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BEDFORD/CAMBRIDGE, Mass. - Stoke Therapeutics (NASDAQ:STOK), a $715 million market cap biotech company with an "EXCELLENT" financial health rating according to InvestingPro, and Biogen (NASDAQ:BIIB) announced Monday that the first patient has been dosed in their global Phase 3 EMPEROR study evaluating zorevunersen for Dravet syndrome, a rare genetic epilepsy disorder.
The trial will assess the investigational antisense oligonucleotide against a sham treatment over 52 weeks in patients aged 2 to 18 years with confirmed SCN1A gene variants. Participants will receive either zorevunersen or a sham procedure, with the primary endpoint measuring change in major motor seizure frequency at week 28. The company’s strong financial position, with a current ratio of 8.41 and minimal debt, positions it well to support this crucial clinical program.
Zorevunersen aims to increase NaV1.1 protein production from the non-mutated copy of the SCN1A gene, potentially addressing the underlying cause of Dravet syndrome rather than just managing symptoms. The drug has received FDA Breakthrough Therapy Designation and orphan drug status from both U.S. and European regulators.
"Given the severity of this disease and the limitations of current treatments, the substantial and durable reductions in seizures and continuing improvements in cognition and behavior support our belief that zorevunersen may improve outcomes for patients with Dravet syndrome," said Barry Ticho, Chief Medical Officer of Stoke Therapeutics, in the press release.
The EMPEROR study has initiated in the United States, United Kingdom, and Japan, with plans to expand to Europe. Key secondary endpoints include durability of seizure reduction at week 52 and changes in behavior and cognition measured by Vineland-3 subdomains.
Dravet syndrome affects approximately 38,000 people across the U.S., UK, EU-4 and Japan, according to company estimates. The condition causes severe seizures and significant cognitive and behavioral impairments, with over 90 percent of patients continuing to experience seizures despite current treatments.
Under their collaboration agreement, Stoke retains commercial rights in North America while Biogen holds rights for the rest of the world. The market has responded positively to Stoke’s progress, with the stock gaining nearly 27% over the past six months. InvestingPro analysis reveals strong analyst support, with multiple additional insights available to subscribers, including detailed valuation metrics and growth projections.
In other recent news, Stoke Therapeutics has garnered attention with several developments. The company received a Buy rating from Jefferies, which set a price target of $30, citing the potential of its epilepsy drug, zorevunersen, ahead of Phase III clinical trial data expected in the second half of 2027. H.C. Wainwright also maintained a Buy rating, adjusting its price target to $35 from $47 after Stoke announced the Phase 3 EMPEROR trial would begin around the second quarter of 2025. This trial is crucial for validating data from an open-label extension study that showed significant improvements in patients treated with zorevunersen. The OLE data revealed that the drug not only reduces seizure frequency but also positively impacts behavior and cognition in patients with Dravet syndrome. These findings were based on a regimen involving a 70 mg loading dose followed by a 45 mg maintenance dose, potentially leading to an 80-87% reduction in seizure frequency over eight months. Jefferies and H.C. Wainwright both highlight the drug’s potential in the competitive Dravet syndrome market.
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