Rapp Therapeutics

The company is a clinical-stage biopharmaceutical firm focused on discovering and developing transformational small molecule medicines for patients suffering from central nervous system (CNS) disorders. Incorporated in Delaware, the organization is dedicated to advancing understanding and treatment of these conditions.

Neuronal receptors are complex protein assemblies, consisting of receptor principal subunits and receptor associated proteins (RAPs). RAPs play crucial roles in regulating receptor expression and function. The founders of the company have made pioneering discoveries related to RAP function, forming the basis of the RAP technology platform. This deep expertise in RAP biology offers the opportunity to explore previously inaccessible targets and develop CNS drugs specific to receptor variants and neuroanatomical regions associated with certain diseases. RAP-219, the most advanced product candidate, is an AMPA receptor (AMPAR) negative allosteric modulator (NAM). RAP-219 aims to achieve neuroanatomical specificity through its selective targeting of a RAP known as TARPg8, associated with the neuronal AMPAR, a clinically validated target for epilepsy. While AMPARs are widely distributed in the CNS, TARPg8 is expressed only in discrete regions, including the hippocampus, a key site involved in focal epilepsy. Phase 1 trials in healthy adults have been completed to assess the safety and tolerability of RAP-219, and a Phase 2a proof-of-concept trial in adult patients with drug-resistant focal epilepsy is planned for mid-2024, with topline results expected in mid-2025. RAP-219 also shows therapeutic potential in peripheral neuropathic pain and bipolar disorder, with Phase 2a trials expected to begin in the second half of 2024 and in 2025, respectively. Another TARPg8 targeted molecule, RAP-199, with differentiated chemical and pharmacokinetic properties, is expected to enter a Phase 1 trial in the first half of 2025.

Beyond TARPg8, there are two advanced discovery-stage nicotinic acetylcholine receptor (nAChR) programs stemming from the RAP technology platform. The first discovery-stage nAChR program includes modulators of a6 nAChRs, being developed for the treatment of chronic pain. The second discovery-stage nAChR program involves modulators of a9a10 nAChRs, targeted for the treatment of hearing disorders. The RAP technology platform continues to be leveraged to discover additional product candidates.