BioXcel at Canaccord Genuity: Strategic Insights on Phase 3 Trials

On Tuesday, 12 August 2025, BioXcel Therapeutics (NASDAQ:BTAI) participated in Canaccord Genuity’s 45th Annual Growth Conference, offering a strategic overview of its upcoming Phase 3 trial data for BXCL501. The company emphasized both its financial stability and the challenges of preparing for an sNDA submission, while also discussing commercialization strategies and intellectual property protection.

Key Takeaways

• BioXcel is preparing for an sNDA submission for BXCL501 following the Phase 3 trial data readout.
• The Serenity at Home trial is pivotal, focusing on safety and efficacy in treating agitation at home.
• BioXcel holds a strong patent portfolio, ensuring market exclusivity until February 21943.
• The company is financially stable, with $18 million in cash and an additional $15 million received.
• BioXcel is exploring strategic partnerships to enhance commercialization efforts.

Operational Updates

• The Serenity at Home trial has gathered data from 2,200 agitation episodes over 12 weeks, involving 200 patients.
• The primary focus is on safety, with repeat dosing efficacy being explored.
• There are currently no FDA-approved therapies for treating agitation in schizophrenia and bipolar disorder at home.

Future Outlook

• BioXcel is preparing for a meeting with the FDA on August 20th to confirm sNDA submission requirements.
• The company plans to initiate a second Phase 3 trial for Alzheimer’s agitation, pending CRO selection and resource availability.
• BioXcel is considering a price point of up to $1,400 per prescription for BXCL501, with details to be finalized after the data readout.

Commercialization and Market Opportunity

• BioXcel sees significant potential for BXCL501 in both home and retail settings.
• The company is open to strategic partnerships to leverage existing footprints for broader patient reach.
• With 13 patents in the Orange Book, BioXcel maintains robust intellectual property protection.

Financial Status

• BioXcel has eliminated all financial covenants as of August 15th.
• The company is financially stable, with $18 million in cash and an additional $15 million received through ATM and warrant exercises.

Q&A Highlights

• The emphasis on safety in the Serenity at Home trial was reiterated, with the primary objective being the safety of BXCL501.
• BioXcel’s intellectual property was highlighted as a competitive advantage, ensuring market exclusivity for the foreseeable future.
• The company continues to evaluate its commercialization options, focusing on leveraging its clinical trial and regulatory approval capabilities.

Readers are encouraged to refer to the full transcript for a detailed account of the conference call.

Full transcript - Canaccord Genuity’s 45th Annual Growth Conference:

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Afternoon, everyone. I’m Sumant Kulkarni, a senior biotechnology analyst here at Canaccord Genuity. And I’d like to thank all our clients for your continuous support of our equity research effort here. I’m very pleased to have BioXcel Therapeutics presenting at our conference. It’s a really interesting time and an important time for the company ahead of one of their key Phase III data sets that’s coming up any day now in August is what you’ve said.

I want to steal some thunder from you because you have a product that is out in the market already. It’s a GalMe. It’s for agitation. So we’d like to hear how you’d like to make America calm again with your product. With that, I’ll turn it over to you, Wimal, to give a small intro, and then we’ll go right into Q and A.

Wimal, Unknown, BioXcel Therapeutics: Thank you, Sumant. Good afternoon, everyone. Thanks for joining us today. As Sumant mentioned that we have a very important catalyst upcoming this month. This is about our pivotal Phase C trial related to schizophrenia and bipolar agitation in the home setting.

So we already have our product approved in the in care setting, it’s called as EGALMI. And two doses are approved, one hundred and twenty microgram and one hundred and eighty microgram. For home setting, we got alignment with the FDA to test one hundred and twenty microgram where we have already proven safety and efficacy in an in care setting, and now we are showing the safety in the home setting. Safety is the primary endpoint because we had two pivotal trials. We have proven the safety and efficacy in care.

Efficacy, we want to show the repeat dosing, over repeat dosing, so that’s an exploratory endpoint. That’s very exciting because for the first time we have conducted a trial in a home setting. In addition, as we all know, that there are no FDA approved therapies for treatment of agitation for schizophrenia bipolar in the home setting. In addition, as we continue to assess the commercial opportunity, And we put out a deck yesterday that originally we mentioned there are twenty three million episodes. But as we do market research and we’re triangulating with literature as well as our own clinical trial data, we believe opportunity is bigger than the twenty three million episodes.

And that is coming from our trial where we have already collected the data for 2,200 episodes over a twelve week period, and it’s a 200 patient trial. That’s one focus, clearly, in the very near term. In addition, we are preparing for sNDA package, what is required to file the sNDA. So we have an upcoming meeting with the FDA again this month. So we are excited about that, that what is package requirement to file an sNDA.

And in addition, for our Alzheimer’s program, where we already have one positive Phase C trial, we are evaluating currently options with CROs to initiate that trial. We already have alignment with the FDA to initiate a Phase C trial there. And it will be a second confirmatory Phase C trial. So current focus near term is on schizophrenia, bipolar in the home setting.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Thanks, Bimal. We also have a Senior VP of Medical and Clinical Affairs here, Dushan Kostich. So he’s kind of the guy in the hot seat right now, given all the stuff that you have coming up with the FDA. So I’ll start with the Serenity At Home trial. How good of a competitor is that going to be from an in home experience perspective to what you’ve seen in an in care setting?

Wimal, Unknown, BioXcel Therapeutics: So the best comparator we can think is some of the antibiotics that are used in the hospital setting and they are used in the home setting. I would say that comes to our mind.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. What specific metrics do you expect to report when you have these data out?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Yes, so as Vimal mentioned, the primary objective of the study is safety. And so in terms of safety, the primary endpoint is the incidence of adverse events, particularly drug related adverse events. We are particularly paying attention on the adverse events that are linked to the pharmacodynamic effects of the compound, which means something like somnolence, to some degree dry mouth, mouth tingling, dizziness, and then also some of the adverse effects that we have seen in the clinic, nausea and abdominal discomfort at lower rates. So, adverse events are really the primary objective and the primary endpoint, and again, as Vimal mentioned, we are evaluating the repeat dose efficacy over twelve weeks. So, are exploratory endpoints measuring patient reported outcomes.

So, patient and caregiver reported CGIs, global clinical indicators, and those are exploratory endpoints. So, some of that will be included in the top line data as well. Got

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: it. So, this is a neuropsychiatric trial. These trials typically tend to have placebo effects. You have an approved product out there on the market already, which had really good results in the SERENITY trial. How do you expect the placebo effect to change, if at all, in an in home setting versus in care setting?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: So that’s an interesting question because it’s true, and the placebo effect really, when you look across the psychiatry trials, it varied a lot depending on the patient population and on the designs of the trial. However, we should keep in mind that again, the efficacy of the compound has been proven before, so the importance of placebo effect is somewhat lower than what you would expect in a regular pivotal phase three trial. Having said that, we will see how placebo performs, we don’t really know, we have never done a placebo controlled trial in the home setting, so we don’t really know how patients will react to placebo versus the active compound. There can be differences in terms of the environment, whether they are in the emergency room or at home, and you can argue both ways which environment is more conducive to them experiencing this placebo effect. So we’ll see when the data come out.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: I think in your press release you mentioned that you’d seen up to the point you put out that release, you had about 2,200 episodes of agitation treated. How representative is that of a real world experience with number of agitation episodes over that time frame?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Right. So when you sort of do a rough calculation and compare it to our market research as well as the only published scientific manuscript in that area, we see that we are in about the same ballpark. So we think it’s very representative.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Okay. And given these patients are at home, they’re administering the film themselves. This is as close to real world as you can get, But Right. Is there any difference because these patients happen to be a trial that they’ll be somewhat more compliant or anything like that we should be thinking about?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Whether they’re more compliant because they’re in the trial, that has generally not been so much a case in psychiatry trials. We don’t we don’t really know, but they are basically choosing when to take it themselves. So this is not like a chronic therapy where they are supposed to take it every day. They are supposed they are taking this product when they feel they need it. So from that perspective, I think it’s really like a real world experience.

Wimal, Unknown, BioXcel Therapeutics: And also I like to add what Dushan said that we are seeing vast majority of patients completing the trial over twelve week period. That’s an indicator that there is an unmet need and they are getting some benefit from the trial.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So let’s move on now to the interactions with the FDA. You have a meeting coming up on the twentieth of this month. What do you specifically expect to discuss at that meeting? And is it fair to assume that you have this dataset coming and you’ll because you have the meeting on the twentieth that we’ll see the data before that?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: I don’t think we can we can go into the exact date of when the data are coming out. This meeting with the FDA, like most of the pre sNDA meetings, are mainly around the format and the content of the sNDA application, so it’s pretty operational from that standpoint. But we are also planning to discuss to confirm that our understanding of what’s needed for submission of the sNDA that we got from the meeting with FDA last year in April, that that is our correct understanding. So we need to conf so we don’t need to, but we are planning to confirm that, But really the main purpose of the meeting is for the format and the content of the sNDA.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: On the FDA’s interactions with you and the eventual ability to get this product in an in home setting, what’s the key thing that the FDA needs to see from you to enable that use?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: So the key thing is that the primary objective is the safety. So they really need to see the safety of this study, of the Serenity at Home study, and as we said, the primary objective is safety. So I think as long as we are showing that the safety incidence of adverse events is comparable to what they had seen in the clinic, I think we are in a good place. We are also evaluating the repeat dose efficacy over twelve weeks. We had already shown the trends of repeat dose efficacy over seven days in a controlled setting with an approved dose of Egami that was our post marketing commitment.

We announced those results. So, over seven days, there was a definite indication, proof of a trend of the similar efficacy, whether the patients are getting one or seven doses. We are hoping to extend similar findings, so we are planning, we are evaluating these similar findings in the at home setting with the currently studied dose at home.

Wimal, Unknown, BioXcel Therapeutics: And I will add to that, that we are not only studying these patients, like patients are not taking drug alone, so there is a patient population that is taking drug alone, and then there are certain population in the range of twenty percent, twenty five percent who have their diets, their caregiver. So FDA wanted to see that these patients in real life may be living alone or they are living with a caregiver, so we’ll be presenting the data from both cohorts.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So moving on to the FDA environment. Every day, if you pick up like a trade rag, you see something or the other is going on at the agency. There’s lots of uncertainty. So within that context, has the composition of the team that you’ve been interacting with at the FDA stayed largely the same? Are they

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: It’s just stayed largely the same. Yes, we haven’t seen any changes.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Okay. So this is a safety trial primarily. What are the key risks that you see for the FDA to approve this product or not for in home use?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Right now, I think we have a pretty good understanding with the FDA what’s needed. We will, as I said, we will confirm that understanding on August 20. And we don’t really foresee any big risk, but we will know when the data come out, what the adverse event profile looks like.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So we have data coming in August. What’s the quickest you can submit a supplemental new drug application?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Well, it’s an sNDA, so it’s supplemental, so it’s not as involved as the NDA. I don’t think I want to really commit to any number of months, and we will give guidance once we have the top line data and look at the data and then we will decide and give guidance how long it’s going to So

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: this is pretty important. We’ve seen the commercial deck that you put out, I think it was a couple of days ago. In that context, this seems like a really pivotal moment for the company in terms of trials, not just a pivotal program, but for the company. How are you thinking about your quality and level of strategic interactions with partners in industry after these data sets come out?

Wimal, Unknown, BioXcel Therapeutics: As we all know, the neuropsychiatric area, the assets which are derisked are attractive. And one of the things we learned that the bigger potential for this product is in the home setting, in a retail setting. That interest starts growing as we are coming close to the data readout.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So on the market opportunity, in your slide deck, you have some nice data points for us. One is that I think you mentioned that you could support a pricing of up to $1,400 per prescription. How many films would that entail?

Wimal, Unknown, BioXcel Therapeutics: That’s a great question. We’ve been thinking that depending on number of episodes and how many film we put it in a package and price it, and after data readout, we will go and talk to the peers and validate our assumptions. Currently, has a package of 10, where 10 films are packaged. And we will come up with a packaging for the home setting that makes sense. What we learn in terms of what the real world frequency of agitation looks like.

And you’ve also said, I think, share of sixteen percent in agitation and schizophrenia and bipolar disorder. I guess, would you go on a limb and say what kind of peak sales you might expect at what point? I think that’s the matrix we have given, what we collected the data in our market research. We’ll continue to refine this research. And then after the data readout, we will be in a position to say what the peak sales will look like for this product.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Also going back to the commercial deck, it’s interesting that you use the term prodromal for agitation. You see some people might want to take this film when they think an episode is coming. What I guess if you are allowed to take this product at in home, would prodromal be a part of the label? Or how are you thinking about that? Or is it just prescription and then it’s up to the patient to use the product?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Yeah. It’s going to be a prescription. It’s not going to be part of the label because it has not been studied that way, but it should be noted that the way it has been studied in the protocol, it says that you should take this when you feel that you need some treatment for agitation. So that can mean a lot of things, and we’ll see how FDA interprets that and how they put it in the label. But we’ll see what’s going to happen.

We know that as we already put out publicly, a majority of the patients took the product when appropriate when they were having maybe at least moderate agitation, but we’ll see how the data pans out overall and how FDA interprets it. I think that’s going to be interesting. But it’s definitely true, a number of patients experience some symptoms that is telling them that agitation is coming, and as you could see I think in the deck that we showed, it’s about twenty two percent in our market research. However, if you look at the European survey, it goes up to over seventy percent. So that could be helpful for patients.

Wimal, Unknown, BioXcel Therapeutics: This is a similar situation like in migraine, Sumant, where you have some aura and you start feeling. Says agitation is this state which can escalate very fast. So patient who get agitation, they start feeling this inattention, and they know from their prior experience that if they don’t do something about it, this will escalate. And agitation goes from what you said, prodromal stage to mild and mild if it is not controlled to moderate and severe, And that’s part of the reason certain percentage of patients end up in the institutional setting. So having a treatment at the home and with this study, we’re demonstrating a lot of episodes, like two thousand two hundred.

It’s going to be probably higher by the time we have all the database logged and cleaned up. So we will have plenty of data to show that these patients, when they take it and how they feel and what benefit they see from the drug, and what the safety profile looks like, which is a primary endpoint in the trial.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So let’s fast forward to a time when this SND is approved. You brought up the migraine market. Having covered several of those companies. It’s very evident that, that market was very promotion sensitive or is very promotion sensitive. It requires your product may require some education.

How are you thinking commercialization? Are you confident you can do this yourselves? Or would you need a strategic partner in The US to take this product to the next level?

Wimal, Unknown, BioXcel Therapeutics: That’s a great question. Currently, our organization is designed to run the clinical trial and get a regulatory approval. We are a small organization. And we’ll continue to leverage our key capability of running the clinical trials and regulatory submission and getting approval. In terms of the commercialization, we are evaluating what our options are.

And it could be with a potential partner who already have an established footprint so that we can bring this medication to as larger or broader number of patients as we can. So that work is in progress, and we’ll provide an update on that. So if you look

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: at your product, it is a five zero five(two) product. It’s approved. It could target a potentially very large market. So when you are discussing this product with Strategix, how focused are they on the exclusivity from the FDA versus the intellectual property that the product has?

Wimal, Unknown, BioXcel Therapeutics: Currently, we have 13 patents in our Orange Book. So we have a very robust, we believe, intellectual property. And this has been developed over a period of time. And it’s a range of different types of intellectual property that we believe will allow us to maintain the market exclusivity until 02/1943. So this product has a very long patent life.

So I think having the intellectual property and being in the Orange Book is really helpful, and also in addition to that, it’s a film which is very specifically manufactured, And we are the one who first time manufactured the film where we did the micro deposition because drug is so potent on the film. So we have very unique characteristics in terms of the manufacturing as well as strong, we believe, intellectual property. So I’ll

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: move on now to agitation in Alzheimer’s. You have this product in development for acute agitation in Alzheimer’s as well, potentially large market. Just before we get to that, what percentage of your patients in serenity at home are above 65 years of age?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: So, we have a certain number of patients 65. Actually, our trial enrollment criteria, inclusion criteria included patients up to 75 years of age. The data are still being cleaned, so I would be uncomfortable giving you a precise percentage.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: But it would still contribute to a potential safety number eventually when you have to ask for approval in Alzheimer’s?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: Well, we would see how the FDA looks at it because they look at it as different indications. So we would see how they would look at it. I can’t tell you right now.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: All right. So if I remember correctly, you have breakthrough product therapy designation in Alzheimer’s. Given we’ve seen the FDA make some positive comments on wanting to get products through approval processes and other things like that very quickly relative to previous times, what’s the potential for this product to be filed on the basis of the one trial that you have so far? So

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: we have no really indication that that’s going to be the case. We are continuing to evaluate and we are continuing to monitor how this develops. And as we have breakthrough designation, obviously, we have opportunities to talk to the FDA on a more frequent basis. But for the time being, we are just assuming that what was true a year ago is still true today, which is that we will need a confirmatory second phase three trial.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Got it. So what will be your specific next steps on agitation in Alzheimer’s?

Dushan Kostich, Senior VP of Medical and Clinical Affairs, BioXcel Therapeutics: That would be so right now, we we have the protocol. We submitted protocol to the FDA, FDA made some minor comments, we incorporated those comments, so we have a protocol that they agreed on, ready to go. We are currently selecting a CRO, So finishing the selection of the CRO and triggering the trial will be the next steps. And once we submit the sNDA, we will have the internal resources that are currently devoted to the bipolar schizophrenia. We will have those internal resources freed and able to initiate the Alzheimer’s trial.

So it’s basically ready to go very soon.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: So we have a few seconds left, but I want to go back to a very important eight ks you filed recently. You also had your second quarter results out this morning. So in that eight ks, I’m just confirming, is it fair to assume that you now have no more financial covenants left as of the August 15 deadline?

Wimal, Unknown, BioXcel Therapeutics: That’s correct.

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: Okay. And could you remind us as to your latest cash and the share count?

Wimal, Unknown, BioXcel Therapeutics: I think we announced that we have this morning $18,000,000 of cash, and then we received additional proceeds through the ATM and some exercise of the warrants in the range of $15,000,000

Sumant Kulkarni, Senior Biotechnology Analyst, Canaccord Genuity: All right. Thanks. So I guess we’ll just wait for the data and look forward to your next steps. Thank you. Thanks Thank again for tuning into the webcast and for attending.

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