Rapport Therapeutics presented an update on its clinical pipeline at the 12th Annual Cantor Fitzgerald Global Healthcare Conference, with investors focusing on the breadth of its neuroscience portfolio and its lead asset for focal onset seizures.
CEO Abe Ceesay and CFO Troy Ignelzi discussed RAP-219, a TARP gamma-8 AMPA modulator built around the company’s receptor-associated protein (RAPP) platform. Phase II data in focal onset seizures showed a 77.8% reduction in clinical seizures and a 24% seizure-freedom rate measured from Day 1 through Day 56. Patients in that trial were heavily medicated at baseline — 70% were taking three to four medications plus a device, totaling four to five therapies. Phase III populations are expected to be on two to three medications, Ceesay noted.
Most Phase II patients have enrolled in the open-label extension, with an initial data cut anticipated in the fourth quarter showing four to six months of continuous exposure. Two Phase III trials for focal onset seizures are now underway following interactions with the U.S. FDA, Chinese FDA, EMA and PMDA. Enrollment is projected to span 18 to 40 months, with specific guidance planned for 2025.
Rapport also outlined plans for a bipolar mania Phase II study, expected to release data in October. The inpatient monotherapy trial is designed to detect a placebo-adjusted difference of four points on the Young Mania Rating Scale, with the alpha level adjusted down from 0.1 to 0.05 and a modest sample-size increase. The protocol excludes first-episode patients and those with mixed depression-man"ia features; participants undergo a background medication washout. If the results are positive, the company plans two additional Phase III studies.
An unrelated long-acting injectable program is currently in IND-enabling studies, with first human pharmacokinetic data expected in 2027. The compound has low solubility, high potency and an inherent oral half-life of 22 days, with no drug-drug interactions anticipated. Filed as a supplemental new drug application, market research suggests the LAI could double the epilepsy market opportunity and extend patent exclusivity from 2041 to 2046 or 2047.
On the financial side, Rapport ended the second quarter with a little over $430 million in cash, funding the company through the second half of 2029 and covering the Phase III timeline assumptions. The stock traded in the $43.89 to $45.77 range at the time of the conference, reflecting a market capitalization of $2.11 billion. Shares were up 96% over the past year. Analyst consensus rates the stock a "Strong Buy" with price targets between $50 and $80, implying roughly 35% upside.
