Rapport Therapeutics (RAPP) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Pipeline overview and strategic focus

• Precision neuroscience approach leverages receptor-associated proteins, with RAP-219 as lead program showing unprecedented phase II results in focal onset seizures.

• Upcoming bipolar mania data readout expected in Q4, with plans for a long-acting injectable formulation to expand opportunities.

• Integrated discovery pipeline aims for a self-sustaining, regenerative portfolio.

Bipolar mania program insights

• RAP-219 targets excessive glutamate in the corticolimbic network, aligning with disease biology and clinical precedent.

• Study focuses on acutely manic patients, excluding first-episode and mixed-feature cases; all participants are on monotherapy after washout.

• Phase II study powered for a four-point placebo-adjusted difference in YMRS; protocol adapted to lower alpha and increase sample size for potential registrational use.

• If successful, two additional phase III studies are planned, with similar design but larger size.

• No signals of severe neuropsychiatric adverse events observed; safety monitored by a data safety committee.

Focal onset seizure (FOS) program and clinical progress

• Phase II data showed 77.8% reduction in clinical seizures and a 24% seizure freedom rate, supported by objective biomarkers.

• Tolerability profile is highly differentiated, avoiding common adverse events seen in traditional anti-seizure medications.

• Open label extension ongoing, with most patients enrolled and four to six months of continuous exposure data expected in Q4.

• Phase III studies launched globally, including China, with positive regulatory interactions and sufficient cash runway into 2029.

• Patient severity in phase III expected to be lower (two to three background meds) compared to phase II.