Acadia Pharmaceuticals Inc. · NASDAQ:ACAD
Acadia Pharmaceuticals — Q4 2022 Earnings Presentation
30 slides from Acadia Pharmaceuticals's earnings presentation, Feb 27, 2023
Slide 1: Fourth Quarter and Full Year 2022 Earnings Call
Fourth Quarter and Full Year 2022 Earnings Call February 27, 2023
Slide 2: 4Q and Full Year 2022 Earnings Call Agenda
4Q and Full Year 2022 Earnings Call Agenda Introduction Mark Johnson | Vice President, Investor Relations CEO Opening Remarks Steve Davis | Chief Executive Officer Financial Update Mark Schneyer | Chief Financial Officer NUPLAZID Update Brendan Teehan | Chief Operating Officer, Head of Commercial Ka
Slide 3: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward-looking statements. These statements relate to future events and involve known and unknown risks, uncertainties and other factors which may cause our actual results, performance or achievements to be materially different from any future r
Slide 4: Opening Remarks
Opening Remarks 4
Slide 5: Four Strategic Priorities for Acadia
Four Strategic Priorities for Acadia NUPLAZID for Parkinson’s Trofinetide for Negative Symptoms Alzheimer’s Disease Disease Psychosis Rett Syndrome of Schizophrenia Psychosis • $517.2M net sales in FY22 • PDUFA Date: • Positive pivotal results: • ACP-204 Phase 1 program 3/12/2023 ADVANCE-1 ongoing •
Slide 6: NUPLAZID Franchise Performance
2021 Full-Year Highlights NUPLAZID® Franchise Performance Net Sales (in millions) Highlights: $500 • PDP franchise cash flow positive since 2019 • $517.2M net sales in FY22; 7% YoY growth $400 • Overall demand relatively steady YoY • LTC channel growth improved; up 5% YoY $300 • Office-based channel
Slide 7: Trofinetide for the Treatment of Rett Syndrome
Trofinetide for the Treatment of Rett Syndrome PDUFA: March 12, 2023 Promise of Trofinetide ▪ No FDA approved therapies specifically for Rett ▪ A rare and severe neurodevelopmental disorder ▪ Positive Phase 3 Lavender study results achieved on co-primary endpoints and key secondary endpoint ▪ Improv
Slide 8: Next Wave of Breakthroughs in CNS
Next Wave of Breakthroughs in CNS Negative Symptoms of Schizophrenia • Positive ADVANCE-1 study complete • ADVANCE-2 study expect to complete enrollment mid-year with TLR in early 2024 Alzheimer’s Disease Psychosis • ACP-204 Phase 1 program ongoing • Plan to evaluate in Phase 2 trials in patients wi
Slide 9: Finance Update
Finance Update
Slide 10: 4Q22 and FY22 Financial Highlights
4Q22 and FY22 Financial Highlights Millions, Except EPS 4Q22 4Q21 YoY FY22 FY21 YoY (GAAP) (GAAP) Change (GAAP) (GAAP) Change Total Revenue $136.5 $130.8 4% $517.2 $484.1 7% R&D $75.7 $67.1 13% $361.61 $239.4 51% SG&A $104.4 $105.8 -1% $369.1 $396.0 -7% Net Income (Loss) ($41.7) ($43.1) ($216.0) ($1
Slide 11: FY23 Financial Guidance
FY23 Financial Guidance FY23 Guidance Commentary • Midpoint of the range reflects ~1% growth volume YoY and includes ~2.5% net price growth YoY NUPLAZID® Net Sales $520 to $550M • Parkinson’s disease psychosis (PDP) guidance only • No sales guidance for Trofinetide • Increased due to IRA inflation c
Slide 12: NUPLAZID for the Treatment of PDP
NUPLAZID® for the Treatment of PDP
Slide 13: New Real-World Evidence
New Real-World Evidence Safety Publications: Retrospective Analyses 1,2: COMPARED TO ATYPICAL ANTIPSYCHOTICS*, PATIENTS TREATED WITH NUPLAZID HAD LOWER ALL - CAUSE MORTALITY M OSHOLDER ET AL . P UBLICATION (PD PATIENTS ) L AYTON ET AL . P UBLICATION (PDP PATIENTS ) Overall analysis 360 days: 23% low
Slide 14: Trofinetide for Rett Syndrome
Trofinetide for Rett Syndrome
Slide 15: Rett Syndrome
Rett Syndrome HIGH UNMET NEED No FDA-approved drug for the treatment of Rett syndrome ESTIMATED 6,000 - 9,000 PATIENTS IN THE U.S.1 Debilitating Symptoms2: • Fine and gross motor impairment • Loss of verbal and nonverbal communication • Hand stereotypies • Seizures • G.I. symptoms, including severe
Slide 16: Phase 3 Lavender Study Design
Phase 3 Lavender Study Design Pivotal, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Double-Blind Treatment Period (12 weeks) Co-primary 187 young efficacy endpoints females Trofinetide* (5–20 years) 1. RSBQ with Rett 2. CGI-I Placebo Syndrome Key secondary efficacy endpoint Pre-t
Slide 17: Positive Phase 3 Lavender Study Results
Positive Phase 3 Lavender Study Results Rett Syndrome Behavioural Questionnaire (RSBQ) Clinical Global Impression – Improvement (CGI-I) Change from Baseline CGI-I Score Change from Baseline Placebo Trofinetide* Week 12 Score Placebo Trofinetide* Mean -1.7 -5.1 CGI-I 3.8 3.5 p-value 0.0175 p-value 0.
Slide 18: Lilac: Open-Label Extension Study
Lilac: Open-Label Extension Study Open-Label Treatment Period (40 weeks) Primary objective to assess safety 154 young females from Also includes the Lavender Trofinetide* same efficacy Study endpoints as Lavender (e.g. RSBQ and CGI-I) Pre-treatment End of baseline Treatment Patients may continue to
Slide 19: Lilac Top-Line Results: Efficacy Assessments (RSBQ)
Lilac Top-Line Results: Efficacy Assessments RSBQ LAVENDER LILAC (12 weeks) (40 weeks) Placebo-controlled Open-label extension RSBQ mean RSBQ mean change Dose Group Dose Group change (12 wks) from Lavender baseline to end of Lilac (52 wks) Baseline Trofinetide -5.1 Trofinetide -7.3 RSBQ scores impro
Slide 20: Lilac Top-Line Results: Efficacy Assessments (CGI-I)
Lilac Top-Line Results: Efficacy Assessments CGI-I LAVENDER LILAC (12 weeks) (40 weeks) Placebo-controlled Open-label extension CGI-I score CGI-I score New Baseline for Lilac Dose Group Dose Group Lavender Baseline (at 12 wks) (at 40 wks) Trofinetide 3.5 Trofinetide 3.1 CGI-I improvements: • Trofine
Slide 21: Lilac Top-Line Results: Safety and Tolerability
Lilac Top-Line Results: Safety and Tolerability Adverse Event (AE) Summary Discontinuations Adverse Events >10% observed: • Discontinuations due to AE of diarrhea was 21% • Diarrhea (74.7%); • 96% were mild-to-moderate • Overall 46% of patients discontinued • Vomiting (28.6%); • 100% were mild-to-mo
Slide 22: Key Launch Initiatives for Trofinetide
Key Launch Initiatives for Trofinetide Drive Disease State Awareness and Education 1 • Understand Rett syndrome and the unmet needs of core symptoms, such as hand wringing, lack of purposeful eye gaze and communication Hire Rare Disease Commercial Team and Patient Identification 2 • Hiring seasoned
Slide 23: R&D Update
R&D Update
Slide 24: Addressing Significant Unmet Needs in CNS
Addressing Significant Unmet Needs in CNS Program Indication Preclinical Phase 1 Phase 2 Phase 3 Registration Marketed Parkinson’s NUPLAZID® Disease (pimavanserin)1 Psychosis Trofinetide2 Rett Syndrome Negative Pimavanserin Symptoms of Schizophrenia Alzheimer’s ACP-204 Disease Psychosis SYNGAP1; ASO
Slide 25: Negative Symptoms of Schizophrenia
Negative Symptoms of Schizophrenia HIGH UNMET NEED No FDA-approved treatment for the negative symptoms of schizophrenia patients receiving treatment in the U.S. >700K have persistent negative symptoms Negative symptoms include apathy, lack of emotion, social withdrawal, restricted speech, and blunte
Slide 26: Clinical Development Program for the Negative Symptoms of Schizophrenia
Clinical Development Program for the Negative Symptoms of Schizophrenia 34 MG Dose1,2 ADVANCE-1 Results Primary Endpoint (Overall – all 3 doses tested) Improvement in NSA-16 vs. placebo at 26 weeks At Week 26: Two-sided p-value = p=0.043 p=0.0065 (unadjusted) p=0.031 (unadjusted) Patients on 34 mg v
Slide 27: Investing in New Opportunities in Alzheimer's Disease Psychosis: ACP-204
Investing in New Opportunities in Alzheimer’s Disease Psychosis: ACP-204 Program Objectives • Leverage 5HT-2A benefits and favorable safety/tolerability profile • Reduce risk of QT prolongation • Optimize the efficacy profile • Potentially improve onset of action Clinical Development • Phase 1 progr
Slide 28: Closing Remarks
Closing Remarks 28
Slide 29: Upcoming Development Milestones
Upcoming Development Milestones Rett Syndrome Negative Symptoms Alzheimer’s Disease of Schizophrenia Psychosis Pimavanserin Trofinetide ACP-204 Phase 3 PDUFA Date ADVANCE-2 Study Phase 1 Results: 3/12/2023 Complete Enrollment: 1H23 Mid-2023 Initiate ADP Study: TLR: 1H24 2023 Profitable PDP franchise
Slide 30: Q&A Session
Q&A Session
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