Rapport Therapeutics Conference Call Summary Company Overview - Company: Rapport Therapeutics (NasdaqGM:RAPP) - Industry: Precision Neuroscience - Focus: Development of therapies for neurological conditions using receptor-associated proteins Key Points and Arguments Company Vision and Strategy - Rapport Therapeutics aims to be a leading precision neuroscience company, leveraging receptor-associated proteins to create highly specific compounds for various neurological conditions [8][9] - The company was formed through a collaboration between Third Rock and Johnson & Johnson, bringing significant institutional knowledge to its programs [9] Lead Program: RAP-219 - Description: RAP-219 is a novel forebrain-restricted TARP gamma-8 AMPA modulator targeting focal onset seizures [10][11] - Financial Position: As of September 30, 2025, the company had approximately $513 million, providing a cash runway into the second half of 2029 [10] - Clinical Progress: Positive phase two data for RAP-219 was released in September 2025, with plans to enter pivotal studies in Q2 2026 [10][13] Market Opportunities - Focal Onset Seizures: Approximately 1.8 million patients in the U.S. with a market opportunity of $15 billion [16] - Primary Generalized Tonic-Clonic Seizures: Around 800,000 patients with a $7 billion market opportunity [16] - Bipolar Mania: 1.5 million patients with a $40 billion market opportunity [16] - Long-Acting Injectable (LAI): Expected to be the first of its kind in epilepsy, providing significant revenue potential [15][16] Clinical Data and Efficacy - Phase 2A Trial Results: - 71% median reduction in long episodes (electrographic seizures) [27] - 77.8% reduction in clinical seizures, with 72% of patients achieving a 50% reduction [28] - 24% of patients achieved clinical seizure freedom, a significant outcome compared to historical placebo rates [29] - Tolerability: The drug was well tolerated with a low discontinuation rate of 10% [30] Future Developments - Bipolar Mania Study: Expected data in the first half of 2027 [35] - Long-Acting Injectable: Expected to enter IND-enabling studies with initial human PK data anticipated in 2027 [34][35] - Discovery Pipeline: Focused on nicotinic receptors with promising candidates for chronic pain and migraine [39] Regulatory and Market Positioning - Rapport Therapeutics has received positive feedback from the FDA, allowing for accelerated development of RAP-219 [13][32] - The company is positioned to potentially lead in the market with a new mechanism of action and favorable safety profile compared to existing treatments [44][46] Additional Important Information - The company emphasizes the importance of addressing treatment-resistant patients, with 30%-40% of focal onset seizure patients being refractory to current medications [16][18] - The long-acting injectable formulation is expected to mitigate risks associated with missed doses, a significant concern for patients and caregivers [47][48] - The discovery platform aims to create a self-sustaining pipeline, moving beyond single-asset dependency [38] This summary encapsulates the key insights from the conference call, highlighting the strategic direction, clinical advancements, and market potential of Rapport Therapeutics and its lead program, RAP-219.
Rapport Therapeutics (NasdaqGM:RAPP) FY Conference Transcript