Good afternoon, everyone. I'm Sumanth Kulkarni, a Senior Biotechnology Analyst here at Canaccord Genuity. I'd like to thank all our clients for your continued 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 on the market already. It's IGALMI. It's for agitation. 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, Vimal, to give a small intro, and then we'll go right into Q&A.
Thank you, Sumanth. Good afternoon, everyone. Thanks for joining us today. As Sumanth mentioned, we have a very important catalyst upcoming this month. This is about our pivotal Phase 3 trial related to schizophrenia and bipolar agitation in the home setting. We already have our product approved in the in-care setting. It's called IGALMI, and two doses are approved, 120 mcg and 180 mcg. For the home setting, we got an alignment with the FDA to test 120 mcg, where we have already proven safety and efficacy in an in-care setting. 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 the in-care. Efficacy, we want to show the repeat dosing over repeat dosing. 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, there are no FDA-approved therapies for treatment of agitation for schizophrenia and bipolar in the home setting. As we continue to assess the commercial opportunity, we put out a deck yesterday that originally we mentioned there are 23 million episodes. 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 23 million episodes. That is coming from our trial where we have already collected the data for 2,200 episodes over a 12-week period, and it's a 200-patient trial. That's one focus clearly in a very near term. In addition, we are preparing for a SNDA package, what is required to file the SNDA.
We have an upcoming meeting with the FDA again this month. We are excited about that, what is the package requirement to file an SNDA. In addition, for our Alzheimer's program, where we already have one positive Phase III trial, we are evaluating currently options with CROs to initiate that trial. We already have alignment with the FDA to initiate a Phase III trial there, and it will be a second confirmatory Phase III trial. Current focus near term is on schizophrenia and bipolar in the home setting.
Thanks, Vimal. We also have a Senior VP of Medical and Clinical Affairs here, Dusan Kostic. He's kind of the guy in the hot seat right now, given all the stuff that you have coming up with the FDA. 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?
The best competitor 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.
Got it. What specific metrics do you expect to report when you have these data out?
Yeah, as Vimal mentioned, the primary objective of the study is safety. In terms of safety, the primary endpoint is the incidence of adverse events, particularly drug-related adverse events. We are particularly paying attention to 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 also some of the adverse effects that we have seen in the clinic, including nausea and abdominal discomfort at lower rates. Adverse events are really the primary objective and the primary endpoint. Again, as Vimal mentioned, we are evaluating the repeat dose efficacy over 12 weeks. There are exploratory endpoints measuring patient-reported outcomes, so patient and caregiver-reported CGIs, global clinical indicators. Those are exploratory endpoints. Some of that will be included in the top-line data as well.
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?
That's an interesting question because it's true. The placebo effect, really, when you look across the psychiatric trials, 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. The importance of the placebo effect is somewhat lower than what you would expect in a regular pivotal Phase III 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. We'll see when the data come out.
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 the real-world experience with the number of agitation episodes over that time frame?
Right. 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. We think it's very representative.
OK. Given these patients are at home, they're administering the film themselves. This is as close to real-world as you can get. Is there any difference because these patients happen to be in a trial that they'll be somewhat more compliant or anything like that we should be thinking about?
Whether they're more compliant because they're in the trial, that has generally not been so much a case in psychiatric trials. We don't really know. They are basically choosing when to take it themselves. This is not like a chronic therapy where they are supposed to take it every day. They are taking this product when they feel they need it. From that perspective, I think it's really like a real-world experience.
I'd like to add what Dusan said, that we are seeing a vast majority of patients completing the trial over a 12-week period. That's an indicator that there is an unmet need and they are getting some benefit from the trial.
Got it. Let's move on now to the interactions with the FDA. You have a meeting coming up on the 20th of this month. What do you specifically expect to discuss at that meeting? Is it fair to assume that you have this data set coming, and because you have the meeting on the 20th, that we'll see the data before then?
I don't think 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, is mainly around the format and the content of the SNDA application. It's pretty operational from that standpoint. 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 the FDA last year in April, that that is our correct understanding. We are planning to confirm that. The main purpose of the meeting is for the format and the content of the SNDA.
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?
The key thing is the primary objective is the safety. They really need to see the safety of this study, of the SERENITY At-Home trial. As we said, the primary objective is safety. 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 12 weeks. We had already shown the trends of repeat-dose efficacy over seven days in a controlled setting with the approved dose of IGALMI. That was our post-marketing commitment. We announced those results. 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.
We are evaluating these similar findings in the at-home setting with the currently studied dose at home. I will add to that that we are not only studying these patients. Patients are not taking drugs alone. There is a patient population that is taking drugs alone. There are certain populations in the range of 20% - 25% who have their diets, their caregiver. FDA wanted to see that these patients in real life may be living alone or they are living with a caregiver. We will be presenting the data from both cohorts.
Right. 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. Within that context, has the composition of the team that you've been interacting with at the FDA stayed largely the same, or are there any?
It has stayed largely the same. We haven't seen any changes.
OK. 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?
Right now, I think we have a pretty good understanding with the FDA what's needed. We will, as I said, confirm that understanding on August 20. We don't really foresee any big risk. We will know when the data come out, what the adverse event profile looks like.
We have data coming in August. What's the quickest you can submit a supplemental new drug application?
It's an SNDA. 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. 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 take.
This is pretty important. We've seen the commercial deck that you put out, I think, 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?
As we all know, the neuropsychiatric area, the assets which are de-risked are, like, you know, attractive. One of the things we learned is 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.
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?
That's a great question. We've been thinking that depending on the number of episodes and how many films we put in a package and price it. After data readout, we will go and talk to the payers and validate our assumptions. Currently, IGALMI has a package of 10, where 10 films are packaged. We will come up with a packaging for the home setting that makes sense with what we learn in terms of what the real-world frequency of agitation looks like.
You've also said, I think, peak share of 16% in agitation in schizophrenia and bipolar disorder. I guess, would you go out 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. After the data readout, we will be in a position to say what the peak sales will look like for this product.
Also, going back to the commercial deck, it's interesting that you use the term prodromal for agitation, right? You see some people might want to take this film when they think an episode is coming. I guess if you are allowed to take this product in-home, would prodromal be a part of the label? How are you thinking about that? Is it just prescription, and then it's up to the patient to use the product?
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. 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. That can mean a lot of things. We'll see how FDA interprets that and how they put it in the label. 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. We'll see how the data pans out overall and how FDA interprets it. I think that's going to be interesting. It's definitely true. A number of patients experience some symptoms that are telling them that agitation is coming.
As you could see, I think in the deck that we showed, it's about 22% in our market research. However, if you look at the European survey, it goes up to over 70%. That could be helpful for patients.
Right.
This is a similar situation like a migraine, Sumanth, where you have some aura and you start feeling. This agitation is a state which can escalate very fast. Patients who get agitation start feeling this inner tension, and they know from their prior experience that if they don't do something about it, this will escalate. Agitation goes from what you said, prodromal stage, to mild, and if it is not controlled, to moderate and severe. That's part of the reason a certain % of patients end up in the institutional setting. Having a treatment at the home and with this study, we're demonstrating a lot of episodes, like, you know, 2,200. It's going to be probably higher by the time we have all the database logged and cleaned up. 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.
OK. Let's fast forward to a time when this SNDA 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. Your product may require some education. How are you thinking about commercialization? Are you confident you can do this yourselves? Would you need a strategic partner in the U.S. to take this product to the next level?
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. It could be with a potential partner who already has an established footprint so that we can bring this medication to as large or a broader number of patients as we can. That work is in progress, and we'll provide an update on that.
Right. If you look at your product, it is a 505(b)(2) product. It's approved. It could target a potentially very large market. 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?
Currently, we have 13 patents in our Orange Book. We have a very robust, we believe, intellectual property. This has been developed over a period of time. It's a range of different types of intellectual property that we believe will allow us to maintain the market exclusivity until 2043. This product has a very long patent life. I think having the intellectual property and being in the Orange Book is really helpful. In addition to that, it's a film which is very specifically manufactured. We are the ones who first time manufactured the film where we did the microdeposition because the drug is so potent on the film. We have very unique characteristics in terms of manufacturing as well as strong, we believe, intellectual property.
I'll move on now to agitation and Alzheimer's. You have this product in development for acute agitation and Alzheimer's as well, potentially large market. Just before we get to that, what % of your patients in the SERENITY At-Home trial are above 65 years of age?
We have a certain number of patients over 65. Actually, our trial enrollment criteria, inclusion criteria, included patients up to 75 years of age. The data is still being cleaned. I would be uncomfortable giving you a precise %.
It would still contribute to a potential safety number eventually when you have to ask for approval in Alzheimer's.
We would see how the FDA looks at it because they look at it as different indications. We would see how they would look at it. I can't tell you right now.
All right. 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?
We have no real indication that that's going to be the case. We are continuing to evaluate, and we are continuing to monitor how this develops. As we have breakthrough designation, obviously, we have opportunities to talk to the FDA on a more frequent basis. 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 III trial.
What will be your specific next steps on agitation and Alzheimer's?
Right now, we have the protocol. We submitted the protocol to the FDA. The FDA made some minor comments. We incorporated those comments. We have a protocol that they agreed on, ready to go. We are currently selecting a CRO. Finishing the selection of the CRO and triggering the trial will be the next steps. 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. It's basically ready to go very soon.
We have a few seconds left, but I want to go back to a very important 8-K you filed recently. You also had your second quarter results out this morning. In that 8-K, I'm just confirming, is it fair to assume that you now have no more financial covenants left as of the August 15 deadline?
That's correct.
OK. Could you remind us of your latest cash and the share count?
I think we announced that we have this morning $18 million of cash. We received additional proceeds through the ATM and some exercise of the warrants in the range of $15 million.
Got it. Thanks. I guess we just wait for the data and look forward to your next steps. Thank you.
Thank you.
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