BioXcel Therapeutics Inc (BTAI) 2023 Q4 法說會逐字稿

Good morning.

And welcome to the BioXcel Therapeutics conference call, which will include an update on the company's late-stage clinical programs and the discussion of financial results for the fourth quarter and full year 2023.

(Operator Instructions)

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 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 those forward-looking statements.

Risk factors that may affect future results are detailed in the company's quarterly report on Form 10-Q for the quarter ended September 30, 2023, which can be found at www.BioXcelTherapeutics.com or on www.SEC.gov and which will be updated in its annual report on Form 10-K for the year ended December 31, 2023.

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.

Will be joined in the Q&A session by Dr. Frank Yocca, our Chief Scientific Officer; Matt Wiley, Chief Commercial Officer, and Dr. Rob Risinger, Chief Medical Officer of Neuroscience.

It's now my pleasure to turn the call over to Dr. Mehta.

Please go ahead.

Thank you, operator.

Good morning and thank you for joining us.

This is an exciting time for BioXcel Therapeutics.

First, we are very pleased with the progress of our TRANQUILLITY program.

As I have communicated previously, this is a top priority for capital allocation.

Second, we are focused on advancing the SERENITY program.

Both programs provide significant opportunities for us to address the unmet need in treating patients struggling with episodes of acute agitation.

During today's call, we will dedicate our prepared remarks to update on TRANQUILLITY and SERENITY.

These late-stage clinical program represent groundbreaking opportunities to advance our goal of bringing new treatment options to patients.

As a reminder, there are no currently approved acute treatment for agitation associated with Alzheimer's disease or for bipolar disorder or schizophrenia in the at-home setting.

This condition represents a large untapped markets.

All of us at BioXcel Therapeutics are motivated to develop new treatment options for the greater number of patients and caregivers.

Vince will now provide a detailed update on TRANQUILITY and SERENITY.

After this, I will briefly discuss several corporate updates, which will then review our financial results before we open the call for Q&A.

With that, I will turn the call over to Vince.

Thank you, Vimal.

So I appreciate the opportunity to speak with all of you this morning and provide an update on our two late-stage clinical programs with BioXcel Fiber One, let me begin with TRANQUILLITY where we are evaluating Fiber One as an acute treatment for agitation associated with Alzheimer's dementia or for short AED.

As Vimal said, TRANQUILLITY remains a top priority to bring a novel treatment to this large underserved patient population.

Every year, Estimates show that millions of Americans with Alzheimer's disease experience, agitation.

Physicians often aim to address this with neuropsychiatric drugs used off label because there are no approved treatments specifically for acute agitation episodes.

This area of neuroscience drug development for an episodic treatment is novel and uncharted, and we believe we are uniquely positioned in this space.

At BioXcel Therapeutics, we have the gallery already on the US market as an acute approved acute treatment for bipolar disorder and schizophrenia related agitation.

This provides us with a strong foundation from which we are expanding into development in AED in our TRANQUILITY program.

And then to development in different settings in the bipolar and schizophrenia populations through the SERENITY program.

For TRANQUILITY, we have completed now a Type B breakthrough therapy designation meeting with the FDA on February 20th.

This meeting followed the one we had in October of last year.

Let me remind you that we are not in a position to provide detailed information about our overall program plans until after we receive and review the FDA's final meeting minutes.

However, we can share and know that based on the FDA's feedback, we plan to generate additional Phase 3 efficacy and safety data in care facilities to expand the database beyond the 70 patients who have been treated with 60 micrograms of fiber, one in TRANQUILITY one and two to date, we plan to generate these data, including repeat efficacy data in a variety of relevant care settings and to use PEC as the primary efficacy measure as used in the prior TRANQUILITY two study.

To remind everyone, in November 23, we announced that we were planning to conduct a Phase 3 trial in the at-home setting with safety as the primary objective or otherwise known as TRANQUILITY trial.

Given the priority to expand the database to generate additional efficacy data in care facilities, the company is now reevaluating the timing for initiating TRANQUILITY at home.

Lastly, the FDA has indicated that we will need to generate long-term safety data.

However, given the lack of any regulatory precedent for episodic treatment of MDD and patients, we plan to further engage with the FDA regarding this requirement.

We look forward to receiving final meeting minutes from the agency and sharing more details with you later this month.

We're extremely motivated by this potential opportunity to bring a much needed acute treatment option to patients developing novel episodic treatments with no regulatory precedent comes with new learnings to create an optimal development path.

At this time, we believe we have a clear path forward to that point.

We've had multiple meetings with the FDA for this uncharted regulatory path and have greatly appreciated the agency's guidance as we move forward to advancing our clinical program.

Let's now turn to SERENITY where we are evaluating the potential at-home use of fiber, one for agitation associated with bipolar disorders or script schizophrenia.

On March the 6th, we completed a Type C meeting with the FDA this fall, the one we conducted last November.

Based on FDA feedback, we plan to evaluate 120 microgram dose of Fiber One.

As a reminder, an approved dose of [Academy] in the at-home setting an amendment to our SERENITY three-part two protocol, which the company had voluntarily paused as we discuss the FDA's proposed changes to the protocol in light of the results of [3.23] Part one and our current plan is to move forward with this study as amended with safety as the primary objective and efficacy measures as exploratory endpoints.

Keep in mind, the label for Academy currently includes the limitation on use, noting the lack of efficacy or safety data beyond 24 hours.

We believe that our ability to seek labeling without the current LOU will depend in part.

On the number of agitation episodes we observed for the three month follow-up period.

To support our pivotal trial with the 120 microgram dose.

We are also planning to conduct a small study, approximately 30 patients to evaluate the correlation between patient reported or informing reported efficacy with train greater reported efficacy using PEC scores.

We expect to provide additional information about the program plans after receiving and reviewing final meeting minutes from the FDA.

And with that, I will turn the call back over to Vimal.

Thank you, Vince.

We are also pleased with progress in other areas of the business on January 1, the permanent J-code for the government became effective and our commercial generation efforts continue to progress.

In addition, we continue building a robust intellectual property portfolio and strengthening our long-term patent protection.

Finally, we are pleased to have received fast-track designation from the FDA for BXCL701.

I would like to end my remarks by emphasizing that we remain focused on strengthening the company's balance sheet.

To this end, we are actively exploring multiple financial options to extend our cash runway and fund our key clinical program in order to create maximum value for our shareholders.

I would now like to turn the call over to Rich, who will review our fourth quarter and full year 2023 financial results.

Thank you, Vimal.

Net revenue from [IGALMI] was $376,000 for the fourth quarter 2023 compared to $238,000 for the same period in 2022.

Net revenue was $1.4 million for the full year 2023 compared to $375,000 for 2022.

Research and development expenses were $9.9 million for the fourth quarter of 2023 compared to $32.5 million for the same period in 2022.

Research and development expenses were $84.3 million for the full year of 2023 compared to $91.2 million for 2022.

The decrease expenses for both fourth quarter and the full year -- primarily attributed to decreased clinical trial activity associated with the wind-down of SERENITY three and TRANQUILITY two studies, a decrease in chemical manufacturing and control costs and a decrease in personnel related to the company's reprioritization in August of 2023.

Selling, general and administrative expenses were $9.6 million for the fourth quarter of 2023 compared to $20.7 million for the same period in 2022.

SG&A expenses were $83.4 million for the full year 2023 compared to $68.8 million for 2022.

Increase costs for the full year, primarily attributable to an increase in legal and professional fees.

Cost associated with personnel and related expenses to support the commercialization of [Nozomi] in the US prior to the company's reprioritization.

BioXcel Therapeutics had a net loss of $22.3 million for the fourth quarter of 2023 compared to a net loss of $54.8 million for the same period in 2022.

In the same year for the full year 2023, the company reported a net loss of $179 million compared to a net loss of $165.8 million for the full year 2022.

Loss for 2023 includes approximately $18.6 million in noncash stock-based compensation.

Total cash expenditures for 2023 totalled approximately $155 million.

Cash and cash equivalents totaled $65.2 million as of December 31, 2023 compared to $193.7 million as of December 31, 2022.

Company estimates says the current cash and cash equivalents will fund its operations through mid 2024.

The estimated cash runway does not include any potential financing activities being undertaken by the.

Now I'd like to turn the call back to Vimal.

Thank you, Rich.

We would now like to open the call for questions.

Operator?

(Operator Instructions)

Greg Harrison, Bank of America.

Hey, good morning guys.

Thanks for taking my questions.

So after your recent FDA meetings, it seems you have more clarity for the TRANQUILITY programs.

Would you say now you have two separate path to SNDA and Alzheimer's one in the CARE study and one at-home?

And then how are you thinking about funding the program and the company as a whole as you get closer to the end of your cash runway is, are there any specific avenues that you're exploring?

Thanks

Good morning, Greg.

This is Vince.

Thanks for the question.

And so to answer directly, yes, we believe so.

So obviously, the TRANQUILITY efficacy and pack primary study and also the safety home study would be run with registrational intent.

So yes, in terms of funding, I would refer back to that.

So as I, Greg stated in my opening remarks, we are looking at multiple options to fund the company.

Now having a clarity from the FDA clearly tells us what other trials we need to do to capture these large market opportunities, both in the TRANQUILITY and SERENITY and as we speak, we are very actively working on those wells.

Great.

Thanks again.

Robyn Karnauskas, Truist Securities.

Thank you for taking my questions.

So I have two to after the recent meetings with the FDA, you've chosen the 120 milligram dose that and does that mean that would allow for expansion and at-home setting as a follow for that?

Yes, morning, Robyn.

And so I think the choice of 120 microgram dose and we believe is a good choice for the following reasons and efficacy it's fundamentally established for that does that's clearly an approved dose of [economy], one of two approved doses of

[economy].

And so yes, we believe that with that, again, a registrational intended study with safety as a primary and is what we need to go back to the FDA with data in hand.

And following up on that a little bit.

So you'll give them a minute.

Have you spoken to the FDA clearly about that dose and you'll give them a little later point.

Yes.

Sorry to interrupt you.

Absolutely.

We've spoken to the FDA.

We have crystal clarity.

I think on the choice of dose.

Okay.

Then last question, sorry for three that's bad, like funding the program like Vimal, like how do you think about you have a lot going on you talked about like give a lot of options.

How do we think about and this assay, you know, you have to find a lot of these programs are giving you feedback title How do investors think about the FastClick?

What actually how do you think about the options on the table for you?

So Robyn, good morning.

In terms of the priority we will focus on TRANQUILITY the trial that Vince has discussed because that's can help us on a path to a potential SNDA in the care setting.

As discussed on this call, and also on the SERENITY program, we have a clarity what dose we can take it forward for a potential registration trial.

So our trial, we have a tremendous experience in the company.

We have done more than 11 placebo-controlled double-blinded trials.

These trials are capital efficient and the opportunity we have in front of us is very large, but we want to be very focused and as I mentioned, TRANQUILITY will be that our price priority followed by SERENITY.

Great.

Thank you.

Graig Suvannavejh from Mizuho Securities.

Hey, good morning.

Thanks for taking my questions and for the clarity on two, please for me, I don't know if the first one was already asked.

My apologies just on the on TRANQUILITY at-home or just the TRANQUILITY program overall that pivots to needing to collect and now efficacy data, can you tell us kind of what changed versus a previous plan just to focus on study on safety.

And then secondly, while I realize that you've got to figure out the financing for the company on a go forward basis, I'm just trying to get a sense of whether you can provide us clarity around how much you think it might take to get to complete TRANQUILITY at home in order to be able to get it in a position to be able to file an SNDA.

And similarly, what you think that total cost would be for completing the SERENITY program, it's just important just to in light of your current cash just to for investors to know what more funds you need to get to really the next revenue inflection point.

Thank you.

Yep.

Good morning, Graig.

I'll take the first part of that question is obviously an important question.

So after we met with FDA last year and we began to think about the design of the study in depth and in detail and begin to engage with our advisers and KOLs.

It became clear that using non validated caregiver assessed instruments would be challenging and would frankly, carry risk.

So with that better understood, we went back to the FDA to rediscuss what additional court additional efficacy unquote meant [AM] And obviously, we've now agreed on formal efficacy analysis with PEC as a primary endpoint and clearly an endpoint we've used before with success.

So hopefully that provides clarity.

And good morning, Graig.

This is Vimal.

Our financing solution is very relevant.

Now having that clarity from the FDA was very important for us to know what financing we need to do.

So regarding the TRANQUILITY trial efficacy trial that Vince mentioned is under actively being designed and we will be able to share more details about it once we have received meeting minutes, which we expect after 30 days from our meeting on February 20.

In terms of the seniority at-home trial, we have tremendous experience with SERENITY one, two and

[three and 83].

We have a very good idea in terms of the timing as well as capital requirement for that trial.

And as I said in the beginning, for patient costs are low for these trials.

And overall, these trials are capital efficient and they can be delivered in a reasonable time.

So we are working on all of that and we will come back and lay out the plan for TRANQUILITY as well as SERENITY.

And we will be having conversations what the capital requirements are based on our current cash position and how much cash than we need to extend using the means we have, which are equity investment versus monetization of any assets we have or flash partnering.

But all these things are being pursued in parallel, and we will be able to provide a clarity on the plan and financing as we progress.

Okay.

Thank you vimal.

Sumant Kulkarni, Canaccord Genuity.

Morning.

Thanks for taking my question.

So you mentioned in your prepared remarks that for labelling purposes, the limitation of use is over a 24 to 48-hour period and you need to generate more data over three months.

And when you're doing so what's the risk that you might find that agitation is not necessarily episodic?

And in that case, you still think you might pursue Fiber One as a rescue therapy.

So I think the first way to answer that is that's not our expectations, but I'm going to ask Dr. Risinger, our CMO to comment a little bit further on that?

I mean, I guess it revolves around your expectations for frequency.

Yes.

I think I mean not to get into the weeds, but the CMAI as a tally of episodes Italia have on certain number of episodes plus severity.

The FDA has agreed to our pack as a pivotal endpoint for acute agitation.

And we would all like to understand what the relationship is with a chronic condition with episodic or episodes of acute agitation.

So we hope to be able to speak a little bit to that data.

But the reality is these patients have acute episodes that need treatment even now and even with every treatment that is used, whether it's on-label or off-label.

And then my follow-up is a financial question, what do all the latest updates mean with respect to your ability to be in line with the covenants associated with the prior financing?

Or will you need to reverse those again?

Good question Sumant.

We are always in discussion with our strategic partner and they are very supportive of facilitating and having company be able to get financing because they really believe in this asset.

And they see that this is a large market opportunity.

So it's a very good partnership and we work together to chart out the optimal path.

I think most important from this call is that we have a clarity from a FDA perspective what we need to do in terms of our path forward for these two programs.

Thank you.

Ram Selvaraju, H.C. Wainwright.

Thanks very much for taking my questions.

Can you hear me?

We can.

Okay.

So I just wanted to see if you could offer some clarity whether quantitative or qualitative regarding the paused study and how much data from that study that was collected before it's paused.

You expect it to be usable in furtherance of the development plans for 501.

I'm going to ask Dr. Risinger, our CMO to comment on that as far as you can.

When you submit for an NDA, all data in all patients who were ever treated with the medication is use.

And so to that extent, safety data from that trial is entirely submitted to the FDA.

With respect to whatever information was collected on the activity profile, is any of that information likely to be useful in potentially offsetting future development costs?

Or I'm just trying to ascertain to what extent you have to recapitulate anything if indeed you do.

I think the pregnant pause here is we're trying to -- I think I captured the gist of your question in that.

Can that data be then sort of rolled into another study and typically you can't do that.

It just has to be separate studies.

That doesn't mean it doesn't offset cost because in the end, FDA wants to see exposures and safety.

And that's what we have that safety data is probably is in the final analysis, a minor cost consideration.

Okay.

And then just really quickly, do you have any updates on your plans for [OncoSec Cellectis] this time?

Thank you.

Ram, we have formally started the process to explore the opportunities, whether it's partnering or on monetization company was extremely focused right now on getting the plan for the neuroscience business, which is TRANQUILITY and SERENITY.

Having that clarity, we will have more time to dedicate to focus on oncology Actel because as you know, if we can monetize or partner that can help bring non-dilutive financing to company.

Thank you very much.

Thank you.

Colin Bristow, UBS.

Hey, Good morning guys.

And thanks for the updates on where you put it sort of piggybacking on a few of the questions that would be nice.

Firstly, on the TRANQUILITY program, can you give us any indication on onset atrial size or perhaps what you see sort of timing.

It feels like this is now going to be a back half initiation.

So is it reasonable for us to assume that we'll probably see data in the back half of '25.

And secondly, on the existing agile launch, sales are down sequentially now acknowledging there's no sales effort to prove the surprised you can tell us why sales are not at least staying flat.

And then just a point of clarification on the credit agreement.

Are you again acknowledging you didn't satisfy one of the requirements.

Is it reasonable to expect that we'll get an update shortly after you cement things with FDA?

Thank you.

Good morning Colin, so I'll take the first of your of your three-parter um in terms of timelines that we clearly want to have the meeting minutes in hand and digested before we make any definitive statements there and with that, I'll hand over to

--.

Sure.

Good morning, this is Matt.

So to answer your question about Q4 versus Q3.

In Q3, remember that we had a fulsome commercial effort for most of that quarter.

In Q4, we saw revenues increased 10% versus Q3.

We saw our cartons shipped increased by about 8%.

That's a pretty tall order considering the fact that we have six corporate account directors in the field versus the 70 reps plus managers we had in the field previously in addition to the cats.

So the cat team is really punch above their weight to get to this revenue.

We've seen that occur on some of the contracting efforts in Q4 begin to matriculate.

We're seeing more of that in Q1, and we expect to see continued growth.

Great.

Thanks.

And then on the credit.

Hi, Colin.

This is Vimal.

As I mentioned, that we are closely partnering with our strategic financing partners, portal, key and [QIA] and any questions or any condition that needs to be met.

They're very collaborative with us and we update those amendments if needed.

So if there is anything needed we will update you what is needed on the credit

[side].

Thank you.

That's helpful.

Samir Devani, Rx Securities.

Hi, guys.

Thanks for taking my questions.

I've got just a couple and so I just want to make sure I understand the TRANQUILITY program strategy correctly for is it now the plan to do to get the label expanded further in care facilities and then do the subsequent OUTCOMES trial.

And if that's the case, what's the marketing strategy in that scenario?

And then the second question is just on and maybe this is for Richard, as OpEx is significantly lower in the quarter than I had.

I'm just wondering if Q4 is a good run rate for Q1?

Thanks very much.

Yes, hi, morning.

This is Vince again.

I'll take the first part of your question.

So that is a good way to look at it, right.

And I think it's also probably reasonable to say that in principle or in theory, we could run both studies together.

I think your description is our the way we look at it, and I think that's a sensible way to approach it.

Terms of the run rate, Samir, yes, it's probably a good marker of plus or minus a few dollars, but generally a pretty good marker.

And it does, Matt, I'll just dovetail to what Vince outlined just now, market entry strategy in this market.

Do you think about going into the care setting.

A lot of those are contracted through IDNs.

So we're actively engaged with them now and it's going to take a much smaller footprint to engage with those care facilities and snus versus the home setting.

So we feel that building a beachhead there makes a really good commercial sense.

That's great.

Thanks very much.

Sumant Kulkarni, Canaccord Genuity.

Thanks for taking the follow-up.

So this might be somewhat of a premature question, but it's still an important one, I think both from a financial modelling perspective, what are your latest thoughts about eventual commercial pricing of Fibre One in the Alzheimer's agitation setting, as it appears that the product might be under-priced currently given the potential value intuitive?

Yes.

So that's a great question, and I appreciate it.

I mean, right now, when you look at the price point of Rexulti, it's just over $1,400 a month.

So there does seem to be some pricing flexibility in that market and one that we continue to examine.

Thank you.

We reached end of our question-and-answer session.

I'd like to turn the floor back over to management for any further or closing comments.

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

Have a great day.

Thank you.

That does conclude today's teleconference and webcast.

You may disconnect your line at this time and have a wonderful day.

We thank you for your participation today.