Summary of Rapport Therapeutics (RAPP) 2025 Conference Company Overview - Company: Rapport Therapeutics (RAPP) - Industry: Biotechnology, specifically in precision neuroscience and epilepsy treatment - Key Personnel: Abe Cisse (CEO), Choi Ignelzi (CFO) [1][2] Core Points and Arguments - Vision: Rapport aims to be a leading precision neuroscience company, focusing on receptor associated proteins to develop targeted therapies [4][5]. - Lead Program: RAP-two 19, an AMPA TARP gamma-eight modulator, targets specific brain areas to modulate AMPA receptors, aiming to reduce adverse effects (AEs) associated with traditional anti-seizure medications [5][6]. - Clinical Trials: - Currently conducting a Phase 2a proof of concept study for RAP-two 19 in focal epilepsy, fully enrolled with 30 patients, with data expected in September 2025 [7][8]. - Plans to explore RAP-two 19 in bipolar mania and peripheral neuropathic pain, with a long-acting injectable formulation also in development [8][9][10]. - Differentiation: RAP-two 19 is designed for once-daily dosing with a long half-life, aiming to minimize drug-drug interactions (DDIs) and improve patient adherence [12][14][15][16]. Important Data and Metrics - Phase 1 Studies: Completed four studies with 100 healthy volunteers, showing a favorable tolerability profile and high receptor occupancy (up to 85%) without significant sedation or motor impairment [23][24]. - Efficacy Expectations: The primary endpoint for the Phase 2a study is a 30% reduction in long episodes of seizures, with a target responder rate of at least 40% [38][39]. - Safety Profile: No serious adverse events (SAEs) reported; mild AEs observed were transient and resolved on their own [45][46]. Additional Insights - Biomarker Utilization: The study leverages an implantable RNS device to capture EEG activity, providing an objective measure of efficacy compared to traditional seizure diaries [28][30]. - Regulatory Pathway: The company plans to conduct two registrational trials for NDA submission, with the potential to start in 2026 [51][52]. - Market Positioning: The combination of efficacy, tolerability, and administration profile positions RAP-two 19 as a potentially best-in-class therapy for epilepsy [41][44]. Conclusion Rapport Therapeutics is advancing its lead program RAP-two 19 with a focus on precision neuroscience, aiming to address significant unmet needs in epilepsy treatment through innovative clinical trial designs and a differentiated safety profile. The upcoming data readout in September 2025 will be critical for the company's future development plans and market positioning.
Rapport Therapeutics (RAPP) 2025 Conference Transcript