Executives from Praxis Precision Medicines (NASDAQ:PRAX) used a fireside chat at the Guggenheim Emerging Outlook Biotech Summit 2026 to outline near-term regulatory plans and discuss commercial considerations for its central nervous system (CNS) pipeline, with a particular focus on essential tremor and epilepsy programs.
Essential tremor: framing market size and unmet need
Chief Executive Officer Marcio Souza said the company’s recent work, including outreach to the field, reinforced what he described as the large scale of essential tremor (ET) and the limitations of current treatment options. He cited prevalence estimates of roughly 2% to 2.5% of the U.S. population and argued that “there is nothing really right now that is effective” and safe enough for patients who require treatment.
In discussing market activity, Guggenheim analyst Yatin Suneja referenced research indicating about 80,000 ET prescriptions per month written by neurologists alone. Souza 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. He added that script data may undercount Medicare utilization and suggested the true prescription volume could be higher, noting ET’s older patient demographic. Souza 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.
Commercial approach, sales force considerations, and patient outreach
Souza 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, and said those learnings would be amplified in commercialization through increased patient messaging and expansion of a centralized database to facilitate patient-physician discussions.
On access, he said the company is focused on minimizing payer-related friction at launch, adding that payer hurdles are typically less severe in settings where approved treatments are limited, but that Praxis remains “very cognizant” of coverage dynamics.
Pricing: expected ranges and patient affordability
Souza separated affordability considerations by payer type. 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. He added that, based on the company’s checks so far, he did not expect access limitations, and characterized the copay for the drug itself as “relatively small,” while noting that older patients face broader healthcare cost burdens.
When asked about price levels, Souza 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.
Regulatory timing: two NDAs expected around mid-February
Souza 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.
On whether Praxis would seek Priority Review for the ET drug (ulixacaltamide hydrochloride), Souza 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.
Epilepsy programs: relutrigine, EMERALD, vormatrigine, and elsunersen
Discussing relutrigine, Souza said the NDA package is “much smaller” than ET by nature, citing fewer studies and patients. He described the initial focus as SCN2A and SCN8A, where he said benefits are “very clear,” given the severity of patients with uncontrolled seizures since birth and significant developmental delays.
Souza provided updates on the EMERALD study in developmental and epileptic encephalopathies (DEE), stating the trial started “not that long ago,” is progressing rapidly, and that Praxis expects to finalize the study in the second half with a likely readout this year. He said that, if positive, EMERALD could support a label expansion and that Praxis believes it could be a strong fit for the FDA’s STAR program concept, potentially enabling a faster supplemental NDA timeline than typical—while noting these expectations assume approval of the initial NDA.
On commercialization needs, Souza said Praxis expects a small specialized field force for relutrigine and related activities such as medical affairs, noting limited overlap between adult neurology and pediatric epilepsy. He estimated the addressable SCN2A/SCN8A U.S. population at roughly 3,500 to 10,000 patients and said most are already identified due to early and severe disease presentation and widespread genetic diagnosis in the U.S.
For vormatrigine in focal onset seizures, Souza reiterated that the bar for placebo-adjusted efficacy is not “that high” and said prior RADIANT results were “very impressive” on both short- and long-term measures. He said prior guidance for POWER1 remains intact, describing an efficacy range where 40% to 50% for the drug alone would be “quite exquisite,” and said Praxis is confident discontinuation will be better managed in POWER1 than in RADIANT. He characterized the opportunity as another multi-billion dollar addition to the portfolio and said POWER3 could be designed to support eventual movement toward first-line use.
On elsunersen, Souza said Praxis anticipates an NDA next year and does not believe it would cannibalize the relutrigine SCN2A market. He argued monotherapy is unlikely to be realistic for some patients and said combination therapy is expected, with the company viewing the two drugs as complementary.
Chief Financial Officer Tim Kelly said Praxis has about $1.5 billion on its balance sheet and expects its cash runway to extend into 2028, which he said is likely sufficient to reach break-even while supporting planned launches. On priority review vouchers, Kelly said the company views two as a conservative estimate, tied to relutrigine and elsunersen.
About Praxis Precision Medicines (NASDAQ:PRAX)
Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on discovering and developing precision therapies for disorders driven by neuronal excitability. The company applies translational neuroscience and genetic insights to design small molecule drugs that target specific ion channels and receptor subtypes implicated in neurological and psychiatric conditions. Its research aims to address unmet needs in rare epilepsies, essential tremor, treatment-resistant depression and other central nervous system (CNS) disorders.
The company’s pipeline includes several lead candidates at various stages of development.
