Praxis Precision Medicines Provides Vormatrigine Program Update
Rhea-AI Summary
Praxis Precision Medicines (NASDAQ: PRAX) reported topline Phase 2/3 POWER1 results for vormatrigine in focal onset seizures. The primary endpoint, percent change in monthly seizure frequency, was not met. A key secondary endpoint, 50% responder rate, was achieved, with more pronounced seizure reduction at 30 mg in the study’s second half.
Vormatrigine was generally well-tolerated, with adverse event-related discontinuations under 10%, and about 90% of treated patients entered and remain in the open-label extension. Praxis is pausing enrollment in POWER2 while reassessing the vormatrigine program and continues preparing planned launches for relutrigine and ulixacaltamide.
AI-generated analysis. Not financial advice.
Positive
- Secondary 50% responder rate endpoint was achieved in POWER1
- Seizure reduction was more pronounced on 30 mg dose in later study period
- Adverse event-related discontinuations were under 10%, indicating favorable tolerability
- Approximately 90% of vormatrigine patients transitioned to and remain in the OLE study
Negative
- POWER1 study did not meet its primary endpoint of seizure frequency reduction
- Enrollment in the POWER2 vormatrigine study is paused for program reassessment
Market Reaction – PRAX
Following this news, PRAX has declined 10.62%, reflecting a significant negative market reaction. Our momentum scanner has triggered 4 alerts so far, indicating moderate trading interest and price volatility. The stock is currently trading at $299.55. This price movement has removed approximately $1.11B from the company's valuation.
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Key Figures
Market Reality Check
Peers on Argus
PRAX is down 0.75% while key biotech peers show mixed moves: AVXL +2.71%, EYPT +0.59%, SANA +5.43%, NKTR -0.15%, ZBIO -0.86%, indicating a stock-specific reaction to the vormatrigine update.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| May 07 | Earnings and pipeline | Positive | -1.4% | Q1 2026 results, $1.4B cash, NDA progress, epilepsy readout timelines. |
| May 05 | Inducement equity grants | Neutral | +1.4% | Restricted stock unit grants to new employees under 2024 Inducement Plan. |
| May 04 | Earnings call scheduling | Neutral | -1.3% | Announcement of Q1 2026 earnings date and investor conference participation. |
| Apr 14 | Ulixacaltamide NDA accepted | Positive | +8.4% | FDA acceptance of NDA for ulixacaltamide in essential tremor with PDUFA date. |
| Apr 13 | Phase 3 data showcase | Positive | +0.3% | Expanded Essential3 Phase 3 data presentations and Breakthrough Therapy designation. |
Recent clinically positive or regulatory milestones (e.g., NDA acceptances) have generally seen aligned positive price reactions, while broader corporate updates with strong fundamentals have shown at least one divergence.
Over the last few months, Praxis reported multiple late-stage CNS milestones. In April 2026, FDA acceptance of the ulixacaltamide NDA with a January 29, 2027 PDUFA date and Essential3 Phase 3 data supported a positive move. Additional Essential3 data at AAN 2026 was followed by a modest gain. In May 2026, Q1 results highlighted a $1.4B cash position and 2028 funding runway, yet the stock slipped. Today’s vormatrigine POWER1 miss contrasts with earlier epilepsy and tremor successes, adding nuance to the pipeline narrative.
Market Pulse Summary
This announcement reported that the POWER1 study in highly refractory focal onset seizures did not meet its primary endpoint, although a 50% responder secondary measure and higher-dose signal were observed, with discontinuations under 10% and about 90% of patients entering the OLE. Praxis paused POWER2 enrollment to reassess vormatrigine, while previously advancing ulixacaltamide and relutrigine toward PDUFA dates. Investors may watch for updated development plans, balance across the CNS portfolio, and how future data clarify vormatrigine’s risk–benefit profile.
Key Terms
focal onset seizures medical
adverse event medical
open label extension medical
phase 2/3 medical
double-blind medical
randomized medical
multicenter medical
anti-seizure medications medical
AI-generated analysis. Not financial advice.
POWER1 Study in highly refractory patients with focal onset seizures did not meet its primary success measure
Secondary measure, the
Vormatrigine was generally well-tolerated; adverse event-related discontinuations were less than
Approximately
Praxis is pausing enrollment in the POWER2 study to reassess the vormatrigine program and determine potential modifications
BOSTON, June 01, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced results from the Phase 2/3 POWER1 study evaluating vormatrigine in patients with focal onset seizures (FOS).
“While the results for POWER1 were not what we hoped for, we are encouraged by the signal we saw on the higher dose arm, the low discontinuation rate and solid safety profile,” said Marcio Souza, President and Chief Executive Officer of Praxis. “We will take some time to review these results to ensure we have the best path forward for developing vormatrigine and the ongoing POWER2 study. We continue to focus on preparing for the planned launches for relutrigine and ulixacaltamide.”
POWER1 (NCT06999902) is a double-blind, randomized, multicenter Phase 2/3 trial that evaluated the safety and efficacy of vormatrigine in adults with FOS who are concurrently taking at least 1 but no more than 3 acceptable anti-seizure medications (ASMs). Patients were randomized to receive either once daily 20 mg/day of vormatrigine for 6 weeks and then once daily 30 mg/day of vormatrigine for another 6 weeks or once daily placebo for 12 weeks. The primary endpoint was the percent change in monthly seizure frequency from baseline.
About Vormatrigine
Vormatrigine is a next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium-channels in the brain that is currently being developed as a once daily, oral treatment for adult focal onset seizures and generalized epilepsy. Preclinical data demonstrate vormatrigine is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy. In vitro, vormatrigine has demonstrated superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of vormatrigine have demonstrated unprecedented potency in the maximal electroshock seizure (MES) model, a highly predictive translational model for efficacy in focal epilepsy. Data from patients in the RADIANT study demonstrated a robust seizure reduction and generally safe and well tolerated profile.
About Praxis
Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four late-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, Instagram, LinkedIn and Twitter/X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the development of our product candidates, including vormatrigine, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.
The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2025 and other filings made with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of Its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.
Investor Contact: Praxis Precision Medicines investors@praxismedicines.com 857-702-9452 Media Contact: Dan Ferry Life Sci Advisors Daniel@lifesciadvisors.com 617-430-7576