Praxis Precision Medicines to Showcase Updates from Largest Epilepsy Pipeline of Precision Epilepsy Programs at the 2024 American Epilepsy Society Annual Meeting
Praxis Precision Medicines (NASDAQ: PRAX) announced its participation in the 2024 American Epilepsy Society Annual Meeting, where it will present data from three epilepsy programs. The company will showcase updates through five poster presentations featuring their clinical-stage assets, including relutrigine and vormatrigine, described as the most potent and selective anti-seizure medications developed to date. The presentations will focus on preclinical and clinical data, including results from the EMBOLD study in childhood epilepsies. The event will be held from December 6-10, 2024, in Los Angeles, with Praxis exhibiting at booth #1235 and hosting a scientific exhibit.
Praxis Precision Medicines (NASDAQ: PRAX) ha annunciato la sua partecipazione al 2024 American Epilepsy Society Annual Meeting, dove presenterà dati di tre programmi per l'epilessia. L'azienda mostrerà aggiornamenti attraverso cinque presentazioni con manifesti che presenteranno le proprie risorse in fase clinica, inclusi relutrigine e vormatrigine, descritti come i farmaci anti-sequestro più potenti e selettivi sviluppati fino ad oggi. Le presentazioni si concentreranno su dati preclinici e clinici, inclusi i risultati dello studio EMBOLD nelle epilessie infantili. L'evento si terrà dal 6 al 10 dicembre 2024, a Los Angeles, con Praxis che esporrà allo stand #1235 e ospiterà una mostra scientifica.
Praxis Precision Medicines (NASDAQ: PRAX) anunció su participación en el 2024 American Epilepsy Society Annual Meeting, donde presentará datos de tres programas de epilepsia. La empresa mostrará actualizaciones a través de cinco presentaciones de póster que destacarán sus activos en etapa clínica, incluyendo relutrigine y vormatrigine, descritos como los medicamentos antiepilépticos más potentes y selectivos desarrollados hasta la fecha. Las presentaciones se centrarán en datos preclínicos y clínicos, incluidos los resultados del estudio EMBOLD en epilepsias infantiles. El evento se llevará a cabo del 6 al 10 de diciembre de 2024 en Los Ángeles, con Praxis exhibiendo en el stand #1235 y organizando una exhibición científica.
프락시스 정밀 의학(Praxis Precision Medicines, NASDAQ: PRAX)은 2024 미국 간질 학회 연례 회의에 참가하여 세 가지 간질 프로그램의 데이터를 발표할 것이라고 발표했습니다. 이 회사는 relutrigine과 vormatrigine을 포함한 임상 단계 자산을 특징으로 하는 다섯 개의 포스터 발표를 통해 업데이트를 선보일 예정입니다. 이들 약물은 현재까지 개발된 가장 강력하고 선택적인 항경련제입니다. 발표는 소아 간질에서의 EMBOLD 연구 결과를 포함한 전임상 및 임상 데이터에 집중할 것입니다. 이 행사는 2024년 12월 6일부터 10일까지 로스앤젤레스에서 열리며, 프락시스는 부스 #1235에서 전시하고 과학 전시를 주최할 것입니다.
Praxis Precision Medicines (NASDAQ: PRAX) a annoncé sa participation à la 2024 American Epilepsy Society Annual Meeting, où elle présentera des données de trois programmes sur l'épilepsie. L'entreprise mettra en avant des mises à jour à travers cinq présentations sous forme de poster mettant en lumière ses actifs en phase clinique, y compris relutrigine et vormatrigine, décrits comme les médicaments anti-crise les plus puissants et sélectifs développés à ce jour. Les présentations se concentreront sur des données précliniques et cliniques, y compris les résultats de l'étude EMBOLD sur l'épilepsie infantile. L'événement se déroulera du 6 au 10 décembre 2024 à Los Angeles, avec Praxis exposant au stand #1235 et organisant une exposition scientifique.
Praxis Precision Medicines (NASDAQ: PRAX) gab die Teilnahme am 2024 American Epilepsy Society Annual Meeting bekannt, wo Daten aus drei Epilepsieprogrammen präsentiert werden. Das Unternehmen wird Aktualisierungen über fünf Posterpräsentationen zeigen, die ihre klinischen Vermögenswerte, einschließlich relutrigine und vormatrigine, vorstellen, die als die leistungsstärksten und selektivsten Antikonvulsiva gelten, die bisher entwickelt wurden. Die Präsentationen werden sich auf präklinische und klinische Daten konzentrieren, einschließlich der Ergebnisse der EMBOLD-Studie zu Epilepsien bei Kindern. Die Veranstaltung findet vom 6. bis 10. Dezember 2024 in Los Angeles statt, wobei Praxis am Stand #1235 ausstellt und eine wissenschaftliche Ausstellung veranstaltet.
- Promising results reported from relutrigine EMBOLD study in childhood epilepsies
- Company advancing multiple clinical-stage assets in epilepsy treatment
- Development of potentially leading anti-seizure medications (relutrigine and vormatrigine)
- None.
BOSTON, Nov. 26, 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 that that it will present preclinical and clinical data from three of its epilepsy programs at the American Epilepsy Society (AES) Annual Meeting, being held from December 6 to 10, 2024 in Los Angeles, California.
“At Praxis, we are on the brink of transformative change with a leading pipeline that includes relutrigine and vormatrigine, the most potent and most functionally selective anti-seizure medications developed to date,” said Steven Petrou, chief scientific officer and co-founder of Praxis. “Following promising results with the relutrigine EMBOLD study in particularly challenging childhood epilepsies, we are confident that these future therapies will redefine treatment for DEEs as well as focal and generalized epilepsy. This year at AES we will be sharing the latest advances across our epilepsy portfolio through an exciting lineup of activities.”
Praxis will have multiple options for visitors to learn more about its portfolio:
- Exhibiting at booth #1235, where visitors can interact with members of the Praxis team
- Presenting five unique posters covering three of its clinical-stage assets, detailed below
- Hosting a scientific exhibit featuring its leading portfolio of precision epilepsy programs, detailed below
Poster Presentations
- Saturday December 7, 12:00 p.m. – 2:00 p.m. PST
- Location: South Hall H, Level 1
1.398. Vormatrigine Demonstrates Potent Antiseizure Activity Across Three Acute Models with Highest Predictive Validity for Focal Onset Seizures
1.525. Emergency Use Case of Relutrigine, a Next-Generation Sodium Channel Functional State Modulator, in an Infant with SCN2A-DEE and Refractory Seizures and Recurrent Status Epilepticus
1.526. Clinical Updates from the Elsunersen Emergency Use Program: A Novel ASO for Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy
1.527. Establishing the Predictive Validity of Preclinical Seizure Models in Generalized Epilepsies: An Extension of the Praxis Analysis of Concordance Framework
1.528. Relutrigine Demonstrates Robust Seizure Reduction and Seizure Freedom in DEEs: Results from the EMBOLD Study
Materials will be made available on the Resources page of the Praxis website following presentation at AES 2024: https://praxismedicines.com/resources/.
Scientific Exhibit
- Monday December 9, 2:00 p.m. – 5:00 p.m. PST
- Location: Room 403B, Level 2
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 in a heavily pre-treated population a well-tolerated, robust, short- and long-term improvement in motor seizures 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, 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.com/.
About Vormatrigine (PRAX-628)
Vormatrigine is a next-generation, functionally selective small molecule targeting the hyperexcitable state of NaV 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 demonstrates vormatrigine is differentiated from standard of care, with the potential to be best-in-class for focal onset seizures. 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 the PRAX-628-101 study demonstrated that vormatrigine can be safely dosed in healthy subjects to greater than 15 times the predicted human equivalent of the rodent MES EC50, a translational indicator that suggests a therapeutic window with unprecedented magnitude relative to approved therapies.
About Elsunersen (PRAX-222)
Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-onset SCN2A-DEE to treat seizures and other symptoms in patients with gain-of-function SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models. Data from the EMBRAVE study demonstrated well-tolerated, significant and sustained seizure reduction in patients with SCN2A-DEE. Elsunersen has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD) from the FDA, and ODD and PRIME designations from the European Medicines Agency (EMA) for the treatment of SCN2A-DEE. The Elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), and RogCon, Inc. To learn more about the EMBRAVE study, please visit https://www.embravestudy.org/.
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.
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