Rapport Therapeutics (RAPP) FY Conference Summary Company Overview - Company: Rapport Therapeutics (RAPP) - Industry: Biotechnology, specifically focusing on epilepsy and related neurological disorders Key Points and Arguments Clinical Development Progress - Rapport has made significant progress since going public a year ago, focusing on the clinical development of its lead program, RAP 219 [2][3] - Completed phase one studies, including multiple ascending dose studies and a human PET study, which have increased confidence in the pharmacology and specificity of RAP 219 [3][27] - Currently conducting a phase 2a proof of concept study in refractory focal epilepsy patients, utilizing an implantable neurostimulation device for diagnostics [3][6] Study Design and Rationale - The study design aims to produce compelling proof of concept results that are translatable to a phase three registration setting [5] - The study leverages an EEG biomarker correlated with clinical seizures, addressing the limitations of traditional clinical seizure diaries [6][9] - The trial includes an eight-week retrospective data collection period followed by a 28-day prospective period and an eight-week treatment phase [7][31] Data Expectations and Efficacy Measures - Data readout is expected in September, with a focus on achieving a 30% reduction in long episodes for 40% of participants, correlating to a 50% reduction in clinical seizures [19][22] - The study is designed to ensure a robust set of baseline characteristics, with a median clinical seizure count of 10, which positions the study favorably compared to other trials [48] Pipeline and Future Development - Rapport is also developing RAP 219 for bipolar disorder and neuropathic pain, with plans to initiate a bipolar study in Q3 [34][36] - The neuropathic pain program is currently on clinical hold due to requested protocol changes by the FDA [36][38] - A long-acting injectable formulation of RAP 219 is being explored, which could transform treatment for epilepsy patients [42][45] Financial Position - The company reported a cash position of $285 million, sufficient to fund activities through at least the end of 2026, covering the upcoming phase three trials and ongoing bipolar studies [51] Risks and Confidence - Confidence in the upcoming data readout is supported by a robust preclinical package and a high concordance rate between long episodes and electrographic seizures [47][49] - The company is optimistic about the translatability of the phase 2a results to phase three studies, with plans for an end-of-phase two meeting with the FDA following positive results [24][33] Additional Important Insights - The study design has received positive feedback from the epilepsy community, highlighting its objective analysis capabilities [9][17] - The long half-life of RAP 219 (8-14 days) is seen as beneficial for patient compliance, reducing the risk of breakthrough seizures [32] - The company aims to differentiate RAP 219 from existing treatments through its efficacy, dosing flexibility, and lack of sedation [23][27]
Rapport Therapeutics (RAPP) FY Conference Transcript