Rapport Therapeutics (Nasdaq: RAPP) announced positive Phase 1 results for RAP-219, a drug candidate for central nervous system disorders. The trials demonstrated that RAP-219 achieved target receptor occupancy within five days and showed a favorable tolerability profile. The company also announced the resignation of their chief medical officer, Bradley Galer.
These results are important because they suggest RAP-219 could offer a meaningful improvement over existing treatments for focal epilepsy and other CNS disorders, which often have significant side effects that limit their effectiveness. RAP-219’s targeted mechanism, focusing on the TARPγ8 protein primarily found in the hippocampus and cerebral cortex, may minimize side effects often seen with less selective drugs impacting the cerebellum and brainstem. The quick achievement of target engagement suggests a faster onset of therapeutic action, a potential benefit for patients.
Across four Phase 1 trials involving 100 healthy volunteers, RAP-219 demonstrated good tolerability with no serious adverse events and limited low-grade adverse events, including no reported sedation or motor impairments. Data from the PET trial confirmed the targeted activity of RAP-219 and its concentration in relevant brain regions. The MAD-2 trial further supported the safety and tolerability profile, showing that therapeutic drug levels could be reached quickly. A Phase 2a proof-of-concept trial for RAP-219 in focal epilepsy is ongoing, with topline results anticipated in mid-2025.
The positive Phase 1 results strengthen the case for RAP-219 as a potential treatment for focal epilepsy and other CNS disorders. Confirmation of the drug’s targeted action and tolerability profile increases its potential to address unmet needs for patients suffering from these conditions. The upcoming Phase 2a results will be crucial in determining the clinical efficacy of RAP-219 and its potential path toward becoming a valuable new therapeutic option.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
