ACADIA Pharmaceuticals (ACAD) investor relations material

Strategic vision and business priorities

• Focused on building a leading neurological and rare disease company, leveraging a robust commercial franchise and expanding late- and early-stage pipelines, with a mission to deliver first- or best-in-class therapies for underserved patients.

• Business development targets late-stage assets in neurological and broader rare diseases, as well as expansion into endocrine, metabolic, nephrology, cardiovascular, and immunology areas, leveraging a strong balance sheet.

• AI-powered analytics and data innovation are being deployed to enhance commercial targeting, clinical trial monitoring, regulatory document development, and precision medicine.

• Core pillars include globalization, patient empowerment, and investment in precision medicine to accelerate R&D and commercial execution.

• Expansion into Europe and other global markets, with DAYBUE approvals and launches planned, including a new powder formulation (DAYBUE STIX).

Pipeline and R&D milestones

• Five new Phase 2 or Phase 3 study starts are anticipated by the end of 2027, with four molecules in the pipeline targeting large markets and a combined peak sales potential estimated at $11 billion.

• Advancing Remlifanserin (ACP-204) for Alzheimer's and Lewy body dementia psychosis, with phase II top-line results expected between August and October 2026 and ongoing Phase 2/3 trials.

• Initiated phase II studies for ACP-211 in major depressive disorder and ACP-204 in Lewy body dementia psychosis; ACP-211 targets Major Depressive Disorder with a ketamine-like mechanism but aims for minimal in-office monitoring.

• Phase III trofinetide trial underway in Japan, with results anticipated within a six-month window spanning into 2026.

• ACP-711 for essential tremor to begin phase II in Q4 2026; ACP-271 to start first-in-human studies in Q1 2026, with both addressing essential tremor, tardive dyskinesia, and Huntington's disease.

Commercial execution and growth drivers

• NUPLAZID remains the only FDA-approved treatment for Parkinson's disease psychosis, with a 25% market share, ~97,000 patients treated, and patent protection through 2038.

• Direct-to-consumer campaigns and a 30% increase in field force or customer-facing teams are expected to drive further NUPLAZID growth.

• DAYBUE is the first and only FDA-approved therapy for Rett syndrome, with strong persistency, over 2,000 patients treated since 2023, and a new oral powder formulation (STIX) approved in December 2025.

• DAYBUE STIX offers improved convenience and dietary compatibility, expected to drive new patient starts and re-engagement.

• Both brands to benefit from expanded sales teams and international launches, particularly in Europe and other global markets.