® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com August 2025 BXCL501 Commercial Opportunity in the At-Home Setting for Bipolar Disorders and Schizophrenia NASDAQ: BTAI |
The potential for BXCL501 in the at-home setting reflects significant unmet need and attractiveness to patients Anticipated Episodic Patient Choice (Future Market) (% of Episodes, Market research, n=240) 80% 20% BXCL501 Other • Our market research indicates approximately 3 episodes of agitation per month in the home setting – Previous estimate of 23M annual episodes was based on historic claims data (~1.2 episodes per month) – Due to the lack of approved treatment options, the claims data likely underestimate the true episode frequency – Consequently, the total addressable market (TAM) is significantly higher than previously reported • Based on the target product profile, HCPs indicated that they would treat ~16% of their patients with BXCL501 • HCPs believe that the most significant unmet need is the lack of effective and fast-acting treatments for the at-home setting • Patients are the primary stakeholder for the treatment of their agitation episodes – Patients feel that they lack control over their thoughts and actions during agitation episodes, which occur approximately three times per month on average – Patients want to treat the episode as soon as it begins and before it escalates in severity • Patients research suggests that they would use BXCL501 in 80% of the episodes experienced at-home • Pricing flexibility in the market may allow up to $1,400+ per prescription • Attractive market access factors The At-Home Market for BXCL501in Schizophrenia and Bipolar Disorders: Summary 2 |
A behavioral emergency with highly variable clinical definitions and diagnoses1-3 Agitation Characteristics Emotional signs and symptoms • Hyperresponsiveness • Racing thoughts • Emotional tension Agitation is characterized by1 Behavioral signs and symptoms • Inability to stay calm or still • Motor and verbal hyperactivity • Communication impairments 3 1. Martínez-Raga J, et al. Front Psychiatry. 2018;9(54). 2. Roppolo LP, et al. J Am Coll Emerg Physicians Open. 2020;1(5):898-907. 3. Zeller SL, Rhoades WP. Clin Ther. 2010;32(3):403-425. |
35% 53% 12% Mild Moderate Severe Schizophrenia (n=20) 35 Episodes per year (~3/month) Bipolar Disorder (n=60) 38 Episodes per year (~3/month) 1Source: InVibe Feb 2023 Q4: In the past month, about how many agitation episodes [IF PATIENT SHOW “have you” IF CG SHOW “has your loved one”] experienced? Q5. Of the [XX] agitation episodes you experienced in the past month, how many would you categorize as mild, moderate, or severe? 2Roberts et al. BMC Psychiatry (2018) 18:104 Severity Number of episodes/year Mild 22.4 Moderate 15.4 Moderate-intense 6.8 Severe 2.9 Total 47.5 (~4/month) Published Survey (N=583)2 BXCL501 Serenity at Home Pivotal Phase 3 Trial Data from more than 2,200 episodes collected 15% 49% 36% Mild Moderate Severe Primary Market Research (N=80)1 Episode Frequency in Patients Experiencing Agitation Agitation episodes expected to occur 3-4 times per month based on multiple data sources 4 |
Potentially reduced ER visits, hospitalizations, or first responder interventions At-Home Intervention: Early Intervention May Prevent Severity Escalation 5 PRE-AGITATION AGITATION INITIATION MILD MODERATE SEVERE OPTIMAL INTERVENTION BXCL501 CLINICAL DEVELOPMENT AT HOME SETTING 23M EPISODES1 1Number of schizophrenia/bipolar patients with agitation: Kwong, M et al., Presented at the Academy of Managed Care Pharmacy Nexus 2021, October 18-21, 2021; Number of episodes per patient: Symphony claims data 16M EPISODES HOSPITAL SETTING PRE-AGITATION AGITATION INITIATION MILD MODERATE SEVERE The market opportunity at home is expected to be much larger than 23 M annually originally estimated |
The available drugs either work too slowly or have unacceptable side effects.” – Psychiatrist 0% 10% 20% 30% 40% 50% 60% Key unmet needs in SCZ 16% 12% View lack of current effective and fast acting treatments to be the primary unmet need Are concerned with the side effects of current treatments Mention lack of access to effective treatments Other than the sedative effects of antipsychotics there really aren't any good options for managing agitation without just making the patient drowsy.” – Psychiatrist 0% 10% 20% 30% 40% Key unmet needs in BP 16% 15% View lack of current effective and fast acting treatments to be the primary unmet need Are concerned with the side effects of current treatments Mention poor compliance with medication as a key issue Market seeks effective, fast-acting, more tolerable treatment for agitation in bipolar and schizophrenia agitation for at-home use Agitation as an Unmet Need Source HCP Demand Study Oct 2022 Quantitative research n=90 38% 48% “ “ 6 |
BXCL501 Peak Market Share Based on Target at-home Product Profile Agitation associated with ~16% Bipolar Disorder & Schizophrenia Methodology: Current Management: Respondents provide information about their patients and the treatment of each indication, including: Perceived unmet need, Estimated patient insurance mix, Guidelines used in treatment, Involvement of patients' caregivers Current Allocation: Respondents estimate their current use of pharmaceutical treatments in the last 100 episodes of acute agitation for each indication. BXCL501 Evaluation: Respondents review the BXCL501 TPP for each indication and evaluate: Their perception of the profile, The patient types best suited for BXCL501,The likelihood of prescribing BXCL501 in the future Future Allocation: Respondents repeat the allocation exercise assuming BXCL501 is available (in low and high doses for AD) in the following scenarios: Independent of cost sharing, Preferred Tier, Non-preferred Tier Source: Certara Demand Study October 2022 Quantitative research n=90 Allocation exercise assumptions: • Percent of last 100 adult patients with breakthrough agitation seen the community setting who would receive BXCL501 • “Community setting” = patients seen in: Provider office, clinic, outpatient mental health center, assisted living facility and supportive housing • 10 films per prescription HCPs view lack of effective, tolerable, and fast acting treatments to be the primary unmet need; in addition, no products are currently approved in the at-home setting HCP Preference Estimate 7 |
Patients experiencing agitation indicate that they are not typically diagnosed, nor treated, for this symptom. This creates an opportunity and need for HCP education and market conditioning. Recognition and Treatment of Agitation 8 Q1. Have you ever been diagnosed with agitation specifically? This can be a related but separate diagnosis to other mental health conditions Q2. Have you ever been prescribed a treatment specifically for agitation episodes? Q3. What was the treatment you were prescribed for agitation episodes? Source: InVibe Feb 2023 41% 59% Yes No 35% 65% Yes No Agitation Diagnosis (% of Total PT/CGs, n=80) Prescribed a Treatment for Agitation (% of Total PT/CGs, n=80) |
[I]t feels like there's something inside of me telling me there's something wrong, and I can't sit still, and I feel like my body's about to jump out of my skin. It's just really annoying, and you get short-tempered because of it and angry and snappy at people. It's hard to describe because you can't get it to go away, and it's just there, and you're stuck with it, and there's nothing you can do to make it go away.” (Q1, R10, PT, SCZ) Overall Quality Descriptors Beyond their control Different from ordinary fear and anger. Feeling a lack of agency over their own thoughts and actions Likely to escalate Emotional experience of agitation likely makes it harder to make concrete, proactive decisions on when to seek help Patient Quote Difficult for patients to articulate; patients feel a sense of helplessness Patients Perspectives of At-Home Agitation 9 Question 1: Take a moment to describe what it is like to experience an episode of agitation associated with schizophrenia or bipolar disorder. How do you (or your loved one) feel during these times, both physically and emotionally? Source: InVibe July 2022 |
22% 78% Yes No 11% 89% Mild 39% 61% 28% 72% Advance Knowledge of Agitation Episodes (% of Episodes, n=240*) * These data are compiled from 3 episodes described by each patient (N=80 pts/cgs *3 = 240) Caregivers and patients were equally likely to have advance knowledge of an episode (22% for both groups) Patients experience a prodromal phenomenon with agitation incidents, which increases with episode severity. Patient Aura Source: InVibe Feb 2023 10 Moderate Severe |
Patients indicate that they want to manage the episode with BXCL501 either just before or at the first sign of escalation in the at-home setting. Episode Intervention Target I would love to be able to have it available when I knew an episode was coming. That it would help bite it in the butt, so to say. That would be such a benefit for me.” (VR4, R8, PT, BPD, age 57) Best Time To Take Igalmi (% PTs/CGs n=80) 53% 37% 7% 3% When an episode is coming When an episode begins When an episode is most severe Not sure “ Source: InVibe Feb 2023 11 |
Medications Used to Treat Agitation Episodes (Current Market) (% of Episodes, n=240) Anticipated Uptake for BXCL501 (Future Market) (% of Episodes, n=240) 80% 33% 17% 14% 13% 6% 4% 0% 20% 40% 60% 80% 100% BXCL501 Anti-anxiety Anti-depressants Mood stabilizers Anti-psychotic Other None 31% 24% 20% 15% 10% 24% 0% 20% 40% 60% 80% 100% Anti-anxiety Anti-psychotic anti-depressants Mood stabilizers Other None Source: InVibe Feb 2023 Based on a target product profile, patients say they would use BXCL501 for 80% of their episodes Patients Preference Estimate 12 |
$1,419.00 $1,110.88 $315.00 $0.00 $200.00 $400.00 $600.00 $800.00 $1,000.00 $1,200.00 $1,400.00 $1,600.00 Rexulti Auvelity BXCL501 for BP/SCHZ Agitation At-Home Pricing Landscape* Price per 30 days of Therapy Current and development agitation products (chronic) cost over $1400/month. These products have community psychiatry indications in Major Depressive Disorder as well, demonstrating pricing elasticity in psychiatric retail markets. Retail Agitation Markets & Pricing Considerations 13 BXCL501 retail market pricing opportunity |
Provisional Targeting Model 14 Among the highest deciles of Bipolar & Schizophrenia patient Dx volume, 70% are managed by psychiatrists, with 8.1k total HCP targets. 50-70 discrete field FTEs can cover these targets Sources: Clarivate Claims, FY2021; Definitive December 2022 BP/SCZ Patient Dx Volume (Decile >5) National Distribution of 8.1k HCP Targets by Decile Psychiatry 70% All Other Specialties 30% |
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