Rapport Therapeutics (RAPP) TD Cowen 46th Annual Health Care Conference summary

Key achievements and recent progress

• Released proof-of-concept data for RAP-219 in refractory focal-onset seizures, showing a 72% reduction in electrographic seizures and a 78% reduction in clinical seizures, with a 24% seizure freedom rate in September 2025.

• Positive FDA interaction enabled acceleration of phase III program initiation to Q2 2026, ahead of previous guidance.

• Phase III will consist of two parallel trials with three dose levels (0.25, 0.75, 1.25 mg), focusing on refractory patients with breakthrough seizures.

• Proof-of-concept study population was highly refractory, with 70% on three or more medications, supporting data translatability to broader phase III population.

Clinical development and trial design

• Placebo-adjusted seizure reduction bar is 30–35%, with non-placebo adjusted reduction expected at 50–55%.

• Emphasis on efficacy, tolerability, and ease of use (once-daily dosing, minimal drug-drug interactions) to drive broad adoption.

• Phase II safety profile showed only mild to moderate AEs, with no severe events; psychiatric AEs (mainly anxiety) were consistent with background rates and not associated with aggression.

Market opportunity and strategic outlook

• Market research with over 100 neurologists and epileptologists positions RAP-219 as best-in-class for efficacy, tolerability, and dosing.

• Estimated $2.5 billion opportunity in refractory focal-onset seizures, with potential for multi-billion dollar revenue at branded pricing.

• Development of a long-acting injectable (LAI) formulation for RAP-219 underway, with first human PK results expected in 2027.

• LAI seen as transformational by KOLs due to improved adherence and reduced risk of breakthrough seizures.