All right. Good morning, everybody. Nice to see all of you. Thank you so much for being here today. My name is Chris Schott. I'm an associate in the healthcare group at JP Morgan. Today, I have the pleasure of introducing Vimal Mehta, Co-founder and CEO of BioXcel Therapeutics. Vimal is planning on doing about a 20-minute presentation, followed by Q&A, and he'll have a few members of his management team up here to help out with Q&A. Thank you very much.
Thank you, Chris. Good morning, everyone. I'm very pleased to be here to provide the update on BioXcel Therapeutics and the exciting year we have ahead. First of all, I like to thank everyone who is attending as well as JP Morgan for inviting us for this opportunity. BioXcel Therapeutics is a AI-driven, a company that's developing transformative medicines in neuroscience and immuno-oncology. Those are the two key focus areas, and underlying platform is our artificial intelligence platform. Before I begin, I like to remind everyone that I will be making forward-looking statements. Also another disclaimer which pertains to our recent approval of the product, IGALMI. What is really unique about BioXcel Therapeutics? We're building a completely new biopharma model and using a, I call it as a drug re-innovation approach.
We are taking advantage of all the information that's out available for a clinical candidate, whether it's stuck in the clinic for any reason or it went all the way to the market, but its full potential was not exploited. That's exactly what we are trying to do, and we want to bring transformative medicines to the patient by doing three things: optimizing the R&D, like, you know, efficiency, shortening the development time, and increasing the probability of success. Those are the three things we set up when we became a public company in March of 2018, and now we have a approved product which I'm going to talk about it. Our neuroscience franchise is focused on acute treatment of agitation. Agitation is a very large, huge unmet medical need. It's under-diagnosed and underserved.
There has been no innovation over several years or a decade where new therapy has not come in this area. We are very pleased that our first drug, IGALMI, got approved. It's for acute treatment of agitation resulting from schizophrenia, bipolar I or bipolar II. So three indications in that. We continue to expand the potential. It's a pipeline within a product. We have Alzheimer's-related agitation that will expand this to cover almost 139 million episodes that happen for these three indications we are focusing on. Our second product is in immuno-oncology. We announced today that we have positive phase II data. Due to the embargo, we couldn't describe the data in a quantitative manner. We'll be providing the full data at the ASCO GU. We have a couple of presentations scheduled for ASCO GU.
We have established OnkosXcel to maximize the value for our shareholders, considering now we were a AI company in 2018 and a drug discovery and development company. Now we have MSL, we have commercialization. We have full capability AI to commercialization. We want to focus going forward as a long-term value for our shareholders, being a leading neuroscience company, continue to utilize our AI-based platform. In short term, in 2023, we have multiple catalysts. We have four data catalysts. Two are pivotal trials. One is TRANQUILITY II, which is related to Alzheimer's-related agitation. SERENITY III, which is related to at-home use of this drug like BXCL501, because currently IGALMI is approved for under the medical supervision, so it will allow us to take that drug home. SERENITY III trial is focused on that.
It's an exciting year, 2023. Not only that, in 2022, we got the approval in April, and we launched the drug. Now we have about six months of experience, market experience in a market, nascent market where there are no commercial analogs, so we're building our own experience in agitation. Our disruption is part of our is in our DNA and whatever we do or what we are trying to build a new model for the biopharmaceutical company. When we became a public company in 2018, to our knowledge, we were the first AI company that was developing drugs in the neuroscience and immuno-oncology space. Today, like you know, from the first IND to approval, it was three and a half years, and to launch, it's four years.
We have been able to go based on our model, what we have set out the company, that we can use AI, we can use existing data, knowledge, and write a new chapter on the drugs or the failed pipeline. We have been able to bring a drug to the market. That's a gold standard. We have met the FDA gold standard that we can bring a drug to the market using our platform and our team. AI, I know there may be a lot of AI, like, you know, buzzword. What we use AI is a augmentive intelligence. We are augmenting our team.
We have integrated our AI platform, combined with our drug discovery, development, translation, and clinical team to bring these drugs to the marketplace in very high unmet need areas. I believe that we got the first oral film approved for this indication for agitation related to bipolar schizophrenia, agitation. Then we built the full commercial integrated team to launch this product. As you can imagine, all of this is happening in last four years. You can look at our record, how much money we have raised and how much we have spent. All of this has been done in a very capital efficient manner. I'm very pleased to share the story that where we are.
In addition, we did a strategic financing to continue to expand the market for our product, lead product BXCL501, as well as continue to do the commercialization of IGALMI. What is in front of us in terms of the opportunity? We have 139 million episodes that happen. Our WAC price is about $105 for the product. If you look at it's a $15 billion market opportunity that company is very well set up to capitalize on it. What allowed us to achieve all of this is our drug discovery and development engine. We start with AI or big data, identify multiple opportunities that could be very interesting and that meet the high unmet medical need. When we selected agitation, we did the analysis, and agitation was one of the biggest healthcare burden for the society.
There was no innovation, and patients and SGPs needed a drug that can help them. We chose that. Once we have identified our candidate, we come up with multiple opportunities. We select using preclinical and translational work. Once we have built confidence in that, we go to the clinic for a human proof of concept. That's our third part of the process. At that point in time, our investment is limited because first two part of the process, I talked to you from AI all the way to translational work, can be six-12 month process. Once we have built the confidence, we go to the clinic, conduct the clinical trial. Once we know the signal, we design a phase three pivotal trial. That's exactly what was done for approval of our current product.
If everything is good, we submit the NDA and get the approval. We have proven it once. Second time, now today we announced that 701, we have a human proof of concept. That's where we are turning cold tumors to hot. That's a very novel concept. That's completely new mechanism. We have a large safety database, 800 patient, and it's well-tolerated in combination with Keytruda. We have proven second time. We started with two drugs when we did the IPO, and both drugs are progressing forward. One is already approved and another one has a human proof of concept. This is just one snapshot to show you that how we have a global map of disease, state mechanism, underlying biology, drugs using the AI platform.
What literally AI allows us to do is crunch millions of publication, millions of data points all together, convert into a metadata relationship map, and allows you to look for unique insights. What does it mean? If something is already in a publication, human eye can catch it, humans can read one publication a day. Machines don't get tired. They continuously can read a lot more than what humans can read. That's exactly using this process, using this kind of information, we identified BXCL501, which was an agitation we selected. Our strategic focus is on stress-related symptoms, I'm just showing you one example, what we use to continue to build sustainable R&D pipeline. We have a BXCL502, we'll be bringing BXCL503 and BXCL504.
AI will allow us to build a sustainable R&D pipeline outside 501, even though 501 is a pipeline within a product. Here is a timeline map so that you can see how we got to the approval and launch. IND got approved in Q4 of 2018. We did our initial work, clinical work, like as I said, human proof of concept, and then registration trial within 18-month period. We had a positive data in two registration trials, SERENITY I and II. We started the program in Alzheimer's related agitation to see how broad is the potential of this drug. We got a Breakthrough Therapy designation from the FDA. We submitted a NDA in Q2 of 2021, drug got approved in April last year. Now it's in the marketplace.
We did the trade launch. At this point in time, I'm very pleased to say it's helping patient, it's helping healthcare providers. We're getting a lot of information, and I will be sharing some anecdotes from the marketplace today. What are we trying to solve in terms of the medical problem? Using all this technology, all our capability, we are solving the agitation market. Agitation is a huge healthcare burden. It's a problem for the patient, it's a problem for the healthcare provider, it's a problem for the family. If these patients are not treated, somebody gets hurt. This is one of the biggest reason for the workers comp. It's not the construction workers get hurt, it's the healthcare worker, they get hurt. It's a huge unmet need. Why do I have this slide? I just want you to make a comparison.
Depression in nineties'80s was not a big market until the new drugs like Prozac and all that came. The innovation happened. There were the tool available for the physicians to treat these patients. They became rapidly expanded and became big markets. Today, there are more than 300 prescriptions in the depression market. We think we have brought an innovation to the market by getting the IGALMI approved. We think that it's underserved and under-diagnosed. Any projections we do when we go back, look at the claims data, we do more market research, we learn from the field intelligence, market keep expanding, whatever our original assumptions were. We are learning. We are in a learning phase. We are establishing the market literally.
There are no commercial analogs for this because there has not been a product approved for over a decade in this space. We are very happy that IGALMI has been approved, and now it is in the marketplace. You can see our evolution as a company from AI to drug discovery, development, and a commercial organization as of today. The opportunity in front of us is really, really huge. It's almost 139 million episodes. They happen every day. They happen in hospitals, they happen in ALF, they happen in nursing homes, they happen at home. These patients, if they cannot be managed at home, they end up in the ALF or nursing home if they are elderly patient. If these are schizophrenia, bipolar patient, they end up in the emergency room.
It's a huge problem for the society, we truly believe, and that's why we are working very hard that we can make a societal impact. Like, you know, if we can bring a drug that can really help patients, that can help healthcare providers, as well as the family member. Currently, on this map of 139 million agitation episodes? IGALMI is already approved, it's already launched, and I'm going to talk more about it, and we are capturing that market of 16 million episodes. Expansion opportunity is at home for the schizophrenia and bipolar patient. There are about 23 million episodes that happen at home. If we can bring a drug for at-home setting, that may even reduce the burden for people not to go to the emergency room because they can treat their agitation at home.
We are expanding the market, and we are in alignment with the FDA, what we need to do to expand this label. SERENITY III trial was designed. It is on track, and we will be announcing the data from this trial in first half of 2023. In addition, almost 100 million episodes in Alzheimer's related agitation, there is no current approved therapy. Anything that is used to manage agitation has a black box warning, like antipsychotic, benzodiazepine. We have a completely novel mechanism, it, we believe target the causal mechanism of agitation. We are very excited about TRANQUILITY II data that is expected again in first half of 2023. We have two pivotal data readouts in first half of 2023. We have built a very depth in our pipeline.
As you can see, that 501 is a pipeline within a product, and then we have a BXCL502 that's targeting another market, which is chronic agitation. 501 is for more for acute agitation. You can see the impact within 20 minutes with our drug. In addition, we are developing some wearable device to predict agitation, so that this device plus our drug can be used to prevent when a patient get agitation in early stages. If you can get those signals, then you can give IGALMI when agitation has not escalated. We are developing that, and you will be hearing about it, updates in our R&D forums about our efforts in those areas. 701, we announced the data today, positive phase II data, so we are extremely excited about that.
Just to recap on our catalysts that are in front of us, TRANQUILITY II, SERENITY III, and MDD, which is we are using 501 as adjunctive treatment, as an accelerant for antidepressant. All three datasets will come in first half of 2023. In addition, we already talked about 701, that we will have full presentation of our data at the ASCO GU, and that's part of the reason we couldn't put more information in the press release. Few words on IGALMI commercialization. We have a very broad label for IGALMI to cover mild, moderate, and severe, so we're covering the whole spectrum, three different indications like schizophrenia, bipolar I or bipolar II. We believe IGALMI represents a game-changing potential because there is nothing in the marketplace. There's no competition. It's completely a novel mechanism, new innovation.
This is a snapshot where when we launched in 2022, we did a trade launch in July. We have a six month of experience under our belt. We started with 26 reps, and the goal was to learn and understand how the market will receive our product, and did some marketing efforts and everything else to build the confidence that this is. Market is real, IGALMI potential is real. Now we have expanded to full 70 people to cover the whole U.S. geographies. We have corporate account directors to unlock the market access. We are going with marketing efforts, enhanced marketing efforts to create the awareness of the product. We are introducing free trial program, so hospitals can get experience in using IGALMI because. Which is very impactful. We have seen our experience.
We have a $81 billion data lake or claims data that we are using for a precise targeting of the marketplace. In overall conclusion, we have a very good reception, market reception, positive from the STPs and the hospitals. Market dynamics are very favorable. Doctors, physicians, nurses, all of them, they get hurt if this patient is not treated, and that can result in a morale for the nurses. They're like, you know, treating these patients, that's a big issue if one of their colleagues get hurt or legal liabilities associated with that. Market drivers are very favorable for us. Market access is accelerating as we are progressing, and now we have a full coverage of the marketplace. We're covering all 1,700 hospitals as of December first.
We have full team in play. There is a very strong momentum for IGALMI building in 2023. What I like to do it is share with you our experience from one of the hospitals who was a early adopter. These were the early adopter, and how in one hospital IGALMI has progressed. That will give you a feel for that when we have market access, and as we're getting market access, what the momentum is building for IGALMI in the marketplace. We are targeting, just to remind everyone, 1,700 hospitals.
We decided to add IGALMI to our formulary at the institution because as a pharmacy director, we know that not one drug works perfectly for every patient. This gave us a new treatment option and a new formulation that we felt could empower our patients to be part of the treatment modality. The adoption rate has been growing pretty steadily at our institution. Originally, it was brought in for our psych practitioners. They were the original people to request it through formulary. Their use was fairly quick, and they liked the results that they saw when they were utilizing it, being able to assess the patient, being able to empower the patient to be part of the treatment options. That organically grew to our ED physicians because they were consulted to go take care of some agitated patients in the ED.
The ED physicians got to see it work, felt it was effective as well. They started asking questions of how they may be able to utilize that agent and get it stocked up in their automated dispensing machine, Pyxis machine on their unit. That has now led to our hospitalists evaluating this as well because they have seen it work in the ED. We feel the value proposition with IGALMI has been the ability to assess and treat our patients without completely making them very sedated. That has helped us to either be able to discharge a patient out of the ED, or if they are admitted to our psych unit, it allows our patients and our physicians to work through their treatment programs quicker and more effectively without having to deal with just pure agitation.
That was just a preview for you from one hospital who adopted the IGALMI, and things are picking up. The momentum is picking up. Now we have 1,700 hospitals. We're continuously getting market access, and we're seeing similar kind of a pattern in other hospitals also. We are extremely excited for IGALMI in 2023. Just few words on our Alzheimer's related agitation. As I said, there's no currently approved therapy. All agents have black box warning. We have a Breakthrough Therapy designation. Our strategy for expanding 501 has been expand the medical setting, like at home, expand the indication, which is Alzheimer's related agitation, and then expand the geographical expansion. Currently, our focus was U.S. We'll continue to explore what the opportunity is in U.S. and Japan through strategic partnerships.
That's a goal we have set up for ourselves that we'll continue to look into these three areas to expand the potential of our agitation franchise. Just to give you some feel, Alzheimer's agitation, why there are 100 million episodes. As the dementia progresses, the agitation episodes start changing. In the beginning, you may have a episodic situation where there are three episodes a month. That can change as your dementia changes. It could become three episodes a week. As you further progress in dementia, it can become a chronic situation where you have agitation literally every day. You can see on this slide, we have BXCL501 positioned for episodic as well as intermittent chronic. PRN use. As and when needed, this drug is given. You don't want to put too much burden on the patient.
They're already on 10-12 drugs. This drug basically helps them when they have that kind of a situation and calms them so that that situation can be managed. We are developing another mechanism. It's completely novel, BXCL502, for chronic agitation. We want to be leader in this space and we wanna make sure that we continue to do innovation in this space, including the wearable, which you will hear more in 2023. Currently, we are doubling our market entry strategy. Once we have the TRANQUILITY II readout, we will provide how we want to enter the market in the Alzheimer's related agitation. As you can understand, the lecanemab got approved last Friday. It will help with the cognition. We are targeting agitation.
One of the biggest reason for patient to go to the ALF and nursing home is the agitation. We are targeting a very huge problem. There are about 70% of the patients in Alzheimer's get agitation episodes. Overall, just to wrap up, our opportunity in front of us is 139 million episodes. IGALMI 16 at home setting with SERENITY will capture 23 million episodes, and with TRANQUILITY program, another 100 million. Overall, a very large opportunity for the company, $15 billion. Let me now turn to immuno-oncology. It never gets much attention, today we had a press release, like, you know, outlining that we saw a positive top-line data in cold tumor. Like, these are small cell neuroendocrine prostate cancer patients, and they are on platinum therapy.
They are really, really sick, and we have been able to show that 701 combined with Keytruda can make difference in these patients' life. You will hear more at the ASCO GU, the full data from our PIs. Today, I'm very pleased to see that this asset is progressing forward. What does it mean for the business, basically? OnkosXcel has been established, and we want to follow the same path we have done in neuroscience that have a strategy for efficient development path to bring the drugs in the oncology space. Our focus is hard-to-treat tumors, the cold tumors, convert cold tumors to hot. We think that's where the biggest unmet medical need is, and that's where you can help patients. We are targeting that.
Obviously, team is very experienced, both from oncology as well as like, you know, building business and creating value for the shareholders. We are well-positioned to deliver on the mission, to fulfill the mission of OnkosXcel. This is our lead product, BXCL701, which I talked about, that we did a press release today. What is really exciting about it's a novel mechanism. It's one of the most advanced oral innate immunity activator. We haven't spent $0.5 billion or $1 billion to prove this mechanism, probably one tenth of that, to show this mechanism can work. Because this asset was stuck in a phase III. Previous company has spent $125 million. It never went anywhere because there was no immunotherapy. It was combined with chemotherapy.
Once immunotherapy came, AI platform guided us, this is an area to focus, we chose this mechanism, DPP8 and DPP9 inhibition. OnkosXcel will continue to focus on this mechanism and on this biology. We have a clinical proof of concept. Safety and tolerability has been established. We all know in innate immunity space, there is a scarcity of asset, and we have the one of the most advanced oral agent. There are multiple M&A have happened in this space, and we are very excited to present full data set in small cell neuroendocrine cancer. Just to remind everybody, when we presented our interim data with 15 patients at Prostate Cancer Foundation, our response rate was 33%, mean duration response of nine months, and drug was well-tolerated and also stable disease. You will hear all of that for the full trial at the ASCO GU.
It's a monumental year for the company with four data readouts in two therapeutic areas and a launch of the product. A lot happening with the business, but we are very focused. These are our accomplishments for 2022. With a strong cash balance on our balance sheet and a strategy, and with the IGALMI picking up in the marketplace, we are in a very good position to execute on these milestones. With that, I would like to thank you everyone for joining us, and I will open this session for Q&A.
Well, Vimal, I've got a question, after today's announcement. Can you just speak a little bit more to what's next for the platform and, you know, what you're excited about in 2023?
Today's announcement was for our lead asset in oncology, and we were waiting for this data, and we are very pleased to see that it's making difference, converting cold tumors to hot. Now we have set up because data was looking when we looked at the interim data. We have set up OnkosXcel Therapeutics, and now we will be unleashing the value for our shareholders because currently we don't think that being valued because neuroscience business has moved so fast and every focus of everyone has been around our IGALMI and the expansion of our product. We are looking for strategic option. They include licensing, they include partnering, they include M&A or they include financing. We are looking for all those options, and we will be exploring those options and come back which option we adopt.
It's a very exciting phase for the company.
On IGALMI, what has allowed you to go from IND to approval and launch in only four years?
That was the reason we were able to do that fast were two reasons. One, agitation is such a under-diagnosed and unsolved market that FDA is very supportive in approving a drug in this space. Second, we were able to identify a mechanism that targets and treat the agitation despite any underlying neuropsychiatric condition using our AI platform. AI platform allowed us to identify an asset that has so much data and that were de-risked. By de-risking upfront and then designing a strategy to translate when we came up with the 501 concept. What is really unique about 501? It's a approved drug. It's a Precedex. It's called dexmedetomidine. It was sold as a sedative anesthetic by Pfizer and Hospira. It made a lot of money for them, but they never explored its full biology.
AI platform guided us that this could treat agitation. We are doing micro-dosing. We are using 10 to 20 times lower dosage, we are treating agitation. this drug is in a surgical unit, given only in a surgical unit. It's a IV. We converted that into a sublingual thin film that you put it under the tongue. It has a mucoadhesive, and it dissolves, and it is a very rapid onset of action. I will say new indication, micro-dosing and sublingual film, all three things got aligned. drug is behaves very well in every clinical trial that we have conducted it as a dose-dependent response. our trials from the human proof of concept to phase III trial, SERENITY I and II, were positive. Everybody who has looked at the data, they said you normally don't see this kind of a data in neuropsychiatric.
We were very pleased, and FDA looked at the data safety, and they were able to approve the drug. It's a combination of AI platform, performance of the drug, our team expertise to be able to move the drug so fast, and high unmet medical needs. All of that got aligned together to get this drug approved in 3.5 year and then launch.
Thank you. Are there any other questions from the audience here? If not, I've got one more. Maybe just taking a step back and looking at the overall business model, what would you say is differentiated with BioXcel relative maybe to some of your competitors?
Traditional drug discovery or development normally require 10-12 years, $100s of millions or billions to get a drug to the finish line, because you're trying to start with a very novel biology, and that is needed. It takes that long to collect the data that you can get a drug approved. What is different about business model is we're taking advantage of the existing knowledge that is there. In case of BXCL501, we took all the advantage of Precedex because it was in the marketplace for 20 years. We know a lot about it. It has been in the patient for that 20 years.
All of that gave us lot of information to figure out what indication to go in and then how to design the trial and what to expect in terms of the safety and tolerability, which is big deal, and how to achieve the efficacy that we will need to get the drug approval. I will say that I call it as a drug reinnovation, Rajan. It's a very innovative way of doing it, that you take advantage of all the information that is sitting there. Just imagine, 701 was another company that has this asset in the phase III, Point Therapeutics, and they had spent $125 million. It was a public company. They failed in their phase III trial, and this asset was sitting there. We purchased it.
We figured out using AI that it's an innate immunity activator, and it needs to go in this direction. We used AI to figure out which tumor type to go. It turned out to be right that like, you know, all the pieces got aligned. What I'm trying to say it is that clear competitive advantage is being able to utilize the existing data that exists. This is my belief that the billions of dollars of assets sitting from phase II to phase III, and if we can use the AI engine on it, we can create a lot of drugs and opportunities. This is just the tip of the iceberg, what we are showing. We needed to prove our model, and we are very excited today.
We have proven our model, and we'll continue to create new drugs using this engine and bring transformative medicines to patients both in neuroscience and immunology.
Great. Oh, here's a question.
Thanks for taking the question. For the Alzheimer's agitation, how should we think about the in-hospital setting usage versus maybe a broad usage in all settings? Thank you.
I will start, and then I will pass it on to my colleagues, Rob and Matt. Alzheimer's, we are conducting the trials in ALF. This is residential settings, basically, where these patients reside, and also in the nursing homes. When their dementia progresses, they cannot be managed in assisted living facility, it escalates in terms of the care center. We are proving to the FDA outside the hospital setting. This will be a retail prescription product. With that, I can pass it on to Rob and Matt, if you'd like to add more.
Sure. In terms of the current indication, for IGALMI in schizophrenia and bipolar disorder, they're often seen in the emergency room. Patients do have agitation episodes above the emergency room, if you will. They come to the emergency room agitated, and they may continue to be agitated, for example, if they're admitted to the inpatient unit. Similarly, patients with agitation, whether it be at home or assisted living or nursing home, demented patients with agitation are also brought to the emergency room. Whether they're sent directly back to where they came from or escalating, for example, to a greater level of care. You know, grandma at home gets agitated. You can't manage her sundowning. Often she's admitted because she was agitated and then go to an assisted living or a nursing home.
There's a sort of pattern if you follow the patient and what happens. Our trials are designed for TRANQUILITY I, II, III. Trials are designed to demonstrate efficacy in the assisted living and nursing home, knowing that there's mild to moderate to severe. You know, there's a range of dementia. We're demonstrating efficacy across that range. I'll let Matt speak to why we're doing it this way and how this is actually a strategic decision.
Well, thanks, Rob. What we've seen in previous research is that up to 20% of the AD patients with agitation wind up in the emergency department, which I think is going to be helpful for us commercially, because in many cases, their first introduction to IGALMI could be in the emergency setting, and then coming back out in the community builds a very nice commercial bridge, to the retail setting.
All right. Thank you, everybody. Vimal, I don't know if you have any closing comments, but we're close on time, so thank you very much.
Thank you very much for joining us today. If you have any questions, we are here all day for our one-on-one meeting, and we'll be very happy to address.