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

Good afternoon. I'm Carter Gould, senior biopharma analyst here at Barclays. I'm delighted to welcome BioXcel to the Barclays virtual Global Healthcare Conference. BioXcel has been one of those stories where the company just sort of kept their head down in the past 2 years, continue to execute, and it's good to see their lead asset, BXCL501, come into greater focus from investors over the past couple of months. We're joined today by the company's CEO and Founder, Vimal Mehta. He will provide the presentation today. Vimal?

Thank you, Carter. Good afternoon. And thank you for joining us today. As a reminder, I will be making forward-looking statements regarding our clinical programs and business outlook in addition to regulatory product development. That's on Slide #2. Coming back to Slide #3. For those who are following the slide, it's a virtual presentation, so I will do my best to keep my narrative aligned with the slide numbers. BioXcel Therapeutics is a clinical-stage company utilizing artificial intelligence approaches to identify and advance novel opportunities in neuroscience and immuno-oncology. During this presentation, I would like to focus in on our lead neuroscience candidate, BXCL501, our proprietary sublingual thin formulation of dexmedetomidine. We believe this program has the ability to become a neuroscience franchise as we are currently exploring its potential in 4 indications with our first set of Phase III pivotal trials reading out in mid of this year. For 701, our trials remain ongoing as we continue to investigate this immuno-oncology candidate in a range of solid tumors. Slide #3 (sic) [ #4 ] outlines our approach that we use AI, our artificial intelligence platform, to identify new candidates. And BXCL501 currently is in pivotal trial, Phase III trial for 2 indications, schizophrenia and bipolar, so agitation resulting from schizophrenia and bipolar. And I will be referring them as SERENITY I and SERENITY II trials. And the other trial that is ongoing is a Phase Ib/II dementia trial. And the data readout for both trials are expected in mid of 2020. Yesterday, we had our earnings call. And I'd just like to summarize for everybody, the key messages that both the trials are on track, and we expect the data readouts in mid-2020. We have added additional $60 million in a public offering that happened in February to the -- our balance sheet. And that gives us quite a bit of operational flexibility to move these programs and prepare for their commercial readiness if we have a positive Phase III data from our SERENITY I and II trials. In addition, we got approval from the FDA to initiate opioid withdrawal trial -- opioid withdrawal symptoms trial. And we are planning for it, and we'll provide a more granular feedback in terms of the initiation, enrollment and the data readout on the opioid withdrawal trial. So that's the kind of a very high-level update on the 501 program. 701 program, which is targeting rare forms of the tumor, we have multiple trials ongoing. And we will share as we go along when the data readouts are expected from those trials. Moving to the Slide #4 (sic) [ #5 ]. As you can see that we are developing a neuropsychiatric franchise in terms of acute agitation. We have depth in our portfolio and the breadth. If you look at in terms of the first NDA and follow-on sNDA, we are looking at 4 indications currently and delirium is in the clinical planning stage. We are developing a formulation for KalmPen, which will be for severely agitated patients and also developing a wearable device in combination with 501 for pre- and post-agitation in dementia. In addition, we are developing an agent with BXCL501 in combination approach to tackle and target the chronic agitation in dementia. Slide #5 (sic) [ #7 ]. I'm going to now describe the BXCL501 program and then dive into the clinical programs that are ongoing and their design and the data readout. Agitation is a common phenomenon and it is associated with multiple psychiatric conditions. There are about 9.4 million patients who suffer from -- each year from some of the indications that we have outlined we are focusing on. And it's a large health care burden. And all these patients experience multiple episodes depending on their disease condition. FDA definitely recognized that and gave us a fast track designation to develop drug BXCL501 for acute treatment of agitation. And we believe that current treatments are suboptimal. Most of the time, antipsychotic drugs are being used. And they obviously have a lot of side effects. And particularly in dementia, they have black box warning. In addition, some of the injectables are used. They are invasive and riddled with multiple side effects. So we believe that BXCL501, we are bringing a novel mechanism and novel treatment for -- treatment of acute agitation across multiple neuropsychiatric conditions. Slide #7 (sic) [ #10 ] outlines at a very high level the mechanism. We believe that our drug directly targets the causal agitation as the underlying API, Dex, acts at the alpha-2 receptor and preventing the release of norepinephrine. In terms of differentiation of our product, it's highly differentiated from current treatment. It's easy to administer, non-traumatic experience for the patient, rapid onset of action observed in our clinical studies. It's noninvasive and it's a novel mechanism. And we are taking advantage of the low-dose pharmacology of this drug. So Slide #8 (sic) [ #11 ] gives the -- a little bit picture about the film. It's a sublingual thin film, which is our proprietary immediate delivery sublingual thin film product. It has muco-adhesion so that as soon as you put it under the tongue, it sticks. And this technology is very adaptable and it is very flexible technology, the way we have done the drug. It's not a monolithic film, it is like we have a drug added on top of the film for immediate release. We have done manufacturing for the drug, like for multiple registration batches. And drug is on-site for the SERENITY I and SERENITY II trials as well as for our dementia trial, which I will refer as TRANQUILITY. And we are in the process of commercial scaleup for product launch. And we -- that formulation is on track, and we feel very good about it. Just to remind everyone, Slide #9 (sic) [ #12 ], our design for the study when we did ascending dose study Phase Ib. And primary endpoint was reduction impact from the baseline at 2 hours. That continues to be our endpoint in our registration trial. Slide #10 (sic) [ #13 ] outlines that we saw dose-dependent responses. In the picture, we are showing like a couple of doses, placebo plus 80 mcg and 180 mcg. In the table, we are showing all the doses that were used and primary endpoint at 28 -- at 2 hours. The most notable is that we had almost 90% patient responding with 180-microgram dose and 67% patients responded with 120-microgram dose. And in terms of the -- we got statistically significant responses with both the doses and some of the lower doses that are in the table. So that data allowed us to go to the FDA and develop the plan for our Phase III. Slide 11 (sic) [ 14 ] outlines the drug was well tolerated and it was safe. And all subjects in the trial, 100% were able to self-administer the film. That was a very good finding in our study, that even though we always think it's a caregiver drug, will be given by the caregiver, but patients were self-administering the drug. Slide 12 (sic) [ 15 ] outlines the design of the study, where we have like 2 trials. SERENITY I in schizophrenia patients, up to 375 patients. And similarly, bipolar -- agitated bipolar patients, same, up to 375 patients. There are 3 arms to the studies, one is placebo and 2 different doses, 120 microgram and 180 microgram. And endpoint is exactly the same what it was in our Phase Ib study, a change from baseline in PEC score. And we -- I want to reiterate that trials are going well in terms of the enrollment, and we expect to announce our data in mid-2020. Slide 13 (sic) [ 16 ] kind of outlines that as we move towards the commercial preparation in terms of where the drug will be sold, like what are the centers. And we believe it will be in emergency room department, general and psychiatric; in the hospitals where there's a medical, neuro and psych ward; urgent care centers; and some mental health clinics, which are called crisis care centers. So it will be more or less institutional sales force that will be selling this product in these kind of institutional settings. Now moving on to our dementia trial. Like 501 is being developed for like acute treatment of agitation. And we believe the dementia progression starts with pre-agitation state as the disease progresses and then it becomes more episodic and then some chronic agitation and ultimately chronic agitation. So we are focusing initially with 501 in the episodic and sub-chronic. And also, we are developing a wearable device with 501 to be able to tackle the pre-agitation state. The agitation associated with dementia is a very large market. Only in U.S., there are about 6 million patients in 2020. And it is going to grow to 8 million patients by 2030. And 70% of these patients experience agitation once they have one of these diseases. And episodes tend to increase in frequency as dementia progresses. And there is a very high unmet medical need because there are no, as far as we are aware, approved FDA therapies for treatment of this agitation. Slide 16 (sic) [ 18 ] outlines our Phase Ib/II study in dementia. We are like -- the patients are all more than 65-year-old. We are doing an ascending dose study, it's an adaptive design. There is a placebo arm control and we are measuring 3 different endpoints. Pittsburgh Agitation Scale, we are measuring impact, and Cohen-Mansfield Agitation Inventory scale, which has been modified for this study. Just to remind everyone that this kind of a study, we are not aware that has been done before. So we are conducting this study in the real-life setting in the long-term care centers, where a lot of these patients reside. And we are doing evaluation over a 24-hour period but like using -- but efficacy endpoints, we are measuring multiple endpoints that we can utilize with FDA to determine the next step in the process in terms of what the late-stage development will be and what endpoints we will be using in our pivotal trial. So the data readout from these trials are on track to be announced in mid-2020 as well. Our fourth indication is opioid withdrawal symptoms. Slide #18 (sic) [ #19 ] outlines that there are about 2.1 million patients. Almost out of that, 1.2 million patients undergo opioid use disorder treatment. They try to quit, try to do this 3, 4 times a year. And in our trial, we are using 2 films per day for 5 to 10 days. And if required, like we can go for more than 2 films also. So that will be determined during the trial. So that's kind of a high-level commercial opportunity on opioid withdrawal. And next slide, 18 (sic) [ 20 ] outlines that -- what the design is. It's -- evaluation period is 5 days. We are testing -- we'll be testing multiple doses, ascending dose study with a placebo control. And the endpoints for efficacy will be measuring the pharmacokinetics, safety and tolerability in addition to COWS and SOWS, which are Clinical Opiate Withdrawal Scale or Short Opiate Withdrawal Scale of Gossop. At this point in time, we are in the preparation stage to launch this study. And as we launch it and we have enrollment starting with the study, we'll provide more guidance on the data readout -- top line data readout from this study. Slide #19 (sic) [ #21 ] outlines at a very high level our product life cycle strategy more going and developing at depth. We are continuing with our mission to get to the first NDA in schizophrenia and bipolar with SERENITY I and II trials on. Dementia trial is ongoing and opioid is getting ready for launch. So that's the mild-to-moderate agitation patients. For pre-agitation, we are developing and testing Apple Watch currently. And for severe agitation, we are developing an IM formulation, what we call as KalmPen. So that kind of concludes with Slide #20 (sic) [ #22 ] the BXCL501 plan. Again, to reiterate that we are looking forward for the data readouts in mid of 2020 from both the trials, SERENITY and TRANQUILITY trials: schizophrenia and bipolar, SERENITY; and dementia, TRANQUILITY trial. And opioid withdrawal trial is expected to start soon. And then for our SERENITY trial, then we are starting -- we will start preparing to submit our first NDA for BioXcel Therapeutics. So that concludes the BXCL501 section. I'm going to quickly now, in the next few minutes, introduce BXCL701. It's a potentially first-in-class oral I-O therapy. Slide 22 (sic) [ 24 ] outlines its dual mechanism in terms of the immune activation and immune evasion. And we believe 701 is designed to stimulate the innate immunity system, facilitating a strong adaptive anticancer immune response, turning cold tumors to hot. That's on Slide #20 (sic) [ #24 ]. The ongoing trials. There are 3 ongoing trials, treatment emergent trial, where we are doing Phase Ib -- I/II trial for 701 in combination with KEYTRUDA. We are in the dose optimization stage. And once that has been completed, it will go to the efficacy. And we expect the data readout by the end of 2020. Pancreatic trial with our partners, Nektar and Pfizer, they are currently conducting their trial with bempeg and avelumab. And once we have that data, we'll initiate the triple combination phase. And MD Anderson trial, which is a solid tumor response to checkpoint inhibitors, has been initiated. And we expect data readouts, which are on Slide #24 (sic) [ #29 ] from 2 trials, neuroendocrine prostate cancer this year as well as MD Anderson this year, and expect to initiate the pancreatic triple combination phase this year. So 2020 is a data-rich year for the company. We have multiple data readouts for BXCL501 program as well as BXCL701 program. I finally like to thank all of you for joining me today to hear more about the exciting progress we have made with our clinical programs and business. I look forward to continuing to provide updates on all of our programs and activities throughout 2020. We will be very happy to answer any questions in follow-up calls, which we'll be very happy to set up with anybody who is interested. And with that, I'd like to wish you have a great day. And thank you again for joining us today.

Thanks again, Vimal.