Efficacy and Safety of Ulixacaltamide in Essential Tremor: Topline Phase Three Results from Essential3 Study One (Parallel-Design Study)
Salima Brillman1, Alexander Shtilbans2, Jill Farmer3, Monique Giroux4, Melanie Fridley4, Kimberly Crimin4, Minao Tang4, Taylor Crush4, Claudio Santos4, Steven Petrou4, Alyssa Wyant4, Megan Sniecinski4, Marcio Souza4
1Parkinson's Disease and Movement Disorders Center of Silicon Valley, Palo Alto, CA, USA, 2Hospital for Special Surgery, Department of Neurology, New York, NY, USA, 3BoroNeuro, Hopewell, NJ, USA, 4Praxis Precision Medicines, Boston, MA, USA
Objective:
Evaluating the efficacy, safety, and tolerability of ulixacaltamide vs. placebo in adults with essential tremor (ET) as part of the pivotal Phase 3 Essential3 Program.
Background:
An estimated 7 million people in the US live with ET, a progressive neurologic disorder with involuntary tremor presenting significant disruption to daily activities and quality-of-life. Existing treatment options are limited, with high discontinuation rates due to poor tolerability and limited efficacy. Ulixacaltamide is a selective T-type calcium channel (TTCC) modulator in development for ET. The Essential3 Program comprised two simultaneous 12-week registrational studies, with blinded assignment: Study 1 (parallel-group design) and Study 2 (randomized withdrawal), conducted using a decentralized model, with optional long-term safety extension.
Design/Methods:
Study 1 was a randomized, double-blind, placebo-controlled trial enrolling participants aged 18–85 years with ET (symptoms ≥3 years; if on ET medication(s), stable dose for ≥28 days prior to screening). Participants were randomized 1:1 to ulixacaltamide hydrochloride 60mg QD (after 2-week titration) or placebo for 12 weeks. The primary endpoint was change from baseline to Day 56 in mADL11 (TETRAS-ADL items 1-11 with modified score). Secondary endpoints included rate of disease improvement and Patient/Clinical Global Impression scores. Safety assessments included TEAEs, labs, ECGs.
Results:
A total of 473 participants were enrolled. The primary endpoint was met, with topline results to be presented.
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.
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