Stoke Therapeutics Stock Price, News & Analysis

Stoke Therapeutics, Inc. (Nasdaq: STOK) is a biotechnology company focused on restoring protein expression by harnessing the body’s potential with RNA medicine. The company is classified under pharmaceutical preparation manufacturing and is headquartered in Bedford, Massachusetts. Using its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke develops antisense oligonucleotides (ASOs) designed to selectively restore naturally occurring protein levels in genetic diseases.

Core focus and therapeutic areas

Stoke’s first medicine in development is zorevunersen, an investigational antisense oligonucleotide being evaluated as a potential first-in-class, disease-modifying treatment for Dravet syndrome. Dravet syndrome is described by the company as a severe developmental and epileptic encephalopathy characterized by recurrent seizures and significant cognitive and behavioral impairments. Most cases are associated with mutations in one copy of the SCN1A gene, which lead to insufficient levels of NaV1.1 protein in neuronal cells in the brain.

Zorevunersen is designed to increase functional NaV1.1 protein production in brain cells from the non‑mutated (wild‑type) copy of the SCN1A gene. According to Stoke, this differentiated mechanism of action aims to reduce seizure frequency beyond what has been achieved with anti‑seizure medicines and to improve neurodevelopment, cognition, and behavior. The company states that zorevunersen has demonstrated the potential for disease modification in patients with Dravet syndrome and is being evaluated in a global Phase 3 study.

Proprietary RNA medicine platform

Stoke’s TANGO platform is used to develop antisense oligonucleotides (ASOs) that selectively restore naturally occurring protein levels. The company describes its initial focus as diseases of the central nervous system and the eye that are caused by a loss of approximately 50% of normal protein levels, a mechanism referred to as haploinsufficiency. Stoke reports that proof of concept for its approach has been demonstrated in other organs, tissues, and systems, which it views as supporting broad potential for the platform.

Lead program: Zorevunersen for Dravet syndrome

Zorevunersen is the centerpiece of Stoke’s current pipeline. It is being studied in the EMPEROR Phase 3 study (NCT06872125), a global, double‑blind, sham‑controlled trial in children and adolescents with Dravet syndrome who have a confirmed SCN1A variant not associated with gain‑of‑function. In this study, participants are randomized 1:1 to receive zorevunersen via intrathecal administration or a sham comparator for a 52‑week treatment period following an 8‑week baseline period. Eligible participants are offered ongoing treatment in an open‑label extension (OLE) study.

The primary endpoint of EMPEROR is the percent change from baseline in major motor seizure frequency at week 28 in patients receiving zorevunersen compared with sham. Key secondary endpoints include durability of effect on major motor seizure frequency and improvements in behavior and cognition as measured by Vineland‑3 subdomains (expressive communication, receptive communication, interpersonal relationships, coping skills, and personal skills). Additional endpoints include safety and global impression of change measures reported by clinicians and caregivers, as well as developmental scales such as the Bayley Scales of Infant Development.

Stoke has reported multi‑year clinical data from Phase 1/2a and ongoing OLE studies of zorevunersen in Dravet syndrome. The company describes durable reductions in major motor seizure frequency on top of standard‑of‑care anti‑seizure medicines, along with continuing improvements in cognition and behavior over time. Analyses comparing treated patients to natural history data have been highlighted by Stoke as supporting the disease‑modifying potential of zorevunersen.

Regulatory designations and collaboration for zorevunersen

According to company disclosures, zorevunersen has been granted orphan drug designation by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has also granted rare pediatric disease designation and Breakthrough Therapy Designation for the treatment of Dravet syndrome with a confirmed mutation in SCN1A not associated with gain‑of‑function. As part of Breakthrough Therapy Designation, Stoke has held multidisciplinary meetings with the FDA to discuss the ongoing clinical development of zorevunersen and potential expedited regulatory pathways.

Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under this collaboration, Stoke retains exclusive rights for zorevunersen in the United States, Canada, and Mexico, while Biogen holds exclusive commercialization rights in the rest of the world. The companies have jointly announced milestones such as the dosing of the first patient in the global Phase 3 EMPEROR study and data presentations at major epilepsy conferences.

Additional pipeline: STK‑002 for Autosomal Dominant Optic Atrophy (ADOA)

Beyond Dravet syndrome, Stoke is developing STK‑002, a proprietary antisense oligonucleotide in clinical development for Autosomal Dominant Optic Atrophy (ADOA). ADOA is described as the most common inherited optic nerve disorder and a rare disease that causes progressive and irreversible vision loss in both eyes, often beginning in the first decade of life. An estimated 65% to 90% of ADOA cases are caused by variants in the OPA1 gene, many of which lead to haploinsufficiency and reduced OPA1 protein expression.

STK‑002 is designed to upregulate OPA1 protein expression by leveraging the non‑mutant (wild‑type) copy of the OPA1 gene, with the aim of maintaining or improving vision in people with ADOA. Stoke reports that it has generated preclinical data demonstrating proof‑of‑mechanism and proof‑of‑concept for STK‑002, and that a Phase 1 study, known as the OSPREY study, is underway. STK‑002 has received orphan drug designation from the FDA as a potential new treatment for ADOA.

Stoke has also sponsored the FALCON natural history study, a multicenter, 24‑month prospective study of people with OPA1‑associated ADOA. Data from FALCON have provided insights into disease etiology, progression, and clinical assessments, and the company states that these findings informed the design of the OSPREY Phase 1 study of STK‑002.

Research focus on haploinsufficiency diseases

Across its programs, Stoke emphasizes a focus on diseases caused by a loss of about half of normal protein levels, or haploinsufficiency. In addition to central nervous system and eye diseases, the company notes that proof of concept for its TANGO approach has been demonstrated in other organs, tissues, and systems. Stoke has also indicated that it is working to expand its pipeline, including lead optimization efforts to identify a clinical candidate targeting SYNGAP1, described as a severe and rare genetic neurodevelopmental disease.

Exchange listing and corporate governance

Stoke Therapeutics’ common stock trades on the Nasdaq Global Select Market under the ticker symbol STOK, as reflected in its SEC filings. The company files current reports on Form 8‑K and amendments on Form 8‑K/A that cover topics such as financial results, executive leadership changes, collaboration updates, clinical milestones, and shareholder voting outcomes. For example, the company has disclosed the appointment of its Chief Executive Officer and related employment and change‑of‑control agreements, as well as decisions regarding the frequency of advisory votes on executive compensation.

Position within biotechnology and rare disease

Within the broader biotechnology landscape, Stoke positions itself as a company dedicated to RNA‑based medicines that aim to restore protein expression rather than replace genes or supply exogenous proteins. Its programs are concentrated in rare, severe genetic diseases where there is a high unmet medical need, such as Dravet syndrome and ADOA. The company highlights multi‑year clinical and natural history data, regulatory designations, and collaborations as key elements of its development strategy.

Key themes for STOK stock research

• RNA medicine platform: Use of antisense oligonucleotides and the TANGO approach to increase expression from the non‑mutated allele in haploinsufficiency disorders.
• Lead asset risk and opportunity: Zorevunersen’s clinical profile in Dravet syndrome, including seizure reduction and potential cognitive and behavioral benefits, along with its regulatory designations and Phase 3 trial design.
• Rare disease focus: Concentration on severe, inherited conditions of the central nervous system and eye, including Dravet syndrome and ADOA.
• Partnerships: Strategic collaboration with Biogen for zorevunersen development and commercialization, with regional rights delineated between the parties.
• Pipeline breadth: Advancement of STK‑002 for ADOA and research activities aimed at additional targets such as SYNGAP1.
• Regulatory and clinical milestones: Ongoing Phase 3 EMPEROR study in Dravet syndrome and Phase 1 OSPREY study in ADOA, supported by natural history and OLE data.

Frequently asked questions about Stoke Therapeutics (STOK)

• What does Stoke Therapeutics do?
  

  Stoke Therapeutics is a biotechnology company that develops RNA‑based medicines intended to restore protein expression in genetic diseases. Using its TANGO approach, the company designs antisense oligonucleotides to selectively increase naturally occurring protein levels, with an initial focus on diseases of the central nervous system and the eye caused by haploinsufficiency.

• What is Stoke Therapeutics’ lead drug candidate?
  

  The company’s lead investigational medicine is zorevunersen, an antisense oligonucleotide in development as a potential first‑in‑class, disease‑modifying treatment for Dravet syndrome. It is being evaluated in the global Phase 3 EMPEROR study in children and adolescents with Dravet syndrome who have a confirmed SCN1A variant not associated with gain‑of‑function.

• How is zorevunersen designed to work?
  

  Zorevunersen is designed to increase functional NaV1.1 protein production in brain cells from the non‑mutated copy of the SCN1A gene. By boosting NaV1.1 levels, the goal is to reduce seizure frequency beyond what has been achieved with anti‑seizure medicines and to improve neurodevelopment, cognition, and behavior in people with Dravet syndrome.

• What regulatory designations has zorevunersen received?
  

  According to Stoke, zorevunersen has received orphan drug designation from the FDA and EMA, rare pediatric disease designation from the FDA, and Breakthrough Therapy Designation from the FDA for the treatment of Dravet syndrome with a confirmed SCN1A mutation not associated with gain‑of‑function.

• What is the EMPEROR study?
  

  The EMPEROR Phase 3 study (NCT06872125) is a global, double‑blind, sham‑controlled trial evaluating the efficacy, safety, and tolerability of zorevunersen in children ages 2 to under 18 with Dravet syndrome and a confirmed SCN1A variant not associated with gain‑of‑function. Participants are randomized to receive intrathecal zorevunersen or a sham comparator, and the primary endpoint is percent change from baseline in major motor seizure frequency at week 28.

• What is STK‑002 and which disease does it target?
  

  STK‑002 is a proprietary antisense oligonucleotide in clinical development for Autosomal Dominant Optic Atrophy (ADOA), a rare inherited optic nerve disorder that leads to progressive and irreversible vision loss. STK‑002 is designed to upregulate OPA1 protein expression from the non‑mutant copy of the OPA1 gene with the aim of maintaining or improving vision. It is being studied in the Phase 1 OSPREY trial and has received orphan drug designation from the FDA.

• What is the FALCON study and why is it important?
  

  FALCON is a 24‑month, multicenter, prospective natural history study of people with OPA1‑associated ADOA. It evaluated changes in structural and functional ophthalmic assessments over time. Stoke reports that FALCON is the largest prospective natural history study in ADOA and that its findings have informed the clinical development of STK‑002, including the design of the OSPREY Phase 1 study.

• Which partners does Stoke Therapeutics work with?
  

  Stoke has a strategic collaboration with Biogen to develop and commercialize zorevunersen for Dravet syndrome. Under this agreement, Stoke retains exclusive rights in the United States, Canada, and Mexico, while Biogen holds exclusive rights in the rest of the world.

• On which exchange does STOK trade and what is the security type?
  

  Stoke Therapeutics’ common stock, with a par value of $0.0001 per share, is listed on the Nasdaq Global Select Market under the trading symbol STOK, as disclosed in the company’s SEC filings.

• Where is Stoke Therapeutics headquartered?
  

  The company states that it is headquartered in Bedford, Massachusetts.