Rapport Therapeutics (NasdaqGM:RAPP) 2025 Conference Transcript

Rapport Therapeutics Conference Call Summary Company Overview - Rapport Therapeutics is focused on precision neuroscience, leveraging receptor-associated proteins to enhance treatment efficacy and tolerability for patients [2][3] Key Programs - The lead program, RAP-219, is a TARP gamma-8 AMPA modulator, showing promising results in epilepsy and ongoing programs in bipolar mania [2][3] - RAP-219 has demonstrated a 78% median reduction in clinical seizure frequency and a 24% seizure freedom rate in a proof of concept study [11][12] FDA Engagement and Study Plans - The company plans to meet with the FDA by the end of 2025 for an end of phase two meeting, aiming to start phase three studies by Q3 2026 [4][5] - Two parallel registrational studies are planned, following a standard protocol for focal epilepsy [4][5] Safety and Efficacy Insights - The biological thesis focuses on modulating the AMPA receptor through TARP gamma-8, which is expressed in key brain structures related to focal seizures [6][8] - The tolerability profile from phase two data showed a 10% discontinuation rate, indicating best-in-class tolerability for anti-seizure medications [9] - Concerns regarding adverse events (AEs) related to psychiatric disorders were addressed, with no significant findings in their trials [10] Patient Population and Efficacy Expectations - The proof of concept study involved a highly refractory patient population, with 70% on three or four anti-seizure medications [16] - The efficacy results are expected to translate well into the broader phase three patient population, despite potential variability [18][13] Bipolar Mania and Other Indications - The company is exploring RAP-219 for bipolar mania, supported by the drug's mechanism targeting excitatory processes in the limbic structure [24][26] - Enrollment for the bipolar trial is progressing well, with data expected in 2027 [26] Market Opportunity - The potential market opportunity for RAP-219 in epilepsy is estimated to be multi-billion dollars, with expected utilization similar to existing treatments like Keppra [29] Upcoming Milestones - Key updates expected include the end of phase two meeting with the FDA, additional data analyses at AES in December 2025, and the initiation of phase three studies in 2026 [30][31] - The company has sufficient cash reserves, approximately $500 million, to fund operations through 2029 [32]