Detailed launch readiness for relutrigine and ulixacaltamide, with PDUFA target action dates of December 27, 2026 and January 29, 2027
Shared updated long-term efficacy data for both relutrigine and ulixacaltamide
Outlined plans to extend ulixacaltamide into Parkinson’s disease, Restless Legs Syndrome, and Autism Spectrum Disorder, with studies initiating in 2027
Introduced a broadened pipeline with multiple assets in Pain, KCNT1 DEEs and the Solidus™ antisense oligonucleotide (ASO) Platform
BOSTON, Nov. 12, 2026 (GLOBE NEWSWIRE) — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today hosted its Investor Day in New York. Management presented its commercial strategy and launch plans for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) and ulixacaltamide in essential tremor (ET), and provided an update on its expanded development pipeline.
“Praxis is preparing to launch the first therapies designed specifically for SCN2A and SCN8A DEEs and for ET, if approved. Today we showed how our teams are ready to bring both treatments to patients in the coming months. We also provided exciting updates about our portfolio, including expanding ulixacaltamide to new indications, our sodium channel program in pain and additional programs in rare epilepsies. With the launches approaching quickly and the expanded pipeline we introduced today, we are well positioned to become the world’s preeminent CNS company over the next decade,” said Marcio Souza, president and chief executive officer.
Investor Day Highlights
Commercial Strategy and Launch Readiness
Praxis detailed its commercial strategy and launch readiness for relutrigine (SCN2A and SCN8A DEEs) and ulixacaltamide (ET). The FDA has set PDUFA target action dates of December 27, 2026 for relutrigine and January 29, 2027 for ulixacaltamide. Key opinion leaders joined management to discuss the unmet need and treatment landscape in each indication.
- Praxis outlined its commercial investments in data, patient services and field team hiring and training, which lay the foundation for the upcoming ulixacaltamide and relutrigine launches and for future launches.
- Hiring for the field force is on track and building momentum ahead of the upcoming launches:
- The ulixacaltamide field team of about 300 will be fully hired before year-end to cover about 19,000 target health care professionals (HCPs).
- The relutrigine field team is fully hired and has profiled 100% of its top 10 accounts per region and 100% of Tier 1 and Tier 2 target HCPs ahead of the PDUFA date.
Ulixacaltamide
- Praxis provided an updated population-based prevalence estimate for ET of about 9 million people in the U.S., including about 2 million who are seeking treatment today and who represent Praxis’ initial commercial focus.
- Provided sensitivity analyses of the Essential3 primary endpoint, including key sensitivity analyses requested by FDA during the New Drug Application (NDA) review, which were consistent with the primary result.
- Shared an early analysis of patients in the Essential3 Long-Term Safety Study who completed recent efficacy assessments after receiving ulixacaltamide for approximately 95 weeks, which showed durable and improving ADL response over time, further supporting ulixacaltamide’s use as a chronic therapy.
Relutrigine
- Praxis shared updated open label extension data from the EMBOLD study in SCN2A- and SCN8A-DEEs, showing continued reduction in seizures through 18 months with 86% reduction in seizures from baseline.
- For the ongoing EMERALD study in broad DEEs, shared the baseline characteristics showing that patients had a median of 58 seizures in the previous 28 days at baseline and were on a median of 3 anti-seizure medications, highlighting the severity of the studied population.
Pipeline Updates
Movement Disorders Expansion
- Praxis plans to develop and initiate studies in 2027 with ulixacaltamide in Parkinson’s disease (about 1 million patient prevalence), restless legs syndrome (5 to 8 million patient prevalence) and autism spectrum disorder (8 million patient prevalence).
- Praxis will advance two new molecular entities that optimize the t-type calcium channel mechanism of action for the treatment of movement disorders, with expected first in human studies in 2027.
- Praxis continues to make progress on its recent collaboration with Remagine Labs to develop an iontophoretic transdermal patch for ulixacaltamide. The patch is designed to improve accessibility for ulixacaltamide and is planned to enter the clinic in 2027.
Pain
- Praxis introduced a two-pronged approach utilizing its expertise in sodium channel modulation with a pan-sodium channel strategy and a NaV1.8 isoform biased strategy for developing therapies for chronic and acute pain indications.
- Praxis is utilizing its proprietary Praxis Analysis of Concordance (PAC) framework in pain to identify an optimized set of pre-clinical models for screening and de-risking development candidates.
- PRAX-215 is a pan-sodium channel molecule currently being evaluated in a Phase 1 SAD/MAD study with a readout expected in 2027 and is being carried forward into chronic and acute pain trials.
- PRAX-110 is an isoform biased molecule and is expected to enter the clinic in 2028.
Other Pipeline Updates
- Praxis introduced PRAX-206 in KCNT1 developmental epilepsies, which has shown strong pre-clinical data and is planned to enter the clinic in early 2027.
- Shared a portfolio of six preclinical ASO programs being advanced within the Solidus™ platform, in addition to elsunersen, which is in pivotal development.
Webcast and Presentation
A replay of the Investor Day webcast and a copy of the presentation are available in the Events and Presentations section of the Praxis investor website.
About Ulixacaltamide
Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide has received Breakthrough Therapy Designation from the FDA and is the most advanced program within Praxis’ Cerebrum™ small molecule platform.
About Relutrigine
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 (RPDD) from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome; as well as Breakthrough Therapy Designation (BTD) from the FDA, and ODD from the European Medicines Agency for the treatment of SCN2A-DEE and SCN8A-DEE. To learn more about the EMERALD study, please visit Emerald | Resilience Studies.
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 patients in the RADIANT study demonstrated a robust seizure reduction and generally safe and well tolerated profile. To learn more about the POWER2 study, please visit POWER studies.
About Elsunersen
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. The elsunersen program is ongoing under a collaboration with Ionis Pharmaceuticals, Inc., and RogCon, Inc. Elsunersen holds Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease Designations from the FDA, and Orphan Drug and PRIME designations from the EMA. To learn more about the EMBRAVE3 study, please visit Embrave | Resilience Studies.
About Praxis
Praxis Precision Medicines is a fully integrated, leading central nervous system (CNS) precision neuroscience 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 across a range of conditions, with four late-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, LinkedIn and X/Twitter.
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 development of Praxis’ product candidates, the anticipated timing of regulatory submissions, approvals and interactions, the potential market opportunity and commercial potential of Praxis’ product candidates, Praxis’ commercial launch plans and readiness, and the anticipated timing of clinical trials and plans to initiate new clinical programs, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “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: the timing of and Praxis’ ability to obtain regulatory approval of its product candidates; Praxis’ ability to build commercial capabilities and successfully launch and commercialize its product candidates, if approved; the potential addressable market sizes for its product candidates; uncertainties inherent in clinical trials and preclinical development; the expected timing of clinical trials, data readouts and the results thereof; 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, 2025 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 LifeSci Advisors Daniel@lifesciadvisors.com 617-430-7576
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