Rapport Therapeutics (RAPP) The Citizens Life Sciences Conference 2026 summary

Scientific and clinical innovation

• RAP-219 targets TARP gamma-8, a receptor-associated protein linked to AMPA receptors, enabling selective activity in key brain regions and reducing adverse events common with other anti-seizure drugs.

• Preclinical and clinical studies demonstrated unprecedented efficacy and a wide therapeutic index, with PET imaging confirming target engagement in relevant brain areas for focal onset seizures.

• Phase 2 trial used a novel biomarker approach with implantable neurostimulation devices, achieving a 72% reduction in biomarker episodes and a 78% median reduction in clinical seizures, far surpassing expectations.

• Safety profile was highly favorable, with low discontinuation rates, no serious adverse events, and most adverse events being mild.

Phase 3 development and global strategy

• Two parallel global Phase 3 trials are planned, each enrolling about 330 subjects, with low-mid and mid-high dose strategies and straightforward titration schemas.

• No known drug-drug interactions and once-daily dosing are expected to facilitate broad enrollment and appeal.

• A new partnership with Tenacia in China will add Chinese sites to the Phase 3 program, accelerating global development and market access.

Market opportunity and competitive landscape

• Focal onset seizures represent a large market, with 30%-40% of 1.8 million patients considered refractory, translating to a $15 billion branded market opportunity.

• Multiple innovative drugs are needed due to widespread polypharmacy; RAP-219 is viewed as potentially best-in-class by physicians.

• Recent positive Phase 3 results from a competitor reinforce confidence in the predictive value of strong Phase 2 data.