Acadia Pharmaceuticals Inc.

Overview

Acadia Pharmaceuticals is a biopharmaceutical company focused on neurological and rare disease communities. Its two commercial franchises are NUPLAZID (pimavanserin) for Parkinson’s disease psychosis (PDP) and DAYBUE (trofinetide) for Rett syndrome. The company maintains a pipeline of early- and late-stage programs across neurology and rare diseases and pursues product expansion, internal development, business development, and international commercialization.

Core franchises and current products

• NUPLAZID (pimavanserin)

  • Mechanism: selective serotonin inverse agonist/antagonist targeting the 5-HT2A receptor.
  • Indication: approved in the U.S. for treatment of Parkinson’s disease psychosis (PDP).
  • Other clinical evaluations: negative symptoms of schizophrenia and irritability in autism spectrum disorder (pediatric).
  • Intellectual property: U.S. and foreign patent portfolio, with Orange Book-listed patents and patent protection on formulations and methods of use through 2026–2038 with extensions.

• DAYBUE (trofinetide)

  • Mechanism: peptide-based therapy related to the IGF-1 pathway; targets core symptoms of Rett syndrome.
  • Approvals and regulatory status:
    • U.S.: FDA-approved March 2023; available since April 2023.
    • Canada: Health Canada approved October 2024.
    • Israel: Ministry of Health approved December 2025.
    • Europe: Marketing authorization application filed January 2025; CHMP issued a negative trend vote February 2026 with plans for potential re-examination.
    • Japan: Phase 3 planned to support JNDA submission; results expected late 2026/early 2027.
  • New formulation: DAYBUE STIX (oral solution powder) launched December 2025.
  • Market context: U.S. prevalent population estimated at 6,000–9,000 Rett patients; Europe estimated at 9,000–12,000.
  • IP and licensing: exclusive North American rights licensed from Neuren (2018); licensing expanded in 2023 to cover outside North America and NNZ-2591.
  • Post-approval/compliance: includes nonclinical carcinogenicity studies and REMS considerations as part of the regulatory framework.

Pipeline and late-stage programs

• Remlifanserin (ACP-204)

  • Indications: Alzheimer’s disease psychosis (ADP) and Lewy Body Dementia psychosis (LBDP).
  • Stage: Phase 2 (RADIANT for ADP; ILLUMERA for LBDP).
  • Timing: RADIANT enrollment expected to complete in H1 2026; topline results forecasted around Aug–Oct 2026.

• ACP-211

  • Indication: major depressive disorder (MDD), with exploration in treatment-resistant depression (TRD).
  • Stage: Phase 2 initiated Q4 2025.
  • Timing: topline results expected 2Q–3Q 2027.

• ACP-711

  • Indication: essential tremor (in collaboration with Saniona A/S).
  • Stage: Phase 2 planned for late 2026; collaboration includes milestone and royalty structure.

• ACP-271

  • Indications: tardive dyskinesia and Huntington’s disease.
  • Stage: Phase 1 planned for 2026.

• ACP-2591

  • Indications: cGP analogue for Rett syndrome and Fragile X syndrome.
  • Stage: Phase 1 development; global rights from Neuren.

• Antisense Oligonucleotide (ASO) program

  • Collaboration with Stoke Therapeutics focused on SYNGAP1 syndrome and discovery work for CNS genetic neurodevelopmental diseases.

Global presence, regulatory and commercial approach

• Headquarters: San Diego, California; significant operations in Princeton, New Jersey; European headquarters in Zug, Switzerland.
• Commercial model: uses a network of third-party distributors and pharmacies to market and sell products.
• Geographic focus: U.S. remains central for NUPLAZID and DAYBUE, with active expansion efforts in the EU, Japan, Canada, Israel, and other international markets.

Market context and patient population

• Parkinson’s disease psychosis (PDP) in the U.S.: approximately 130,000 patients treated annually with an atypical antipsychotic; about 20% of those patients are treated with NUPLAZID.
• Rett syndrome prevalence: U.S. estimated 6,000–9,000; Europe estimated 9,000–12,000.

Financial highlights

• Net product sales (NUPLAZID and DAYBUE): $1,071.5 million for 2025; $957.8 million for 2024.
• One-time/non-operating event: sale of a Rare Pediatric Disease Priority Review Voucher (PRV) in December 2024 for $150 million (before fees and expenses). Under the license agreement, Acadia paid Neuren one-third of the net proceeds.

Summary

Acadia Pharmaceuticals focuses on neurological and rare diseases, with NUPLAZID for PDP and DAYBUE for Rett syndrome as its primary commercial products. The company maintains a pipeline spanning early- and late-stage programs across multiple neurodegenerative and neurodevelopmental indications, pursues international regulatory and commercial expansion, and follows a four-capability strategy (Precision Medicine, Data Innovation, Globalization, Patient Empowerment) to drive growth. Revenue from its two core products was $1,071.5 million in 2025, with a one-time PRV sale occurring in 2024.