BEDFORD, Mass. - Stoke Therapeutics, Inc. (NASDAQ:STOK), a biotechnology company valued at $583.7 million, announced today that it has reached an agreement with health regulators from the U.S., Europe, and Japan on the design of its upcoming Phase 3 EMPEROR study. According to InvestingPro data, the company maintains a strong financial position with a healthy current ratio of 5.09, indicating robust liquidity to fund its clinical programs. This trial will investigate zorevunersen as a potential treatment for Dravet syndrome, a severe form of epilepsy in children.
The study, slated to begin in mid-2025, will assess the efficacy of zorevunersen in reducing major motor seizure frequency and improving behavior and cognition in children and adolescents aged 2 to less than 18 years. This global, randomized, double-blind, sham-controlled trial aims to enroll around 150 patients with Dravet syndrome. Participants must have a confirmed variant in the SCN1A gene not linked to a gain of function.
Dravet syndrome is a debilitating condition with high unmet medical needs, characterized by frequent, prolonged, and drug-resistant seizures. The disease also results in developmental and cognitive impairments, impacting the quality of life for patients and their families. No disease-modifying therapies are currently approved for Dravet syndrome.
Zorevunersen, an investigational antisense oligonucleotide, has been designed to increase the expression of the NaV1.1 protein by targeting the non-mutant copy of the SCN1A gene. This could potentially address the genetic cause of Dravet syndrome. The drug recently received the FDA's Breakthrough Therapy Designation, which is intended to expedite the development and review process for drugs that could significantly improve treatment over existing options.
Clinical data supporting the Phase 3 dosing regimen showed an 87% median reduction in convulsive seizure frequency and improvements in cognition and behavior in patients who received the treatment as part of earlier trials. These patients were already on the best available anti-seizure medicines. The company's promising clinical results have caught analysts' attention, with InvestingPro reporting that three analysts have recently revised their earnings estimates upward, despite the company not yet being profitable.
The EMPEROR study will also consider key secondary endpoints, such as the durability of the effect on seizure frequency and improvements in various aspects of cognition and behavior. The trial is expected to last 60 weeks, with results anticipated by the end of 2027.
Stoke Therapeutics hosted a webcast and conference call for analysts and investors earlier today to discuss the regulatory alignment for the Phase 3 study of zorevunersen.
This news article is based on a press release statement from Stoke Therapeutics.
In other recent news, Stoke Therapeutics has been making significant strides in the biotechnology industry. The company's drug candidate, zorevunersen, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA). This is a significant development, as zorevunersen is aimed at treating Dravet syndrome, a rare form of epilepsy. The FDA's decision was influenced by positive outcomes from Phase 1/2a and open-label extension studies, demonstrating that zorevunersen significantly reduced seizure frequency and improved cognitive and behavioral measures in patients. Stoke Therapeutics is currently in discussions with the FDA and other global regulatory bodies to advance zorevunersen into a global, randomized, controlled Phase 3 registrational study. Furthermore, TD Cowen maintained its Buy rating for Stoke Therapeutics following the release of promising clinical trial results for zorevunersen. These developments underscore Stoke Therapeutics' commitment to advancing RNA-based medicines to address genetic conditions.
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