1. Praxis received Breakthrough Therapy Designation for ulixacaltamide in essential tremor. 2. NDA submission for ulixacaltamide is expected in early 2026. 3. Essential tremor affects about seven million people in the U.S., indicating a significant market. 4. Positive topline results from Phase 3 studies highlight ulixacaltamide's potential advantages. 5. 85% of neurologists report treating patients seeking effective therapies for essential tremor.
FAQ
Why Very Bullish?
The Breakthrough Therapy Designation indicates strong potential for ulixacaltamide, suggesting likely market uptake. Past instances like the approval of new CNS therapies have shown significant stock price increases.
How important is it?
The article highlights a crucial regulatory milestone that significantly enhances PRAX's prospect, showing high relevance to potential investors.
Why Long Term?
With NDA submission planned in 2026, investor interest will grow as regulatory milestones approach, akin to previous drug approval timelines impacting similar biopharma stocks positively.
Praxis Precision Medicines Receives Breakthrough Therapy Designation from FDA for Ulixacaltamide in Treating Essential Tremor
BOSTON, Dec. 29, 2025 (GLOBE NEWSWIRE) — Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company focused on CNS disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for ulixacaltamide HCl. This designation is based on encouraging topline results from the Essential3 Phase 3 program targeting essential tremor (ET), a condition affecting millions of Americans.
Significance of the Breakthrough Therapy Designation
The BTD is a critical milestone that allows for expedited development and regulatory review for drugs intended to treat serious conditions. Preliminary evidence suggests that ulixacaltamide may offer substantial improvements over existing therapies in managing ET.
Marcio Souza, President and CEO of Praxis, stated, “The granting of the Breakthrough Therapy Designation for ulixacaltamide highlights its potential to address the significant unmet needs of patients with ET. Recent positive engagements with the FDA, along with this BTD, will help us expedite this promising treatment for patients.”
Clinical Development Timeline
Praxis is on track to file the New Drug Application (NDA) for ulixacaltamide in early 2026. This follows a successful pre-NDA meeting with the FDA, during which Praxis received pivotal feedback. The NDA's content has been aligned with the FDA's guidelines, paving the way for its formal submission.
The Essential3 program includes two pivotal Phase 3 studies that demonstrated the drug's efficacy, further reinforcing the case for ulixacaltamide in treating ET.
Understanding Essential Tremor
Essential tremor (ET) is the most prevalent movement disorder in the United States, impacting approximately seven million individuals. This condition leads to involuntary rhythmic movements that can significantly disrupt daily activities. The progressive nature of ET often results in increased tremor severity and amplitude over time.
Currently, the only FDA-approved treatment is propranolol, a beta-blocker, which has limited efficacy and is poorly tolerated by many patients. The majority of the ET population lacks effective treatment options, with estimates suggesting that around 2 million patients are actively seeking therapies.
- 77% of ET patients feel their condition is inadequately managed.
- 50% of patients are not receiving any treatment.
- 85% of neurologists' visits concern patients seeking treatment for ET.
- 40% of their patients are left untreated.
These statistics highlight the urgent need for innovative treatments like ulixacaltamide, which could potentially lead to better management of ET symptoms.
About Ulixacaltamide
Ulixacaltamide functions as a highly selective small molecule inhibitor of T-type calcium channels. Its design targets the abnormal neuronal burst firing associated with tremor activity in the Cerebello-Thalamo-Cortical (CTC) circuit. This innovative treatment is currently the most advanced program within the Praxis Cerebrum™ small molecule platform.
About Praxis Precision Medicines
Praxis Precision Medicines is committed to transforming insights from genetic research into actionable therapies for CNS disorders characterized by excitement-inhibition imbalance. The company's portfolio includes a variety of clinical-stage product candidates, focusing on both rare and prevalent neurological disorders.
For further details about their initiatives and product pipeline, visit www.praxismedicines.com and follow Praxis on Facebook, LinkedIn, and Twitter.
Forward-Looking Statements
This article contains forward-looking statements as defined by the Private Securities Litigation Reform Act of 1995, which include predictions about Praxis’ future operations and strategies. These statements are subject to numerous risks and uncertainties, and you should not rely solely on them as definitive. For comprehensive risk factors, refer to the company's filings with the Securities and Exchange Commission.