Praxis Precision Medicines Announces Plans to File an NDA for Relutrigine in SCN2A and SCN8A Developmental and Epileptic Encephalopathies in Early 2026
Rhea-AI Summary
Praxis Precision Medicines (NASDAQ: PRAX) announced that following a planned discussion with the FDA on Dec 11, 2025, the agency confirmed the company may file a New Drug Application (NDA) for relutrigine in early 2026 for SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The company said EMBOLD study data will serve as the basis for efficacy and safety, and described EMBOLD results as statistically significant and clinically meaningful improvements. Management characterized the FDA interaction as collaborative and supportive of the planned NDA timeline.
Positive
- NDA filing confirmed with FDA for early 2026
- EMBOLD trial data identified as basis for efficacy and safety
- Statistically significant and clinically meaningful EMBOLD results
Negative
- Not approved yet; NDA submission and review remain future steps
- Clinical-stage company without commercial product revenue
News Market Reaction – PRAX
In the Dec 11 session, PRAX gained 2.18%, reflecting a moderate positive market reaction.
Data tracked by StockTitan Argus on the day of publication.
Historical Context
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Dec 09 | FDA pathway alignment | Positive | -4.7% | FDA alignment on simplified, accelerated registrational path for elsunersen. |
| Dec 08 | Clinical data update | Positive | +9.3% | Strong relutrigine and vormatrigine seizure reduction data presented at AES. |
| Dec 04 | Topline EMBOLD results | Positive | +3.0% | EMBOLD relutrigine data in SCN2A/SCN8A DEEs with early stop for efficacy. |
| Dec 04 | Pre-NDA meeting | Positive | +3.0% | Positive pre-NDA FDA feedback for ulixacaltamide in essential tremor. |
| Dec 03 | Inducement grants | Neutral | +3.9% | Equity inducement grants to new employees under 2024 Inducement Plan. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Recent clinically positive and FDA-alignment announcements have usually coincided with positive price reactions, with one notable selloff on good news.
Over the last week, PRAX has reported a series of late-stage CNS and epilepsy updates. Positive EMBOLD data for relutrigine in SCN2A and SCN8A DEEs on Dec 4 and AES presentations on Dec 8 both saw gains of 3.05% and 9.27%. FDA alignment on a simplified elsunersen pathway on Dec 9 drew a -4.74% reaction, contrasting with earlier strength. Today’s NDA-planning news for relutrigine builds directly on the EMBOLD topline and prior FDA discussions.
Key Terms
fda regulatory
new drug application (nda) regulatory
developmental and epileptic encephalopathies medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
EMBOLD data to serve as basis of efficacy and safety following discussion with the FDA
BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the successful completion of the planned discussion with the FDA leading to the confirmation to file a New Drug Application (NDA) for relutrigine in early 2026.
“We are very pleased with the collaborative discussions we've had with the FDA, which support our path toward an NDA submission in early 2026,” said Marcio Souza, president and chief executive officer. “Building on the unprecedented and compelling results from the EMBOLD study, where relutrigine demonstrated statistically significant and clinically meaningful improvements, we are advancing toward delivering a much-needed therapy for children living with these devastating diseases.”
About Relutrigine (PRAX-562)
Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathies (DEEs) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in severe DEEs. Relutrigine’s mechanism of precision sodium channel (NaV) modulation is consistent with superior selectivity for disease-state NaV channel hyperexcitability. In vivo studies of relutrigine have demonstrated dose-dependent inhibition of seizures up to complete control of seizure activity in SCN2A, SCN8A and other DEE mouse models. Relutrigine has been generally well-tolerated in three Phase 1 studies and has demonstrated biomarker changes indicative of NaV channel modulation. Data from the Phase 2 EMBOLD study demonstrated a well-tolerated safety profile with robust, short- and long-term improvement in motor seizures in a heavily pre-treated population, alongside maintained seizure freedom in some patients with SCN2A- and SCN8A-DEE. Relutrigine has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD), and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMERALD and EMBOLD studies, please visit ResilienceStudies.com.
About Praxis
Praxis Precision Medicines is a clinical-stage 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 clinical-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 Praxis’ product candidates and the anticipated timing of regulatory submissions and interactions, 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, 2024 and as updated in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as well as 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 Science Advisors Daniel@lifesciadvisors.com 617-430-7576