Rapport Therapeutics Provides Update on RAP-219 Clinical Trials and Financial Results
核心洞察
Rapport Therapeutics is advancing RAP-219, a precision medicine for CNS disorders, with Phase 2a trial in focal epilepsy (搜索) on track for mid-2025 data.
A clinical hold was placed on the RAP-219 trial for diabetic peripheral neuropathic pain (搜索) (DPNP) by the FDA, pending protocol amendments.
The company's MAD-2 and PET trials of RAP-219 are ongoing, with topline data expected in Q1 2025, informing bipolar disorder (搜索) treatment.
Rapport Therapeutics, a clinical-stage biotechnology company, has announced its third-quarter financial results for 2024 and provided an update on its clinical programs, particularly focusing on RAP-219, a small molecule precision medicine targeting central nervous system (CNS) disorders. The company is currently evaluating RAP-219 for focal epilepsy (搜索), peripheral neuropathic pain (搜索), and bipolar disorder (搜索).
RAP-219 Clinical Development
The company's lead program, RAP-219, is designed to selectively target TARPγ8 (搜索), a receptor-associated protein (RAP) associated with the neuronal AMPAR (搜索), a clinically validated target for epilepsy. Rapport Therapeutics is conducting a Phase 2a proof-of-concept trial in focal epilepsy (搜索), with topline results expected in mid-2025. This trial is enrolling 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), serving as a biomarker to clinical seizures.
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 (搜索)." He added that learnings from ongoing clinical activities have strengthened confidence in RAP-219's potential as a treatment for focal epilepsy, peripheral neuropathic pain (搜索), and bipolar disorder (搜索).
Clinical Hold on DPNP Trial
The FDA placed a clinical hold on the Investigational New Drug Application (IND) for RAP-219 in diabetic peripheral neuropathic pain (搜索) (DPNP), requesting additional information and amendments to the protocol design. The company believes it can resolve the clinical hold and will provide an update on the anticipated timing of the Phase 2a trial initiation once available. The clinical hold does not impact the ongoing Phase 2a trial in focal epilepsy (搜索) or the planned proof-of-concept trial in bipolar disorder (搜索).
Ongoing MAD-2 and PET Trials
Rapport is conducting a second multiple ascending dose trial (MAD-2) of RAP-219, expected to be completed in Q4 2024. This trial will assess additional dosing regimens to inform dosing for the Phase 2a trial for bipolar acute mania. A Phase 1 human positron emission tomography (PET) trial is also underway to confirm brain target receptor occupancy and brain region specificity across a range of RAP-219 dosing and exposure levels. Topline results from both the MAD-2 and PET trials are expected in Q1 2025.
Financial Update
Rapport Therapeutics ended the third quarter with $320.7 million in cash, cash equivalents, and short-term investments. The company expects these funds to support operations through the end of 2026.
For the third quarter of 2024, the net loss was $17.5 million, compared to $8.7 million for the prior year period. Research and development expenses were $15.5 million, driven by clinical development costs. General and administrative expenses were $6.1 million, reflecting costs associated with business growth and operating as a public company.
Board of Directors Appointments
Rapport Therapeutics has appointed new members to its Board of Directors: Rob Perez, Raymond Sanchez, MD, Paul Silva, and Wendy B. Young, PhD. These appointments bring expertise in drug discovery, neuroscience clinical development, and operational leadership. Steve Paul MD, founder and chair of Rapport's Board of Directors, expressed appreciation to Jeff Tong, PhD, for his leadership and welcomed the new members, citing their experience as beneficial for realizing the full potential of Rapport's science.
About RAP-219
RAP-219 is a clinical-stage AMPAR (搜索) negative allosteric modulator (NAM) designed to achieve neuroanatomical specificity through its selective targeting of TARPγ8 (搜索). The company believes RAP-219 has the potential to provide a differentiated clinical profile, including improved activity and tolerability, compared to traditional neuroscience medications.