ulixacaltamide (PRAX-944) / Praxis Precision Medicines - LARVOL DELTA

A Breakthrough in Essential Tremor: Phase 3 Evidence for Ulixacaltamide as the First Targeted Therapy (MDS Congress 2026) - "Lunch and refreshments will be provided. Ulixacaltamide is an investigational therapy and has not been approved by any regulatory authority."

P3 data • CNS Disorders • Essential Tremor • Movement Disorders

Ulixacaltamide Demonstrates Superiority Over Placebo as Monotherapy and as Add-on to Propranolol in Essential Tremor: Phase 3 ESSENTIAL3 Study 1 (MDS Congress 2026) - No abstract available

Clinical • Head-to-Head • Monotherapy • P3 data • CNS Disorders • Essential Tremor • Movement Disorders

Ulixacaltamide Demonstrates Broad Functional Improvement Across Individual Domains of Activities of Daily Living in Essential Tremor (MDS Congress 2026) - No abstract available

Clinical • CNS Disorders • Essential Tremor • Movement Disorders

Non-surgical treatment of essential tremor: What's in store? (PubMed, Parkinsonism Relat Disord) - "While ACP-711 is a newly investigated positive allosteric modulator (PAM) of GABA-A receptors, other PAMs (SAGE-324 and PRAX-114) have been withdrawn after failure to demonstrate statistically significant effects in clinical trials. This has also extended to include highly selective Cav3 antagonists (T-type calcium channel blockers): suvecaltamide (JZP385) and NBI-827104/ACT-7099478. By contrast, ulixacaltamide (PRAX-944) has been recently found to be effective and safe in a large phase 3 trial. ES-481, a novel selective antagonist of transmembrane AMPA receptor regulatory protein (TARP)-γ8-dependent AMPA receptors, has been tested in a small trial in Canada, complicated by early participant drop-out...Although many compounds have failed during advanced clinical development, others remain in the pipeline. This renewed interest in the treatment of ET, along with a better understanding of its pathophysiology and clinical trial design, could ultimately lead to newer..."

Journal • Review • Essential Tremor • Movement Disorders • Orthopedics • CAV3

Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET) (clinicaltrials.gov) - P3 | N=1000 | Active, not recruiting | Sponsor: Praxis Precision Medicines | Enrolling by invitation ➔ Active, not recruiting | Trial completion date: Dec 2026 ➔ Jul 2028

Enrollment closed • Trial completion date • Essential Tremor • Movement Disorders

Praxis Precision Medicines and Remagine Labs Announce Strategic Collaboration to Develop Transdermal Ulixacaltamide for Essential Tremor (GlobeNewswire) - "Under the agreement, Praxis and Remagine will collaborate to apply Remagine's proprietary platform, designed for precise, multi-day active transdermal dosing, to ulixacaltamide, complementing Praxis's lead oral program. A transdermal option is intended to broaden the population ulixacaltamide can reach, while strengthening ulixacaltamide's competitive positioning and extending the long-term value of the franchise. Praxis has also made an equity investment in Remagine Labs, underscoring the companies' shared commitment to quickly advancing the program."

Licensing / partnership • Essential Tremor

Cannabidiol reduces atypical absence seizures and epileptic spasms in a Gabrb3+/D120N mouse model of Lennox-Gastaut syndrome. (PubMed, Epilepsia Open) - "Lennox-Gastaut syndrome (LGS) is a rare type of epilepsy that's hard to treat and poses a challenge for developing new drugs. Finding suitable animal models that accurately represent LGS is crucial. This article describes the development of a mouse model of LGS with a genetic mutation that increases seizures and epileptic spasms. We tested how different antiseizure drugs affect the mice. CBD, ethosuximide, and ulixacaltamide reduced seizure incidence and duration. CBD and vigabatrin also reduced spasm frequency in young mice. These promising results suggest the mouse model could be a valuable tool for drug discovery in LGS."

Journal • Preclinical • Absence Seizure Disorder • CNS Disorders • Epilepsy

Recent Highlights and Anticipated Milestones (GlobeNewswire) - "The FDA has accepted Praxis’ NDA for ulixacaltamide for the treatment of ET and has set a target action date under the Prescription Drug User Fee Act (PDUFA) of January 29, 2027...The FDA has accepted with priority review the relutrigine NDA for the treatment of SCN2A and SCN8A DEEs, with a PDUFA target action date of September 27, 2026...Assuming successful initial NDA approval of relutrigine, the EMERALD study, if positive, would serve as the basis for a supplemental NDA submission in 2027."

PDUFA • CNS Disorders • Epilepsy • Essential Tremor

Automated Quantitative Assessment of Archimedes Spirals in Essential Tremor: Development and Validation of a Regulatory-grade Digital Biomarker Pipeline for the Essential3 Program (AAN 2026) - "Participants in two randomized, double-blind Phase 3 studies assessing the impact of ulixacaltamide in essential tremor (parallel-design and randomized-withdrawal; Essential3) drew large and small Archimedes spirals with their dominant hand at baseline, Day 56, and Day 84. Scanned spiral drawings were processed through a pipeline performing automated template removal, skeletonization, and extraction of 12 quantitative features reflecting tremor amplitude, irregularity, and complexity...Conclusions The Essential3 Spiral Analysis Initiative established a fully automated, validated, digital pipeline transforming hand-drawn spirals into quantitative, reproducible digital features. Integration of normative data and advanced machine-learning models establishes a scalable framework for regulatory-grade digital biomarkers of motor function in patients with tremor, suitable for use in large-scale clinical trials."

Biomarker • Essential Tremor • Movement Disorders

Combined Efficacy and Safety of Ulixacaltamide in Essential Tremor: Topline Results from the Phase 3 Essential3 Program (AAN 2026) - "Despite its prevalence, there have been no new treatments for ET since propranolol’s approval ~40 years ago, with most therapies discontinued because of tolerability or modest efficacy. Conclusions Essential3 is the only ongoing Phase 3 Program in ET, designed to evaluate the clinical benefit of pharmacologic TTCC modulation with ulixacaltamide. Positive topline findings highlight ulixacaltamide’s potential as a first-in-class therapy poised to redefine the ET treatment landscape for the first time in several decades."

Clinical • P3 data • P3 data: top line • Essential Tremor • Movement Disorders

Efficacy and Safety of Ulixacaltamide in Essential Tremor: Topline Phase 3 Results from Essential3 Study 1 (Parallel-Design Study) (AAN 2026) - "Conclusions Essential3 is the only ongoing Phase 3 Program in ET, with Study 1 representing the first registrational study exploring the efficacy and tolerability of pharmacologic TTCC modulation with ulixacaltamide. Topline results are anticipated to significantly redefine the ET treatment landscape."

Clinical • P3 data • P3 data: top line • CNS Disorders • Essential Tremor • Movement Disorders

Maintenance of Response and Durability of Effect with Ulixacaltamide in Essential Tremor: Topline Phase 3 Results from Essential3 Study 2 (Randomized Withdrawal Study) (AAN 2026) - "Essential3 is the only ongoing Phase 3 Program in ET, with Study 2 representing the first registrational study exploring the maintenance of effect following pharmacologic TTCC modulation with ulixacaltamide. Findings highlight ulixacaltamide's potential to redefine the treatment landscape and address the persisting impact on daily life in patients with ET."

Clinical • P3 data • P3 data: top line • Essential Tremor • Movement Disorders

Praxis Precision Medicines Announces FDA Acceptance of New Drug Application for Ulixacaltamide HCl in Patients with Essential Tremor (The Manila Times) - "FDA assigned PDUFA target action date of January 29, 2027; No advisory committee meeting expected...The NDA is supported by positive results from the Essential3 Phase 3 program, which comprised two simultaneously enrolled pivotal studies in adults with essential tremor."

FDA filing • PDUFA • Essential Tremor

Recent Highlights and Anticipated Milestones (GlobeNewswire) - "Following a positive pre-NDA meeting with the FDA in December 2025, Praxis has submitted an NDA for the treatment of ET. Praxis will present several oral presentations and posters on ulixacaltamide at the upcoming American Academy of Neurology (AAN) Annual Meeting, taking place April 19 to 22, 2026 in Chicago, IL. Commercial preparations and pre-launch activities are well underway and will accelerate through 2026."

Clinical data • FDA filing • Essential Tremor

What’s Ahead in 2026 and Beyond (GlobeNewswire) - "NDA submission to the FDA is expected by mid-February 2026...NDA submission to the FDA is expected by mid-February 2026 for SCN2A- and SCN8A-DEE...The EMERALD study enrolling broad DEEs is expected to complete in the second half of 2026. Assuming successful NDA approval of relutrigine, the EMERALD study, if positive, would serve as the basis for an sNDA submission by 2027....Clinical data from the EMBRAVE3 study, if successful, would serve as the basis for an NDA submission in 2027."

FDA filing • Trial status • Epilepsy • Essential Tremor

Praxis Precision Medicines Announces the FDA Has Granted Breakthrough Therapy Designation for Ulixacaltamide HCl in Essential Tremor (GlobeNewswire) - "The BTD for ulixacaltamide was based on the positive topline data from the Essential3 program, consisting of two pivotal Phase 3 studies of ulixacaltamide in ET....The Company has aligned with the FDA on the content of the NDA and expects to submit the ulixacaltamide NDA in early 2026."

Breakthrough therapy • FDA filing • Essential Tremor

Praxis Precision Medicines…announced the successful completion of its pre-NDA (New Drug Application) meeting with the FDA, including receipt of written feedback and an in-person meeting (GlobeNewswire) - "Praxis has gained alignment from the agency on the content of the NDA and expects to complete its NDA submission in early 2026."

FDA event • FDA filing • Essential Tremor

Praxis Precision Medicines…announced that HCWainwright analyst Doug Tsao will host an expert call on Deep Dive into Praxis’ Ulixacaltamide Data with statistician Prof. Chuck McCullogh, University of California, San Francisco on November 24, 2025… (GlobeNewswire)

Clinical data • CNS Disorders

Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET) (clinicaltrials.gov) - P3 | N=1000 | Enrolling by invitation | Sponsor: Praxis Precision Medicines | Recruiting ➔ Enrolling by invitation | N=600 ➔ 1000 | Trial completion date: Apr 2025 ➔ Dec 2026 | Trial primary completion date: Sep 2024 ➔ Sep 2025

Enrollment change • Enrollment status • Trial completion date • Trial primary completion date • Essential Tremor • Movement Disorders

Praxis Precision Medicines to Present on Late-Stage Programs across Epilepsy and Movement Disorders at the American Academy of Neurology 2025 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc...today announced that that it will present data from its four late-stage programs in epilepsy and movement disorders at the American Academy of Neurology (AAN) 2025 Annual Meeting taking place from April 5 to 9, 2025 in San Diego, California."

Clinical data • Essential Tremor

Praxis Precision Medicines Provides Update on Essential3 and Corporate Update (GlobeNewswire) - "Praxis Precision Medicines, Inc...today provided an update on the interim analysis for Study 1 of the Essential3 program of ulixacaltamide in essential tremor (ET), an update on other therapies in development and a financial update...The Independent Data Monitoring Committee (IDMC) overseeing the interim analysis of Study 1 of the Essential3 program has provided Praxis with the outcome of such analysis. Based on the predefined decision framework for Study 1, the IDMC has recommended that the study be stopped for futility, due to the results being unlikely to meet the primary efficacy endpoint under the parameters set by the statistical model...Given the advanced state of enrollment for both Study 1 and Study 2 in the Essential3 program, and in the context of the advice received by the IDMC, Praxis has decided to continue both studies to completion, with topline results expected in the third quarter of 2025."

DSMB • P2 data • P3 data • CNS Disorders • Essential Tremor

Characteristics of Adult Essential Tremor Patients Seeking Participation in a Decentralized US Clinical Trial: Pre-screener Findings from the Essential3 Program Evaluating Efficacy and Safety of Ulixacaltamide (AAN 2025) - "Findings from the largest ET survey to date show that patients seeking participation in a clinical trial report ET as a high-burden, inadequately managed disorder impacting several activities of daily living. Findings also suggest a degree of normalization of ET impact (likely necessitating ongoing patient and physician education) and emphasize the urgent need for innovation in developing effective, well-tolerated treatments."

Clinical • Essential Tremor • Movement Disorders

Essential3: An Innovative Multi-Study Phase 3 Program to Evaluate the Efficacy and Safety of Ulixacaltamide (MDS Congress 2024) - P2, P3 | "In addition to providing evidence of ulixacaltamide safety and efficacy, the Essential3 program is expected to set the foundation for future clinical trial design in ET and other neurologic disorders."

Clinical • P3 data • CNS Disorders • Essential Tremor

Assessing Clinically Meaningful Improvement Associated with Ulixacaltamide Treatment in Adults with Essential Tremor on Concomitant Propranolol: Findings from Essential1 (AAN 2024) - P2 | "Building on findings from Essential1 and focusing on measures determined to be most meaningful to patients, these results highlight the potential for ulixacaltamide to revolutionize the treatment of ET, with evidence of meaningful improvement both alone and in combination with chronic propranolol use."

Clinical • Essential Tremor • Movement Disorders

An Innovative Multi-study Phase 3 Program to Evaluate the Efficacy and Safety of Ulixacaltamide: The Future of Clinical Trial Design in Essential Tremor (AAN 2024) - P2, P3 | "In addition to providing evidence of ulixacaltamide safety and efficacy, the Essential3 program is expected to set the foundation for future clinical trial design in ET and other neurologic disorders."

Clinical • P3 data • CNS Disorders • Essential Tremor • Movement Disorders