Rapport Therapeutics Announces Q3 Financials and Business Update

Rapport Therapeutics, Inc., a clinical-stage biotechnology company specializing in small molecule precision medicines for central nervous system (CNS) disorders, has revealed its financial outcomes for the third quarter of 2024, alongside major business advancements. The company is progressing well with its lead program, RAP-219, particularly in its Phase 2a proof-of-concept trial for focal epilepsy, and anticipates top-line data from ongoing trials in early 2025.

The company has enhanced its Board of Directors with the addition of several industry experts, including Rob Perez, Raymond Sanchez, Paul Silva, and Wendy B. Young. These individuals bring extensive experience in drug discovery, neuroscience clinical development, and operational leadership, contributing valuable insights as Rapport Therapeutics seeks to expand its pipeline and achieve growth.

Rob Perez, currently operating partner at General Atlantic and former CEO of Cubist Pharmaceuticals, has a robust background in commercial leadership and has spearheaded significant drug launches and international expansions. Raymond Sanchez, a seasoned psychiatrist with over two decades in academia, medicine, and the pharmaceutical industry, adds his expertise in CNS and analgesia, having played a pivotal role in the regulatory approvals of multiple compounds. Paul Silva, an accomplished finance and operations executive, has been instrumental in transforming Vertex Pharmaceuticals into a high-growth company and brings his financial acumen to the team. Wendy B. Young, with over 30 years in drug discovery and development, has a proven track record in advancing clinical candidates and has been recognized for her contributions to the biotechnology field.

RAP-219, designed to target TARPγ8 associated with neuronal AMPAR, is seen as a promising treatment for focal epilepsy, peripheral neuropathic pain, and bipolar disorder. Ongoing clinical trials include a second multiple ascending dose trial (MAD-2) and a Phase 1 positron emission tomography (PET) trial to confirm brain target receptor occupancy. The company is also developing a long-acting injectable formulation of RAP-219, potentially offering improved patient adherence for anti-seizure medication.

In the realm of focal epilepsy, recruitment for the Phase 2a trial is on track, with the trial enrolling patients with drug-resistant focal epilepsy who have an implanted responsive neurostimulation (RNS) device. The RNS device aids in capturing intracranial electroencephalography data, providing biomarkers to measure clinical seizures. Results are anticipated in mid-2025.

For peripheral neuropathic pain, the company’s Investigational New Drug (IND) application for a Phase 2a trial in diabetic peripheral neuropathic pain (DPNP) has been placed on clinical hold by the FDA, pending additional protocol design information. However, this hold does not affect the ongoing focal epilepsy trials or the planned bipolar disorder trial.

Looking ahead, Rapport Therapeutics plans to initiate a Phase 2a trial in bipolar disorder patients experiencing acute mania in 2025. Additionally, the company is advancing its RAP-enabled nicotinic acetylcholine receptor (nAChR) discovery-stage programs, targeting chronic pain and hearing loss.

Financially, the company reported a net loss of $17.5 million for the third quarter of 2024, compared to $8.7 million in the same period the previous year. Research and development expenses increased to $15.5 million, driven by clinical development and pipeline progression costs. General and administrative expenses rose to $6.1 million, reflecting business growth and public company operational costs. Rapport Therapeutics concluded the quarter with $320.7 million in cash, cash equivalents, and short-term investments, which are projected to fund operations through the end of 2026.

RAP-219 stands out due to its selectivity in targeting TARPγ8, which is mainly expressed in the hippocampus and cortex. This specificity is expected to provide a better clinical profile with fewer side effects compared to conventional neuroscience medications. With its lead program and additional preclinical and discovery-stage initiatives, Rapport Therapeutics is poised to make significant advancements in treating CNS disorders.