Phase 2a follow-up period data for RAP-219 in focal onset seizures (FOS) demonstrated sustained seizure reduction, including a 90% median reduction in clinical seizures over baseline in weeks 9-12
RAP-219 Phase 2 trial in bipolar mania topline results now expected in the fourth quarter of 2026, ahead of previous 1H 2027 guidance
RAP-219 Phase 3 program in FOS remains on track for initiation in the second quarter of 2026
Pipeline programs, including RAP-219 long-acting injectable formulation, RAP-219 in primary generalized tonic-clonic seizures, and <ALPHA>6<BETA>4 nAChR in chronic pain and migraine, continue to advance
Strategic collaboration and license agreement entered into with Tenacia Biotechnology to develop and commercialize RAP-219 in Greater China across indications
Ended the first quarter of 2026 with $476.8 million in cash, cash equivalents and short-term investments, excluding restricted cash, expected to fund operations into the second half of 2029
BOSTON and SAN DIEGO, May 07, 2026 (GLOBE NEWSWIRE) -- Rapport Therapeutics, Inc. (Nasdaq: RAPP) ("Rapport" or the "Company"), a clinical-stage biotechnology company dedicated to the discovery and development of small molecule precision medicines for patients with neurological or psychiatric disorders, today reported financial results for the quarter ending March 31, 2026, and provided a business update.
"We entered 2026 with strong momentum across the RAP-219 development program, highlighted by compelling new follow-up Phase 2a data recently presented at AAN that further reinforces RAP-219's treatment effect in focal onset seizures and builds confidence as we enter our Phase 3 trials," said Abraham N. Ceesay, chief executive officer of Rapport. "With multiple important development milestones ahead, including continued advancement of our epilepsy franchise, progress in bipolar mania, and development of our long-acting injectable formulation, we believe we are well positioned to continue building value across our pipeline."
CORPORATE HIGHLIGHTS
RAP-219 in Epilepsy
-- Positive Phase 2a Follow-up Period Data Reinforces RAP-219's Sustained
Activity in Focal Onset Seizures. In April 2026, Rapport presented new
8-week follow-up period data from its Phase 2a trial of RAP-219 in
patients with drug-resistant FOS at the American Academy of Neurology
$(AAN)$ Annual Meeting.
-- Therapeutic levels of RAP-219 were sustained, resulting in
continued biomarker and clinical responses in the 8-week follow-up
period (weeks 9-16).
-- RAP-219 continued to demonstrate clinically meaningful
improvements in long episodes (LEs) and clinical seizures during
the follow-up period, with an 80% median reduction in LEs and 90%
median reduction in clinical seizures compared to baseline in
weeks 9-12 and a 68% median reduction in LEs and 59% median
reduction in clinical seizures compared to baseline in weeks
13-16.
-- Based on pharmacokinetic $(PK)$ data collected across the Company's
Phase 1 and Phase 2 trials and further supported by population PK
modeling, RAP-219 is now estimated to have a 22-day half-life,
compared to the prior reported estimate of 14 days.
-- Phase 3 Program in FOS. Following feedback from an end-of-Phase 2 meeting
with the U.S. Food and Drug Administration (FDA) in December 2025, the
Company accelerated the initiation of its Phase 3 program in FOS, which
is on track to begin in the second quarter of 2026.
-- Open-label Trial Initiated. The Company is enrolling patients from its
Phase 2a FOS trial into an open-label, long-term safety trial. Data from
the trial is expected to be released in the second half of 2026.
-- Expansion into Primary Generalized Tonic-Clonic Seizures (PGTCS).
Building on the robust clinical data generated in FOS, Rapport plans to
initiate a Phase 3 trial of RAP-219 in PGTCS in the first half of 2027,
expanding its epilepsy franchise into the most common type of generalized
seizure.
Additional Pipeline Updates
-- Bipolar Mania Phase 2 Trial Topline Results Expected Ahead of Plan.
Enrollment in the Phase 2 trial is progressing well and topline results
are now expected in the fourth quarter of 2026, ahead of the previous
guidance of first half of 2027. Additionally, the Company modified the
trial's statistical analysis plan and increased target enrollment,
enabling the trial to potentially be considered as confirmatory evidence
of effectiveness. Following completion of the Phase 2 trial, and subject
to the results, the Company plans to engage with the FDA in an
End-of-Phase 2 meeting to align on the design of a potential Phase 3
program to support a New Drug Application for the treatment of bipolar
mania.
-- Long-Acting Injectable Formulation Development Continues. Development of
RAP-219's long-acting injectable formulation continues to advance, with
IND-enabling activities underway and initial Phase 1 pharmacokinetic data
expected in 2027.
-- <ALPHA>6<BETA>4 Program Advancing Toward Clinic. Rapport continues
IND-enabling activities for its <ALPHA>6<BETA>4 nAChR agonist development
candidate, which is being developed as a potential novel non-opioid
treatment for chronic pain and migraine.
Business Updates
-- Strategic Collaboration with Tenacia Biotechnology. In March 2026,
Rapport entered into a strategic collaboration and license agreement with
Tenacia Biotechnology (Hong Kong) Co., Ltd. (the Tenacia License
Agreement) for the development and commercialization of RAP-219 in
Greater China across indications, including FOS and bipolar mania.
FIRST QUARTER 2026 FINANCIAL RESULTS
-- Net Loss: Net Loss for the first quarter of 2026 was $19.9 million, as
compared to $24.1 million for the prior year period.
-- Collaboration Revenue: Collaboration revenues were $20.0 million for the
first quarter of 2026, as compared to zero for the prior year period due
to the execution of the Tenacia License Agreement in the first quarter of
2026.
-- Research and Development (R&D) Expenses: R&D expense was $32.7 million
for the first quarter of 2026, as compared to $19.6 million for the prior
year period. The increase in R&D expense was primarily driven by
operational costs related to clinical development and costs to support
the progression of the Company's overall pipeline.
-- General and Administrative (G&A) Expenses: G&A expense was $11.5 million
for the first quarter of 2026, as compared to $7.5 million for the prior
year period. The increase in G&A expense was primarily driven by costs
associated with the growth of the business.
-- Cash Position: The Company ended the first quarter of 2026 with $476.8
million in cash, cash equivalents and short-term investments, excluding
restricted cash, compared to $490.5 million as of December 31, 2025.
-- Cash Runway: The Company expects that cash, cash equivalents, and
short-term investments as of March 31, 2026, will enable it to fund its
operating expenses and capital expenditure requirements into the second
half of 2029.
About RAP-219
RAP-219 is an investigational and potential first-in-class, clinical-stage TARP<GAMMA>8-specific AMPA receptor (AMPAR) negative allosteric modulator (NAM). Whereas AMPARs are distributed widely in the central nervous system, the receptor associated protein (RAP) TARP<GAMMA>8 is expressed only in discrete brain regions, including the hippocampus and neocortex, where focal seizures often originate. By contrast, TARP<GAMMA>8 has minimal expression in the hindbrain, where drug effects are often associated with intolerable adverse events. With this precision approach, the Company believes RAP-219 has the potential to provide a differentiated profile as compared to traditional neuroscience medications. Due to the role of AMPA biology in various neurological disorders and the selective targeting of TARP<GAMMA>8, the Company believes RAP-219 has pipeline-in-a-product potential and is evaluating the compound as a potential treatment for patients with focal onset seizures, primary generalized tonic-clonic seizures and bipolar mania. A long-acting injectable formulation of RAP-219 is also in development and could be the first of its kind in epilepsy.
About Rapport Therapeutics
Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing small molecule precision medicines for patients with neurological and psychiatric disorders. The Company's founders made pioneering discoveries related to the function of receptor associated proteins (RAPs) in the brain, which form the basis of Rapport's RAP technology platform. The platform enables a differentiated approach to generate precision small molecule product candidates with the potential to overcome many limitations of conventional neurology drug discovery. Rapport's precision neuroscience pipeline includes the Company's lead investigational drug, RAP-219, which is designed to achieve neuroanatomical specificity through selective targeting of a RAP expressed only in discrete regions of the brain. The pipeline is anchored by the Company's epilepsy portfolio, including FOS and primary generalized tonic-clonic seizures, as well as bipolar mania. The Company is also advancing additional discovery and preclinical programs leveraging its platform, including in chronic pain and migraine and in hearing and vestibular disorders.
Availability of Other Information About Rapport Therapeutics
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