Praxis Precision Medicines announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to its lead candidate, ulixacaltamide HCl, for the treatment of essential tremor. The designation, awarded on December 29, 2025, is based on topline data from the company’s Essential3 Phase 3 program, which demonstrated a clinically meaningful reduction in tremor amplitude and improved patient‑reported outcomes compared with placebo.
Ulixacaltamide is a selective small‑molecule inhibitor of T‑type calcium channels, a mechanism that targets abnormal neuronal burst firing in the cerebello‑thalamo‑cortical circuit that underlies essential tremor. The drug’s mechanism offers a distinct therapeutic profile from the only FDA‑approved treatment, propranolol, which has limited efficacy and tolerability. The essential tremor market is sizable—affecting roughly seven million U.S. patients—and is projected to grow to more than $300 million by 2035, creating a multi‑billion‑dollar opportunity for a first‑in‑class therapy.
The BTD accelerates ulixacaltamide’s development and regulatory review, allowing Praxis to file a New Drug Application earlier than otherwise possible. Management indicated that the company is preparing to submit the NDA in early 2026 following a successful pre‑NDA meeting with the FDA in December. The designation is a key catalyst for Praxis, whose valuation is heavily tied to ulixacaltamide’s success. The company’s recent financing has bolstered its cash position, providing a runway into 2028, while ongoing R&D investments in other CNS candidates—such as vormatrigine and relutrigine—demonstrate a broader pipeline strategy.
President and CEO Marcio Souza emphasized that the BTD “further underscores ulixacaltamide’s potential to address the substantial unmet need in patients with essential tremor.” He added that the company’s “positive interactions with the FDA, together with this BTD, are enabling us to advance this promising treatment faster to patients.” Analyst coverage has been largely positive, with a consensus rating of “Strong Buy” or “Moderate Buy” and significant upside potential.
The market reaction to the BTD was robust, with Praxis shares surging to a new 52‑week high and a 17% pre‑market increase reported by some outlets. Investors cited the expedited development pathway and the strong topline data as primary drivers of the rally, while the broader essential tremor market’s size and limited treatment options reinforced the commercial upside. The BTD also signals to investors that Praxis’s lead asset is on a clear path to regulatory approval, potentially unlocking the multi‑billion‑dollar opportunity that has long been a cornerstone of the company’s growth strategy.
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