Summary of Rapport Therapeutics Investor and Analyst Day Company Overview - Company: Rapport Therapeutics - Event: Inaugural Investor and Analyst Day - Focus: Lead candidate RAP-two 19 for epilepsy treatment Key Points and Arguments Industry and Product Focus - Lead Candidate: RAP-two 19, a TARP gamma eight AMPA modulator, aimed at treating focal onset seizures in epilepsy [3][19] - Market Size: Focal epilepsy affects approximately 1.8 million patients in the U.S., with 30-40% being drug-resistant [32] - Pipeline: Fully enabled pipeline including RAP-two 19 and two late-stage discovery programs targeting chronic pain and hearing disorders [19][27] Clinical Trials and Results - Phase Two Trial: Ongoing trial for RAP-two 19 in focal onset seizure patients, fully enrolled with results expected in September [21][26] - Phase One Results: Completed four Phase I studies with 100 healthy volunteers; RAP-two 19 was safe and well-tolerated [52][53] - Receptor Occupancy: Achieved target receptor occupancy of 50-70% at lower doses than predicted, indicating potential for effective treatment [56][58] Scientific Insights - Mechanism of Action: RAP-two 19 selectively modulates AMPA receptors in specific brain regions, potentially reducing side effects associated with traditional anti-seizure medications [38][39] - Preclinical Efficacy: Demonstrated significant efficacy in animal models with a favorable safety profile compared to existing treatments [44][46] Management and Strategy - Management Team: Comprised of experienced professionals in neuroscience and drug development, emphasizing a strategic approach to building the company [15][17] - Vision: To create a leading precision neuroscience company, addressing the limitations of conventional small molecules in treating neurological disorders [24][25] Additional Important Content - Unmet Needs: Despite numerous treatment options, the number of treatment-resistant epilepsy patients remains unchanged, highlighting the need for innovative therapies [92][93] - Patient Population: The trial population is representative of refractory focal epilepsy patients, enhancing the translatability of results to broader populations [87] - Long-Acting Injectable: Potential development of a long-acting injectable formulation for RAP-two 19, which could transform treatment for epilepsy patients [29][30] Conclusion Rapport Therapeutics is positioned to address significant unmet needs in the epilepsy treatment landscape with its innovative lead candidate RAP-two 19, backed by a robust clinical and scientific foundation. The company's strategic focus on precision neuroscience and the experienced management team further enhance its potential for success in the market.
Rapport Therapeutics (RAPP) 2025 Investor Day Transcript