Essential3 is the first positive Phase 3 program in essential tremor; ulixacaltamide has received FDA Breakthrough Therapy Designation, with data selected for plenary presentation at AAN 2026
Praxis to present 3 oral presentations and 12 posters at AAN 2026
Visit Praxis at Booth #2324 for an in-booth speaker showcase featuring Phase 3 Essential3 results for ulixacaltamide, plus scientific exchange on essential tremor.

BOSTON, April 13, 2026 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ:PRAX), a fully integrated, leading central nervous system (CNS) precision neuroscience biopharmaceutical company, today announced it will present expanded analyses from the Phase 3 Essential3 program of ulixacaltamide in essential tremor, alongside new data from its advancing CNS pipeline, at the 2026 AAN Annual Meeting, taking place April 18–22, 2026, in Chicago, Illinois.

Essential tremor affects approximately 7 million people in the United States and is one of the most common movement disorders, with few effective treatment options. Ulixacaltamide, a differentiated and highly selective small molecule inhibitor of T-type calcium channels, has received FDA Breakthrough Therapy Designation based on results from the Essential3 study, the first positive Phase 3 program in essential tremor.

"Essential tremor affects millions of people and has seen little therapeutic innovation in decades," said Steven Petrou, Ph.D., co-founder and president of research and development. "We believe the Essential3 program delivers meaningful improvements in daily function for patients with ET. As we advance ulixacaltamide, we remain committed to developing therapies that that addresses the full range of unmet need across neuroscience."

Praxis Presence at AAN 2026

Praxis will present 15 scientific presentations at AAN 2026, led by a plenary presentation highlighting the Essential3 program in essential tremor. Additional presentations include clinical updates from its elsunersen ASO program alongside posters spanning its broader CNS pipeline.

Booth #2324: Praxis team members will be available throughout the meeting to discuss Phase 3 Essential3 data for ulixacaltamide and the unmet need and disease burden in essential tremor, and will host an in-booth speaker showcase focused on Essential3 results. Additional discussions will include the broader CNS pipeline.

Essential3 Plenary Presentation

Tuesday, April 21, 2026 | 9:15 AM–9:30 AM CT | W375de Clinical Trials Plenary

PL5-001: Maintenance of Response and Durability of Effect with Ulixacaltamide in Essential Tremor: Topline Phase 3 Results from Essential3 Study 2 (Randomized Withdrawal Study)

Featured Oral Presentations

Sunday, April 19, 2026 | 4:06 PM–4:18 PM CT | W196b Movement Disorders: Technological Advances in Diagnostics and Therapeutics

S11.004: Automated Quantitative Assessment of Archimedes Spirals in Essential Tremor: Development and Validation of a Regulatory-grade Digital Biomarker Pipeline for the Essential3 Program

Tuesday, April 21, 2026 | 5:18 PM–5:30 PM CT | W192c Epilepsy: Basic Science and Mechanisms

S29-010: Clinical Updates from the Elsunersen Emergency Use Program: A Novel ASO for Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy

Essential Tremor Posters

Monday, April 20, 2026

17-002: Efficacy and Safety of Ulixacaltamide in Essential Tremor: Topline Phase 3 Results from Essential3 Study 1 (Parallel-Design Study) | 11:45 AM–12:45 PM CT | Hall F, Neighborhood 17
17-003: Combined Efficacy and Safety of Ulixacaltamide in Essential Tremor: Topline Results from the Phase 3 Essential3 Program | 11:45 AM–12:45 PM CT | Hall F, Neighborhood 17

CNS Pipeline Posters

Tuesday, April 21, 2026

11-002: 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 | 8:00 AM–9:00 AM CT | Hall F, Neighborhood 11
11-007: High Seizure Burden and Limited Treatment Persistence in Epilepsy: Findings from the EMPOWER Observational Study and a US Real-world Claims Analysis | 8:00 AM–9:00 AM CT | Hall F, Neighborhood 11
14-004: PAC-PAIN: Application of the Praxis Analysis of Concordance Framework for Establishing the Predictive Validity of Preclinical Pain Models | 8:00 AM–9:00 AM CT | Hall F, Neighborhood 14
7-005: Evaluating the Therapeutic Potential of Emerging Precision Sodium Channel Modulators in Pain | 5:00 PM–6:00 PM CT | Hall F, Neighborhood 7

Wednesday, April 22, 2026

11-009: Complementary Antisense Oligonucleotide Treatment and Precision Sodium Channel Modulation for Early Onset SCN2A Developmental and Epileptic Encephalopathy: Emergency Use Case in a Preterm Infant with Refractory Status Epilepticus | 8:00 AM–9:00 AM CT | Hall F, Neighborhood 11
11-010: POWER1 – A Double-blind, Randomized, Multicenter Phase 2/3 Study Evaluating the Efficacy and Safety of Vormatrigine in Adults with Focal Onset Seizures | 8:00–9:00 AM CT | Hall F, Neighborhood 11
10-003: Vormatrigine Rapidly Reduces Seizures in Adults with Treatment-resistant Epilepsy: Results from the RADIANT Study | 11:45 AM–12:45 PM CT | Hall F, Neighborhood 10
10-008: Updates from the First-in-human Phase 1 Clinical Trial Evaluating the Safety, Tolerability, Pharmacokinetics and Food Effect of Vormatrigine in Healthy Participants | 11:45 AM–12:45 PM CT | Hall F, Neighborhood 10
10-009: EMERALD: A Phase 3, Randomized, Multi-center, Double-blind, Placebo-controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants with Developmental and Epileptic Encephalopathies | 11:45 AM–12:45 PM CT | Hall F, Neighborhood 10
10-010: Efficacy and Safety of Relutrigine in Pediatric Participants with SCN2A- and SCN8A-related Developmental and Epileptic Encephalopathies: Pivotal EMBOLD Cohort 2 Study| 11:45 AM–12:45 PM CT | Hall F, Neighborhood 10

For more information on Praxis's presence at AAN 2026, visit https://praxismedicines.com/2026-aan-annual-meeting.

Materials will be made available on the AAN meeting page and on the Resources section of the Praxis website following presentation at AAN 2026.

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 including multiple programs across movement disorders and epilepsy, with four late-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, LinkedIn and X/Twitter.

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 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 POWER1 and POWER2 studies, please visit POWER studies.

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 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 study, please visit Emerald | Resilience 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. 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 EMBRAVE3 study, please visit Embrave | Resilience Studies.

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 clinical trials, the development of Praxis' product candidates and plans to initiate new clinical programs, the anticipated timing of regulatory submissions and interactions, potential market opportunity and commercial potential of Praxis' product candidates 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; 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, 2025 to be filed 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

Praxis to Showcase Essential3, the First Positive Phase 3 Program in Essential Tremor, at AAN 2026