Praxis to present latest preclinical and clinical advancements across leading epilepsy portfolio at the 2025 American Epilepsy Society (AES) Annual Meeting
BOSTON, Nov. 24, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a clinical-stage biopharmaceutical company translating genetic insights into therapies for central nervous system (CNS) disorders driven by neuronal excitation-inhibition imbalance, today announced they will present the latest preclinical and clinical data and progress on trials across its precision epilepsy pipeline at the American Epilepsy Society Annual (AES) Meeting, December 5-9, 2025, in Atlanta, Georgia.
"The AES meeting is a cornerstone for the epilepsy community, bringing together science, clinical innovation, and patient advocacy," said Steven Petrou, chief scientific officer and co-founder of Praxis. "We're excited to continue showcasing the strength of our precision epilepsy pipeline and the potential to meaningfully impact the lives of people living with epilepsy. Our presentations at AES reflect the continued translation of our science into clinical progress and reinforce our commitment to advancing transformative therapies."
Praxis Presence at AES 2025
Praxis will showcase its precision epilepsy programs through multiple poster and late-breaking presentations, a dedicated scientific exhibit, and interactive sessions at Booth #217. Attendees are invited to connect with the Praxis team to learn more about the company's latest research and commitment to advancing therapies for people living with epilepsy.
BOOTH #217: Praxis team members will be available to discuss preclinical and clinical data as well as progress on trials across its precision epilepsy pipeline, including updates on vormatrigine, relutrigine, and elsunersen.
SCIENTIFIC EXHIBIT: A walkthrough meeting showcasing all of Praxis' conference posters, with team members available to discuss clinical updates and progress for all programs. Sunday, December 7, 2:00–5:00 PM (ET) | Rooms B207 and B208, Georgia World Congress Center
POSTER and LATE-BREAKING PRESENTATIONS: Showcasing the latest data across Praxis's leading pipeline of precision epilepsy programs. Additional presentation details are provided below.
Saturday, December 6 | 12:00 – 2:00 PM (ET) | GWCC, POSTER HALL B2
- 1.503 (Late Breaker): Vormatrigine Rapidly Reduces Seizures in Adults with Treatment-Resistant Epilepsy: Full Results from the RADIANT Study
- 1.38: Relutrigine Demonstrates Sustained Seizure Reduction with Continued Exposure on Top of Standard of Care: Results from the EMBOLD Open Label Extension
Sunday, December 7 | 12:00 – 1:30 PM (ET) | Room B206 (Basic Science Poster Highlights)
- 3.189: PAC-DEE: An Extension of the Praxis Analysis of Concordance Framework for Establishing the Predictive Validity of Preclinical Seizure Models across Broad Developmental and Epileptic Encephalopathies
Monday, December 8 | 12:00 – 1:45 PM (ET) | GWCC, POSTER HALL B3
- 3.189: PAC-DEE: An Extension of the Praxis Analysis of Concordance Framework for Establishing the Predictive Validity of Preclinical Seizure Models across Broad Developmental and Epileptic Encephalopathies
- 3.303: Complementary Antisense Oligonucleotide Treatment and Precision Sodium Channel Modulation for Early Onset SCN2A DEE: Emergency Use Cases in a Preterm Infant with Refractory Status Epilepticus
- 3.341: Preclinical Findings of Relutrigine, a Functional State Sodium Channel Modulator, Point to Anticonvulsant Potential in Dravet Syndrome with Greater Potency than Fenfluramine
- 3.36: Vormatrigine Exhibits a Favorable Drug-Drug Interaction Profile Supporting Broad Combination Use with Antiseizure Medications
- 3.37: Epilepsy Monitoring of Prospective Seizure Observations with Electronic Records (EMPOWER): A Novel, Prospective, Large-scale, Observational Study Designed to Better Understand the Patient Journey
For more information on Praxis's presence at AES 2025, visit http://praxismedicines.com/aes2025.
Materials will be made available on this page and on the Resources section of the Praxis website following presentation at AES 2025.
About Vormatrigine (PRAX-628)
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 POWER1 Study.
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 Elsunersen (PRAX-222)
Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early-seizure-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, with potential to be the first disease-modifying treatment for SCN2A-DEE. Elsunersen has received ODD and RPDD from the FDA, and ODD and PRIME designations from the European Medicines Agency for the treatment of SCN2A-DEE. The elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc. To learn more about the EMBRAVE study, please visit EMBRAVE Study.
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 LinkedIn, Facebook, Instagram 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 plans to initiate new clinical programs, the anticipated timing of regulatory submissions and interactions and our projected cash runway, 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, 2024 and as updated in the Quarterly Report on Form 10-Q for the period 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 [email protected] 857-702-9452 Media Contact: Dan Ferry Life Science Advisors [email protected] 617-430-7576
