Last reviewed 2026-05-29 · How we verify

STK-001 - Multiple Ascending Doses

Stoke Therapeutics, Inc · Phase 1 active Small molecule ✓ Verified May 2026

STK-001 - Multiple Ascending Doses is a Small molecule drug developed by Stoke Therapeutics, Inc. It is currently in Phase 1 development.

STK-001 is a small molecule being investigated in an open-label study for its safety and pharmacokinetics in children and adolescents with Dravet Syndrome. The study involves administering single and multiple ascending doses of STK-001 to participants with Dravet Syndrome.

Likelihood of approval

9.6% vs 9.6% industry baseline

If approved by FDA: likely 2033–2036

Steps remaining: Phase 2 → Phase 3 → NDA/BLA submission

Confidence: Medium

Why this estimate

Baseline phase 1 → approval rate +9.6pp
Industry-wide phase 1 drugs reach approval ~9.6% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas).

Predicted approval windows by jurisdiction (conditional on FDA approval)

Regulator	Country	Likely year	Lag vs FDA
FDA	US	2033–2036	—
EMA	EU	2034–2037	+0.7 yr
MHRA	GB	2034–2037	+0.7 yr
Health Canada	CA	2034–2038	+0.9 yr
TGA	AU	2034–2038	+1.2 yr
PMDA	JP	2034–2038	+1.5 yr
NMPA	CN	2035–2039	+2.3 yr
MFDS	KR	2034–2038	+1.4 yr
CDSCO	IN	2034–2039	+1.8 yr
ANVISA	BR	2035–2039	+2.3 yr

Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).

Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.

At a glance

Generic name	STK-001 - Multiple Ascending Doses
Sponsor	Stoke Therapeutics, Inc
Modality	Small molecule
Phase	Phase 1

Approved indications

No approved indications tracked.

Common side effects

No common side effects on file.

Key clinical trials

An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome (PHASE1, PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

STK-001 - Multiple Ascending Doses CI brief — competitive landscape report
STK-001 - Multiple Ascending Doses updates RSS · CI watch RSS
Stoke Therapeutics, Inc portfolio CI

Frequently asked questions about STK-001 - Multiple Ascending Doses

What is STK-001 - Multiple Ascending Doses?

STK-001 - Multiple Ascending Doses is a Small molecule drug developed by Stoke Therapeutics, Inc.

Who makes STK-001 - Multiple Ascending Doses?

STK-001 - Multiple Ascending Doses is developed by Stoke Therapeutics, Inc (see full Stoke Therapeutics, Inc pipeline at /company/stoke-therapeutics-inc).

What development phase is STK-001 - Multiple Ascending Doses in?

STK-001 - Multiple Ascending Doses is in Phase 1.

Manufacturer: Stoke Therapeutics, Inc — full pipeline

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing