This section highlights FDA-related milestones and regulatory updates for drugs developed by Stoke Therapeutics (STOK).
Over the past two years, Stoke Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
STK-001, STK-002, and zorevunersen. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
STK-001 - FDA Regulatory Timeline and Events
STK-001 is a drug developed by Stoke Therapeutics for the following indication: Children and Adolescents with Dravet Syndrome.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- STK-001
- Announced Date:
- September 10, 2024
- Indication:
- Children and Adolescents with Dravet Syndrome
Announcement
Stoke Therapeutics, Inc announced highlights from presentations at the 15th European Epilepsy Congress (EEC) related to the Company's work to develop the first disease-modifying medicine for Dravet syndrome.
AI Summary
At the 15th European Epilepsy Congress, Stoke Therapeutics shared promising new data on its work to develop the first disease-modifying treatment for Dravet syndrome. The company presented results from its Phase 1/2a studies and open-label extension studies of zorevunersen (STK-001). Data showed that patients experienced substantial and sustained seizure reductions, along with meaningful improvements in cognition and behavior during the first year of treatment—with further gains observed as treatment continued. The treatment was generally well tolerated in the trials, suggesting that zorevunersen may offer benefits beyond those seen with current anti-seizure medications. These findings are significant because Dravet syndrome currently lacks disease-modifying therapies. The encouraging results have paved the way for the design of a Phase 3 registrational study, underlining Stoke Therapeutics’ commitment to addressing the underlying causes of this severe genetic epilepsy.
Read Announcement- Drug:
- STK-001
- Announced Date:
- September 3, 2024
- Indication:
- Children and Adolescents with Dravet Syndrome
Announcement
Stoke Therapeutics, Inc announced it will present data from its Dravet syndrome clinical program at the 15th European Epilepsy Congress (EEC) taking place September 7 – 11, in Rome, Italy.
AI Summary
Stoke Therapeutics, Inc. recently announced that it will present key data from its Dravet syndrome clinical program at the upcoming 15th European Epilepsy Congress (EEC) in Rome, Italy, from September 7 to 11. The presentations will include findings from both Phase 1/2a and open-label extension (OLE) studies of zorevunersen, an investigational antisense oligonucleotide designed to address the genetic basis of Dravet syndrome.
The data highlights substantial reductions in seizures as well as continued improvements in cognition and behavior among children and adolescents with the condition. The presentations will feature a dedicated platform session and two in-person poster sessions led by respected experts in pediatric epileptology. These sessions represent an important step in showcasing the potential of zorevunersen to change the treatment landscape for Dravet syndrome, paving the way for a future Phase 3 registrational study.
Read Announcement- Drug:
- STK-001
- Announced Date:
- August 7, 2024
- Estimated Event Date Range:
- July 1, 2024 - December 31, 2024
- Target Action Date:
- H2 2024
- Indication:
- Children and Adolescents with Dravet Syndrome
Announcement
Stoke Therapeutics, Inc announced that Company to provide an update on Phase 3 registrational plans for zorevunersen in the second half of 2024 –
AI Summary
Stoke Therapeutics announced that it will provide an update on its Phase 3 registrational plans for zorevunersen in the second half of 2024. The update will include details on the design of a global Phase 3 study as the company works with regulatory agencies around the world. This upcoming announcement is an important milestone as the company aims to advance zorevunersen, an investigational antisense oligonucleotide, for treating Dravet syndrome.
Zorevunersen is being developed as a potential disease-modifying therapy that addresses the genetic root cause of Dravet syndrome by restoring protein expression. The update is especially significant for families, caregivers, and clinicians who have shown strong support for innovative treatments in this area. This planned communication is eagerly awaited by stakeholders interested in the future of therapeutic options for Dravet syndrome.
Read Announcement- Drug:
- STK-001
- Announced Date:
- August 7, 2024
- Indication:
- Children and Adolescents with Dravet Syndrome
Announcement
Stoke Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has removed the Partial Clinical Hold on zorevunersen.
AI Summary
Stoke Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has removed the Partial Clinical Hold on its investigational drug, zorevunersen (STK-001). This milestone clears the way for the company to advance its plans toward a Phase 3 registrational study aimed at treating children and adolescents with Dravet syndrome. Zorevunersen is designed as a disease-modifying therapy that could reduce seizures and improve cognition and behavior by targeting the genetic cause of the condition. The FDA’s decision supports Stoke’s confidence in the potential benefits of zorevunersen. The company plans to provide an update on its Phase 3 study design and further discussions with global regulatory agencies in the second half of 2024. This progress is seen as a critical step forward in offering a new treatment option for patients and families impacted by Dravet syndrome.
Read Announcement
STK-002 - FDA Regulatory Timeline and Events
STK-002 is a drug developed by Stoke Therapeutics for the following indication: Autosomal Dominant Optic Atrophy (ADOA).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- STK-002
- Announced Date:
- August 7, 2024
- Indication:
- Autosomal Dominant Optic Atrophy (ADOA)
Announcement
Stoke Therapeutics, Inc announced that The Company is on track to initiate the Phase 1 study (OSPREY) of STK-002 for the treatment of Autosomal Dominant Optic Atrophy (ADOA) this year.
AI Summary
Stoke Therapeutics, Inc. revealed that it is on track to begin the Phase 1 study, known as OSPREY, of its candidate STK-002 later this year. This early study will evaluate the treatment of Autosomal Dominant Optic Atrophy (ADOA), a rare inherited condition that causes progressive and irreversible vision loss. The trial aims to assess the safety and initial effectiveness of STK-002, an antisense oligonucleotide developed using the company’s proprietary TANGO platform, which is designed to restore proper protein levels. If successful, STK-002 could become the first disease-modifying therapy for ADOA. This milestone is a significant step forward for Stoke Therapeutics as it continues to advance its portfolio of RNA medicine therapies, striving to address unmet medical needs in rare genetic disorders.
Read Announcement
zorevunersen - FDA Regulatory Timeline and Events
zorevunersen is a drug developed by Stoke Therapeutics for the following indication: For the treatment of Dravet syndrome with a confirmed mutation.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- zorevunersen
- Announced Date:
- July 10, 2025
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc and Biogen Inc announced the presentation of data from an analysis that informed the design of the Phase 3 EMPEROR study and evaluated the potential effects of the Phase 3 zorevunersen dosing regimen.
AI Summary
Stoke Therapeutics and Biogen recently presented data that helped shape the design of the Phase 3 EMPEROR study. The analysis focused on the zorevunersen dosing regimen, which consists of two loading doses of 70 mg followed by two maintenance doses of 45 mg. Results at Week 68 showed improvements in cognition and behavior in patients, a finding that stands in contrast to results observed with standard care in natural history studies. These encouraging results have led to the inclusion of key secondary endpoints focused on cognitive and behavioral assessments in the Phase 3 EMPEROR study. The data support the potential of zorevunersen as a first-in-class, disease-modifying treatment for Dravet syndrome by addressing not only seizures but also important neurodevelopmental challenges faced by patients.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- March 18, 2025
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc provided business updates.
AI Summary
Stoke Therapeutics, a biotechnology company focused on RNA medicine, provided a business update centered on its progress with zorevunersen, a potential disease-modifying treatment for Dravet syndrome. The company announced that its global Phase 3 EMPEROR study is on track to begin in the second quarter of 2025. Recent milestones, including positive data supporting the dosing regimen and global regulatory alignment, have boosted community enthusiasm and advanced the study timeline. In addition, an ongoing collaboration with Biogen is enhancing the company’s expertise in neurology and global commercialization, further preparing Stoke for launch readiness. As of December 31, 2024, Stoke reported $246.7 million in cash, cash equivalents, and marketable securities, positioning the company to fund operations through mid-2028. The update also noted that Dr. Edward M. Kaye will be transitioning from his CEO role to an advisory position while remaining on the board.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- February 18, 2025
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Biogen Inc announced a collaboration for the development and commercialization of zorevunersen, a potential first-in-class disease modifying medicine in development for the treatment of Dravet syndrome, in all territories outside the United States, Canada, and Mexico.
AI Summary
Biogen Inc has announced a collaboration with Stoke Therapeutics to develop and commercialize zorevunersen, an investigational antisense oligonucleotide aimed at treating Dravet syndrome. Under the deal, Biogen gains exclusive rights to commercialize zorevunersen in all territories outside the United States, Canada, and Mexico, where Stoke retains full rights. The collaboration marks a significant step for Biogen as it expands its rare disease pipeline, leveraging its global experience in commercializing innovative, disease-modifying medicines. Zorevunersen targets the SCN1A gene, which is the main root cause of Dravet syndrome, a severe condition marked by recurrent seizures and cognitive impairments. A pivotal Phase 3 study, known as EMPEROR, is scheduled to start in Q2 2025, with key data expected by the second half of 2027.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- January 7, 2025
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc. announced alignment with global regulatory agencies on the design of the Company's Phase 3 EMPEROR study of zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet syndrome.
AI Summary
Stoke Therapeutics, Inc. announced that it has achieved global regulatory alignment for its Phase 3 EMPEROR study on zorevunersen, a promising new treatment for Dravet syndrome. The study, designed with the input of the U.S. FDA, the European Medicines Agency (EMA), and Japan’s PMDA, will evaluate the drug’s ability to reduce major motor seizure frequency while also improving behavior and cognition in children and adolescents aged 2 to under 18 years. This research aims to determine if zorevunersen can become the first disease-modifying medicine for Dravet syndrome, addressing the unmet needs of patients who face both severe seizures and developmental challenges. With the planned study set to begin in mid-2025, the company’s progress marks a significant step towards offering more effective treatment options for this challenging condition.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- January 6, 2025
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc announced that its management team will host a webcast and conference call for investors and analysts to discuss successful alignment with global regulatory agencies related to a Phase 3 study of zorevunersen as potentially the first disease-modifying medicine for the treatment of Dravet syndrome.
AI Summary
Stoke Therapeutics, Inc. announced that their management team will host a webcast and conference call on Tuesday, January 7, 2025, at 8:00 am Eastern Time. During the call, investors and analysts will learn about the company’s successful alignment with global regulatory agencies regarding its Phase 3 study of zorevunersen. This study is examining the potential of zorevunersen to become the first disease-modifying medicine for treating Dravet syndrome—a severe genetic epilepsy.
The webcast will be available through Stoke’s Investors & News section, where interested participants can access the live session or register for the dial-in details. A replay of the event will also be available for at least 90 days after the webcast, ensuring that all stakeholders have the opportunity to review the discussion and the insights shared about this promising therapy development.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- December 6, 2024
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc announced new data from an analysis of nine patients treated with an initial 2 or 3 doses of 70mg, followed by 45mg maintenance dosing in the Phase 1/2a and open-label extension (OLE) studies of zorevunersen.
AI Summary
Stoke Therapeutics, Inc. announced promising new data from an analysis of nine patients who received an initial treatment of two or three doses of 70mg of zorevunersen, followed by a 45mg maintenance dose. In these patients from the Phase 1/2a and open-label extension studies, the results showed substantial and long-lasting reductions in convulsive seizure frequency when used alongside the best available anti-seizure medications.
Additionally, patients experienced continuous improvements in cognition and behavior over a period of up to two years of treatment. These encouraging findings support the proposed dose regimen for the upcoming Phase 3 registrational study. Zorevunersen was generally well tolerated, strengthening its profile as a potential disease-modifying therapy for Dravet syndrome.
Read Announcement- Drug:
- zorevunersen
- Announced Date:
- December 4, 2024
- Indication:
- For the treatment of Dravet syndrome with a confirmed mutation,
Announcement
Stoke Therapeutics, Inc announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for zorevunersen for the treatment of Dravet syndrome with a confirmed mutation, not associated with gain-of-function, in the SCN1A gene.
AI Summary
Stoke Therapeutics, Inc. recently received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for zorevunersen. This designation is specific to treating Dravet syndrome in patients who have a confirmed mutation in the SCN1A gene that is not linked to gain-of-function. The breakthrough status highlights the promising clinical data from early studies, which suggested that zorevunersen may significantly reduce seizure frequency and improve cognitive as well as behavioral outcomes when used with current anti-seizure medications.
By helping to restore natural levels of the NaV1.1 protein, zorevunersen aims to address the root cause of the condition rather than just its symptoms. The FDA’s decision is expected to expedite further development and review, offering hope for a more effective treatment option for patients with this challenging and severe form of epilepsy.
Read Announcement
