Praxis Precision Medicines Announces Positive Pre-NDA Meeting with FDA for Ulixacaltamide in Essential Tremor
Rhea-AI Summary
Praxis Precision Medicines (NASDAQ: PRAX) completed a pre-NDA meeting with the FDA for ulixacaltamide in essential tremor and received written feedback plus an in-person discussion. The company said it gained alignment with the agency on NDA content and expects to submit the NDA in early 2026. Praxis highlighted momentum from the positive Essential3 program, where ulixacaltamide showed statistically significant, clinically meaningful improvements in daily functioning. Management framed the FDA feedback as advancing the program toward a potential new treatment for millions living with essential tremor.
Positive
- FDA alignment on NDA content after pre-NDA meeting
- Company expects NDA submission in early 2026
- Ulixacaltamide showed statistically significant functional improvements in Essential3
Negative
- NDA not yet submitted; regulatory approval still required
- Clinical-stage program means no marketed product or revenue yet
Key Figures
Market Reality Check
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 03 | Inducement grants | Neutral | +3.9% | Inducement RSU awards to new employees under 2024 Inducement Plan. |
| Nov 24 | Scientific meeting | Positive | -3.5% | Upcoming AES presentations on preclinical and clinical epilepsy portfolio data. |
| Nov 20 | Fireside chat | Positive | -13.3% | Analyst-hosted fireside chat on ulixacaltamide clinical data interpretation. |
| Nov 06 | Inducement grants | Neutral | +4.7% | RSU awards to new employees under Nasdaq Listing Rule 5635(c)(4). |
| Nov 05 | Investor conferences | Positive | -4.1% | Participation in multiple November investor conferences and webcasts. |
Recent news has often seen mixed reactions: several seemingly positive pipeline or visibility updates were followed by negative moves, while routine inducement grant notices saw modest gains.
Over the last month, Praxis issued multiple updates highlighting corporate activity and clinical communication. Investor and scientific outreach included epilepsy data presentations at AES and a fireside chat focused on ulixacaltamide, yet those events coincided with declines of -3.46% and -13.33%. In contrast, routine inducement grant announcements on Nov 6, 2025 and Dec 1, 2025 were followed by gains of 4.68% and 3.93%. Today’s positive pre‑NDA FDA alignment for ulixacaltamide fits the broader narrative of advancing late‑stage programs after the Essential3 results.
Regulatory & Risk Context
Short interest at 9.45% of float with 7.03 days to cover indicates moderate positioning that could amplify moves in either direction if sentiment shifts.
Market Pulse Summary
This announcement highlights successful pre-NDA interactions with the FDA and plans to submit an ulixacaltamide NDA in early 2026 for essential tremor, building on prior Essential3 efficacy data. In recent months, Praxis has raised substantial capital, advanced multiple CNS programs, and increased scientific and investor outreach. Investors may focus on the eventual NDA content, regulatory review outcomes, cash usage trends, and any further clinical or safety updates related to ulixacaltamide and the broader pipeline.
AI-generated analysis. Not financial advice.
Praxis confirms plans to submit the essential tremor NDA for ulixacaltamide in early 2026
BOSTON, Dec. 04, 2025 (GLOBE NEWSWIRE) -- Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced the successful completion of its pre-NDA (New Drug Application) meeting with the FDA, including receipt of written feedback and an in-person meeting. Praxis has gained alignment from the agency on the content of the NDA and expects to complete its NDA submission in early 2026.
“We are very pleased with the collaborative discussions we recently had with the FDA and remain on track to submit Praxis’ first NDA in early 2026. Building on the strong momentum from the positive Essential3 program, where ulixacaltamide demonstrated statistically significant and clinically meaningful improvements in daily functioning, the FDA feedback moves us closer to delivering a much-needed therapy to the millions of people living with essential tremor who currently lack effective and safe treatment options,” said Marcio Souza, president and chief executive officer.
About Essential Tremor (ET)
Essential tremor is the most common movement disorder, affecting roughly seven million people in the United States alone, representing a multi-billion-dollar commercial opportunity. ET is characterized by involuntary rhythmic movement in the upper limbs, with or without tremor in other body locations such as the head, vocal cords, or legs. These tremors significantly disrupt daily living and are progressive in nature, with increases in tremor severity and amplitude commonly observed over the course of the disease. Propranolol, a beta-blocker, is the only approved pharmacotherapy for ET, offering limited efficacy and poor tolerability and is also contraindicated for comorbidities that affect a significant share of the ET population. Other beta blockers and anti-convulsants are used off-label, though similarly are characterized by limited efficacy and tolerability. The vast majority of patients are left without a treatment option, with an estimated minimum of 2 million patients seeking treatment. In a patient survey, up to
About Ulixacaltamide
Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide is the most advanced program within Praxis’ Cerebrum™ small molecule platform.
About Praxis
Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating insights from genetic epilepsies into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance. Praxis is applying genetic insights to the discovery and development of therapies for rare and more prevalent neurological disorders through our proprietary small molecule platform, Cerebrum™, and antisense oligonucleotide (ASO) platform, Solidus™, using our understanding of shared biological targets and circuits in the brain. Praxis has established a diversified, multimodal CNS portfolio including multiple programs across movement disorders and epilepsy, with four clinical-stage product candidates. For more information, please visit www.praxismedicines.com and follow us on Facebook, Instagram, LinkedIn and Twitter/X.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the development of Praxis’ product candidates and the anticipated timing of regulatory submissions and interactions, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.
The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended December 31, 2024 and as updated in its Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as well as other filings made with the Securities and Exchange Commission. Although Praxis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on information and factors currently known by Praxis. As a result, you are cautioned not to rely on these forward-looking statements. Any forward-looking statement made in this press release speaks only as of the date on which it is made. Praxis undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future developments or otherwise.
Investor Contact: Praxis Precision Medicines investors@praxismedicines.com 857-702-9452 Media Contact: Dan Ferry Life Science Advisors Daniel@lifesciadvisors.com 617-430-7576
FAQ
What did Praxis announce about ulixacaltamide and the FDA on December 4, 2025?
When does Praxis expect to submit the ulixacaltamide NDA (PRAX)?
What clinical evidence did Praxis cite for ulixacaltamide in essential tremor (PRAX)?
Does the FDA meeting mean ulixacaltamide is approved for essential tremor (PRAX)?
How might the pre-NDA meeting affect PRAX investors and timeline?
