Praxis的癫痫药物太好了，无法继续使用，FDA接下来将召开会议

Praxis Precision Medicines Inc. (NASDAQ:PRAX) stock rose Friday after the company shared results from the registrational cohort of the EMBOLD study evaluating relutrigine for the treatment of patients with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs).

The Data Monitoring Committee recommended stopping the study early for efficacy.

The FDA has confirmed a meeting to review the data and discuss next steps in the coming weeks. Praxis will make a determination on the timing for filing an NDA for relutrigine after the meeting.

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The EMBOLD topline results will be presented at the American Epilepsy Society Annual Meeting at the Georgia World Congress Center on Saturday.

On Thursday, Praxis Precision Medicines also completed 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.

Analyst Commentary and Blockbuster Potential

HC Wainwright wrote on Friday that relutrigine has been overlooked and underappreciated by investors for some time, though it is expected to begin to change after EMBOLD data.

Analyst Douglas Tsao sees relutrigine as a blockbuster in the SCN8A and SCN2A populations alone, which could expand much further in the broader DEE population being tested in the EMERALD study.

HC Wainwright anticipates priority review for relutrigine, with approval next year is highly likely.

“We can’t recall an instance where a biotech company got its first two approvals of drugs developed organically and both with blockbuster potential within a span of just a few months. The success of EMBOLD also significantly derisks and accelerates the development of relutrigine in broader DEEs,” HC Wainwright analyst wrote on Friday.

The analyst increased the probability of success for relutrigine to 80% from 60% and raised the projected peak sales to $2.8 billion from $760 million.

The analyst maintains the Buy rating for Praxis Precision, while increasing the price forecast from $258 to $340.

Strong Pipeline Momentum

In August, Praxis shared topline results from its Phase 2 RADIANT study evaluating vormatrigine in patients with focal onset seizures and generalized epilepsy.

The trial showed a median seizure reduction of around 56.3%, with 60% of the patients achieving at least a 50% reduction in seizures.

PRAX Price Action: Praxis Precision Medicine shares were up 30.84% at $248.56 at the time of publication on Friday. The stock is trading at a new 52-week high, according to Benzinga Pro data.

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