Core Insights - Rapport Therapeutics, Inc. announced positive results from its PET and MAD-2 trials for RAP-219, indicating its potential as a treatment for focal epilepsy and other CNS disorders [1][2][3] Trial Results - In the PET trial, RAP-219 achieved target receptor occupancy (RO) associated with maximal efficacy within five days of dosing, confirming the neuroanatomical expression of TARPγ8 [1][10] - The MAD-2 trial demonstrated that RAP-219 was generally well tolerated, with no serious adverse events and favorable tolerability across various dosing regimens [2][11] - The ongoing Phase 2a trial of RAP-219 in focal epilepsy is on track, with topline data expected in mid-2025 [1][7] Drug Profile - RAP-219 is designed as a selective AMPA receptor negative allosteric modulator targeting TARPγ8, which is enriched in the hippocampus and cerebral cortex, potentially leading to improved tolerability compared to traditional treatments [8][9] - The drug has a long half-life of 8-14 days and minimal drug-drug interactions, making it suitable for patients on multiple medications [8] Company Overview - Rapport Therapeutics focuses on developing small molecule precision medicines for CNS disorders, leveraging its RAP technology platform to create differentiated product candidates [9] - The company is advancing RAP-219 for multiple indications, including focal epilepsy, diabetic peripheral neuropathic pain, and bipolar mania [9]
CORRECTION -- Rapport Therapeutics Announces New Phase 1 Data, Further Supporting RAP-219's Transformative Potential for CNS Disorders