Praxis Precision Medicines Announces Positive, Best-in-Disease Topline Results in Patients with Focal Onset Seizures from the RADIANT Study of Vormatrigine
Praxis Precision Medicines (NASDAQ: PRAX) announced positive topline results from its Phase 2 RADIANT study of vormatrigine for focal onset seizures. The study demonstrated a 56.3% median reduction in seizure frequency over 8 weeks, with 22% of patients achieving complete seizure freedom in the final 28 days of treatment.
Key highlights include 54% of patients achieving 50% response in the first week of treatment. Vormatrigine showed a favorable safety profile and demonstrated advantages as a fast-acting, no-titration, once-daily oral drug with no food requirements. The company plans to complete its pivotal POWER1 study in Q4 and initiate POWER2 study based on these results.
Additional data will be presented at the 36th International Epilepsy Congress on August 31, 2025, with plans to present full study results at the American Epilepsy Society Annual Meeting in December 2025.
Praxis Precision Medicines (NASDAQ: PRAX) ha annunciato risultati positivi preliminari dal suo studio di Fase 2 RADIANT su vormatrigine per le crisi epilettiche a esordio focale. Lo studio ha mostrato una riduzione mediana del 56,3% nella frequenza delle crisi nell鈥檃rco di 8 settimane, con il 22% dei pazienti che ha raggiunto la completa libert脿 da crisi negli ultimi 28 giorni di trattamento.
I punti salienti includono il 54% dei pazienti che ha ottenuto una risposta del 50% gi脿 nella prima settimana di trattamento. La vormatrigine ha evidenziato un profilo di sicurezza favorevole e vantaggi come farmaco orale a somministrazione giornaliera unica, rapido nell鈥檃zione, senza necessit脿 di titolazione e senza restrizioni alimentari. L鈥檃zienda prevede di completare lo studio cardine POWER1 nel quarto trimestre e di avviare lo studio POWER2 basandosi su questi risultati.
Ulteriori dati saranno presentati al 36掳 Congresso Internazionale sull鈥橢pilessia il 31 agosto 2025, con l鈥檌ntenzione di presentare i risultati completi dello studio all鈥橝merican Epilepsy Society Annual Meeting nel dicembre 2025.
Praxis Precision Medicines (NASDAQ: PRAX) anunci贸 resultados positivos preliminares de su estudio de Fase 2 RADIANT sobre vormatrigina para crisis focales. El estudio mostr贸 una reducci贸n mediana del 56,3% en la frecuencia de crisis durante 8 semanas, con un 22% de los pacientes alcanzando libertad total de crisis en los 煤ltimos 28 d铆as de tratamiento.
Los aspectos destacados incluyen que el 54% de los pacientes logr贸 una respuesta del 50% en la primera semana de tratamiento. Vormatrigina mostr贸 un perfil de seguridad favorable y ventajas como un medicamento oral de acci贸n r谩pida, sin necesidad de titulaci贸n y con una sola dosis diaria, sin requerimientos alimentarios. La compa帽铆a planea completar su estudio pivotal POWER1 en el cuarto trimestre e iniciar el estudio POWER2 bas谩ndose en estos resultados.
Se presentar谩n datos adicionales en el 36潞 Congreso Internacional de Epilepsia el 31 de agosto de 2025, con planes de presentar los resultados completos del estudio en la Reuni贸n Anual de la American Epilepsy Society en diciembre de 2025.
Praxis Precision Medicines (NASDAQ: PRAX)電� 甑唽 氚滌瀾 旃橂毳� 鞙勴暅 vormatrigine鞚� 2靸� RADIANT 鞐瓣惮鞐愳劀 旮嶌爼鞝侅澑 欤检殧 瓴瓣臣毳� 氚滍憸頄堨姷雼堧嫟. 鞐瓣惮 瓴瓣臣 8欤� 霃欖晥 氚滌瀾 牍堧弰臧� 欷戩暀臧� 旮办 56.3% 臧愳唽頄堨溂氅�, 旃橂 毵堨毵� 28鞚� 霃欖晥 22%鞚� 頇橃瀽臧� 鞕勳爠頃� 氚滌瀾 鞛愳湢毳� 雼劚頄堨姷雼堧嫟.
欤检殧 雮挫毄鞙茧電� 旃橂 觳� 欤检棎 54%鞚� 頇橃瀽臧� 50% 氚橃潙鞚� 氤挫瀯鞚� 韽暔霅╇媹雼�. Vormatrigine鞚� 鞎堨爠靹� 頂勲頃勳澊 鞖办垬頃橁碃 牍犽ジ 鞛戩毄, 鞖╇焿 臁办爤 攵堩晞鞖�, 頃橂（ 頃� 氩� 氤奠毄頃橂姅 瓴疥惮 鞎诫搿滌劀 鞁濎偓 鞝滍暅鞚� 鞐嗠姅 鞛レ爯鞚� 鞛呾頄堨姷雼堧嫟. 須岇偓電� 鞚� 瓴瓣臣毳� 氚旐儠鞙茧 4攵勱赴鞐� 欷戩稊鞝� POWER1 鞐瓣惮毳� 鞕勲頃橁碃 POWER2 鞐瓣惮毳� 鞁滌瀾頃� 瓿勴殟鞛呺媹雼�.
於旉皜 雿办澊韯半姅 2025雲� 8鞗� 31鞚� 鞝�36須� 甑牅 臧勳 頃欗殞鞐愳劀 氚滍憸霅� 鞓堨爼鞚措┌, 2025雲� 12鞗� 氙戈淡 臧勳 頃欗殞 鞐半須岇潣鞐愳劀 鞝勳泊 鞐瓣惮 瓴瓣臣毳� 氚滍憸頃� 瓿勴殟鞛呺媹雼�.
Praxis Precision Medicines (NASDAQ: PRAX) a annonc茅 des r茅sultats positifs pr茅liminaires de son 茅tude de phase 2 RADIANT sur la vormatrigine pour les crises focales. L鈥櫭﹖ude a montr茅 une r茅duction m茅diane de 56,3% de la fr茅quence des crises sur 8 semaines, avec 22% des patients ayant atteint une libert茅 compl猫te de crises lors des 28 derniers jours de traitement.
Les points cl茅s incluent 54% des patients ayant obtenu une r茅ponse de 50% d猫s la premi猫re semaine de traitement. La vormatrigine a pr茅sent茅 un profil de s茅curit茅 favorable et des avantages en tant que m茅dicament oral 脿 action rapide, pris une fois par jour sans titration ni contraintes alimentaires. La soci茅t茅 pr茅voit de terminer son 茅tude pivot POWER1 au quatri猫me trimestre et de lancer l鈥櫭﹖ude POWER2 sur la base de ces r茅sultats.
Des donn茅es suppl茅mentaires seront pr茅sent茅es lors du 36e Congr猫s International de l鈥櫭塸ilepsie le 31 ao没t 2025, avec l鈥檌ntention de pr茅senter les r茅sultats complets de l鈥櫭﹖ude lors de la r茅union annuelle de l鈥橝merican Epilepsy Society en d茅cembre 2025.
Praxis Precision Medicines (NASDAQ: PRAX) gab positive Zwischenergebnisse aus der Phase-2-Studie RADIANT mit Vormatrigin bei fokalen Anf盲llen bekannt. Die Studie zeigte eine median 56,3%ige Reduktion der Anfallsh盲ufigkeit 眉ber 8 Wochen, wobei 22% der Patienten in den letzten 28 Tagen der Behandlung anfallsfrei waren.
Wichtige Highlights sind, dass 54% der Patienten bereits in der ersten Behandlungswoche eine 50%ige Ansprechrate erzielten. Vormatrigin zeigte ein g眉nstiges Sicherheitsprofil und Vorteile als schnell wirkendes, einmal t盲glich oral einzunehmendes Medikament ohne Titrationsbedarf und ohne Nahrungsbeschr盲nkungen. Das Unternehmen plant, die entscheidende POWER1-Studie im vierten Quartal abzuschlie脽en und basierend auf diesen Ergebnissen die POWER2-Studie zu starten.
Weitere Daten werden auf dem 36. Internationalen Epilepsiekongress am 31. August 2025 pr盲sentiert, mit der Absicht, die vollst盲ndigen Studienergebnisse auf der Jahrestagung der American Epilepsy Society im Dezember 2025 vorzustellen.
- Achieved 56.3% median reduction in seizure frequency over 8 weeks
- 22% of patients reached complete seizure freedom in last 28 days
- Rapid response with 54% of patients achieving 50% response in first week
- Favorable safety profile with well-tolerated treatment
- Differentiated profile as fast-acting, no-titration, once-daily oral drug
- Additional long-term efficacy data still pending
- Full study results not yet presented
- Pivotal POWER1 study completion still pending
Insights
Praxis reports exceptional Phase 2 results for vormatrigine in epilepsy with 56.3% seizure reduction and rapid onset of action.
The Phase 2 RADIANT study results for vormatrigine demonstrate remarkable efficacy in treating focal onset seizures. The 56.3% median reduction in seizure frequency over 8 weeks substantially exceeds typical benchmarks for anti-seizure medications (ASMs). Even more striking is that 22% of patients achieved complete seizure freedom in the final 28 days of treatment鈥攁 gold standard outcome in epilepsy care.
What truly differentiates vormatrigine is its rapid onset of action, with over 54% of patients achieving a 50% response in the first week. Current sodium channel blockers typically require weeks of titration before reaching therapeutic effectiveness. The lack of titration requirement, once-daily dosing, and food-independent administration collectively represent significant clinical advantages over existing treatments.
The company's characterization of these results as "best-in-disease" appears justified based on the efficacy metrics. For context, most approved ASMs demonstrate median seizure reductions in the 20-40% range in similar patient populations. The favorable safety profile mentioned further enhances vormatrigine's potential clinical value.
The POWER1 pivotal study completion expected in Q4 and planned initiation of POWER2 suggest the company is advancing rapidly toward potential regulatory submission. For the 3+ million patients with common epilepsy in the U.S., many requiring polytherapy, a single agent with this efficacy profile could significantly improve treatment paradigms if these results are replicated in Phase 3 studies.
Dosing with vormatrigine over 8 weeks led to
Approximately
Rapid and sustained response, with over
Vormatrigine was generally well tolerated and continues to demonstrate favorable safety profile
BOSTON, Aug. 04, 2025 (GLOBE NEWSWIRE) -- , 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 topline results from the Phase 2 RADIANT study evaluating vormatrigine in patients with focal onset seizures.
鈥淲e are thrilled by the best-in-disease topline results of this first cohort for the RADIANT study and for the potential to deliver a fast-acting, highly efficacious and tolerable therapy to patients living with focal epilepsy. Many of the over three million patients living with common epilepsy in the U.S. need to manage multiple anti-seizure medications, and vormatrigine is the only drug in development aiming to become first line and address the market at large.鈥�
鈥淭hese findings build on our earlier clinical data showing a differentiated profile for vormatrigine as a fast-acting, no-titration, once-daily oral drug with no requirement to be taken with food, and a favorable DDI profile, all of which are unseen in ASMs currently in the market or in development. We are well on track to complete the pivotal, 12-week POWER1 study in Q4 and, based on the results from RADIANT, expect to initiate the POWER2 study shortly. We are deeply grateful to the patients, caregivers and clinicians who are contributing to the vormatrigine trials,鈥� said Marcio Souza, president and chief executive officer of Praxis.
鈥淲hile several sodium channel therapies are already used to treat focal onset seizures, there remains substantial room for improvement within this class. A next-generation sodium channel blocker has the potential to represent an important step forward in addressing that gap. I'm encouraged by the initial results of the RADIANT study and hopeful that, in the future, we may be able to offer patients another effective treatment option,鈥� said Jacqueline French, MD, Professor at NYU Langone Health's Comprehensive Epilepsy Center.
Scientific Presentations
Additional data will be presented at the 36th International Epilepsy Congress on August 31, 2025, in Lisbon, Portugal.
Praxis has submitted a late-breaker abstract to present the full study results at the American Epilepsy Society Annual Meeting in December 2025 in Atlanta, Georgia.
Conference Call
Praxis will discuss the study results, as well as its second quarter 2025 financial results and business highlights on a conference call taking place today, August 4, 2025 at 8:30 am ET. Individuals may register for the conference call by clicking the . Once registered, participants will receive dial-in details and a unique PIN which will allow them to access the call. An audio webcast will be accessible through the under the Investor Relations section of the Company鈥檚 website.
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 demonstrates 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 the first cohort of patients in the RADIANT study demonstrated a robust seizure reduction and generally safe and well tolerated profile. To learn more about the POWER1 study, please visit .
About the RADIANT study:
Link to the RADIANT study 听听
About Praxis鈥�
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鈩�, and antisense oligonucleotide (ASO) platform, Solidus鈩�, 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听听and follow us on听,听,听听补苍诲听.
Forward-Looking Statements听
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鈥� future expectations, plans and prospects, including, without limitation, statements regarding the clinical development and potential benefits of vormatrigine, the anticipated timing of our clinical trials and our participation in upcoming events and presentations, as well as other statements containing the words 鈥渁nticipate,鈥� 鈥渂elieve,鈥� 鈥渃ontinue,鈥� 鈥渃ould,鈥� 鈥渆ndeavor,鈥� 鈥渆stimate,鈥� 鈥渆xpect,鈥� 鈥渁nticipate,鈥� 鈥渋ntend,鈥� 鈥渕ay,鈥� 鈥渕ight,鈥� 鈥減lan,鈥� 鈥減otential,鈥� 鈥減redict,鈥� 鈥減roject,鈥� 鈥渟eek,鈥� 鈥渟hould,鈥� 鈥渢arget,鈥� 鈥渨ill鈥� or 鈥渨ould鈥� 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鈥� 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鈥� 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:听 Praxis Precision Medicines听 [email protected]听 857-702-9452听 听 Media Contact: Dan Ferry Life Science Advisors [email protected] 617-430-7576
FAQ
What were the key results of Praxis Precision Medicines' (PRAX) RADIANT study for vormatrigine?
How quickly does vormatrigine work for seizure control?
What are the advantages of vormatrigine compared to existing seizure medications?
When will Praxis (PRAX) present the complete RADIANT study results?
What is the market potential for vormatrigine in epilepsy treatment?
