ADDED
Our business is highly dependent on the success of our product candidates, particularly RAP-219 for focal onset seizures.
The regulatory approval processes of the Food and Drug Administration, European Medicines Agency, Medicines and Healthcare products Regulatory Agency and other comparable regulatory authorities are lengthy, time-consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed.
Business Overview We are a clinical-stage biotechnology company dedicated to the discovery and development of small molecule precision medicines for patients with neurological or psychiatric disorders.
Whereas AMPARs are distributed widely in the central nervous system ( CNS ), TARP 8 is expressed only in discrete regions, including the neocortex and mesial temporal lobe, where focal onset seizures ( FOS ) often originate.
Due to the role of AMPA biology in various neurological disorders and our precision approach of selectively targeting TARP 8, we believe RAP-219 has pipeline-in-a-product potential and we are evaluating it as a potentially transformational treatment for patients with FOS, primary generalized tonic-clonic seizures ( PGTCS ), and bipolar mania.
Several Phase 1 trials in RAP-219 have been conducted in healthy adult volunteers, including a single ascending dose ( SAD ) trial; a multiple ascending dose ( MAD ) trial; a second MAD trial ( MAD-2 ); and a positron emission tomography ( PET ) trial, which utilized a companion PET radiotracer to confirm brain target receptor occupancy ( RO ) and brain region specificity across a range of dosing and exposure levels.
In September 2025, we announced positive topline results from our Phase 2a proof-of-concept trial of RAP-219 in adult patients with drug-resistant FOS.
The trial demonstrated a statistically significant reduction in long episodes an objective electrographic biomarker for clinical seizure reduction compared with baseline over the 8-week treatment period.
In the trial, RAP-219 also demonstrated a statistically significant and clinically meaningful reduction in clinical seizures compared with baseline.
In December 2025, we presented post-hoc analysis from the Phase 2a proof-of-concept trial showing treatment with RAP-219 had consistent effects in the first and second four-week segments of the treatment period.
REMOVED
Our business is highly dependent on the success of our product candidates, particularly RAP-219 for focal epilepsy.
The regulatory approval processes of the Food and Drug Administration ( FDA ), European Medicines Agency ( EMA ), Medicines and Healthcare products Regulatory Agency ( MHRA ) and other comparable regulatory authorities are lengthy, time-consuming and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed.
Business Overview We are a clinical-stage biotechnology company dedicated to discovering and developing small molecule precision medicines for patients with neurological or psychiatric disorders.
Whereas AMPARs are distributed widely in the central nervous system ( CNS ), TARP 8 is expressed only in discrete regions, including the hippocampus and neocortex, where focal seizures often originate.
Due to the role of AMPA biology in various neurological disorders and our precision approach of selectively targeting TARP 8, we believe RAP-219 has pipeline-in-a-product potential and we are evaluating it as a potentially transformational treatment for patients with focal epilepsy, bipolar disorder, and peripheral neuropathic pain.
A total of four Phase 1 trials in RAP-219 have been conducted to date in healthy adult volunteers: a single ascending dose ( SAD ) trial; a multiple ascending dose ( MAD ) trial; a second MAD trial ( MAD-2 ), to assess dosing regimens that may accelerate time to reach therapeutic exposure; and a human positron emission tomography ( PET ) trial, which utilized a companion PET radiotracer to confirm brain target receptor occupancy and brain region specificity across a range of dosing and exposure levels.
In January 2025, we announced results from our PET and MAD-2 trials of RAP-219.
Data demonstrated that neuroanatomical specificity can be achieved through RAP-219 s selective targeting of TARP 8.
In Cohort 1 of the human PET trial, which used the dosing regimen utilized in our ongoing Phase 2a trial in patients with refractory focal epilepsy, RAP-219 achieved target receptor occupancy associated with maximal seizure protection in preclinical models within five days and was generally well tolerated, which we believe further supports the use of such dosing regimen in the Phase 2a trial .
We are currently conducting a Phase 2a proof-of-concept trial in adult patients with refractory focal epilepsy, for which we expect to report topline results in the third quarter of 2025.