Rapport Therapeutics (RAPP) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Company vision and technology

• Aims to be a leader in precision neuroscience using a receptor-associated protein platform, originating from a decade of research at J&J and academic discovery at UCSF.

• Lead program RAP-219 targets gamma-8 TARP AMPA modulation, enabling highly specific receptor targeting in the forebrain.

• Platform enables drugging previously undruggable targets and supports a pipeline of additional programs.

Lead program RAP-219 and clinical development

• RAP-219 addresses focal epilepsy, targeting a population of 1.8 million in the US, with 30–40% experiencing breakthrough seizures despite current therapies.

• Demonstrates efficacy and improved tolerability by selectively modulating forebrain AMPA receptors, avoiding hindbrain-related side effects.

• Preclinical and early clinical data show strong efficacy and a differentiated safety profile, with no treatment-related adverse events at high receptor occupancy.

• Proof-of-concept study uses RNS device in focal epilepsy patients, measuring reduction in long episodes as a biomarker for seizure reduction.

• Enrollment supported by partnership with NeuroPace; data expected mid-2025, with registrational studies to follow.

Market opportunity and pipeline expansion

• RAP-219's addressable market is large and underserved, with potential for multibillion-dollar opportunity.

• Once-daily dosing, long half-life, and lack of drug-drug interactions position RAP-219 favorably for both patients and physicians.

• Plans to expand into neuropathic pain and bipolar disorder, with proof-of-concept studies enabled by IPO proceeds.

• Exploring a long-acting injectable formulation, which could be transformative for epilepsy and applicable to other indications.