Praxis Precision Medicines is advancing ulixacaltamide into a phase 3 clinical trial program, Essential3 (NCT06087276), to assess its safety and efficacy in treating essential tremor (ET). The innovative program incorporates two simultaneous, 12-week, decentralized, pivotal studies involving approximately 600 adults aged 18-85 years diagnosed with ET.
The Essential3 program is designed as a decentralized study, combining in-home and telehealth visits to evaluate ulixacaltamide (60 mg QAM) against placebo. Researchers will also assess the maintenance and durability of the drug's effect in responders following randomized withdrawal (RW).
Mechanism of Action
According to Marcio Souza, MD, president and chief executive officer at Praxis Precision Medicine, ulixacaltamide targets specific T-type calcium channels that are overreactive in the brain networks regulating movement, particularly within the cerebro-thalamo-cortical circuitry linked to tremor. "What this drug has that’s quite particular is its potency, selectivity, and the way it’s delivered, ensuring better tolerability," Souza noted.
Ulixacaltamide's selective action on these T-type calcium channels aims to avoid effects on other calcium channels elsewhere in the body. The drug delivery system is designed to ensure sustained coverage throughout the day while minimizing tolerability issues. Previous phase 1/2 studies have demonstrated the drug's potential.
Trial Endpoints and Design
The primary endpoints for the two studies include the change in TETRAS-Activities of Daily Living (ADL) score and the proportion of participants maintaining response following randomized withdrawal. Secondary endpoints encompass responder rates and mADL11 change after 12 weeks, changes in TETRAS-ADL, clinician and patient measures of severity, and comprehensive safety assessments.
With significant patient interest, nearly 75,000 individuals have expressed interest in participating in the study to date.
