Praxis Precision Medicines, Inc. (NASDAQ: PRAX) announced positive topline results from its Essential3 Phase 3 program evaluating ulixacaltamide HCl for the treatment of essential tremor (ET). The company reported that both pivotal studies met their pre-specified primary endpoints, with all key secondary endpoints also achieved, and ulixacaltamide was generally well tolerated with no drug-related serious adverse events.
In the parallel-group study (Study 1), patients treated with ulixacaltamide demonstrated a mean improvement of 4.3 points from baseline in the Modified Activities of Daily Living 11 (mADL11) at Week 8 (p<0.0001). All key secondary endpoints, including rate of disease improvement over 12 weeks, Patient Global Impression of Change (PGI-C), and Clinical Global Impression of Severity (CGI-S), were statistically significant (p<0.001).
In Study 2, a randomized-withdrawal design, patients maintained superior treatment effects on ulixacaltamide versus placebo (p=0.0369), with the first key secondary endpoint — rate of disease improvement — also demonstrating a statistically significant benefit (p=0.0042).
“Patients in the Essential3 program had been living with essential tremor for an average of 30 years, with worsening symptoms and no effective treatment options,” said Marcio Souza, President and CEO of Praxis Precision Medicines. “The strong results underscore the large unmet need for a therapy like ulixacaltamide. We look forward to discussing a potential NDA with the FDA in the near term.”
The Essential3 program enrolled over 700 patients across two studies using a decentralized design in the United States. Study 1 was a 12-week, double-blind, placebo-controlled trial evaluating change in mADL11 at Week 8. Study 2 enrolled stable responders to ulixacaltamide and assessed maintenance of effect during a four-week randomized-withdrawal phase.
Ulixacaltamide is a selective T-type calcium channel inhibitor designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical circuit, which is associated with tremor activity. The therapy is the most advanced program in Praxis’ Cerebrum™ small molecule platform.
Essential tremor is the most common movement disorder in the U.S., affecting roughly 7 million people, and currently has limited treatment options. Propranolol is the only approved therapy, with limited efficacy and tolerability, leaving a substantial patient population untreated.
Following the announcement, PRAX shares surged more than 200% in early trading, with roughly 2.8 million shares traded, far exceeding the three-month daily average of 449,000 shares. Praxis has submitted a pre-NDA meeting request to the FDA and plans to continue advancing ulixacaltamide toward regulatory submission.
