Rapport Therapeutics, a clinical-stage biotechnology company, announced its third-quarter 2024 financial results and provided an update on its clinical programs, highlighting the progress of RAP-219 and changes to its Board of Directors. The company remains focused on developing precision medicines for central nervous system (CNS) disorders.
RAP-219 Clinical Development
The lead program, RAP-219, is currently under evaluation for focal epilepsy, peripheral neuropathic pain, and bipolar disorder. RAP-219 is designed to selectively target TARPγ8, a receptor-associated protein (RAP) linked to the neuronal AMPAR, a validated target for epilepsy.
Abraham N. Ceesay, chief executive officer of Rapport, stated, "We are pleased with the progress we’re making with RAP-219, particularly as we continue the execution of our Phase 2a proof-of-concept trial in focal epilepsy." He added that ongoing clinical activities have strengthened confidence in RAP-219's potential as a transformational treatment.
Focal Epilepsy Trial
Rapport Therapeutics is actively recruiting patients for a Phase 2a proof-of-concept trial of RAP-219 in focal epilepsy. This trial is on track, with topline data expected in mid-2025. The study enrolls adult patients with drug-resistant focal epilepsy who have an implanted responsive neurostimulation (RNS) device. The RNS device captures intracranial electroencephalography data and records the frequency of long episodes (LEs), which serve as a biomarker for clinical seizures. A 30% reduction of LEs has been shown to predict meaningful changes (≥ 50% reduction) in clinical seizure frequency.
The company will present four poster presentations at the Annual Epilepsy Society meeting in December, focusing on RAP-219, including preclinical data, SAD/MAD trial results, food effect data, and analyses correlating RNS-measured long episode and clinical seizure frequency.
Peripheral Neuropathic Pain
The FDA recently placed a clinical hold on the Investigational New Drug Application (IND) for RAP-219 in diabetic peripheral neuropathic pain (DPNP), requesting additional protocol information. The clinical hold does not affect the ongoing Phase 2a trial in focal epilepsy or the planned trial in bipolar disorder. Rapport Therapeutics believes it can resolve the clinical hold and will provide updates on the Phase 2a trial initiation timeline when available.
Bipolar Disorder Program
Rapport Therapeutics plans to initiate a Phase 2a trial in bipolar disorder patients with acute mania in 2025.
Board of Directors Expansion
Rapport Therapeutics has appointed Rob Perez, Raymond Sanchez, MD, Paul Silva, and Wendy B. Young, PhD, to its Board of Directors. These individuals bring expertise in drug discovery, neuroscience clinical development, and operational leadership. Jeff Tong, PhD, has resigned from the Board.
Financial Update
The company reported a net loss of $17.5 million for the third quarter of 2024, compared to $8.7 million for the same period in the prior year. Research and development expenses were $15.5 million, up from $7.6 million in the prior year, driven by clinical development and pipeline progression costs. General and administrative expenses increased to $6.1 million from $2.0 million, due to business growth and public company operational costs.
Rapport Therapeutics ended the quarter with $320.7 million in cash, cash equivalents, and short-term investments, which is expected to fund operations through the end of 2026.
