Skyrocketing Success of 180% for Epilepsy Pill amid Optimistic Mid-Stage Results

In a groundbreaking development for the epilepsy community, Rapport Therapeutics' investigative therapy, RAP-219, has shown promising results in a Phase II trial. The news was reported by Heather McKenzie, a Neuroscience, Clinical research, and Rapport Therapeutics expert, on a platform that allows for easy sharing on Twitter, LinkedIn, Facebook, Email, and Print.

The trial enrolled 30 patients with drug-resistant focal onset seizures and an implanted responsive neurostimulation (RNS) system. RAP-219 reduced seizures by nearly 78% in these patients, with 24% being seizure-free for the entire eight-week treatment period. The Phase II trial was the first to test a novel antiseizure medication in focal seizure patients using the RNS system with an objective biomarker of seizure activity.

Analysts have praised the results, calling them a "best case scenario" and a "best/bull case outcome." Jefferies analysts wrote that the results signaled a differentiated $1 billion+ epilepsy pill, while Stifel noted that the data far exceed expectations and safety/tolerability looks in-line with widely prescribed anti-seizure medications.

Rapport Therapeutics' shares gained more than $25 in value on Monday morning, a nearly 180% rise, following the release of the Phase II data. The company plans to move RAP-219 into two Phase III pivotal trials in the third quarter of 2026.

Jacqueline French, principal investigator of the Phase IIa study and professor in the department of Neurology at NYU Langone Health's Comprehensive Epilepsy Center, expressed confidence in RAP-219's potential as a highly effective anti-seizure medicine for drug-resistant focal seizure patients. The long episode results of RAP-219 more than doubled Rapport's target of around 40%.

It's important to note that no specific researcher or institution is mentioned as involved with the Phase II study of RAP-219 by Rapport Therapeutics. The company itself is referenced as conducting the study. Stifel also mentioned that while there will be some differences in the trial population for Phase III, the data's impressive results and safety/tolerability profile bode well for the future of this potential treatment.

Patients involved in the trial reported that RAP-219 was generally well-tolerated during the trial. Heather McKenzie's article provides a comprehensive overview of this significant development in the fight against epilepsy, offering hope for those suffering from drug-resistant focal seizures.

Skyrocketing Success of 180% for Epilepsy Pill amid Optimistic Mid-Stage Results