Rapport Therapeutics Announces Positive Phase 1 Data for RAP-219, Confirming Neuroanatomical Specificity and Tolerability

RAPP
September 19, 2025
Rapport Therapeutics announced positive results from its positron emission tomography (PET) trial and second multiple ascending dose (MAD-2) trial for RAP-219. The PET trial confirmed the restricted neuroanatomical expression of TARPγ8 and demonstrated that RAP-219 achieved and exceeded target receptor occupancy within five days of dosing. This supports the dosing regimen currently used in the ongoing Phase 2a trial for focal epilepsy. The MAD-2 trial showed that RAP-219 was generally well tolerated, with faster titration and higher exposures compared to the initial MAD trial. Across a total of four Phase 1 trials involving 100 healthy volunteers, RAP-219 exhibited a favorable tolerability profile with no serious adverse events and only three treatment discontinuations attributed to treatment-emergent adverse events. These Phase 1 results reinforce the company's belief in RAP-219's distinct profile and its potential to deliver transformative outcomes for patients by selectively targeting TARPγ8 associated AMPA receptors. The company expects topline data from its Phase 2a trial in focal epilepsy in mid-2025, building on this positive early-stage validation. The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.