Praxis Precision Medicines Plans Dual NDA Filings for CNS Therapies by Mid-February

Praxis Precision Medicines presented at the Guggenheim Emerging Outlook Biotech Summit 2026 and outlined plans to submit New Drug Applications for ulixacaltamide and relutrigine by mid-February 2026. Both therapies have received Breakthrough Therapy Designation from the FDA.

The Chief Executive Officer said Praxis remained on track to file around mid-February and emphasized that the company was preparing "two NDAs being filed on the same office with the FDA at the same time, give or take," describing them as "neck and neck" and "days apart." He said there were no missing components in the applications and no indication Praxis would miss the filing guidance it had provided publicly.

Ulixacaltamide was granted Breakthrough Therapy Designation by the US Food and Drug Administration in December 2025, following a successful Essential3 program. The CEO stated that the granting of the Breakthrough Therapy Designation for ulixacaltamide "further underscores its potential to address the substantial unmet need in patients with ET."

The CEO cited prevalence estimates of roughly 2% to 2.5% of the U.S. population for essential tremor and argued that "there is nothing really right now that is effective" and safe enough for patients who require treatment. He said his view is that between 1 million and 2 million patients, on a rolling one- to three-year basis, are actively seeking treatment for ET in the United States. Research indicates about 80,000 ET prescriptions per month written by neurologists alone.

The CEO pointed to results from Praxis's Essential3 program, stating that both studies were positive, and emphasized the importance of translating ET trial endpoints into functional benefits that clinicians and patients can understand. He also highlighted a familial component, stating that 70% of ET patients have one to two family members with the condition, which he said could expand market engagement over time.

On pricing, the CEO said it was "a little early" to put a specific number on the table, but he discussed a framework in which $20,000 per year would be "too low" and suggested that "40-60" (in thousands of dollars annually) could be a "sweet spot," with potential to go higher depending on launch dynamics. He also said an analog such as tardive dyskinesia pricing could be a useful benchmark.

For the privately insured population, which he estimated at roughly 30% to 40% at launch, he said copay programs could support "minimal to no" patient copays, which would be incorporated into launch costs. He described Medicare affordability as more complex due to benefit design and timing during the year, and said Praxis was evaluating approaches such as initiation programs that may not be considered a paid drug under current rules.

On whether Praxis would seek Priority Review for ulixacaltamide, the CEO said the company was evaluating the request in the context of workload, shared FDA resources, and what would be best for both the company and the agency. While he expressed confidence that Priority Review would be granted if requested, he said the decision also depends on what maximizes the overall economic and humanitarian benefits for the asset.

The CEO said ET would require broad geographic coverage, describing the need to "solve for zip codes" rather than target only a limited number of physicians. He discussed internal planning around field force sizing, indicating the company does not want launch execution constrained by insufficient capacity. He also cited the company's clinical trial recruitment experience in movement disorders, claiming Praxis enrolled patients "about 5-7 times faster" than others by understanding online and office-based patient behavior.

An investment firm raised its target price on Praxis Precision by 5.3% to $800 from $760 and reiterated its Buy recommendation on the stock. The update comes as the firm reassessed its estimates, through consultations with key opinion leaders, on the potential impact of ulixacaltamide. It now thinks that the drug can take $5 billion to $10 billion in peak sales from the $15+ billion essential tremors market.

Praxis Precision Medicines holds a $1.50 billion cash position with a funding runway into 2028. The company is a clinical-stage biopharma company that focuses on developing therapies targeting central nervous system disorders and genetic epilepsies. The company is based in Boston, Massachusetts, and was founded in September 2015.