Q4 2023 BioXcel Therapeutics Inc Earnings Call
[music].
Unknown Executive: 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. At this time, all participants are to listen only in mode.
Good morning, and welcome to the bio cell 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.
At this time, all participants are a listen only mode.
Unknown Executive: If during the conference call you require operator assistance, please press star zero on your telephone. After the formal remarks, there will be a question and answer session. If you would like to submit a question, you may press star 1 on your telephone. 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 the future financial or business performance of the company, actual results could differ materially from those anticipated in those forward-looking statements.
During the conference call you require operator assistance. Please press star zero on your telephone keypad. After the formal remarks will be a question and answer session. If you'd like to register a question you May Press star one on your telephone keypad gesture.
Just to remind everyone certain matters discussed in today's conference call and or answers that maybe 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 30th 2023 which can be found at www dot biologics cell therapeutics dot com or on Www Dot C C dot Gov, and which will be updated.
Unknown Executive: 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 operated by Dr. Vimal Mehta. Executive Officer, Dr. Vince O'Neill, Chief of Product Development and Medical Officer, and Richard Steinhart, Chief Financial Officer. You will be joined in the Q&A session by Dr. Frank Yocca, 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.
And 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. Jim on meta.
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 Q&A session by Dr. Frank JAKO, 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. Try meta. Please go ahead.
Thank you operator, good morning, and thank you for joining us.
This is an exciting time for buyer comes out of your rigs plus we are very pleased with the progress of our Tango program.
Vimal D. Mehta: First, we are very pleased with the progress of our Tranquility 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 for treating patients struggling with episodes of acute agitation. During today's call, we will dedicate our prepared remarks to updates on tranquility and serenity. These late-stage clinical programs represent groundbreaking opportunities to advance our goal of bringing new treatment options to patients. As a reminder, there are no currently approved acute treatments for agitation associated with Alzheimer's disease or for bipolar disorder or schizophrenia in the at-home setting. These conditions represent large, untapped markets.
As I have communicated previously this is a top priority capital allocation second we are focused on that one thing thats hidden entity program.
Both programs provide significant opportunities for us to address the unmet need and do you think patients struggling with that preserves of acute agitation.
During today's call, we will dedicate our prepared remarks.
And frankly, it instead entity.
These late stage clinical programs represent ground breaking up agenda DS.
Advance our goal of bringing new treatment options to patients as a reminder, there are no currently approved acute menswear agitation associated with Alzheimer's disease or for bipolar disorder schizophrenia in the at home setting these cars.
In addition represent large untapped markets.
Vimal D. Mehta: All of us at BioXcel Therapeutics are motivated to develop new treatment options for the greatest number of patients and caregivers. VINs will now provide a detailed update on Tranquility and Serenity. After this, I will briefly discuss several corporate updates and then review our financial results before we open the call for Q&A. With that, I will turn the call over to VINs. Thank you, Vimal.
All of us at by itself that our buildings are motivated to develop new treatment options for the greatest number of patients and caregivers.
Vince will now provide a detailed update on <unk> and CIT entity. After this I will briefly discuss several corporate update and Vince will then review our financial results before we open the call for Q&A with that I will turn the call over to Ben Thank.
Thank you Bill 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 bio <unk> cell fiber one.
Dr. Vince O'Neill: 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 501. Let me begin with Tranquility, where we are evaluating 5.0.1 as an acute treatment for agitation associated with Alzheimer's Dementia, or for short, AED. As Vimal said, tranquility 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.
Begin with tranquility, well, we're evaluating 501 as an acute treatment for agitation associated with Alzheimer's dementia or for short 80.
It's been all said tranquility remains a top priority to bring a novel treatment to this large underserved patient population.
Every year estimate show that millions of Americans without timeless disease experience agitation.
Physicians often aimed to address this with neuropsychiatric drugs used off label because there are no approved treatments specifically for acute agitation episode.
Dr. Vince O'Neill: This area of neuroscience drug development for an episodic treatment is novel and uncharted, and we believe we are uniquely positioned to address it. At BioXcel Therapeutics, we have EGALMI on the U.S. market as an approved acute treatment for bipolar disorder and schizophrenia-related agitation. This provides us with a strong foundation from which we are expanding into development for AAD in our Tranquility Program and into development in different settings in the bipolar and schizophrenia populations through the Serenity Program. For Tranquility, we have completed a type B breakthrough therapy designation meeting with the FDA on February 20. This meeting followed the one we had in October of last year.
The theory of neuroscience drug development for an episodic treatment is novel and uncharted and we believe we are uniquely positioned in this space at bio cell therapeutics, we have the Gal me already on the U S market is in a pure approved acute treatment of bipolar disorder and schizophrenia related agitation. This provides us.
With a strong foundation from which we are expanding into development and a D&O 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 following the one we had in October of last year.
Dr. Vince O'Neill: 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 date. However, we can share now 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 501 in Tranquility 1 and 2 to date. We plan to generate these data, including repeat efficacy data, in a variety of relevant care settings and to use PENC as the primary efficacy measure, as used in the prior Tranquility tool.
Mind, 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 who the based on the Fda's feedback we plan to generate additional phase III efficacy and safety data.
In care facilities to expand the database beyond the 70 patients who've been treated with 60 micrograms of fiber one in tranquility wanting to to date, we plan to generate these data, including repeat efficacy data in a variety of relevant care settings and to use pink as the primary.
Efficacy measure is used in the prior tranquility to study.
Dr. Vince O'Neill: To remind everyone, on 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, otherwise known as Tranquility at Home. And given the priority to expand the database to generate additional efficacy data in care facilities, the company is now re-evaluating the timing for initiating Tranquility at Home. Lastly, the FDA has indicated that we will need to generate long-term safety data.
To remind everyone in November 23, we announced that we were planning to conduct a phase III trial in the at home setting with safety as the primary objective.
Otherwise known as tranquility at home.
Given the priority to expand the database to generate additional efficacy data in care facilities. The company has 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 AED in patients we plan to further engage with the FDA regarding this requirement.
Dr. Vince O'Neill: However, given the lack of any regulatory precedent for episodic treatment of AAD in 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... 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.
We look forward to receiving final meeting minutes from the agency and sharing more details with you later this month.
Extremely motivated by this potential opportunity to bring a much needed acute treatment option to patients developing novel episodic treatments with no regulatory Preston comes with new learnings to create an optimal development path. At this time, we believe we have a clear path forward.
Dr. Vince O'Neill: At this time, we believe we have a clear path forward. To that point, we've had multiple meetings with the FDA on this uncharted regulatory path and have greatly appreciated the agency's guidance as we move forward to advancing our clinical trials. Let's now turn to Serenity, where we are evaluating the potential at-home use of 501 for agitation associated with bipolar disorders or schizophrenia. On March 6th, we completed a Type C meeting with the FDA.
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 slower to advancing our clinical program.
Let's now turn to serenity well, we are evaluating the potential at home use a final one for agitation associated with bipolar disorders of scripts schizophrenia.
On March the sixth we completed a type C meeting with the FDA. This fall with the one we conducted last November based on FDA feedback we plan to the all year. The 120 microgram dose a final one.
Dr. Vince O'Neill: This follows the one we conducted last... Based on FDA feedback, we plan to evaluate the 120-microgram dose of 501, as a reminder, an approved dose of Igalme, in the at-home setting as an amendment to our Serenity 3 Part 2 protocol, which the company had voluntarily paused as we discussed the FDA's proposed changes to the protocol in light of the results of Serenity 3 Part 1. Our current plan is to move forward with this study as amended with safety as the primary objective and efficacy measures as exploratory. Keep in mind that the label for Igalme currently includes a limitation on use, noting the lack of efficacy or safety data beyond 24 hours.
As a reminder, unapproved does have a gal me.
In the at home setting and an amendment to our survey 93, part two protocol, which the company had voluntarily paused.
As we discussed the Fda's proposed changes to the protocol in light of the results of three anti <unk> power one.
And our current plan is to move forward with this study as amended with safety is the primary objective and efficacy measures as exploratory endpoints.
Keep in mind the label for a gallon me currently includes the limitation on us, noting the lack of efficacy or safety data beyond 24 hours, we believe that our ability to seek labeling with the current L. O you will depend in part.
Dr. Vince O'Neill: We believe that our ability to seek labeling without the current LOU will depend, in part, on the number of agitation episodes we observe over the three-month follow-up period. To support our pivotal trial with the 120-microgram dose, we're also planning to conduct a small study, approximately 30 patients, to evaluate the correlation between patient-reported or informant-reported efficacy with trained rater-reported efficacy using P We expect to provide additional information about the program plans after receiving and reviewing the final meeting minutes from the FDA. And with that, I will turn the call back over to Bill. Thank you, Vince.
The number of agitation episode as we observe over the three months follow up period.
To support our pivotal trial with 120 microgram dose. We're also planning to conduct a small study approximately 30 patients to evaluate the correlation between patient reported or informant reported efficacy with trained rate of reported efficacy using peg 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 them.
Thank you Vince.
Vimal D. Mehta: We are also pleased with progress in other areas of the business. On January 1st, the permanent J-code for EGALMI became effective, and our commercialization 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 BioXcel 701. I would like to end my remarks by emphasizing that we remain focused on strengthening the company's balance sheet.
We are also pleased with progress in other areas of the business on January 1st the permanent J code for regarding mean became effective and our commercial position efforts continued to progress.
In addition, we continue building a robust intellectual property portfolio and strengthening our long term burden protection.
Finally, we are pleased to have received fast track designation from the FDA for <unk> 701.
I would like to end my remarks by emphasizing that we remain focused on strengthening the company's balance sheet towards this end, we are actively exploring multiple financial options to extend our cash runway and find out what the key clinical program in order to create maximum.
Richard I. Steinhart: To this end, we are actively exploring multiple financial options to extend our cash runway and fund our key clinical programs 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.
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 rich. Thank you Bill.
Richard I. Steinhart: Thank you, Vimal. Net revenue from El Gamy was $376,000 for the fourth quarter of 2003, 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 decreased expenses for both the fourth quarter and the full year were primarily attributable to decreased clinical trial activity. Associated with the winding down of Serenity 3 and Tranquility 2 studies, a decrease in chemical manufacturing and control costs, and a decrease in personnel related to the company's reprioritization in August of 2023.
Net revenue from El Ghali was $376000 for the fourth quarter.
Compared to 238000 for the same period in 2022.
Net revenue was $1 4 million the full year 2023 compared to 375000 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 in expenses for both fourth quarter and the full year were primarily primarily.
While activity associated with the wind down of Serenity, III and tranquility to sudden decrease in chemical manufacturing and control cost and a decrease in personnel related to the company's re prioritization in August of 2023.
Richard I. Steinhart: 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. The increased costs for the full year are generally attributable to an increase in legal and professional fees, costs associated with personnel, and related expenses to support the commercialization of Othami in the U.S. 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.
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.
Increased costs for the full year.
Attributable to an increase in legal and professional fee costs associated with personnel and related expenses to support the commercialization of the economy and the U S.
Prior to the company's re prioritization.
<unk> 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.
Vimal D. Mehta: 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. The loss for 2023 includes approximately $18.6 million in non-cash stock-based compensation. Total cash expenditures for 2023 totaled approximately $155 million. Cash and cash equivalents total $65.2 million as of December 31, 2023, compared to $193.7 million as of December 31, 2022. The company estimates that its current cash and cash equivalents will fund its operations through mid 2024. This estimated cash runway does not include any potential financing activities that may be undertaken by the company. Now I'd like to turn the call back to Vimal. Thank you, Rich. We would now like to open the call to questions. Operator.
But at the same here 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.
Noncash stock based compensation.
Total cash expenditures for 2023 total approximately $155 million.
Cash and cash equivalents totaled $65 2 million as of December 31.
Three compared to $193 7 million as of December 31, 2022.
The company estimates that its current cash and cash equivalents will fund its operations through mid 2024. This estimated cash runway does not include any potential financing activities may be undertaken by the company.
Now I would like to turn the call back to Bill.
Thank you rich.
I'd now like to open the call for questions operator.
Unknown Executive: Thank you. We will now be conducting a question and answer session. If you would like to be placed in the question queue, please press star 1 on your telephone keypad.
Thank you well now be conducting a question and answer session if you'd like to be placed in the question queue. Please press star one on your telephone keypad. We ask you. Please limit yourselves to one question and one follow up once again Thats star one to be placed into the question queue. Please ask one question and one follow up her first question.
Unknown Executive: We ask that you please limit yourselves to one question and one follow-up. Once again, that's star 1 to be placed in the question... and please ask one question and one follow-up. Our first question today is coming from Greg Harrison from Bank of America. Your line is now live.
Today is coming from Greg Harrison from Bank of America. Your line is now live.
Unknown Executive: Hey, good morning, guys. Thanks for taking the questions. So after your recent FDA meetings, it seems you have more clarity about the Tranquility Programs. Would you say now that you have two separate paths to SNDA? and Alzheimer's.
Hey, good morning, guys. Thanks for taking my questions.
So after your recent FDA meetings. It seems you have more clarity.
So the tranquility programs would.
Would you say now you have two separate paths to F N D. A.
And all the farmers.
Unknown Executive: One in the care setting and one at home. And then how are you thinking about funding the program and the company as a whole as you get both to the end of your cash runways? Are there any specific avenues that you're exploring? Thank you. Good morning, Greg. This is Ben.
One in the care study now 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.
Good morning, Greg. This is Ben Thanks for the question so to answer directly yes, we believe so so obviously the tranquility efficacy.
Unknown Executive: Thanks for the question. So, to answer it directly, yes, we believe so. Obviously, the Tranquility, Efficacy, PEC Primary Study, and also the Safety at Home Study would be run with registrational intent. So, yes, in terms of funding, I would refer back. So, as Graig stated in my opening remarks, we are looking at multiple options to fund the company.
<unk> Pak primary study and also the safety at home study would be run with Registrational intent. So yes in terms of funding I would refer back to them.
So as I grabbed you stated in my opening remarks, we are looking at multiple options to.
Doug I mean, now having a clarity 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 said entity and as we speak we are very actively working on those for us.
Unknown Executive: Now, having clarity from the FDA clearly tells us what the trials we need to do to capture these large market opportunities, both in tranquility and serenity. And as we speak, we are very actively working on those. Great, thanks again. Our next question is coming from Robyn Karnauskas from Truist Security. Your line is now live. All right, thank you for taking my question. So I have two.
Great. Thanks again.
Thank you. Our next question is coming from Robin Karnofsky from Jewish Securities. Your line is now live.
Alright. Thank you for taking my question so our Q.
Unknown Executive: After the recent meetings with the FDA, you chose the 120 milligram dose, but does that mean that it would allow for expansion in the at-home setting? I have a follow-up for the morning, Robyn. So I think the choice of 120 microgram dose is a good choice for the following reasons. Efficacy is fundamentally established at that dose. That's clearly an approved dose of EGALMI, one of two approved doses of EGALMI.
The recent meetings with the FDA, you've chosen 120 milligram dose but.
And does that mean that would allow for expansion and awesome setting I have a follow up for that.
Yeah morning, Robyn and so I think the choice of 120 microgram dose them. We believe is a good choice for the following reason efficacy is fundamentally established for that does that is clearly an approved dosing began may one of two approved doses of IV gallon me.
Unknown Executive: So yes, we believe that with that, again, a registrational intended study with safety as the primary objective is what we need to go back to the FDA with data in, and follow up on that a little bit. So, you'll give them a minute. Have you spoken to the FDA clearly about that? and you'll give them the minutes later at some point.
So yes, we believe that with that again, a registrational intended study was safety as a primary.
<unk> is what we need to go back to the FDA with data in hand.
And following up on that a little bit.
You'll give them.
Have you spoken to the FDA clearly about that.
And you'll get the look.
Unknown Executive: 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 that, that's bad, like funding the program. Vimal Mehta, Robert Risinger, Frank Yocca, Frank Yocca, Robert Risinger, The fact that, you know, you have to fund a lot of these programs is giving you feedback. How do investors think about the fact that, Actually, how do they think about the options on the table?
Peter I'm point.
Yes, sorry to interrupt you yeah, absolutely we've had 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 demo like how do you think about you know you have a lot going on you talked about like you have a lot of options how do we think about it.
And the fact that you know you have to find a lot of these programs. They are giving you feedback.
So how do investors think about the fact they are.
And actually how did you think about the options on the table for you.
Unknown Executive: So Robyn, good morning. In terms of the priority, we will focus on Tranquility, the trial that Vince has discussed, because that 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 clarity on what dose we can take forward for a potential registration trial. In our trials, we have tremendous experience in the company. We have done more than 11 placebo-controlled double-blinded trials. These trials are capital efficient.
So robin good morning.
The <unk>.
In terms of the priority we will.
Focus on tranquility trial that Vince has discussed because that can help us on a path to a potential is India.
In the care setting as discussed on this call and also on the Serenity program and we have a clarity what doors, 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 control.
Blinded trial these times of capital efficient and opportunity we have in front of with is very large, but we want to be very focused and as I mentioned tranquility will be there tough prior.
Unknown Executive: And the opportunity we have in front of us is very great, but we want to be very focused. And as I mentioned, Tranquility will be the top priority, followed by Serenity. Great, thank you. Questions? Graig Suvannavejh.
Followed by San annuity.
Great. Thank you.
Thank you. Our next question is coming from Greg So when others from Mizuho Securities. Your line is that life.
Unknown Executive: U.S. Securities Authorization Hey, good morning. Thanks for taking my questions and for the clarity. To please forgive me, I don't know if the first one has already asked my apologies, just on that.
Hey, good morning, Thanks for taking my questions and for the clarity.
Two please for me I don't know if the first one was already asked my apologies just on the.
Unknown Executive: Tranquility at Home, or just the Tranquility Program overall, the pivot to needing to collect 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 Transcribed by https://otter.ai, 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 SMDA and, similarly, what you think that total cost would be for completing the Serenity program. It's just important, in light of your current cash, just for investors to know what more funds So I'll take the first part of that question. It's obviously an important question.
[noise] tranquility at home or just the tranquility program overall, the pivots to needing to collect now efficacy data can you tell us kind of what changed versus the previous plan just to focus on 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 S. N D E and similarly, what you think that total cost would be for.
Completing the Serenity program. Its just important just in light of your current cash just to for investors to know what what more funds you need to get them to really the next revenue inflection point. Thank you.
Yep Good morning, Greg So I'll take the first part of that question is obviously an important question. So after we met with the FDA last year and we began to think about the design of the study and in depth and in detail and begin to engage with our advisors and kols. It became clear that using non validated caregiver assessed.
Unknown Executive: So after we met with the FDA last year and began to think about the design of the study in depth and in detail and began to engage with our advisors and KOLs, it became clear that using non-validated caregiver assessments would be challenging and would frankly carry risks. So with that better understood, we went back to the FDA to rediscuss what additional, quote, additional efficacy, unquote, meant. And obviously, we've now agreed on formal efficacy analysis with PEC as a primary endpoint, clearly an endpoint we've used before with success. So hopefully, that provides clarity. Good morning, Greg. This is Vimal.
<unk> would be challenging and would frankly carry risk so with that better understood. We went back to the F. D. A to rediscover what additional quote additional efficacy unquote meant M and obviously, we've now agreed on formal efficacy analysis with Pat because the primary endpoint clearly an endpoint we've used.
Before with with success so far.
Hopefully that provides clarity.
Unknown Executive: The funding question is very relevant. Now, having the clarity from the FDA was very important for us to know what funding we need to do. So, regarding the tranquility trial, the efficacy trial that Vince mentioned, it's 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 Serenity at-home trial, we have tremendous experience with Serenity 1, 2, and Serenity 3. We have a very good idea in terms of the timing as well as capital requirements for that trial. And as I said in the beginning, our patient costs are low for these trials. And overall, these trials are capital efficient, and they can be delivered within a reasonable time.
Good morning, Greg This is being.
Financing goes shouldn't is very relevant now having the clarity from the FDA.
It's very important for us to know what financing we need to do so.
Guarding the tranquility trial I forget the fact that Vince mentioned, if they're not 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 <unk>.
In terms of the C N N V at home trial.
We have tremendous experience with salinity weren't doing 283, we have a very good idea in terms of their timing as well as capital requirement for that trial and as I said in the beginning part of patient costs are low for these trials and overall these ties, though capital efficient and they can be delivered.
The Navy's Naval time, so we are working on all of that and we will come back and lay out the plan for a triangular D as well as say the entity and we will be having conversation would be capital requirements are based on our current cash position and how much cash then we need to extend using the means.
Unknown Executive: So we are working on all of that, and we will come back and lay out the plan for Tranquility as well as for Serenity. And we will be having conversations about the capital requirements based on our current cash position and how much cash runway we need to extend using the means we have, which are equity investment versus monetization of any assets we have or slash partnering. But all these things are being pursued in parallel, and we will be able to provide clarity on the plan and financing as we progress. Thank you. Your next question is coming from Sumant Kulkarni from... Thanks for taking my questions. So you mentioned in your prepared remarks that for labeling purposes, it is for 24 to 48 hours, and you'll need to generate more data over three months.
We have which are a good investment what is the monetization of any assets, we have or slash partnering but all these things are being pursued in parallel and we will be able to provide the clarity on that plan and financing as we progress.
Okay. Thank you for them.
Okay.
Thank you. Our next question is coming from multiple Kearney from Canaccord Genuity. Your line is now live.
Good morning, Thanks for taking my question.
So you mentioned in your prepared remarks that for labeling purposes. The limitation of use is about a 24 to 48 up you get and you need to generate more data with three months.
Unknown Executive: When you do 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 501 as a risk? So, I think the first way to answer that is that it's not our expectations, but I am going to ask Dr. Risinger, our CMO, to comment a little bit further on that. I mean, I guess it revolves around our expectations for frequency.
When you're doing so what's the risk that you might find it agitation is not necessarily episodic and in that case, you still think you might pursue 501 as a rescue therapy.
Yeah.
So I think the first way to answer that is that is not our expectations, but I'm gonna have stopped rising out of our CMO to comment a little bit further on that I mean, I guess it revolves around our expectations for frequency.
Robert Risinger: Yes, I think, I mean, not to get into the weeds, but the CMAI is a tally of episodes. It's a tally of a certain number of episodes plus severity. The FDA has agreed to our PEC as a pivotal endpoint for acute agitation, and we would all like to understand what the relationship is between a chronic condition with episodic or episodes of acute agitation.
Yes, I think I mean, not to get into the weeds, but the CMA I as a tally of episodes it's Italia.
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 episodic or episodes of acute agitation.
Robert Risinger: So we hope to be able to speak a little bit about that data, but the reality is these patients have acute episodes that need treatment even now and with every treatment that is used, whether it's on-label or off-label. And my follow-up is a financial question. So what do all these latest updates mean with respect to your ability to be in line? Covenants Associated with the Prior Financing, or will you need to rework those?
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 these latest updates mean with respect to your ability to be in line with the covenant covenants associated with the private financing or would you need to be but those again.
Unknown Executive: Good question, Sumant. We are always in discussions with our strategic partners, and they are very supportive of facilitating and having companies 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 clarity from an FDA perspective on what we need to do in terms of our path forward for these two programs. The next question is coming from Ram Selvaraju from H.C. Wainwright. Your line is now. Thanks very much for taking my questions. Can you hear me?
Good questions a month, we are always in discussion with our strategic partner <unk>.
And they are very supportive of facilitating and having come to me be able to get financing because they really believe in this asset and they see that this is a large market opportunity.
It's a very good partnership and we work together to a chart out the optimal pad I think most of them Barton from this call is that we have a clarity from FDA.
FDA perspective, what we need to do in terms of our path forward for these two programs.
Thank you.
Thank you. Your next question is coming from Hamzah <unk> from H C. Wainwright. Your line is now live.
Thanks, very much for taking my questions can you hear me.
We can.
Unknown Executive: We can. Okay, so 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. Collected before its pause, you expect it to be usable in furtherance of the development plans for 501. I'm going to ask Dr. Risinger, CMO, to comment on that, as far as he can. When you submit an NDA, all data from all patients who were ever treated with the medication is used. 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,
So just wanted to see if you could offer some clarity whether quantitative or qualitative regarding the pause study and how much data from that study that was collected before its paws you expect to be usable in further and solves the development plans for final one.
And I'm Gonna have stopped are rising our CMO to comment on that as far as he can.
Well.
When you submit for an NDA all data in all patients who were ever treated with the medication is used and so to that extent safety data from that trial is entirely submitted to the FDA.
Okay.
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 yes, if indeed you do.
Robert Risinger: 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 have to. 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.
Okay.
Yeah, I think the pregnant pause here as 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 costs because in the end the FDA wants to see exposures and safety safety safety and that's what we have that safety data is part of the NDA package.
Robert Risinger: That doesn't mean it doesn't offset costs because, in the end, FDA wants to see exposures and safety, safety, safety, and that's what we have. That safety data is part of the NDA package. But I think, Bram, maybe another way to say it, it 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? Ram, we have formally started the process to explore the opportunities, whether it's partnering or... Monetization The company was extremely focused right now on getting the plans for the neuroscience business, which is Tranquility and Serenity. Having the clarity, we will have more time to dedicate to focus on Oncosexcel because, as you know, if we can monetize our partner, that can help bring non-dilutive financing to Thank you very much.
I think Brian maybe another way to say it probably is in the final analysis, a minor cost consideration.
Okay, and then just very quickly do you have any updates on your plans for Oncall Sextile at this time. Thank you.
Uh huh.
Formally started the process to explore the opportunities whether it's partnering or.
Monetization company was extremely focused right now on getting the plans for the neuroscience, there's another vintage tranquility instead of anybody having that clarity we will have more diamond.
We're dedicated to focus on known cause XL because as you know if we can monetize our partner that can help bring non dilutive financing to the company.
Yeah.
Thank you very much.
Vimal D. Mehta: Thank you. Next question, please. Your next question today is coming from Colin Bristow from UBS. Your line is now live. Good morning, guys.
Thank you next caller.
Thank you our next.
Question today is coming from Colin Bristow from UBS. Your line is now live.
Hey, good morning, guys and thanks for the update.
Unknown Executive: And thanks for the updates. I'm sort of piggybacking on a few of the questions we've already been asked. Firstly, on the Tranquility Program, can you give us any indication of sort of trial size or, perhaps more usefully, sort of timing? It feels like this is now going to be a back half initiation.
What are you sort of piggybacking on a few of the questions may only be NAS.
Firstly on the tranquility program, but can you give us any indication on onto the trial size or perhaps than 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 'twenty five.
Unknown Executive: So is it reasonable for us to assume that we'll probably see data in the back half of 2025? Secondly, on the existing Agaume launch, sales are down sequentially. Now, acknowledging there's no sales effort, is this a surprise to you, or can you tell us why sales are not at least staying flat? And then, just a point of clarification on the credit agreement, you know, 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.
Secondly on the existing agave and wood sales are down sequentially now acknowledging there's no sales.
The.
It surprised you or can you tell us why sales are not at least staying flat.
And then just a point of clarification on the credit agreement.
Again, acknowledging you didn't satisfy one of the requirements is it reasonable to expect.
We'll get an update shortly after you.
Unknown Executive: Thank you. Good morning, Colin. This is Vince.
Things with FCA.
Dr. Vince O'Neill: I'll take the first of your three-parter. In terms of timelines, we clearly want to have the meeting minutes in hand and digested before we make any definitive statements. And with that, I'll hand over to Michael. Sure. Good morning. This is Matt.
Good morning, Collyn. This is Vince I'll take the first of your of your three parter M. In terms of timelines, we clearly want to have the meeting minutes in hand, and digested before we make any definitive statements.
And with that I'll hand over to.
Sure. Good morning. This is Matt so to answer your question about Q4 versus Q3.
Matt Wiley: So to answer your question about Q4 versus Q3, you know, in Q3, remember that we had a fullsome commercial effort for most of that quarter. In Q4, we saw revenues increase 10 percent versus Q3. And we saw our cartons shipped increase by about 8 percent.
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%.
Matt Wiley: 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 cash. So the CAD team has really punched above their weight to get to this revenue. We've seen some of the contracting efforts in Q4 begin to bear fruit, and we're seeing more of that in Q1.
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 hadn't field previously in addition to the cats. So the cat team is really punched above their weight to get this revenue we've seen.
Some of the contracting efforts in Q4 begin to matriculate, we're seeing more of that in Q1.
Matt Wiley: And we expect to see continued growth. Great, thanks. And then on the credit, Hi Colin, this is Vimal. As I mentioned, we are closely partnering with our strategic financing partners, both UKRI and QIA, and any questions or any condition that needs to be met, they are very collaborative with us, and we update those amendments if needed. So if there is anything needed, we will update you on what is needed on the credit account.
And we expect to see continued growth.
Okay.
Great. Thanks, and then on the credit.
Hi, Colin this is.
As I mentioned that we are.
I've closely partnering with our strategic financing partners, Boto, <unk>, and <unk> and any questions or any condition that needs to be met they are already collaborating with us and we.
We update those amendment if needed so if there is any.
<unk> needed we will update you on what is needed on the credit agreement.
Thank you that's helpful.
Vimal D. Mehta: Thank you. Thank you. The next question is coming from Samir Devani from RX Security. Your line is now, And guys, thanks for taking my questions. I've got just a couple.
Thank you next question is coming from Samir Giovanni from Arctic Securities. Your line is now live.
Hi, guys. Thanks for taking my questions I've got just a couple of them. So I just want to make sure I understand the tranquility program strategy correctly is it now the plan to do to get the label expanded its first in.
Unknown Executive: So I just want to make sure I understand the Equality Programme strategy correctly. So is it now the plan to get the label, Care Facilities, and then do the subsequent at-home marketing and so on. And then the second question is just on, and for Richard, OPEX. Lower in the quarter than I had.
In care facilities, and then do the subsequent 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 our Opex is significantly lower in the quarter than I had I'm, just wondering if keith or what's a good run rate for Q1, thanks very much.
Dr. Vince O'Neill: Hi morning, this is Vince. Again, 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. But I think your description is our way of looking at it.
Yeah, Hi morning. This is Vince again I'll take the first part of your question. So that that is a good way to look at it right.
It's also probably reasonable to say that in principle or in theory, we could run both studies together, but I think your description is as ever the way we look at it and I think that's a sensible way to approach it.
Dr. Vince O'Neill: And I think that's a sensible way to approach it. In terms of the runway, Samir, yeah, it's probably a good marker, plus or minus a few dollars, but generally a pretty good marker. This is Matt. I'll just dovetail to what Vince said. Outline just now, market entry strategy in this market. You think about going into the care setting. A lot of those are contracted through IDN, 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 SNFs versus the home setting. So we feel that building a beachhead there would make a really good commercial. Thank you. Our next question is a follow-up from Sumant Kulkarni from Kanakorajanui.
In terms of the runway Zamir Ah yeah, it's a it's probably a good marker plus or minus a few dollars, but generally a pretty good marker.
Yeah. This is Matt I'll, just dovetail to what Vince.
Outlined just now market entry strategy in this market do you think about going into the the care setting a lot of those are contracted through idea and so were actively engaged with them now.
And it's going to take a much smaller footprint and engage with those.
Those care facilities in Smiths versus the home settings. So we feel that building a beachhead there makes a really good commercial sense.
Okay, that's great thanks very much.
Yeah.
Thank you. Our next question is a follow up from Sumit Kulkarni from Canaccord Genuity. Your line is now live.
Matt Wiley: Your line is now live. Thank you for taking the follow up. So this might be somewhat of a premature question, but it's still an important one, I think, from a financial modeling perspective. What are your latest thoughts about eventual commercial pricing of 501 in the Alzheimer's agitation setting as it appears that the product might be underpriced currently given its potential value.
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 modeling perspective. So what are your latest talks about eventual commercial pricing of fiber one in the Alzheimers agitation setting as it appears that the product might be underpriced currently given the potential that you might bring to the table.
Unknown Executive: Yeah, so that's a great question, and I appreciate it. I mean, right now, when you look at the price point of Rexalti, it's just over $1,400 a month.
Yeah, that's a great question and I appreciate it I mean right now when you look at the price point of <unk>. Its just over $1400 a month. So there does seem to be some pricing flexibility in that market and one that we continue to examine.
Matt Wiley: So there does seem to be some pricing flexibility in that market and one that we continue to examine. We've reached the 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...
Thank you we've 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.
Okay.
Thank you everyone for joining us today and for your continued interest in backfill 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.