BioCentury
ARTICLE | Product Development

Rapport’s precision synaptic targeting and innovative trial design deliver in Phase II

Proof-of-concept data set up RAP-219 for Phase III in treatment-resistant epilepsy, while increasing confidence in its pipeline-in-a-product potential

September 9, 2025 1:46 PM UTC

Rapport has achieved Phase II proof of concept for a new approach to precision medicine in the CNS. Instead of broadly blocking glutamate receptors — which drive excitatory transmission throughout the brain — the biotech showed that selectively modulating an anatomically restricted protein that regulates glutamate receptors in certain brain regions reduced seizure frequency in drug-resistant patients without the liabilities of global glutamate inhibition.

Shares of Rapport Therapeutics Inc. (NASDAQ:RAPP) rose 119% to $31.47 on Monday, lifting the company’s market cap to over $1 billion, after RAP-219 met the primary and secondary endpoints in a Phase IIa study in treatment-resistant epilepsy. The biotech sought to extend momentum, proposing a $250 million follow-on financing on Tuesday...