US-based Praxis Precision has decided not to halt its Phase III trial examining its essential tremor (ET) therapy after an independent review insisted it would likely fail to meet its endpoints.
An independent data monitoring committee (IDMC) overseeing the interim analysis of the company’s Essential 3 trial (NCT06087276) evaluating ulixacaltamide has said that the first study in the placebo-controlled trial is unlikely to meet the primary efficacy endpoint under the parameters set by the company’s statistical model.
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Despite this, given the state of enrolment for both the first and second planned studies in the trial, the company plans to push ahead anyway with topline results expected in the third quarter of 2025, to support the submission of a future new drug application (NDA) with the US Food and Drug Administration (FDA).
After the announcement, the company’s stock took a hit, dropping by 40.6% from $65.03 on 27 February at market close to $38.60 on 28 February at market close.
Praxis president and chief executive officer Marcio Souza said: “We are disappointed with and surprised by the outcome of the interim analysis for study one. Following the advice of the committee, we will explore different analysis methods for the final dataset, which is expected in the third quarter of 2025.”
Comprising two studies, the Essential 3 trial is a decentralised study evaluating the efficacy and safety of ulixacaltamide. Study one is a parallel design study that will enrol 400 patients. Patients will receive ulixacaltamide or placebo and the study will evaluate the proportion of participants that respond to the drug, as well as efficacy and safety over 12 weeks. Study two is a randomised withdrawal trial that will include 200 patients. The 12-week study will evaluate the efficacy, maintenance of response, and durability of effect in patients who respond to ulixacaltamide.
Ulixacaltamide is a highly selective small molecule inhibitor of T-type calcium channels that was designed to block the abnormal neuronal burst firing related to the tremors experienced by a patient living with ET.
Research published by the US National Institutes of Health (NIH) estimates that as many as 7.63 million people in the US were living with ET in 2012, comprising approximately 2.2% of the population.
Research by GlobalData estimates that, if approved, ulixacaltamide could go on to bring in approximately $29m by the end of 2026, with that figure expected to rise to $526m by the end of 2030.
GlobalData is the parent company of Clinical Trials Arena.
Souza added: “We remain focused on delivering on major near-term milestones in our other development programs, including the topline results from the Radiant and Power1 studies in focal onset seizures (FOS) and generalised epilepsy with vormatrigine, and the initiation of our Emerald registrational study of relutrigine in developmental epilepsies and encephalopathies (DEEs) by mid-year 2025. We maintain a robust financial position that supports our continued investment in this exciting pipeline.”
Elsewhere in the field of ET trial failures, Sage Therapeutics and Biogen have disclosed topline results from the Phase II KINETIC 2 study, in which SAGE-324 (BIIB124) showed no significant benefit. Meanwhile, Jazz Pharmaceuticals’ suvecaltamide has failed to meet primary or key secondary endpoints in its Phase IIb trial.
