Stoke Therapeutics develops RNA medicines intended to restore protein expression, using its TANGO approach and antisense oligonucleotides for diseases caused by insufficient gene output. Its lead investigational medicine, zorevunersen, is in development for Dravet syndrome, with earlier work directed at rare genetic neurodevelopmental and other haploinsufficiency diseases.
Company news commonly covers zorevunersen clinical data, EMPEROR study updates, financial results, capital resources, collaboration activity, board and governance changes, equity inducement grants under Nasdaq rules, and healthcare conference presentations.
Stoke Therapeutics (Nasdaq: STOK) announced that its executives will present at investor conferences beginning November 10, 2026. Chief Executive Officer Ian F. Smith and Chief Patient Officer Jason Hoitt will present at the Guggenheim Healthcare Innovation Conference in Boston at 1:00 p.m. ET and at the Jefferies London Global Healthcare Conference on November 17 at 4:00 p.m. GMT. Live webcasts and archived replays will be available on Stoke’s investor website.
Stoke Therapeutics (STOK) reported a third-quarter net loss of $73.6 million and updated progress on its clinical development programs. Revenue rose to $11.3 million from $10.6 million a year earlier, while the loss widened from $38.3 million. Cash, cash equivalents and marketable securities totaled $358.8 million at September 30, 2026; Stoke expects funding into early 2028.
In the Phase 3 EMPEROR study of investigational Dravet syndrome treatment zorevunersen, approximately 110 patients reached Week 28 and approximately 20 reached Week 52, with no treatment discontinuations. Stoke anticipates data in Q3 2027 and plans a U.S. New Drug Application in H2 2027. Following FDA discussions, cognition and behavior will be evaluated using four Vineland-3 assessment subdomains. Third-cohort dosing is underway in the Phase 1 STK-002 study for autosomal dominant optic atrophy, with initial safety and efficacy data anticipated in H1 2027.
Stoke Therapeutics (Nasdaq: STOK) will host a webcast and conference call on October 29, 2026, to discuss third-quarter business and financial updates. Management will speak at 4:30 p.m. ET. The webcast will be available through the Investors & News section of Stoke’s website, with an archived replay available for at least 90 days following the event.
Stoke Therapeutics (Nasdaq: STOK) granted stock options to its newly appointed chief information officer and seventeen other employees, effective October 15, 2026.
Stephen Aschettino, Senior Vice President and Chief Information Officer, received options covering 70,000 shares; the other employees received options covering an aggregate of 175,920 shares. The employment inducement awards have a $24.61 exercise price, equal to the October 15 closing share price, and a 10-year term. Vesting occurs over four years: 1/4th on the first anniversary of the applicable vesting commencement date, then 1/48th monthly over the following 36 months, subject to continued employment. The grants fall under the 2023 Inducement Plan and Nasdaq Listing Rule 5635(c)(4).
Stoke Therapeutics (STOK) presented STK-002 preclinical and natural history data supporting its ongoing Phase 1 study in autosomal dominant optic atrophy. This inherited optic nerve disorder causes progressive vision loss. Preclinical treatment increased OPA1 protein expression and improved mitochondrial function in patient-derived cells. The FALCON natural history study linked higher mitochondrial oxidative stress with decreased visual acuity.
Dosing is complete in the first two OSPREY cohorts. Stoke expects third-cohort dosing to finish by year-end 2026, with fourth-cohort dosing to follow subject to ongoing safety assessments and initial safety and efficacy results from these latter cohorts anticipated in the first half of 2027.
Stoke Therapeutics (STOK) completed dosing in the first two cohorts of its Phase 1 OSPREY study of STK-002 for autosomal dominant optic atrophy.
The company expects third-cohort dosing to finish by year-end 2026, with fourth-cohort dosing to follow subject to ongoing safety assessments. Stoke expects cohorts three and four to reach therapeutic dosing levels and anticipates initial safety and efficacy data from those cohorts in the first half of 2027. STK-002 is an investigational treatment designed to increase OPA1 protein levels. Preclinical data and study progress will be presented at the AAO annual meeting, October 9–12 in New Orleans. All eight planned trial sites are active.
Stoke Therapeutics (STOK) completed an FDA meeting on its planned U.S. application for zorevunersen, an investigational treatment for Dravet syndrome. Following the discussion, Stoke plans to assess cognition and behavior in Phase 3 EMPEROR using four prespecified Vineland-3 subdomains: expressive communication, receptive communication, interpersonal relationships and personal skills.
Stoke plans to submit data throughout the first half of 2027, anticipates a Phase 3 readout in the third quarter of 2027, and plans to complete its New Drug Application in the second half of 2027, supporting potential U.S. approval and launch in early 2028. EMPEROR compares zorevunersen with a sham procedure over 52 weeks. Its primary endpoint measures seizure-frequency change at Week 28; secondary endpoints assess durability and adaptive functioning at Week 52. All study data will be analyzed at Week 52.
Stoke Therapeutics (STOK) appointed Bo Cumbo to its board on September 23, 2026, as director Edward Kaye retired. Kaye had served on the board since 2017 and was Stoke’s chief executive officer until March 2025. He will continue to advise the board through the transition. Cumbo has more than 30 years of biopharmaceutical experience and has been president and chief executive officer of Solid Biosciences since 2022.
Cumbo previously served as Sarepta Therapeutics’ chief commercial officer, where he worked on launches of treatments for Duchenne muscular dystrophy. Stoke is evaluating zorevunersen, an investigational treatment for Dravet syndrome, in a Phase 3 study. Stoke’s chief executive said the company is preparing for a potential FDA approval in early 2028. Zorevunersen’s safety and efficacy have not been evaluated by a regulatory authority. Stoke collaborates with Biogen (BIIB) on its development and commercialization.
Stoke Therapeutics (STOK) granted inducement stock options for an aggregate 53,175 common shares to nine new employees effective September 15, 2026, under Nasdaq Listing Rule 5635(c)(4).
The options have an exercise price of $28.15 per share, equal to the closing price of the common stock on the grant date, and a 10-year term. Each award vests over four years: 25% on the one-year anniversary of the applicable vesting commencement date, with the remaining 75% vesting in equal monthly instalments over the next 36 months, subject to continued employment and the terms of the 2023 Inducement Plan and the related option agreements. Stoke’s lead investigational medicine, zorevunersen, is in Phase 3 development for Dravet syndrome.
Stoke Therapeutics (STOK) and Biogen (BIIB) reported long-term clinical data for investigational Dravet syndrome therapy zorevunersen from Phase 1/2a and open-label extension studies, and detailed the ongoing global Phase 3 EMPEROR trial.
Four-year extension data showed durable reductions in seizures and statistically significant improvements in cognition and behavior at 1, 2, 3 and 4 years versus open-label baseline. Exploratory analyses indicated substantial reductions in the most severe seizure types through 3 years and improved quality of life through 28 months. Zorevunersen was generally well tolerated over more than 930 administered doses, with some patients treated for more than 5 years; elevated cerebrospinal fluid protein was common but without serious clinical manifestations or hydrocephalus. The EMPEROR study has completed enrollment in the United States, United Kingdom, Japan and Europe, with 162 patients in the primary analysis population and 34 in Europe, and a Phase 3 data readout is anticipated in Q3 2027 to complete a planned rolling U.S. NDA submission in the second half of 2027.