Rapport Therapeutics Conference Call Summary Company Overview - Company: Rapport Therapeutics (NasdaqGM:RAPP) - Event: FY Conference on September 17, 2025 Industry Context - Industry: Neuropsychiatry and Epilepsy - Market Size: Approximately 1.8 million patients in the U.S. with 560,000 treatment-resistant patients [2][27] Key Points and Arguments Phase 2 Trial Results - Efficacy: The Phase 2 trial demonstrated a median reduction of 78% in clinical seizures and a 24% seizure freedom rate, indicating a best-in-class profile for the anti-seizure medication RAP-219 [5][26] - Patient Demographics: The trial enrolled treatment-resistant focal epilepsy patients, with 70% on three or four anti-seizure medications, confirming the refractory nature of the population [7][8] - Trial Design: The trial utilized a novel design informed by the epilepsy community, leveraging an electrographic biomarker to predict clinical seizure reductions [4][9] Next Steps - Phase 3 Plans: Rapport Therapeutics plans to initiate two parallel Phase 3 studies in Q3 2026, following an end of Phase 2 meeting with the FDA [10][36] - Translatability: The results from the Phase 2 trial are believed to be highly translatable to registrational studies, improving the probability of success [6][9] Safety and Tolerability - Tolerability Profile: The trial reported a 10% discontinuation rate, one of the lowest in proof of concept studies for this patient population, with no severe adverse events greater than grade two [18][24] - Comparison with Other Drugs: RAP-219 showed a differentiated tolerability profile compared to Fycompa, with significantly lower rates of sedation and aggression [19][22] Commercial Potential - Market Opportunity: The peak sales opportunity for RAP-219 has shifted from an estimated $1.5 billion to potentially much higher, with expectations of broad adoption by general neurologists and internists [26][27] - Long-Acting Injectable: There is potential for developing a long-acting injectable formulation, which could transform patient management in epilepsy [29][30] Future Indications - Bipolar and Pain: The Phase 2 results have raised the probability of success for other indications like bipolar mania, due to confirmed target engagement and a favorable tolerability profile [39][40] Financial Position - Cash Balance: As of Q2, the company had $260 million, sufficient to fund operations through the end of 2029, including the initiation of Phase 3 trials and development of a long-acting injectable [37][38] Additional Important Insights - Community Feedback: The company has received positive feedback from the epilepsy community regarding the drug's profile and potential for adoption [26][28] - Enrollment Strategy: The company is focused on enrolling the right patient population for Phase 3 studies, considering competitive context and drug profile [32][33] This summary encapsulates the critical insights from the conference call, highlighting the promising developments for Rapport Therapeutics and its lead candidate RAP-219 in the epilepsy market.
Rapport Therapeutics (NasdaqGM:RAPP) FY Conference Transcript