Praxis Precision Medicines Announces Rare Pediatric Disease Designation Granted for Relutrigine in Dravet Syndrome
Praxis Precision Medicines (NASDAQ: PRAX) has received Rare Pediatric Disease Designation (RPDD) from the FDA for relutrigine in treating Dravet syndrome, a genetic developmental and epileptic encephalopathy. This marks the third RPDD for relutrigine, following previous designations for SCN2A and SCN8A DEEs.
The company plans to initiate an all-DEE trial (EMERALD) in 1H2025. Recent clinical results from the EMBOLD study showed promising outcomes, including a 46% placebo-adjusted monthly motor seizure reduction, over 30% of patients achieving seizure freedom, and a 77% reduction in median seizure rate during long-term extension. Praxis is currently enrolling patients in a second, registrational cohort for SCN2A and SCN8A patients, with topline results expected in 1H2026.
Praxis Precision Medicines (NASDAQ: PRAX) ha ricevuto la Designazione di Malattia Pediatrica Rara (RPDD) dalla FDA per relutrigine nel trattamento della sindrome di Dravet, una encefalopatia epilettica e di sviluppo genetica. Questo rappresenta la terza RPDD per relutrigine, dopo le precedenti designazioni per le DEE SCN2A e SCN8A.
L'azienda prevede di avviare uno studio su tutte le DEE (EMERALD) nella prima metà del 2025. Gli recenti risultati clinici dello studio EMBOLD hanno mostrato esiti promettenti, con una riduzione del 46% delle crisi motorie mensili aggiustata per il placebo, oltre il 30% dei pazienti che ha raggiunto la libertà dalle crisi e una riduzione del 77% nel tasso mediano di crisi durante l'estensione a lungo termine. Praxis sta attualmente reclutando pazienti in una seconda coorte registrativa per i pazienti SCN2A e SCN8A, con risultati preliminari attesi nella prima metà del 2026.
Praxis Precision Medicines (NASDAQ: PRAX) ha recibido la Designación de Enfermedad Pediátrica Rara (RPDD) de la FDA para relutrigina en el tratamiento del sindrome de Dravet, una encefalopatía genética del desarrollo y epiléptica. Este es el tercer RPDD para relutrigina, después de las designaciones anteriores para las DEE SCN2A y SCN8A.
La empresa planea iniciar un ensayo clínico para todas las DEE (EMERALD) en la primera mitad de 2025. Los recientes resultados clínicos del estudio EMBOLD mostraron resultados prometedores, incluyendo una reducción del 46% en las convulsiones motoras mensuales ajustadas por placebo, más del 30% de los pacientes logrando la libertad de convulsiones y una reducción del 77% en la tasa mediana de convulsiones durante la extensión a largo plazo. Praxis está actualmente reclutando pacientes para una segunda cohorte registral para pacientes SCN2A y SCN8A, con resultados preliminares esperados en la primera mitad de 2026.
프락시스 프리시전 메디시스 (NASDAQ: PRAX)는 FDA로부터 드라베 증후군 치료를 위한 리루트리진의 희귀 소아 질환 지정을 받았습니다. 이는 리루트리진에 대한 세 번째 RPDD로, SCN2A 및 SCN8A DEE에 대한 이전 지정에 이어 이루어졌습니다.
회사는 2025년 상반기에 모든 DEE에 대한 임상 시험(EMERALD)을 시작할 계획입니다. EMBOLD 연구의 최근 임상 결과는 플라세보를 기준으로 한 월간 운동 발작 감소율이 46%에 달하고, 30% 이상의 환자가 발작 없는 상태를 달성하며, 장기 연장 동안 중간 발작률이 77% 감소하는 등 유망한 결과를 보여주었습니다. 프락시스는 현재 SCN2A 및 SCN8A 환자를 위한 두 번째 등록 코호트를 모집하고 있으며, 주요 결과는 2026년 상반기에 공개될 예정입니다.
Praxis Precision Medicines (NASDAQ: PRAX) a reçu la désignation de Maladie Pédiatrique Rare (RPDD) de la FDA pour le relutrigine dans le traitement du syndrome de Dravet, une encéphalopathie épileptique et du développement génétique. Cela marque la troisième RPDD pour le relutrigine, après les désignations précédentes pour les DEE SCN2A et SCN8A.
L'entreprise prévoit de lancer un essai clinique sur toutes les DEE (EMERALD) au cours du premier semestre 2025. Les résultats cliniques récents de l'étude EMBOLD ont montré des résultats prometteurs, avec une réduction de 46% des crises motrices mensuelles ajustées pour le placebo, plus de 30% des patients atteignant la liberté de crises et une réduction de 77% du taux médian de crises pendant l'extension à long terme. Praxis est actuellement en train de recruter des patients pour une deuxième cohorte d'enregistrement pour les patients SCN2A et SCN8A, avec des résultats préliminaires attendus au premier semestre 2026.
Praxis Precision Medicines (NASDAQ: PRAX) hat von der FDA die Auszeichnung als Seltene Pädiatrische Erkrankung (RPDD) für Relutrigine zur Behandlung des Dravet-Syndroms, einer genetischen Entwicklungs- und Epilepsie-Enzephalopathie, erhalten. Dies ist die dritte RPDD für Relutrigine, nach den vorherigen Auszeichnungen für die DEE SCN2A und SCN8A.
Das Unternehmen plant, in der ersten Hälfte von 2025 eine klinische Studie für alle DEE (EMERALD) zu starten. Die aktuellen klinischen Ergebnisse aus der EMBOLD-Studie zeigten vielversprechende Ergebnisse, darunter eine um 46% reduzierte monatliche motorische Anfallshäufigkeit, über 30% der Patienten erreichten Anfallsfreiheit und eine 77%ige Reduktion der medianen Anfallshäufigkeit während der Langzeitverlängerung. Praxis rekrutiert derzeit Patienten für eine zweite, registrierungsrelevante Kohorte für SCN2A- und SCN8A-Patienten, wobei die Ergebnisse in der ersten Hälfte von 2026 erwartet werden.
- Received FDA Rare Pediatric Disease Designation for relutrigine in Dravet syndrome
- Clinical data shows 46% placebo-adjusted monthly motor seizure reduction
- 77% reduction in median seizure rate in long-term extension
- Over 30% of patients achieved seizure freedom status
- None.
Insights
Dravet syndrome is a genetic developmental and epileptic encephalopathy (DEE) often caused by a mutation in SCN1A
This is the third Rare Pediatric Disease Designation for relutrigine, adding to those granted for SCN2A and SCN8A DEEs
Praxis plans to initiate an all-DEE trial (EMERALD), inclusive of Dravet syndrome, in 1H2025
BOSTON, Dec. 18, 2024 (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 U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for relutrigine in Dravet syndrome.
“We are thrilled to have been granted rare pediatric disease designation for relutrigine in Dravet syndrome. This milestone reflects our commitment to addressing critical unmet needs in rare neurological disorders and underscores the potential of relutrigine as a meaningful new option for patients and families affected by this debilitating developmental and epileptic encephalopathy. The designation builds upon encouraging clinical data demonstrating relutrigine’s ability to reduce seizure frequency and improve overall seizure control, highlighting our dedication to advancing innovative therapies that can significantly impact quality of life for those with limited treatment options,” said Marcio Souza, president and chief executive officer.
Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions primarily affecting children under 18, with fewer than 200,000 cases in the U.S. A key benefit of obtaining a RPDD is the potential to receive a priority review voucher following FDA approval of a product with RPDD if the marketing application submitted for the product satisfies certain conditions. These vouchers can significantly expedite the review process for future New Drug Applications or biologic license applications.
In September, and updated at the recent 2024 American Epilepsy Society Annual Meeting, Praxis shared results for relutrigine in SCN2A and SCN8A patients in cohort 1 of the EMBOLD study that showed:
- Placebo-adjusted monthly motor seizure reduction of
46% during the double-blind period - Over
30% of patients achieved seizure freedom status while on relutrigine - Meaningful gains observed in alertness, communication and seizure severity
77% reduction in median seizure rate seen for patients in the long-term extension
Based on the results of this study, Praxis has initiated and is enrolling patients in a second, registrational cohort for SCN2A and SCN8A patients, expecting topline results in the first half of 2026.
About Relutrigine (PRAX-562)
Relutrigine is a first-in-class small molecule in development for the treatment of developmental and epileptic encephalopathy (DEE) as a preferential inhibitor of persistent sodium current, shown to be a key driver of seizure symptoms in SCN2A-DEE and SCN8A-DEE. Relutrigine’s mechanism of sodium channel modulation is consistent with superior selectivity for disease state sodium channel (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 blocking effects. Relutrigine has received Orphan Drug Designation (ODD) and RPDD from the FDA, and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMBOLD study, please visit https://www.emboldstudy.org/.
About Dravet Syndrome
Dravet syndrome is a severe, progressive genetic epilepsy that typically begins within the first year of life, marked by frequent, prolonged, and treatment-resistant seizures. Beyond seizures, the condition often leads to intellectual disability, developmental delays, movement and balance difficulties, speech and language impairments, growth abnormalities, sleep disturbances, autonomic nervous system dysfunction, and mood disorders. Classified as a developmental and epileptic encephalopathy, Dravet syndrome is associated with significant cognitive and developmental impairments. Patients with this condition also face an elevated risk of early mortality, including due to sudden unexpected death in epilepsy (SUDEP).
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, 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 and the development of our product candidates, 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; preliminary analyses from ongoing studies differing materially from final data from preclinical studies and completed 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, 2023, its Quarterly Reports on Form 10-Q 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.
FAQ
What are the key clinical results of PRAX's relutrigine in the EMBOLD study?
When will PRAX release topline results for the registrational cohort of relutrigine?
What is the significance of the Rare Pediatric Disease Designation for PRAX's relutrigine?
When will PRAX begin the EMERALD trial for relutrigine?
