RAPP

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 α6β4 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, resultin...

Clinically meaningful efficacy demonstrated in the 8-week follow-up period (weeks 9-16), with a 90% median reduction in clinical seizures over baseline in weeks 9-12 and a 59% median reduction in clinical seizures over baseline in weeks 13-16 New data demonstrate that RAP-219 has a half-life of approximately 22 days RAP-219 was generally well tolerated throughout treatment and follow-up periods BOSTON and SAN DIEGO, April 21, 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 announced results from the follow-up period of its Phase 2a trial of RAP-219 in focal onset seizures (FOS) in a late-breaker podium presentation at the 2026 American Academy of Neurology (AAN) Annual Meeting, taking place April 18–22, 2026 in Chicago. In the Phase 2a trial, patients received RAP-219 for 8 weeks and then entered an 8-week follow-up period after treatment ended to evaluate the effect of RAP-219 on long episodes (LEs) – an objective biomarker of epileptiform activity with >90% median concordance with electrographic seizures in this trial – and clinical seizures. Due to RAP-219's long half-life, therapeutic levels of RAP-219 were sustained, resulting in continued biomarker and clinical responses in the 8-week follow-up period. In the first four weeks of follow-up (weeks 9-12), RAP-219 showed even greater reductions in LEs and clinical seizures than were observed during the 8-week treatment period. Clinically meaningful improvements over baseline continued through the second four weeks of follow-up (weeks 13-16). Consolidated Treatment Period and 8-Week Follow-up Period Results 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 an approximately 22-day half-life, compared to the prior reported estimate of 14 days. RAP-219’s plasma concentrations remained within the targeted therapeutic range over the 8-week follow-up period, with observed concentrations consistent with >75% receptor occupancy in weeks 9-12 and 60% to 75% receptor occupancy in weeks 13-16. Patients experienced an 80% median reduction in LEs and 90% median reductio...

Cormorant Asset Management disclosed a sale of 50,000 shares of Praxis Precision Medicines (NASDAQ:PRAX) in a February 17, 2026, SEC filing, with an estimated transaction value of $9.31 million based on the quarter’s average share price. According to a February 17, 2026, SEC filing, Cormorant Asset Management reduced its stake in Praxis Precision Medicines by 50,000 shares during the fourth quarter of 2025. The estimated value of the shares sold was $9.31 million, based on the average unadjusted closing price for the quarter. The quarter-end value of the PRAX stake increased by $227.00 million, a figure that includes both the impact of trading and price movement. Top holdings after the filing: NASDAQ:PRAX: $280.00 million (12.9% of AUM) NASDAQ:BBOT: $223.84 million (12.7% of AUM) NASDAQ:ABVX: $182.05 million (10.3% of AUM) NASDAQ:EYPT: $151.00 million (8.6% of AUM) NASDAQ:RAPP: $91.85 million (5.2% of AUM) As of Monday, PRAX shares were priced at $305.00, up a staggering 700% over the past year and well outperforming the S&P 500’s roughly 19% gain in the same period. Praxis Precision Medicines develops clinical-stage therapies for central nervous system disorders, with lead candidates targeting major depressive disorder, perimenopausal depression, essential tremor, and severe epilepsy. The firm operates a research-driven biopharmaceutical model, advancing proprietary drug candidates through clinical trials and entering into multiple license and collaboration agreements. It targets patients with neurological and psychiatric conditions, as well as healthcare providers and partners in the biotechnology and pharmaceutical sectors. Praxis Precision Medicines is a Boston-based biotechnology company focused on developing innovative therapies for neurological disorders characterized by neuronal imbalance. The company leverages a diversified pipeline of small molecules and antisense oligonucleotides, supported by strategic partnerships and licensing agreements. Its clinical-stage assets and specialized focus position it to address significant unmet medical needs in central nervous system therapeutics. When a biotech stock has such a staggering surge in a single year, trimming a position can be less about losing conviction and more about managing risk during a dramatic run. Praxis Precision Medicines has become one of the standout performers in the neuroscience biotec...

RAP-219’s lead program demonstrated robust and consistent activity in patients with focal onset seizures (FOS) in Phase 2a trial; initiation of Phase 3 program expected in the second quarter of 2026 RAP- 219 epilepsy portfolio expanded with new program in primary generalized tonic-clonic seizures (PGTCS) on strength of Phase 2a FOS data Phase 2 trial in bipolar mania, development of a long-acting injectable, and other pipeline programs continued to progress Entered into a strategic collaboration and license agreement with Tenacia Biotechnology (Hong Kong) Co., Ltd. to develop and commercialize RAP-219 in Greater China across indications, including focal onset seizures and bipolar mania Ended the year with $490.5 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations into the second half of 2029 BOSTON and SAN DIEGO, March 10, 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 and psychiatric disorders, today reported financial results for the quarter and full year ended December 31, 2025, and provided a business update. “2025 was a year of strong momentum for Rapport as we advanced our precision neuroscience pipeline and continued building what we believe can become a leading epilepsy portfolio. The compelling Phase 2a results in focal onset seizures strengthened our confidence in the program’s potential best-in-class profile and support our strategy to advance RAP-219 across multiple opportunities within epilepsy,” said Abraham N. Ceesay, chief executive officer of Rapport. “We also continued to advance several other important initiatives, including our Phase 2 bipolar mania trial, development of a long-acting injectable formulation, and additional pipeline candidates - applying our precision medicine approach to areas of significant unmet need. During the year, we also significantly strengthened our balance sheet through a follow-on offering of our common stock, extending our cash runway and providing us with the financial flexibility to invest in our highest-priority programs.” “With a focused strategy, strong financial foundation, and multiple catalysts ahead, we believe Rapport is well pos...

Positive topline results announced from Phase 2a clinical trial of RAP-219 in patients with focal onset seizures Phase 2 trial of RAP-219 in bipolar mania enrolling patients and on track, with topline results expected in the first half of 2027 Strong balance sheet, bolstered by approximately $269.4 million in net proceeds from recent public offering Ended the quarter with $513.0 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations into the second half of 2029 BOSTON and SAN DIEGO, Nov. 06, 2025 (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 third quarter ending September 30, 2025, and provided a business update. “The positive Phase 2a results for RAP-219 announced in September underscore both the strength of our precision neuroscience platform and the compound’s differentiated clinical potential,” said Abraham N. Ceesay, chief executive officer of Rapport. “Achieving a nearly 78% median reduction in clinical seizures and seizure freedom in nearly one in four patients positions RAP-219 with a potential best-in-class profile for the treatment of drug-resistant focal onset seizures. With a strengthened balance sheet following our recent public offering, we are in a strong financial position to execute our Phase 3 trials in focal onset seizures and continue advancing our bipolar Phase 2 trial toward topline results expected in the first half of 2027. We remain committed to disciplined execution and delivering sustained value for both patients and shareholders.” BUSINESS HIGHLIGHTS RAP-219 Lead Program Focal Onset Seizures (FOS) Successful Phase 2a Topline Results Announced. In September, the Company reported topline results from its single arm Phase 2a clinical trial of RAP-219 in patients with drug-resistant FOS (n=30). The trial met its primary long episode (LE) endpoints with high statistical significance. The Company expects to present further efficacy analyses later in 2025 and additional data, including results from the 8-week follow-up, in 2026. In the 8-week treatment period, 85.2% of patients achieved ≥30% reduction in LEs...

Earlier this week, Rapport Therapeutics announced positive Phase 2a trial results for RAP-219 in refractory focal epilepsy, demonstrating best-in-class efficacy and good tolerability. This milestone attracted renewed analyst optimism, highlighting the significant commercial potential for RAP-219 in central nervous system disorders. We'll examine how the robust clinical data for RAP-219 positions Rapport Therapeutics to reshape its investment narrative around breakthrough treatments. Uncover the next big thing with financially sound penny stocks that balance risk and reward. To see Rapport Therapeutics as a compelling opportunity, investors need to believe in the transformative potential of RAP-219 in central nervous system disorders, not just the latest positive data. This week’s strong Phase 2a results clearly change the short-term outlook: a robust clinical update for RAP-219 moves the company closer to a meaningful commercial pipeline and may ease some near-term doubt over its path to revenue, especially after its recent equity raise. That said, RAP-219 still faces standard industry hurdles, regulatory approval, larger pivotal trials, and the task of scaling up in a crowded neurology market. Previous top risks like lack of revenue, serial losses, and ongoing shareholder dilution remain relevant, but with momentum swinging in their favour, the narrative could shift if the results spark deeper investor or partner interest. The risk for investors is that clinical promise still needs to turn into concrete milestones and, eventually, cash flows. But given past volatility and ongoing losses, investors should not lose sight of dilution risk. Insights from our recent valuation report point to the potential overvaluation of Rapport Therapeutics shares in the market. The Simply Wall St Community’s single retail fair value estimate for Rapport Therapeutics sits at US$51.71, consistent across all submissions. While optimism has firmed up with recent clinical trial success, the ongoing risk of future capital raises is something every investor needs to factor into their view of what the company might ultimately be worth. Investor opinions still vary widely, so explore several viewpoints before deciding. Explore another fair value estimate on Rapport Therapeutics - why the stock might be worth as much as 80% more than the current price! Disagree with this assessment? Cr...

Trial met primary long episode endpoints with high statistical significance, and RAP-219 was generally well tolerated Patients achieved 77.8% reduction in clinical seizures (p=0.01), with 24% achieving seizure freedom for the 8-week treatment period (p<0.0001) Data support advancement of RAP-219 into Phase 3 registrational trials Company to host a conference call today at 8:00 a.m. ET BOSTON and SAN DIEGO, Sept. 08, 2025 (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 announced that the Phase 2a clinical trial of RAP-219 (RAP-219-FOS-201) in patients with drug-resistant focal onset seizures met its primary endpoint, demonstrating a statistically significant reduction in long episodes (LEs) – 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. RAP-219 was generally well tolerated. The Company plans to advance RAP-219 into two Phase 3 pivotal trials in the third quarter of 2026. RAP-219 is a potential first-in-class, investigational TARPγ8-specific AMPAR negative allosteric modulator. “Despite the available therapies, up to 40% of patients with focal epilepsy continue to experience seizures. There is still tremendous need for additional effective anti-seizure medications with novel mechanisms of action. Physicians and patients need new options that deliver meaningful benefits and the potential to offer the promise of seizure freedom,” said Jacqueline French, M.D., principal investigator of the study and professor in the Department of Neurology at NYU Langone Health's Comprehensive Epilepsy Center. “This trial represents the first time a novel antiseizure medication was evaluated in focal seizure patients using the RNS system with an objective biomarker of seizure activity. The magnitude of the reduction in clinical seizure frequency seen in this trial, and the corroboration of the clinical activity from the objective biomarker, give me confidence that a medication like RAP-219 has the potential to be a highly effecti...

BOSTON and SAN DIEGO, Sept. 05, 2025 (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 announced that it will host a conference call and live webcast at 8:00 am ET on September 8, 2025, to report topline results for the Phase 2a trial of RAP-219 in patients with drug-resistant focal onset seizures. Individuals interested in listening to the live conference call may do so through this webcast link or by dialing (800) 715-9871 in the U.S. or (646) 307-1963 for other locations and reference conference ID 4762775. A webcast replay will be available from the “Investors” section of the Company’s website here for 90 days following the completion of the call. About Rapport Therapeutics Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing small molecule precision medicines for patients with neurological or psychiatric disorders. The Company’s founders have made pioneering discoveries related to the function of receptor associated proteins (RAPs) in the brain. Their findings form the basis of Rapport’s RAP technology platform, which 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, designed to achieve neuroanatomical specificity through its selective targeting of a RAP expressed in only discrete regions of the brain. The Company is currently pursuing RAP-219 as a potential treatment for drug-resistant focal onset seizures, bipolar mania and diabetic peripheral neuropathic pain. Additional preclinical and late-stage discovery stage programs are also underway, including targeting chronic pain and hearing disorders. Contact Julie DiCarlo Head of Communications & IR Rapport Therapeutics [email protected]

RAP-219 Phase 2a trial in patients with drug-resistant focal onset seizures is fully enrolled and on track for topline results in September 2025 Phase 2 trial of RAP-219 in bipolar mania has been initiated and is enrolling patients; trial remains on track, with topline results anticipated in the first half of 2027 Ended the quarter with $260.4 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations through the end of 2026 BOSTON and SAN DIEGO, Aug. 07, 2025 (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 second quarter ending June 30, 2025, and provided a business update. “We remain on track to report topline results in September from our Phase 2a trial of RAP-219 in patients with focal onset seizures,” said Abraham N. Ceesay, Chief Executive Officer of Rapport Therapeutics. “This upcoming readout will be a pivotal milestone for our lead program and an opportunity to demonstrate the strength of our precision neuroscience approach. I’m also pleased to share that we have initiated our Phase 2 trial of RAP-219 in bipolar mania, and we are on track to report topline results in the first half of 2027. This is an important step forward as we advance RAP-219 into new areas of high unmet need and further build out our pipeline. Our team remains focused on executing these critical milestones and continuing to invest in our promising precision neuroscience discovery programs. We believe this disciplined approach positions us to deliver transformative treatments for patients and drive long-term value for our shareholders.” BUSINESS HIGHLIGHTS RAP-219 Lead Program Topline Results for RAP-219 Phase 2a Focal Onset Seizure Trial Expected in September 2025. The Phase 2a trial of RAP-219 in drug-resistant focal onset seizures is fully enrolled and on track, with topline results expected in September 2025. This proof-of-concept trial, designed with input from leading epilepsy experts, uses intracranial electroencephalography (iEEG) data from the RNS System to assess RAP-219’s potential effect on long episodes (LEs), an objective biomarker shown to c...

RAP-219 Phase 2a trial in patients with refractory focal epilepsy remains on track with topline results expected in the third quarter of 2025 RAP-219 Phase 2a trial in patients with bipolar mania remains on track to commence in the third quarter of 2025, with topline results expected in the first half of 2027 Company to host inaugural Investor and Analyst Day on June 2, 2025, featuring presentations from management team and key opinion leader Dr. Jacqueline French Ended the quarter with $285.4 million in cash, cash equivalents, and short-term investments, excluding restricted cash, which is expected to fund operations through the end of 2026 BOSTON and SAN DIEGO, May 08, 2025 (GLOBE NEWSWIRE) -- Rapport Therapeutics, Inc. (Nasdaq: RAPP), 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 first quarter ending March 31, 2025, and provided a business update. “2025 represents a transformational year for Rapport as we prepare to deliver our initial proof-of-concept data for RAP-219 from the Phase 2a trial in patients with refractory focal epilepsy and progress our pipeline-in-a-product strategy with the initiation of our bipolar mania trial. Rapport’s first quarter demonstrated our continued ability to execute our clinical development efforts, and we remain confident that we are on track to deliver on our development milestones. Enrollment in the Phase 2a trial in patients with refractory focal epilepsy is progressing as planned, and we are observing a growing level of excitement in the epilepsy community for the data, given the innovative trial design and its potential to evolve epilepsy drug development,” said Abraham N. Ceesay, CEO of Rapport. “The recent PET and MAD-2 trial results further validate the neuroanatomical specificity of TARPγ8 and favorable tolerability profile of RAP-219. When combined with the strong preclinical anti-seizure effects seen, these findings reinforce our confidence in RAP-219’s differentiated precision profile.” BUSINESS HIGHLIGHTS RAP-219 Lead Program PET Data Demonstrated Regionally Selective Expression of TARPγ8 in Epilepsy-Associated Brain Regions. In January, the Company announced results from its healthy volunteer PET trial for RAP-219. The data showed that neur...