Rapport Therapeutics (RAPP) Stifel 2026 Virtual CNS Forum summary

Key program updates

• RAP-219 phase II data in focal onset seizures showed potentially best-in-class results, enabling acceleration of two parallel phase III studies to start in Q2 2026, ahead of previous guidance.

• Phase II study of RAP-219 in bipolar mania is ongoing, with data expected in the first half of 2027; enrollment is progressing well and timelines may be further refined mid-year.

• A long-acting injectable (LAI) formulation of RAP-219 is in IND-enabling studies, with first human PK data anticipated in 2027 and potential for extended IP protection into the late 2040s.

• Discovery programs, including the alpha-6 beta-4 nicotinic program for chronic pain and migraine, are advancing.

• Strategic collaboration with Tenacia opens China for RAP-219 trial enrollment, integrating global development and regulatory pathways.

Clinical development strategy and differentiation

• RAP-219 phase III studies are designed for efficiency, leveraging strong site and community interest, a wide patient funnel, and minimal drug-drug interactions.

• The program benefits from a lack of overlapping mechanisms with most marketed drugs, facilitating broader patient eligibility.

• Integrated global development with China is expected to accelerate enrollment and regulatory submissions.

• Confidence in phase II to phase III translation is based on objective biomarkers, robust seizure reduction, and demographic similarity to contemporary studies.

• Seizure freedom rate of 24% in phase II is highlighted as a strong efficacy signal, with minimal placebo effect expected.

Safety and competitive positioning

• RAP-219 has been tested in hundreds of subjects, with no severe AEs observed in phase II; most AEs are mild to moderate and resolve early.

• The drug is expected to have lower rates of sedation and gait impairment compared to current anti-seizure medications.

• No psychiatric AEs such as aggression or rage have been observed, differentiating RAP-219 from perampanel and other agents.

• Safety profile is being closely monitored in ongoing bipolar mania studies, with no major issues reported to date.

• FDA has not raised any special safety concerns for RAP-219 in end-of-phase II discussions.