Praxis Shares Hit Record High on FDA Breakthrough Nod for Tremor Drug
The U.S. regulator granted Breakthrough Therapy Designation to ulixacaltamide, expediting the path for a novel essential tremor treatment.
Praxis Precision Medicines Inc. (PRAX) shares surged to a new 52-week high on Tuesday after the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to its lead drug candidate, ulixacaltamide, for the treatment of essential tremor.
The stock jumped more than 12%, trading at $303.30 in morning trading and pushing its market capitalization over $6.7 billion. The move followed the company's announcement, which marks a significant regulatory milestone and accelerates the development timeline for a drug targeting the most common movement disorder in the United States.
Essential tremor is a neurological condition that causes involuntary, rhythmic shaking, affecting an estimated seven million people in the U.S. alone. Ulixacaltamide is a highly selective molecule designed to block T-type calcium channels, which are believed to play a role in the abnormal brain activity that causes tremors. According to the company's announcement, the FDA's decision was based on positive results from the company's Phase 3 Essential3 clinical trial program.
The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. To qualify, a drug must show preliminary clinical evidence of substantial improvement over any available therapy on at least one clinically significant endpoint. The designation provides more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review.
"The FDA's decision to grant Breakthrough Therapy Designation to ulixacaltamide is a significant moment for the essential tremor community," said Marcio Souza, president and chief executive officer of Praxis, in a statement. He noted it "underscores the potential of ulixacaltamide to be a major advance in the standard of care."
This regulatory win follows a successful pre-New Drug Application (NDA) meeting with the agency in December 2025. Praxis confirmed it has reached an agreement with the FDA on the contents of its submission package and plans to file its NDA in early 2026, as reported by several news outlets. This clarity on the path to market further de-risks the asset and provides investors with a clear timeline for the next major catalyst.
The market reaction reflects growing investor confidence in the Boston-based biotechnology firm. Shares of Praxis have seen a dramatic rise over the past year, climbing from a 52-week low of $26.70 to Tuesday's record levels. Wall Street analysts remain overwhelmingly bullish on the company's prospects, with 14 analysts rating the stock a "Buy" or "Strong Buy" against only one "Sell" rating. The consensus price target sits at $419.13, suggesting significant further upside from the current trading price. The company's focus on developing therapies for central nervous system disorders has positioned it as a key player in a field with high unmet medical need, and the advancement of ulixacaltamide represents a potential flagship commercial product.