Praxis Precision Medicines Receives FDA Breakthrough Therapy Designation for Relutrigine for the Treatment of Seizures Associated with SCN2A and SCN8A Developmental and Epileptic Encephalopathies
Praxis Precision Medicines (NASDAQ: PRAX) has received FDA Breakthrough Therapy Designation (BTD) for relutrigine in treating seizures associated with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs). The designation was based on compelling Phase 2 EMBOLD trial results showing:
The trial demonstrated 46% placebo-adjusted monthly motor seizure reduction during the 16-week double-blind period, with over 30% of patients achieving seizure freedom. In the 11-month open-label extension, patients showed ~90% seizure reduction and increased seizure-free periods from 3 to 67 days.
The company expects topline results from the registrational EMBOLD cohort 2 study in H1 2026, followed by an NDA filing. Additionally, Praxis has initiated the EMERALD study investigating relutrigine across all DEEs.
Praxis Precision Medicines (NASDAQ: PRAX) ha ottenuto la Designazione di Terapia Sperimentale Innovativa (BTD) dalla FDA per relutrigine nel trattamento delle crisi epilettiche associate alle encefalopatie epilettiche e dello sviluppo (DEE) legate ai geni SCN2A e SCN8A. La designazione si basa sui risultati convincenti dello studio di Fase 2 EMBOLD, che ha mostrato:
Lo studio ha evidenziato una riduzione del 46% delle crisi motorie mensili corretta per il placebo durante il periodo in doppio cieco di 16 settimane, con oltre il 30% dei pazienti che ha raggiunto la libertà dalle crisi. Nell’estensione in aperto di 11 mesi, i pazienti hanno mostrato una riduzione delle crisi di circa il 90% e un aumento dei periodi senza crisi da 3 a 67 giorni.
L’azienda prevede i risultati principali dello studio EMBOLD di registrazione, coorte 2, nella prima metà del 2026, seguiti dalla presentazione della domanda di autorizzazione al mercato (NDA). Inoltre, Praxis ha avviato lo studio EMERALD per valutare relutrigine in tutte le DEE.
Praxis Precision Medicines (NASDAQ: PRAX) ha recibido la Designación de Terapia Innovadora (BTD) por parte de la FDA para relutrigina en el tratamiento de convulsiones asociadas con encefalopatías epilépticas y del desarrollo (DEE) relacionadas con SCN2A y SCN8A. La designación se basa en los resultados convincentes del ensayo de Fase 2 EMBOLD, que mostraron:
El ensayo demostró una reducción del 46% en las convulsiones motoras mensuales ajustada por placebo durante el período de 16 semanas a doble ciego, con más del 30% de los pacientes alcanzando la libertad de convulsiones. En la extensión abierta de 11 meses, los pacientes mostraron una reducción de convulsiones de aproximadamente el 90% y un aumento en los períodos libres de convulsiones de 3 a 67 días.
La compañía espera los resultados principales del estudio EMBOLD de registro, cohorte 2, en la primera mitad de 2026, seguidos por la presentación de la solicitud de aprobación (NDA). Además, Praxis ha iniciado el estudio EMERALD para investigar relutrigina en todas las DEE.
Praxis Precision Medicines (NASDAQ: PRAX)는 SCN2A 및 SCN8A 발달 및 간질 뇌병증(DEEs)과 관련된 발작 치료를 위한 렐루트리진에 대해 FDA 혁신 치료 지정(BTD)을 받았습니다. 이 지정은 2상 EMBOLD 시험의 설득력 있는 결과를 기반으로 합니다:
시험에서는 16주간의 이중맹검 기간 동안 플라시보를 보정한 월간 운동 발작 46% 감소를 보여주었으며, 30% 이상의 환자가 발작 완전 관해를 달성했습니다. 11개월간의 공개 연장 기간 동안 환자들은 약 90%의 발작 감소와 발작 없는 기간이 3일에서 67일로 증가했습니다.
회사는 등록용 EMBOLD 2코호트 연구의 주요 결과를 2026년 상반기에 기대하고 있으며, 이후 신약 승인 신청(NDA)을 진행할 예정입니다. 또한 Praxis는 모든 DEEs를 대상으로 렐루트리진을 조사하는 EMERALD 연구를 시작했습니다.
Praxis Precision Medicines (NASDAQ : PRAX) a obtenu la désignation de thérapie innovante (BTD) de la FDA pour le relutrigine dans le traitement des crises associées aux encéphalopathies épileptiques développementales (DEE) liées aux gènes SCN2A et SCN8A. Cette désignation repose sur des résultats convaincants de l’essai de phase 2 EMBOLD, montrant :
L’essai a démontré une réduction mensuelle des crises motrices ajustée de 46 % par rapport au placebo durant la période en double aveugle de 16 semaines, avec plus de 30 % des patients atteignant une liberté totale de crises. Lors de l’extension en ouvert de 11 mois, les patients ont présenté une réduction d’environ 90 % des crises et une augmentation des périodes sans crises de 3 à 67 jours.
L’entreprise prévoit les résultats principaux de l’étude EMBOLD de cohorte 2 à visée d’enregistrement au premier semestre 2026, suivis par un dépôt de demande d’autorisation de mise sur le marché (NDA). De plus, Praxis a lancé l’étude EMERALD qui évalue le relutrigine sur l’ensemble des DEE.
Praxis Precision Medicines (NASDAQ: PRAX) hat von der FDA die Breakthrough Therapy Designation (BTD) für Relutrigine zur Behandlung von Anfällen im Zusammenhang mit SCN2A- und SCN8A-entwicklungsbedingten epileptischen Enzephalopathien (DEEs) erhalten. Die Bezeichnung basiert auf überzeugenden Ergebnissen der Phase-2-EMBOLD-Studie, die Folgendes zeigten:
Die Studie zeigte während des 16-wöchigen doppelblinden Zeitraums eine 46%ige Reduktion motorischer Anfälle pro Monat, bereinigt um Placebo, wobei über 30% der Patienten anfallsfrei wurden. In der 11-monatigen offenen Verlängerung zeigten die Patienten eine etwa 90%ige Anfallsreduktion und eine Verlängerung der anfallsfreien Intervalle von 3 auf 67 Tage.
Das Unternehmen erwartet die Topline-Ergebnisse der registrierenden EMBOLD-Kohorte 2 Studie in der ersten Hälfte 2026, gefolgt von einer Zulassungsanmeldung (NDA). Zudem hat Praxis die EMERALD-Studie gestartet, die Relutrigine bei allen DEEs untersucht.
- FDA Breakthrough Therapy Designation received for relutrigine, enabling expedited development and review
- Impressive 90% seizure reduction in 11-month open-label extension data
- Significant increase in seizure-free periods from 3 to 67 days
- Strong safety profile with no new safety signals or drug-related serious adverse events
- Expansion of potential market with new EMERALD study across all DEEs
- Final topline results not expected until H1 2026
- NDA filing timeline dependent on successful completion of cohort 2 study
Insights
FDA Breakthrough Therapy Designation significantly accelerates relutrigine's path to approval for rare epilepsies with strong clinical data.
The FDA's Breakthrough Therapy Designation (BTD) for relutrigine represents a substantial regulatory achievement for Praxis Precision Medicines. This designation is only granted when preliminary clinical evidence indicates a drug may offer significant advantages over existing therapies for serious conditions – in this case, rare genetic epilepsies (SCN2A and SCN8A developmental and epileptic encephalopathies).
The clinical data supporting this designation is particularly compelling. The Phase 2 EMBOLD trial demonstrated a
This BTD, combined with the previously received Rare Pediatric Disease Designation, places relutrigine on an expedited regulatory pathway. The company is progressing with their registrational EMBOLD cohort 2 study, with topline results expected by H1 2026, followed by NDA filing. Additionally, Praxis has initiated the EMERALD study to evaluate relutrigine more broadly across all developmental epileptic encephalopathies, significantly expanding the potential patient population.
For patients with these severe, treatment-resistant epilepsies who typically fail multiple therapies, relutrigine could represent a transformative option. The reported improvements in alertness and communication, alongside seizure control, suggest meaningful quality-of-life benefits. The clean safety profile to date further enhances the drug's potential value proposition in these vulnerable pediatric populations.
The Breakthrough Therapy Designation (BTD) was granted based on the highly compelling results from the Phase 2 EMBOLD trial in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs)
The EMBOLD cohort 2 pivotal trial is on track for topline results in H1 2026 with NDA filing to follow
Praxis has recently initiated the EMERALD study investigating relutrigine broadly in DEEs
BOSTON, July 17, 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 that the U.S. Food and Drug Administration (FDA) has granted BTD for relutrigine, a sodium channel functional state modulator for pediatric use for the treatment of patients with SCN2A and SCN8A DEEs.
“This BTD represents a significant milestone for our relutrigine program and further validates its potential. The EMBOLD cohort 1 study supporting our application enrolled the most severe DEE population ever studied and included patients that failed three treatments on average before joining the study. The results from the open-label extension are remarkable, showing an average of both approximately
The BTD enables expedited development and regulatory review for drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that relutrigine may demonstrate substantial improvement on a clinically significant endpoint(s) over existing therapies. The BTD for relutrigine was supported by the positive data from cohort 1 in the Phase 2 EMBOLD study, as well as 11-month data from the open-label extension (OLE) period of the trial. Data from the double-blind period (16 weeks of treatment) showed a placebo-adjusted monthly motor seizure reduction of
The EMBOLD registrational cohort 2 is currently ongoing and continues to enroll, with topline results expected no later than the first half of 2026, followed by a potential NDA filing.
Praxis has recently initiated the EMERALD study evaluating relutrigine in patients across all DEEs. EMERALD is a registrational, 16-week, placebo-controlled study evaluating seizure reduction in patients diagnosed with developmental epilepsies.
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 cohort 1 of the Phase 2 EMBOLD study demonstrated a well-tolerated, 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 epilepsy and movement disorders, with four clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, 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 anticipated timing of our clinical trials, the development of our 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 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
FAQ
What is the significance of FDA Breakthrough Therapy Designation for PRAX's relutrigine?
What were the key results from PRAX's EMBOLD trial for relutrigine?
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What is the EMERALD study that PRAX recently initiated?
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