Rapport Therapeutics (RAPP) Leerink Global Healthcare Conference 2026 summary

Scientific and clinical highlights

• Focus on precision neuroscience, targeting receptor-associated proteins, specifically TARP γ8, which is selectively expressed in brain regions critical for focal-onset seizures and minimally in the hindbrain.

• RAP-219 targets the AMPA receptor via TARP γ8, reducing glutamate-driven excitatory processes and seizures with high selectivity, resulting in a strong risk-benefit profile.

• Phase II proof-of-concept study in refractory focal-onset seizure patients using the RNS System as a biomarker showed a 72% reduction in electrographic seizures and a 78% median reduction in clinical seizures, with a 24% seizure freedom rate.

• The innovative trial design enabled efficient data collection with a small sample size and objective biomarker correlation, supporting rapid and reliable efficacy assessment.

• Over 140 patients have been exposed to RAP-219 across phase I and II studies, with no maximum tolerated dose observed and a favorable tolerability profile.

Safety, tolerability, and dosing

• Adverse events were mild to moderate, mainly dizziness and headache, with a 10% discontinuation rate in phase II.

• Tolerability is linked to Cmax rather than tmax, and slow titration mitigates activating side effects.

• Phase III studies will explore three dose levels with titration periods of 7–9 days, aiming for efficient and safe dose escalation.

Phase III and global development plans

• Two parallel global phase III studies are set to begin in Q2, each enrolling 320–330 subjects, with China included as a recruitment region through a new partnership.

• The China partnership with Tenacia includes $20 million upfront, up to $308 million in milestones, royalties, and Tenacia funding all Chinese development.

• The company expects strong trial enrollment due to competitive positioning, broad eligibility, and global site distribution.