CTAs keep buying Treasuries, gold longs face stop-loss risk: BofA
BOSTON - The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation (BTD) to Praxis Precision Medicines, Inc. (NASDAQ:PRAX) for relutrigine, a treatment for children with SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs), the company announced Thursday. The news comes as Praxis, currently valued at $1.09 billion, maintains a strong financial position with more cash than debt on its balance sheet, according to InvestingPro data.
The designation was based on results from the Phase 2 EMBOLD trial, which showed a placebo-adjusted monthly motor seizure reduction of 46% during the 16-week double-blind period, with over 30% of patients achieving seizure freedom while on relutrigine.
Data from the 11-month open-label extension period demonstrated approximately 90% seizure reduction in patients and an increase in seizure-free periods from 3 days at baseline to a mean of 67 days.
"This BTD represents a significant milestone for our relutrigine program and further validates its potential," said Marcio Souza, president and chief executive officer of Praxis.
The breakthrough designation enables expedited development and regulatory review for drugs intended to treat serious conditions that demonstrate substantial improvement over existing therapies.
Praxis is currently conducting the registrational EMBOLD cohort 2 study with topline results expected no later than the first half of 2026, followed by a potential New Drug Application filing.
The company has also initiated the EMERALD study, which will evaluate relutrigine in patients across all DEEs regardless of etiology.
Relutrigine is a first-in-class small molecule that works as a preferential inhibitor of persistent sodium current. The drug has previously received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA for the treatment of SCN2A-DEE, SCN8A-DEE and Dravet syndrome.
According to the press release statement, no new safety signals or drug-related serious adverse events were observed during the trials, and no dose reductions were needed.
In other recent news, Praxis Precision Medicines has captured the attention of investors with several key developments. The company reported first-quarter 2025 financial results, highlighting operating expenses of $74.7 million and cash reserves of $472 million, which are anticipated to support operations through 2028. Piper Sandler reiterated its Overweight rating on Praxis, maintaining a price target of $270, while Oppenheimer assigned an Outperform rating with a $97 target, both expressing confidence in the company’s strategic direction. Chardan Capital Markets also began coverage with a Buy rating and set a price target of $80, citing the promise of Praxis’s epilepsy treatments.
Praxis is advancing its epilepsy portfolio, with the RADIANT and POWER1 registrational Phase 2/3 trials for treatment-resistant focal onset seizures expected to yield results in 2025. The company’s relutrigine program, which has been granted Rare Pediatric Disease Designation, is also progressing, with the EMERALD trial results anticipated in the first half of 2026. Additionally, Praxis is preparing for the initiation of the Phase 2/3 POWER2 trial later this year. Despite a setback in another program, analysts remain optimistic about the company’s focus on epilepsy treatments, which are advancing into late-stage trials. The company’s financial stability and strategic focus on epilepsy treatments are seen as strong indicators of potential future success.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.