Rapport Therapeutics (RAPP) investor relations material

Pipeline and financing

• RAP-219 TARPγ8 AMPA modulator strategy spans focal-onset seizures (FOS) and bipolar mania, each viewed as a multi-billion-dollar opportunity.

• Recent financing brought cash to ~$815M, extending runway through end-2029 and funding four phase III proof-of-concept trials plus a fifth ongoing trial in primary generalized tonic-clonic seizures.

• December open-label extension update expected to include safety and efficacy data, with 3–6 months of relative exposure and electrographic and clinical seizure measures.

• Long-acting injectable program readout expected in Q3 next year; these milestones are funded.

Epilepsy development

• FOCUS-1 and FOCUS-2 phase III trials are progressing, each targeting ~320 patients; global enrollment includes China through a partnership with neuroscience-focused Tenacia.

• RAP-219 has no drug-drug interactions, which management sees as widening the eligible enrollment pool; the team expects to be positioned to guide on data timing by mid-next year.

• Phase III participants will be more heterogeneous and generally lack RNS devices, but phase II patients were highly refractory, with baseline seizure counts similar to phase III; management expects phase II efficacy to translate.

Safety profile

• About 250 subjects have received RAP-219 across epilepsy and bipolar programs; reported tolerability includes no observed sedation or somnolence, and no metabolic, akathisia or extrapyramidal AEs.

• Management reported no hostility or homicidal ideation signal; in bipolar mania, hostility and irritability were among the domains showing the greatest improvement versus placebo.