BioXcel Therapeutics - Q2 2024

Transcript

Operator (participant)

Good morning and welcome to the BioXcel Therapeutics second quarter 2024 earnings conference call. At this time, all participants are in a listen-only mode. If during the conference call you require operator assistance, please press star zero on your telephone keypad. After the formal remarks, there will be a question-and-answer session. If you would like to register a question, you may press star one on your telephone keypad. Just to remind everyone, certain matters discussed in today's conference call and/or answers that may be given to questions asked are forward-looking statements that are subject to risks and uncertainties related to future events and/or the future financial or business performance of the company. Actual results could differ materially from those anticipated in these forward-looking statements.

Risk factors that may affect future results are detailed in the company's annual report on Form 10-K for the year ended December 31st, 2023, which can be found at www.bioxceltherapeutics.com or on www.sec.gov, and which will be updated in its quarterly report on Form 10-Q for the quarterly period ended June 30th, 2024. As a reminder, today's call is being recorded. Speaking on today's call are Dr. Vimal Mehta, Chief Executive Officer; Dr. Vince O'Neill, Chief of Product Development and Medical Officer; and Richard Steinhart, Chief Financial Officer. They will be joined in the question-and-answer session by Dr. Frank Yocca, Chief Scientific Officer; Matt Wiley, Chief Commercial Officer; and Dr. Rob Risinger, Chief Medical Officer of Neuroscience. It is now my pleasure to turn the call over to Dr. Mehta. Please go ahead.

Vimal Mehta (CEO)

Thank you, Operator. Good morning and thank you for joining us today. At BioXcel Therapeutics, we are focused on expanding our lead asset, BXCL501, into the home setting for bipolar and schizophrenia-related agitation and into a new indication for Alzheimer's-related agitation. We are extremely passionate and motivated to bring this much-needed therapeutic option to millions of patients who are struggling with episodes of agitation. We believe BXCL501 offers a compelling value proposition, and we look forward to advancing the development of our two clinical programs. At this time, we are delighted to reach an important juncture with the Serenity program as we are finalizing development plans for BXCL501 in the at-home setting, where the majority of agitation episodes occur. We truly believe that this is a much-needed treatment option for patients, caregivers, and healthcare providers, as there are no FDA-approved therapies for agitation in the home setting.

The SERENITY At-Home trial could represent a near-term value creation opportunity to potentially expand beyond the institutional setting where IGALMI is currently marketed. Our Tranquility program plans with BXCL501 for Alzheimer's-associated agitation are also progressing. This program could address a significant unmet medical need, and we are pleased to have received Breakthrough Therapy Designation from the FDA for this indication. We believe this program could represent a larger, longer-term growth opportunity to potentially address an estimated 100 million annual episodes of Alzheimer's-associated agitation. Vince will share updates on the Tranquility and Serenity program shortly. Turning to IGALMI, we have seen growing adoption among psychiatric care clinics and behavioral health facilities. This market segment represents a community-adjacent opportunity characterized by strong product fit and lower administrative barriers. Given our small field commercial footprint, we believe targeting this market sector positions IGALMI for continued growth and scalability.

This could also serve as a bridge to the home setting to the at-home setting if approved. We believe this approach, coupled with substantial patent protection that extends to 2043, could enable us to fully realize the potential of IGALMI. Finally, we remain focused on strengthening our balance sheet to support our late-stage clinical trials with the goal of reaching data readouts. We are also evaluating strategic financing alternatives such as royalty monetization, OnkosXcel monetization, as well as strategic partnerships. In summary, plans for our core clinical programs are advancing, our market access strategy with IGALMI is progressing, and we are pleased with the growing patent portfolio. With that, I will turn the call over to Vince.

Vincent O'Neill (CMO)

Thank you, Vimal. So, as Vimal announced, we're pleased to be preparing for initiation of our Serenity At-Home trial, which, as a reminder, is designed to be a double-blind, placebo-controlled multi-center study to evaluate the safety and efficacy of a 120-microgram dose of 501 over a 12-week period. The primary objective will be safety with efficacy measures as exploratory endpoints. We recently received feedback on the trial protocol from the FDA. We've completed the CMC work we believe is sufficient to support the use of child-proof pouches in the home setting and have finally defined the final statement of work and clinical site selection with our CRO, all preparing us for trial initiation. Plans for our Tranquility program are also advancing with the focus on our Tranquility In-Care trial. The proposed trial design is substantially similar to Tranquility 2.

As a reminder, Tranquility In-Care is a double-blind, placebo-controlled multi-center study to evaluate the efficacy and safety of a 60-microgram dose of BXCL501 over a 12-week period. The primary endpoint is expected to be the change in PEC score at 2 hours post first dose. The Tranquility In-Care protocol is in final form, and we're planning to submit it to the agency. Turning to our PMR study with IGALMI, we were pleased to have reported positive top-line results evaluating PRN or as-needed treatment of IGALMI. The study achieved its objective and demonstrated no evidence of tachyphylaxis, tolerance, or withdrawal with the 180-microgram dose, the highest approved dose. Although the study was not statistically powered to evaluate repeat dose efficacy, a reduction in agitation was observed for each episode occurring during the 7-day study period, and no serious adverse events were reported following treatment.

I would now like to turn the call over to Rich, who will review our financial results for the second quarter.

Richard Steinhart (CFO)

Thank you, Vince. Net revenue from IGALMI was $1.1 million for the second quarter of 2024, compared to $457,000 for the same period in 2023. This represented a 141% increase. Sequential quarterly revenue increased 90% in Q2 2024 from the first quarter of 2024. The growth in revenue for both periods was primarily driven by an increase in contracting with psychiatric care clinics and behavioral health facilities. Research and development expenses were $8 million for the second quarter of 2024, compared to $27 million for the same period in 2023. The decreased expenses were primarily attributable to the wind-down of the Serenity 3 and Tranquility 2 and 3 trials, as well as decreased professional fees, personnel, and related costs. Selling, general, and administrative expenses were $9.5 million for the second quarter of 2024, compared to $25.9 million for the same period in 2023.

The expense reduction was primarily attributable to a decrease in personnel and costs associated with the commercialization of IGALMI, compared to the second quarter of 2023, driven by our reprioritization. BioXcel Therapeutics had a net loss of $8.3 million for the second quarter of 2024, compared to a net loss of $53.5 million for the same period in 2023. In the second quarter of 2024, the loss from operations of $17.3 million was offset by unrealized gains related to derivative liabilities. The company used $23.2 million in operating cash during the second quarter of 2024. Cash and cash equivalents totaled $56.3 million as of June 30th, 2024. Finally, to enable us to deliver on our strategic clinical priorities that Vimal and Vince have described earlier, we are continuing to optimize operational efficiencies across our business. Now I'd like to turn the call back to Vimal.

Vimal Mehta (CEO)

Thank you, Rich. We would now like to open the call for questions. Operator.

Operator (participant)

Thank you. We will now be conducting a question-and-answer session. If you would like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate your line is in question queue. You may press star two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star key. One moment, please, while we pull for questions. Your first question comes from Sumant Kulkarni with Canaccord Genuity. Please go ahead.

Sumant Kulkarni (MDand Senior Equity Research Analyst)

Good morning. Thanks for taking our questions. I have three fairly quick ones. On Tranquility In-Care, at what point do you expect to be able to let us know when data might be available? And on that trial, are there any new variables that might make the trial more or less expensive relative to how you've characterized the cost previously?

Vincent O'Neill (CMO)

Morning, Sumant. This is Vince. For Tranquility In-Care, today we're not in a position to provide specific timing. To your second question in terms of cost, we don't obviously typically give specific cost guidance, but we see no reason to have changed our view on the cost of the study recently. It's essentially as previously thought or planned.

Sumant Kulkarni (MDand Senior Equity Research Analyst)

Got it. And then my third one is on Serenity At-Home. How aligned would you say you are with the FDA on that trial? And is the child-proof packaging the key part of enabling that trial to proceed?

Vincent O'Neill (CMO)

I would say we are very aligned with the FDA. As I mentioned in prepared remarks, we did receive feedback from the FDA that has been incorporated into the protocol. Also, as mentioned, CMC work is complete, and the statement of work with the CRO has also been defined. Essentially, the make-ready work for that study is complete.

Sumant Kulkarni (MDand Senior Equity Research Analyst)

And I'll squeeze one last point in. Sorry. Can you talk about any potential areas of discussion that you might have with the FDA on the specific Tranquility In-Care protocol?

Vincent O'Neill (CMO)

I think one point I'll make is obviously Tranquility versus Serenity is an efficacy primary endpoint study. It's obviously a little more complicated, a little longer in duration, and actually a little bit more expensive. We know that the FDA is very interested in repeat dose efficacy, and I think that would be the one point that we want to get right going forward with that protocol.

Sumant Kulkarni (MDand Senior Equity Research Analyst)

Thank you.

Operator (participant)

Next question, Alec Stranahan with Bank of America. Please go ahead.

Mary Daly (Assistant VP and Research Associate)

Good morning. This is Mary Kate on for Alec this morning. And we were just wondering, what are your expectations for the pace of trial execution for the phase 3 trials upon initiation, maybe specifically Serenity here? And maybe could you add additional color on the potential market opportunity for the at-home population here as well? Thank you.

Vincent O'Neill (CMO)

Thanks. And this is Vince. Just to paraphrase your first question, essentially you're asking about the timing of the Serenity At-Home study, correct?

Mary Daly (Assistant VP and Research Associate)

Yes. Yeah. And how long it could potentially take in terms of enrollment once you guys initiate the trial?

Vincent O'Neill (CMO)

Sure. So again, not in a position to give precise timelines today, but I'll actually just repeat what I said a moment ago, and that is that Serenity, of the two studies, Serenity is the less complex study, therefore the faster study, and the less expensive study.

Matthew Wiley (Senior VP and Chief Commercial Officer)

Yeah. Mary Kate, this is Matt. I'll talk a little bit about the market opportunity. So there are 23 million episodes in the at-home market. What we found in market research is that patients who experience agitation with bipolar schizophrenia indicated that they would use BXCL501 in 80% of their episodes, oftentimes at the very beginning or even before those episodes started, so in the prodromal phase of the episode. So the patients are motivated, and clearly we could imagine taking some of the 23 million episodes adding to that as we draw some of those episodes out of the emergency department in the hospital.

Operator (participant)

Next question, Graig Suvannavejh with Mizuho. Please go ahead.

Avantika Joshi (Lead Financial Analyst)

Hi. This is Avantika on for Graig. I just had a couple of questions. The first was, what is the company's current cash runway? And the second is, for both the Serenity and Tranquility studies, are you still planning to finance it yourself? Are you thinking of partnering with someone to actually run the trials? And the last is, what are the gating factors for actually initiating the trials now that you've discussed the protocol with the FDA specifically for Serenity? Thanks.

Richard Steinhart (CFO)

Hi. This is Rich. On the cash runway, we really haven't changed our guidance on cash runway at all. So it remains similar to what we had talked about in the past.

Vimal Mehta (CEO)

Good morning, Avantika. This is Vimal. Regarding your question on the financing, our fundraising strategy is based on funding both trials through various alternatives, as I mentioned. We are exploring all those opportunities in parallel. As you said, both trials are at a point where we already have the feedback, and Serenity At-Home can begin shortly. We are finalizing our plans for the Tranquility In-Care, which Vince already mentioned. Both are strategic priorities, and we are moving them along. As I mentioned in my prepared remarks, we are looking at strategic partnerships as well as a part of this exercise.

Avantika Joshi (Lead Financial Analyst)

Okay. Thank you.

Operator (participant)

Next question, Ram Selvaraju with H.C. Wainwright. Please go ahead.

Raghuram Selvaraju (MD of Equity Research and Senior Healthcare Analyst)

Thanks so much for taking my questions. Three quick ones here. Firstly, just a question on the commercial side. Can you comment on any favorable tailwinds that you see the product experiencing at this juncture and what impact that might have on the cadence of revenue growth and prescription uptake over the course of the second half of 2024? Secondly, with respect to OnkosXcel, can you provide any update with respect to potential spin-out of that entity or other ways in which you could conceivably monetize BXCL701? And then lastly, if you could perhaps comment on your plans for the neurology pipeline beyond BXCL501 and to what extent that might potentially provide you with additional sources of non-dilutive funding. Thank you.

Matthew Wiley (Senior VP and Chief Commercial Officer)

Hey, Ram. So this is Matt again. To answer your question about tailwinds, I mean, one of the things that we've observed over the last 3 quarters is an increase in the interest from these community behavioral health centers. There are about 36,000 of these in the United States. We know from previous market research that psychiatrists have the highest intent to use the product of all the constituents in a hospital setting. So we think that that translates nicely to this community mental health clinic-type setting. We also, based on our experience, we know that the process for acquiring IGALMI does not require the type of administrative hurdles that you typically see in a hospital or IDN setting.

We feel like this is an opportunity that is near-term, low-hanging fruit, and we will continue to progress on that over the balance of the year and into next year as well.

Vincent O'Neill (CMO)

Sure. This is Vince. Morning, Ram. So just to reiterate what you've said, we're obviously committed to monetizing 701. So we have initiated partner outreach through our retained firm, a firm that specializes in this area. And that's in addition to formally assessing the commercial value of our indications. So we're pleased with progress so far, and we'll update you in due course.

Vimal Mehta (CEO)

Good morning, Ram. This is Vimal. Regarding your third question, plans for our neuroscience pipeline. Currently, as I mentioned, we are very focused on moving to late-stage pivotal trials, but we will continue to look at what are the best options to progress the pipeline, which is 502, 503, either through a partnership mechanism or any other ways to move those programs forward.

Raghuram Selvaraju (MD of Equity Research and Senior Healthcare Analyst)

Thank you.

Operator (participant)

Thank you. I would like to turn the floor over to Dr. Mehta for closing remarks.

Vimal Mehta (CEO)

Thank you. Thank you, everyone, for joining us today and for your continued interest in BioXcel Therapeutics. Have a great day.

Operator (participant)

This concludes today's teleconference. You may disconnect your lines at this time and thank you for your participation.