ACADIA Pharmaceuticals (ACAD) investor relations material

Prader-Willi syndrome (PWS) program updates

• Imminent phase 3 COMPASS data readout for ACP-101 (carbetocin) expected in early Q4, likely October.

• ACP-101 is a modified oxytocin analog designed for longer action and greater receptor selectivity, targeting hyperphagia in PWS.

• Prior studies show supportive safety and tolerability, with mixed efficacy; lower doses showed more benefit and fewer side effects.

• The COMPASS trial focuses on a 3.2 mg dose, with HQ-CT as the primary endpoint and secondary/exploratory endpoints including caregiver burden and anxiousness (PAD-Q).

• Stakeholder feedback indicates strong interest in HQ-CT results and anxiousness endpoints, with potential regulatory flexibility depending on data strength.

ACP-204 (5HT2A inverse agonist) development

• ACP-204 is a next-generation 5HT2A inverse agonist, designed to improve efficacy and safety over previous compounds.

• Ongoing phase 2/3 program in Alzheimer’s disease psychosis (ADP) with phase 2 data expected mid-2026; study powered for a medium effect size (0.4).

• Study design includes biomarker-confirmed ADP patients and focuses on efficacy, safety, and convenience (once-daily dosing).

• Lewy body dementia psychosis (LBDP) study enrolling both LBD and Parkinson’s disease dementia psychosis (PDP) patients, with a cap on PDP enrollment.

• Biomarkers and genetic assays are included for learning but not required for LBDP study entry.

ACP-211 and pipeline strategy

• ACP-211, a selectively deuterated R-norketamine, is entering phase 2 for MDD with inadequate response, aiming for oral administration and minimal sedation/dissociation.

• Phase 2 will enroll 150 patients, focus on moderate MDD, and assess efficacy and safety at week 4.

• Early data suggest similar efficacy to ketamine with less motor impairment and minimal dissociation.

• Business development strategy targets phase 2 assets in neuropsychiatry and rare diseases, leveraging internal expertise.

• Additional pipeline catalysts include ACP-271 for dyskinesia and Huntington’s disease (phase 1 start by year-end), Lewy body study initiation, and expansion into Japan.