Praxis Precision Medicines has reported topline data from the Phase IIb Essential1 trial of ulixacaltamide (PRAX-944) to treat essential tremor (ET).
The placebo-controlled, randomised, dose range finding, double-blind trial has been designed for assessing the safety, tolerability, and efficacy of once-a-day 60mg or 100mg ulixacaltamide treatment against a placebo after 56 days.
In the trial, 132 ET patients were randomised and treated.
According to the findings, participants who received treatment with ulixacaltamide showed improvement in the modified Activities of Daily Living (mADL) score relative to the placebo.
Additionally, nominal statistically significant improvements were observed in Clinical Global Impression-Severity (CGI-S) and in Patient Global Impression-Change (PGI-C) scores.
Ulixacaltamide was found to be well tolerated in the trial, and there were no new safety findings.
The company plans to meet with the FDA and commence a Phase III trial to treat ET in the second half of this year, based on the observed efficacy and safety profile.
Praxis Precision Medicines president and CEO Marcio Souza said: “The results from Essential1 illustrate the clear potential of ulixacaltamide as an effective and well tolerated treatment for people with essential tremor.
“The study showed meaningful improvement in patient’s daily functioning while also providing insights to guide the programme’s advancement to Phase III, including a deeper understanding of endpoints and trial design.
“It’s clear to us, based on these results and prior clinical experience, that ADLs are the preferred endpoint for a registrational study for essential tremor. We look forward to meeting with the FDA to discuss the data and our Phase III plans.”
Fatigue, paraesthesia, constipation, anxiety, feeling abnormal, dizziness, and headache are the most commonly reported treatment emergent adverse events (TEAEs) observed in participants who received treatment with ulixacaltamide.
