in neuroscience and immuno-oncology. Our lead products are -- 2 lead products are BXCL501 and BXCL701. 501 is investigational proprietary orally dissolving thin film formulation of dexmedetomidine designed to treat agitation. We are focusing on 4 neuropsychiatric conditions, schizophrenia and bipolar, Alzheimer's disease as well as recently, we have filed IND for adjunctive treatment in major depressive disorder. So we are very excited about the potential and the importance of 501 to treat these diseases. In addition, we have an emerging pipeline, which is BXCL502, candidate that will augment our franchise 501 as a chronic agitation in Alzheimer's disease. So 501 is positioned in Alzheimer's for acute as well as intermittent chronic agitation and 502 will complement to capture the chronic agitation market. In terms of our 701 program, it's -- we believe, it's the most advanced activator of innate immunity. And we are focusing on converting cold tumors to hot and we are focusing on aggressive forms of prostate cancer as well as advanced solid tumors for this program. What is really unique about the company that the approach we have adopted to identify these 2 drugs using our AI engine, and we have built a 4-step process to impact the R&D economics to shorten the development time lines as well as achieve higher probability of success. As you all know that in neuroscience, the success rates quite low. And we -- I will show you that how fast we have been able to move 501 from a drug concept all the way to where we are today. So here is our rapid journey to NDA for 501. And this journey was accomplished in terms of acceptance of our NDA in less than under 3 years from the first-in-human to the NDA acceptance. Our PDUFA date is April 5. Our NDA remains under review by the FDA, and we expect to hear from them by the PDUFA date. In between, if you think about Q1 of 2018, we became a public company. We had 2 positive Phase III trials by mid of 2020. And then we filed our NDA in Q2 of 2021, when it was accepted. In between, we initiated a program that's related to the Alzheimer's disease, and we got a breakthrough therapy designation. And recently, we announced that we have initiated a Phase III program for follow-on sNDA, if we get approval for our first 2 indications, which are for a schizophrenia and bipolar-related agitation. In addition, we have demonstrated that 701 continues to move along, and we have some clinical proof of concept already achieved. So this approach and rapid time lines we have achieved, we have built a very robust pipeline, thanks to our AI-based approach that allows us to identify numerous candidates that we can sift through and then select the ones we want to pursue. Our 5-year vision as a company is to continue to advance our integrated capability encompassing AI, drug discovery, development, and very recently we have built MSL teams as well as commercialization. We believe we have pretty unique capability from AI to commercialization. Our goal is to continue to expand 501 franchise. And I will discuss the 3-pronged approach we have adopted to continue to expand 501. At the same time, we continue to build sustainable R&D pipeline outside of 501, like 502 that is currently under development at BioXcel Therapeutics. Our neuroscience franchise is focused on acute treatment of agitation, and as all of you know, it's a very debilitating situation for patients, very threatening for health care providers. And there are no current optimal treatments to treat this disease. We believe that we have a very unique position; over a decade, there has not been much innovation in this area. And we believe that 501 can provide a very transformative platform for treatment of these patients as well as for the caregivers. It's a very significant commercial opportunity. There are about 7.3 million estimated patients, adults, in schizophrenia and bipolar disorders. And we have mapped out there are about 25 million agitation episodes that happen every year. When you look at Alzheimer's, as you all know, the patient population is doubling over next 20 years from 5.8 million to almost 12 million patients, and as of today we have mapped that could be about 100 million agitation episodes per year. So our drug is designed to be able to treat as and when agitation episode happens, as well as in Alzheimer's, because there are more agitation episodes. So it can be used, and the trial is designed, as a intermittent chronic agitation -- to treat that agitation. Just to give you a flavor of the mechanism, if you're not familiar with the drug, that like it stops release of a chemical, norepinephrine, and that results in the calming effect. And it's a very highly differentiated product. It's easy to administer, non-invasive, non-traumatic, and patients have self-administered the drug. We continue to expand our patent portfolios and expanding the patent in U.S., Europe and Japan. In terms of the portfolio strategy, we have a considerable opportunity. First is we continue to expand the indications. So schizophrenia and bipolar-related agitation, our PDUFA date is April 5. We have recently started a Phase III program for Alzheimer's-related agitation, and also we have filed an IND for major depressive disorder. So all the indication strategies are in place, and we will continue to execute in 2022 on the clinical plan for Alzheimer's as well as major depressive disorder. Our second strategy is to continue to express the geographical expansion in -- particularly in Europe and Japan. And we are planning to file our first MAA sometime in first half of this year. So -- and in addition to that, we will continue to expand the medical settings for these indications. Coming back to the Phase III program in Alzheimer's, there are 2 trials, TRANQUILITY II and III. They have 150 patients each. And there are 2 doses we are testing, 40 and 60 micrograms, and there will be a placebo arm. So we are excited to execute on this program now. Omicron is behind us and clinical trials can move forward very smoothly. We have a lot of experience in this area in conducting the trials in a schizophrenia, bipolar. We conducted a trial TRANQUILITY that was in the Alzheimer's patients in the assisted living facilities. So we have a lot of experience in execution of this. This trial also have a rollover safety study, and we will be providing the clear guidance when to expect the data readout, but currently, I can say that TRANQUILITY data readout will be first TRANQUILITY II followed by the TRANQUILITY III. That's how we have staggered the execution of these 2 trials. Alzheimer's disease opportunity is very broad, as Alzheimer's patients like -- disease progresses, then their agitation episodes can change. So this slide kind of outlines that how we are thinking about capturing the opportunity as and when needed in acute agitation space, as well as a intermittent agitation as and when it happens or multiple episodes of agitation that happen. And these things can happen in different settings. It could be in hospital, it can be assisted living in nursing homes and in the community at home. So we are trying to cover the spectrum of agitation with our Phase III program. You can see 502 on the right side. That's where we are developing that for chronic agitation where patients may need, when they progress to late stage of Alzheimer's, they may need a drug that is needed for every day in case they have a lot more frequent agitations which are almost every day. So I covered already schizophrenia and bipolar, which is like our PDUFA date is April 5. Alzheimer's program has moved to the Phase III program, we have a breakthrough therapy designation in Alzheimer's. Now we wanted to see what are the other indications that we can use 501 to develop further, and using our about 800 to 1,000 patient data in schizophrenia, bipolar, Alzheimer's as well as opioid withdrawal, we found certain key characteristics in terms of what 501 impacts. And those characteristics were anxiety, restlessness, irritability, panic, sleep disturbances. And if you look at the Ham-D scale, that these parameters are very important in treatment of depression in the major depressive disorder patients. So as and when a antidepressant is given, for the first few weeks these patients are anxious. They have suicidal thoughts. And that results in [ poor ] compliance and clinical outcomes. So we believe 501 can fit in and play an important role because it has a very rapid onset of action, and it impacts a lot of key parameters that are part of the depression spectrum. And we have quite a bit of evidence, both preclinical as well as some of the clinical evidence, that this will be a very big opportunity for 501 to focus on, and this is a very high unmet medical need currently. I don't need to go over these stats. It's a -- burden of depression is huge. And as you see that particularly in major depressive disorder it's a very big unmet medical need. So we will be focusing on accelerating the effect of the current standard of care for treatment of major depressive disorder. So here is our current MDD development plan. So we will be -- we have filed a IND. We will be initiating a trial in the healthy volunteers to find the dose. Once we have the dose, we will treat -- find -- like [ treat ] the MDD patients. That's the first part of the plan, and then we will go for a plan proof-of-concept study to see that 501 plus current standard of care, whether SSRIs or SNRIs, and then we will have a placebo plus SSRI or SNRI arm to establish the proof-of-concept that 501 can really accelerate the antidepressant effect of current standard of care. So that's our plan, and then we will lay out our pivotal program around 501. So as I mentioned, that PDUFA date is April 5, and we, as a company, been preparing to get ready for commercial launch and readiness. In that aspect that we have been doing a lot of work for last year, or more than a year, that our MSL team has been in the field talking to the health care providers. And we have been running unbranded campaigns, and then we've been doing a lot of speaker programs as well as attending the conferences, some of them virtually and a lot of them in person. And very recently, I'm pleased to share that our SERENITY II program for bipolar disorders I and II was published in JAMA publication. This is just a snapshot of what feedback we're getting from the market. So over 150 HCPs were shown the marketing materials in a blinded fashion, and there was a very high acceptance in terms of the HCP surveys about positive impression of 501. In addition, almost 40% of them stated that they will prescribe 501. And not only prescribe, they will prescribe it over current treatment options that are being used in terms of the benzodiazepine, antipsychotics and others. These was very telling to us. So we do believe that this -- our 501 can be a very disruptive platform. There has been no innovation, there has been no drug approved over the last 10 years. And we are very excited about the potential of 501 if approved on our PDUFA date, which is April 5. We are ready to launch. We have a Chief Commercial Officer in place, and we have building our national sales team. We have developed pricing strategy. And we will be coming up with all of that once we have the approval from the FDA that our plan to launch this product in the marketplace. In addition to our neuroscience franchise, which is focusing on schizophrenia and bipolar-related agitation, Alzheimer's-related agitation which is in Phase III and just filed a IND around the major depressive disorders, we also have an immuno-oncology franchise, our BXCL701. As I mentioned that it's a first-in-class oral activator of innate immunity. We have published this recently, its mechanism, in JITC. We have multiple orphan drug designations. Mechanism is extremely unique, it's DPP 8/9. And we have a collaboration with MD Anderson for advanced solid tumors to test it. In addition, there are a lot of agents in immuno-oncology that are under development and that can be in the area of adaptive immunity. We focused when we started the program on innate immunity that can bridge with the adaptive immunity. And we believe there is a scarcity of these agents, as you can see with recent transactions that has happened for Forty Seven with Gilead and Trillium with Pfizer. So we believe that 701 is a very good opportunity. And by now, we have obviously demonstrated the clinical proof-of-concept for 701. And this data was recently presented at ASCO GU in February of 2022. There are 2 cohorts in our aggressive form of the prostate cancer. One is the adeno and another one is small cell neuroendocrine cancer. And in SCNC, we demonstrated 33% response rate overall. And -- in combination with KEYTRUDA, and if you look at the NEP checkpoint inhibitor, response rates are under 5% alone in these kind of cold tumors. So we are very encouraged that we have now mechanism established and published. We have demonstrated the safety combining with KEYTRUDA and now we have started seeing the response rate in these cold tumors turning them into hot. So for a company, it's a very exciting year that we have both programs they are moving forward through our pipeline, and they were identified using our AI-based approach. When we started the company, as I mentioned, we became a public company almost 4 years back, and it has been a very rapid development of both the programs. So what to expect ahead? The key catalysts are our PDUFA date upcoming on April 5. Alzheimer's disease, we have initiated the Phase III program, and we will be initiating a trial on major depressive disorder. We already have a IND filed with the FDA after having a pre-IND meeting. Similarly, for the neuroscience program, if we get approval, we are ready to launch the product 501. And our teams, both MSL as well as commercial teams, have been working to prepare for that. And in immuno-oncology, we have recently announced the data for 701, and we expect to have more data readouts around advanced solid tumors. So with that, like I'd like to thank you all for taking out the time to listen to our presentation and your interest in BioXcel Therapeutics. We do have about 4 minutes left. If anybody has any questions, I will be happy to address those questions. If there are no questions, thank you very much for attending this presentation.