Rapport Therapeutics, Inc. · NASDAQ:RAPP
Rapport Therapeutics — Epilepsy Foundation Pipeline Conference 2024
8 slides from Rapport Therapeutics's conference presentation, Sep 25, 2024
Slide 1: RAP-219 a Novel, Potent, and Selective Negative Modulator of AMPAR/TARPg8
RAP-219 a Novel, Potent, and Selective Negative Modulator of AMPAR/TARPg8 Arnold Gammaitoni, Pharm.D. SVP, Medical Affairs Rapport Therapeutics Epilepsy Foundation Pipeline Conference September 25-26, 2024 ©Epilepsy Foundation of America, Inc.
Slide 2: RAP-219 targets AMPA Receptors Expressing TARPg8
RAP-219 targets AMPA Receptors Expressing TARPg8 Rapport’s Platform Most Enriched in Hippocampus, Highly-potent and g-8 specific Discovery Cortex, Amygdala !"#A%&#' !()(***++++,!J+./M Nature (2021) 594:448 N22!34 N*!56 fo rt at o rm "7&787''9&!A7:&#A!J+;<2N22,!=!>>22?!98@M Pl a pp R )%A*8JA( )9:9;;;
Slide 3: RAP-219 Exhibits Precision Neuromedicine Profile
RAP-219 Exhibits Precision Neuromedicine Profile Advantageous Therapeutic Index (TI) vs. Common Non-Sedating Anticonvulsant ASMs • Effective at levels corresponding to 70% receptor occupancy • Selective Targeting = Potential Differentiated TI Therapeutic Index = TD50 (toxic dose) on Rotarod/ED50 (ef
Slide 4: RAP-219 Phase 1 Studies in Healthy Volunteers
RAP-219 Phase 1 Studies in Healthy Volunteers • RAP-219-101 • Single ascending dose study – Completed • RAP-219-102 • First multiple ascending dose study, up to 4 weeks of dosing – Completed • RAP-219-103 • PET imaging study to determine brain target receptor occupancy – Ongoing • RAP-219-104 • Cont
Slide 5: RAP-219 MAD Unblinded Safety Summary
RAP-219 MAD Unblinded Safety Summary At highest dose, no TEAEs above Grade 1 and no treatment-related TEAEs 2024 Epilepsy Foundation Pipeline Conference © Epilepsy Foundation of America
Slide 6: RAP-219 Phase 2a PoC Trial in Focal Epilepsy
RAP-219 Phase 2a PoC Trial in Focal Epilepsy Principal Investigator: Jacqueline French, MD Eligibility Criteria: Trial Goal: • 18-65 years old • Evaluate efficacy of RAP-219 using Long • Implanted RNS System: Episode biomarker • Organized epileptiform activity exceeding a • Implanted > 15 mos. prior
Slide 7: Long Episodes – a Biomarker-based Endpoint Demonstrated to Predict Clinical Response
Long Episodes – a Biomarker-based Endpoint Demonstrated to Predict Clinical Response Change in seizure activity recorded through intracranial EEG (iEEG) predicted ASM clinical response • 30-40% reduction in LEs within 1-4 weeks of new ASM was associated with a >50% seizure “In addition to providing
Slide 8: RAP-219 Phase 2a PoC Trial Schema in Focal Epilepsy
RAP-219 Phase 2a PoC Trial Schema in Focal Epilepsy Trial schema Key Endpoints: • LE frequency responder analysis (% of patients that demonstrate ≥30% reduction in LEs) • Change in LE frequency, estimated EES, clinical seizure frequency, and additional iEEG biomarkers • Clinically meaningful improve
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