Massachusetts-based Stoke Therapeutics STOK has been making meaningful progress with the development of zorevunersen (STK-001), its lead investigational candidate for treating Dravet syndrome, a severe and progressive genetic epilepsy.
Per management, zorevunersen is a novel proprietary antisense oligonucleotide with the potential to become the first disease-modifying therapy to address the genetic cause of Dravet syndrome. The candidate is currently in late-stage development for the given indication.
The FDA granted a Breakthrough Therapy designation to zorevunersen for treating Dravet syndrome in December 2024.
In the past year, shares of Stoke Therapeutics have skyrocketed 218.9% compared with the industry’s rise of 18.7%.
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STOK Rides on Zorevunersen’s Development Progress
The phase III EMPEROR study is currently evaluating the safety, efficacy and tolerability of zorevunersen in children aged two to less than 18 years with Dravet syndrome. Enrollment in the study is expected to be completed in June, 2026. The study is being conducted across the United States, the United Kingdom, Europe and Japan.
The primary endpoint of the study is to measure the percentage change from baseline in major motor seizure frequency at week 28 in patients treated with zorevunersen compared with those receiving sham treatment.
Data from the EMPEROR study is expected to be announced in mid-2027. The results are anticipated to support the completion of the rolling new drug application submission for zorevunersen to the FDA, which is planned to begin in the first quarter of 2027.
Stoke Therapeutics has a strategic collaboration with drug giant Biogen BIIB to develop and commercialize zorevunersen for Dravet syndrome.
In February 2025, Biogen in-licensed outside-U.S. rights to zorevunersen for the treatment of Dravet syndrome. The deal broadened BIIB’s rare disease pipeline.
Besides zorevunersen, STOK is also developing another candidate, STK-002, in an early-stage study for the treatment of autosomal dominant optic atrophy, the most common inherited optic nerve disorder.
As of March 31, 2026, the company had $411 million in cash, cash equivalents and marketable securities, which is expected to fund operations into 2028.
STOK's Zacks Rank & Rising Estimates
Stoke Therapeutics currently has a Zacks Rank #2 (Buy). In the past 60 days, estimates for STOK’s 2026 loss per share have narrowed from $3.41 to $3.25, while the same for 2027 have narrowed from $2.90 to $2.80.
Positive regulatory updates and the successful development of zorevunersen should help the stock gain further momentum in 2026.
Other Stocks to Consider
Some top-ranked stocks in the biotech sector are Immunocore IMCR and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, 2026 loss per share estimates for Immunocore have narrowed from 97 cents to 16 cents, while estimates for 2027 have moved from a loss of 39 cents to earnings of 11 cents. IMCR stock has lost 14.9% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 46.66%.
Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have risen from $1.50 to $2.97, while estimates for 2027 have increased from $2.91 to $4.81. LQDA’s shares have surged 79.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters, while missing the same on the remaining occasion, with the average surprise being 54.40%.
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This article originally published on Zacks Investment Research (zacks.com).