Rapport Therapeutics Reports Positive Phase 1 Data For RAP-219 In CNS Disorders

(RTTNews) - Rapport Therapeutics, Inc. (RAPP), a clinical-stage biotechnology company focused on central nervous system disorders, on Thursday, announced promising results from its Phase 1 PET and MAD-2 trials for RAP-219, a selective AMPA receptor negative allosteric modulator.

The positron emission tomography (PET) trial in healthy volunteers confirmed RAP-219's ability to achieve target receptor occupancy within five days, aligning with preclinical efficacy.

The data showed that TARP8-containing AMPA receptors are primarily expressed in the hippocampus and cerebral cortex, key regions for central nervous system disorders (CNS) disorders while minimizing expression in the cerebellum and brain stem to reduce side effects.

The MAD-2 trial in healthy volunteers showed RAP-219 was well tolerated, with no serious adverse events.

Most treatment-emergent adverse events were mild or moderate, and RAP-219 did not cause sedation or motor impairments, distinguishing it from other CNS medications.

Commenting on the result Steve Paul, M.D., co-founder and chair of Rapport Therapeutics, said "The results further validate RAP-219's differentiated profile and potential to deliver transformative outcomes for patients".

A Phase 2a proof-of-concept trial evaluating RAP-219 in patients with refractory focal epilepsy is ongoing, with topline results expected in mid-2025.

The company also announced that Bradley Galer, M.D., has stepped down as Chief Medical Officer, and a search for his successor is underway.

The company ended the third quarter of 2024, with $320.7 million in cash, cash equivalents, and short-term investments.

RAPP closed Wednesday's (Jan. 08, 2025) trading at $16.69, down 6.16%.

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