BioXcel Therapeutics, Inc.

Good morning and welcome to the BioExcel Therapeutics first quarter 2022 financial results conference call. At this time, all participants are in a listen-only mode. If during the conference you require operator assistance, please press star zero on your telephone keypad. After the presentation, there will be a question and answer session. If you would like to register a question, you may press star one on your telephone keypad. Just to remind everyone, certain matters discussed in today's conference call and or answers that may be given to questions asked are forward-looking statements subject to risks and uncertainties related to future events and or the future financial performance of the company. Actual results could differ materially from those anticipated in these forward-looking statements. Risk factors that may affect results are detailed in the company's most recent public filings with the US Securities and Exchange Commission including by its annual report on Form 10-K for the year ended December 31st, 2021, which can be found at www.bioexceltherapeutics.com or on www.sec.gov, which will be updated in its quarterly report on Form 10-Q for the quarter ended March 31st, 2022. As a reminder, today's conference is being recorded. Joining us on today's call are Dr. Vilma Mehta, Chief Executive Officer, Richard Steinhardt, Chief Financial Officer, Matt Wiley, Chief Commercial Officer, Dr. Rob Reisinger, Chief Medical Officer of Neuroscience, Dr. Frank Yaka, Chief Scientific Officer, and Dr. Vince O'Neill, Chief Medical Officer of Oncology. It is now my pleasure to turn the call over to Dr. Mehta, the CEO of BioXcel Therapeutics. Please go ahead.

Thank you, operator. Welcome, everyone, and thank you for joining our call today to discuss BioXcel's financial performance and business highlights for the first quarter of 2022. As you know, Bioexcel Therapeutics is a biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immunology. It has been an already transformational year for the company, highlighted by our first FDA approval of Egalme, our orally dissolving sublingual film approved last month for the acute treatment of mild, moderate, or severe agitation associated with schizophrenia or bipolar I or II disorders in adults. We are gearing up for commercial launch to deliver this new treatment option to patients and caregivers. Our team has made tremendous progress preparing for the deployment of our U.S. national sales force and have finalized pricing for broad market access. Our chief commercial officer, Matt Wiley, will discuss key commercial activities in more detail shortly. Beyond offering an important treatment option to patients living with schizophrenia or bipolar disorder-related agitation, EGALME's FDA approval validates BioXcel's AI-enabled drug discovery and development platform. which progressed from first in human trials to approval in just under 3.5 years. This approval also lays a strong foundation for the continued evaluation of BXL501 for the treatment of agitation associated with other neuropsychiatric disorders, including Alzheimer's and an adjunctive treatment with major depressive disorder, or MDD. As such, we continue to make progress with our three-pillar portfolio expansion strategy for our 501 franchise, which includes indication expansion, extending our geographic reach, and growing the medical settings where 501 is offered. First, as part of our indication expansion strategy, we view 501 as a pipeline within a product. Our Tranquility program for the acute treatment of agitation in patients with Alzheimer's disease is advancing well. With first patient dosing in our Tranquility 2 trial announced last week, we have designed an innovative strategy to rapidly enroll patients with top-line data expected in the fourth quarter of 2022 or early next year. This, along with Tranquility 3 patient enrollment, initiating in the second half of the year, is helping position the franchise for our supplemental NDA, SNDA. We believe 501 may provide an effective treatment option for Alzheimer's disease-related agitation, an indication for which, despite an estimated 100 million episodes per year in the U.S., there are no FDA-approved therapies. We have positive, robust efficacy, safety, and tolerability data in 40 and 60 microgram dosing regimens and have received breakthrough therapy designation from the FDA, giving us high confidence in our pivotal tranquility program. Further, in regards to our indication expansion strategy, we have advanced our 501 clinical program for potential adjunctive treatment of major depressive disorders and have initiated a phase one multiple ascending dose clinical trial. With approximately 27 million cases a year, MDD remains the most common type of depression in the U.S. Current treatment limitations include slow onset of infection and incomplete responses. And with over 300 million antidepressant prescriptions filled annually, we believe that our own clinical data suggests 501 could be an effective treatment option for these patients. Second, with regard to our geographical expansion strategy, we are well poised to capture market opportunity outside of the U.S. We plan to submit a marketing authorization application to the European Medicine Agency for BXEL 501 for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults in the second quarter of this year. We are also exploring market entry strategies in Japan, which is expected to have Fastest growing prevalence in Alzheimer's disease with an estimated 4.9 million cases by 2026. Our third and final strategic pillar is expanding the medical settings where 501 can be offered. This is our land and expand strategy, and we want 501 to ultimately be accessible to patients regardless of treatment setting. We continue to expand these opportunities with additional new indications, including our Tranquility and MDD programs. Beyond 501, we are strategically augmenting our agitation franchise, beginning with 502 for the chronic treatment of agitation related to dementia. Formulation and translation work is underway for 502, a potent selective antagonist for a GPCR target affecting serotonergic signaling in the cerebral cortex. Shifting to our immuno-oncology franchise, we recently announced the formation of the wholly owned subsidiary Oncosexcel Therapeutics. We expect this business structure allows for further strategic and financial flexibility for our promising Immune Oncology Business, while we continue to advance the clinical development of BXEL-701, our leading IO clinical candidate, which is an orally administered systemic activator of innate immunity being developed for the treatment of aggressive forms of prostate cancer and advanced solid tumors. Following positive phase 2 data for BXCL701 in combination with Ketruda, and now this past February, we continued our phase 2 trial in MCRPC patients with either small cell neuroendocrine carcinoma SCNC or adenocarcinoma phenotype. We expect enrollment to complete in our 28-patient SCNC cohort in the second half of this year. We have also enrolled the first patient in our adenocarcinoma randomized trial expansion evaluating BXCL701 monotherapy versus BXCL701 ketruda combination therapy. We will provide additional updates about Oncosexcel therapeutic subsidiary in the second half of this year. Lastly, in April, we announced a $260 million strategic financing with Oak Tree Capital and Qatar Investment Authority. Full execution with sub-financing will extend our cash runway into 2025 and support our commercial activities for EGALMI along with expansion of clinical development efforts for OB-XEL 501 and our additional neuroscience and immunology programs. With our strengthened cash position, we remain confident in our ability to continue executing on our strategic priorities. In summary, we are incredibly pleased with our progress across key clinical, regulatory, and commercial milestones and realizing our vision of becoming the leading AI-enabled neuroscience company. Now, I would like to turn the call over to Matt Wiley, who will give a commercial update. Matt?

Thanks, Vimal, and good morning, everyone. Today I'll focus on commercial launch readiness, including the deployment of our sales team and market access activities. However, I would first like to discuss our decision on Agalmi pricing. We have taken a measured and clear-eyed approach to determining the wholesale acquisition cost for Agalmi. conducting both qualitative and quantitative research to shape our decision. This process spanned over eight months and evaluated multiple treatment settings, payer types, and potential indications. We also considered the volume of patients subject to additional dosing in our clinical trials, which was approximately 15% across both dosage strengths. With this comprehensive evidence-based research, and all of our stakeholders in mind, we arrived at our final pricing decision for Agami with two clear objectives. First, we believe Agami is a disruptive and important entrant into the agitation market, and we want to ensure it is accessible to as many patients as possible. Second, we want to lay the groundwork to build a sustainable and profitable business. The research shows that the price point we are setting today provides a robust foundation for Agalmi with flexibility to adjust for future potential 501 indications, including Alzheimer's disease-related agitation and major depressive disorder across treatment settings. We are very pleased with this outcome, considering the rapidly evolving payer dynamics. Agalmi's wholesale acquisition cost will be $105 per film across dosage strengths. The 120-microgram and 180-microgram doses of Agalmi will be packaged into heat-sealed foil pouches of 10- and 30-count films per carton. Our market access team is already engaged with target GPOs, IDNs, and interested hospital systems to coordinate scientific exchange and establish Agalmi's value proposition. Agalmi's WAC price finalization will enable more substantive discussions and move us closer to contract negotiations with these stakeholders. This is also a critical milestone in navigating the hospital formulary process, which on average can take six to 12 months. As we have stated in previous calls, there are approximately 25 million bipolar and schizophrenia agitation episodes each year. Approximately two-thirds of this market opportunity presents in the hospital and institutional setting, which presents a focused way to build a commercial beachhead. We have identified and are targeting 1,700 institutions in the United States representing about 75% of the defined agitation market. This includes psychiatric centers, academic medical centers, and community hospitals. Our institutional sales team is undergoing training, which will be completed shortly. This is a highly talented team with an average of over 21 years of industry experience, 14 years in the hospital settings, and an average of eight product launches each. The pedigree of this team will allow us to access target hospital accounts swiftly and leverage stakeholder relationships to successfully navigate the formulary process. We expect that this team will start making initial demand impact in the second half of 2022 and will further amplify demand in 2023. We are excited to launch our field sales efforts in earnest and believe that we have built commercial conditions for considerable long-term market penetration and patient impact. Now, I'll turn the call over to Richard, who will give a financial update.