Stoke Therapeutics, Inc.

Stoke Therapeutics, Inc. is a U.S.-based biopharmaceutical company focused on developing RNA-targeted medicines for severe genetic diseases. Its pipeline includes zorevunersen (STK-001) for Dravet syndrome, STK-002 for autosomal dominant optic atrophy, and additional product candidates developed through its TANGO platform and collaboration structure.

−10,2 %

−3,7 %

+404,5 %

5.28

5.28

— Stoke Therapeutics, Inc.
%
Lead clinical programs70% Drug candidates in human clinical development, including zorevunersen and STK-002.
Collaboration and licensing revenue30% Revenue earned from licensing IP and performing partnered development activities.

Stoke’s direct commercial customers are not yet established because its products are still in development; instead,...

  • Strategic collaboration partnersprimary

    Biogen and Acadia fund, develop, and potentially commercialize selected programs under alliance terms.

  • Neurologists and specialty prescribersprimary

    Physicians treating Dravet syndrome or other rare genetic disorders who would prescribe approved products.

  • Third-party payorssecondary

    Commercial and government payors that would influence access, reimbursement, and uptake.

  • Rare-disease patientsprimary

    Patients with severe genetic disorders who are the intended beneficiaries of the therapies.

Stoke is headquartered in the United States and conducts its core research and development activities from there...

  • •United States is the company’s operating and development base
  • •North America is the intended direct commercialization region
  • •Biogen covers zorevunersen outside North America
  • •Future alliances may extend reach into other large markets
  • •Revenue is collaboration-based rather than geography-based today

Stoke’s strategy centers on advancing its RNA-targeted rare-disease pipeline through clinical development and...

01
Advance zorevunersen toward approvalshort-term

It is the company’s most advanced and strategically important program.

02
Develop and de-risk STK-002 and other pipeline assetsmedium-term

Broader pipeline success reduces dependence on a single lead program.

03
Leverage partnerships for commercializationmedium-term

Alliances extend geographic reach and reduce the need for a large internal sales force.

Stoke is an early-stage biotechnology company, so its value depends heavily on successful clinical development,...

critical

Clinical development failure

Lead programs may not show sufficient safety or efficacy in later-stage trials.

Scope
zorevunersen, STK-002
Materiality
high
high

Regulatory approval risk

FDA or foreign regulators may delay, restrict, or deny approval.

Scope
All product candidates
Materiality
high
high

Partner commercialization risk

Biogen, Acadia, or future partners may not devote enough resources to launch and marketing.

Scope
Ex-North America and partnered programs
Materiality
high
high

Financing and dilution risk

The company expects to need additional capital to fund development and operations.

Scope
Corporate funding
Materiality
high
medium

Intellectual property risk

Protection of RNA platform and product rights is essential to commercialization value.

Scope
TANGO platform, product candidates
Materiality
medium
medium

Healthcare compliance risk

Future sales and distribution arrangements must comply with anti-kickback and fraud laws.

Scope
U.S. commercialization
Materiality
medium
Collaboration and license revenue recognition
Can create large quarter-to-quarter swings in reported revenue
Clinical development cost estimation
Affects operating expense timing and comparability
Stock-based compensation
Influences reported operating loss and dilution analysis
Impairment and valuation judgments
Could affect future earnings if asset values change

Structure Therapeutics Inc.

GPCR · Pharmaceutical Preparations

INTENSITY THERAPEUTICS, INC.

INTS · Biological Products, (No Diagnostic Substances)

Quince Therapeutics, Inc.

QNCX · Biological Products, (No Diagnostic Substances)

Lipocine Inc.

LPCN · Pharmaceutical Preparations

4D Molecular Therapeutics, Inc.

FDMT · Biological Products, (No Diagnostic Substances)

SOLIGENIX, INC.

SNGX · Pharmaceutical Preparations

: 29.4.2026