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Mechanism Behind Ulixacaltamide to Treat Essential Tremor: Marcio Souza

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The president and chief executive officer at Praxis Precision Medicine provided clinical insight on the promising build of ulixacaltamide, an investigational, selective agent in development for essential tremor. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

"What this drug has that’s quite particular is its potency, selectivity, and the way it’s delivered, ensuring better tolerability."

At the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held September 27-October 1, in Philadelphia, Pennsylvania, investigators presented the design of Essential3 (NCT06087276), an innovative multi-study phase 3 program to definitively assess safety and efficacy of ulixacaltimide (Praxis Precision Medicine) in essential tremor (ET). Essential3 incorporates 2 simultaneous, 12-week, decentralized, pivotal studies that will include around 600 adults aged 18-85 years with ET. To date, the company has received interest from nearly 75,000 patients to join the study.

The decentralized study combines in-home and telehealth visits to assess ulixacaltamide (60 mg QAM) vs placebo, and maintenance and durability of effect in responders following randomized withdrawal (RW). In the 2 studies, investigators will use change in TETRAS-Activities of Daily Living score, and the proportion of participants maintaining response following RW, as the primary end points. Secondary end points include responder rates and mADL11 change after 12 weeks, change in TETRAS-ADL, clinician and patient measures of severity, and safety assessments.

At the meeting, NeurologyLive® sat down with Marcio Souza, MD, president and chief executive officer at Praxis Precision Medicine, to discuss the mechanism of action of ulixacaltamide. In the conversation, he noted that the drug targets specific T-type calcium channels that are overreactive in the brain networks regulating movement, particularly in the cerebro-thalamo-cortical circuitry linked to tremor. Ulixacaltamide, which has shown promise in previous phase 1/2 studies, has a selective action on these channels, avoiding effects on other calcium channels elsewhere in the body. Furthermore, Souza spoke on the specialized drug delivery system of ulixacaltamide, which ensures sustained coverage throughout the day without causing tolerability issues.

Click here for more MDS 2024 coverage.

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