Praxis Precision Medicines Achieves Breakthrough in Essential Tremor Treatment

Praxis Precision Medicines has announced a significant milestone in the development of a novel treatment for essential tremor, marking a potential paradigm shift in the management of this common neurological disorder. The company's experimental drug, ulixacaltamide, has demonstrated positive results in two late-stage clinical trials, paving the way for a potential FDA approval application by early 2026.

Clinical Trial Success Drives Stock Surge

The Boston-based biotechnology company revealed that both of its Phase 3 studies met their primary endpoints, showcasing the drug's efficacy in reducing tremor severity and improving patients' ability to perform daily tasks. This announcement led to a dramatic surge in Praxis' stock value, with shares more than tripling and reaching a peak of $200 on Thursday afternoon.

The first study, involving 473 participants, focused on changes in scores on a modified 11-item questionnaire assessing patients' ability to perform self-care tasks. Drug-treated participants showed a significant and clinically meaningful improvement of 4.3 points compared to the control group, with benefits observed from the second week of treatment through the end of the 12-week dosing period.

In the second study, researchers evaluated the drug's sustained efficacy. After an initial eight-week exposure to ulixacaltamide, 80 respondents were randomized to either continue the drug or switch to a placebo. The results showed that over half of the drug arm maintained their response, compared to only one-third in the placebo group.

Potential Impact on Patient Care

Ulixacaltamide could become only the second drug specifically approved to treat essential tremor, a condition affecting an estimated 10 million people in the United States alone. The current standard of care, propranolol, while effective for some patients, is associated with side effects and does not work for all individuals.

Dr. Salima Brillman, co-lead investigator of the late-stage studies and founder of the Parkinson's Disease and Movement Disorders Center of Silicon Valley, emphasized the potential impact of these results: "It represents more than data on a chart — it is a real opportunity to help people regain their independence and improve their daily functioning in meaningful ways."

The drug's mechanism of action targets a "calcium ion channel" protein, aiming to block abnormal firing of neurons in the cerebellum and thalamus, brain regions implicated in essential tremor. This novel approach could offer a more targeted treatment option for patients struggling with the disorder.

Safety Profile and Next Steps

Praxis reported that ulixacaltamide was generally well-tolerated across the trials. The most common treatment-emergent adverse events were mild to moderate, including constipation, dizziness, euphoria, brain fog, headache, insomnia, and tingling sensations. Severe events were documented in 6% of the first study's drug arm and 7% of the second study.

With these positive results in hand, Praxis plans to submit an approval application to the FDA by early 2026. The company also intends to share more detailed data from its studies at upcoming medical conferences and in peer-reviewed publications, further solidifying the scientific basis for ulixacaltamide's potential approval and eventual market entry.