VX-670

Phase 2Small molecule

Vertex Pharmaceuticals

VX-670 is one of 4 small molecule programs targeting Myotonic Dystrophy. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Myotonic DystrophyActive, not recruiting

Myotonic Dystrophy Type 1 (DM1)Enrolling by invitation

Myotonic DystrophyPhase 2

Active, not recruiting

Myotonic Dystrophy Type 1 (DM1)Phase 2

Enrolling by invitation

Competition

Competitive Landscape

4 competitors in Myotonic Dystrophy

#DrugCompanyPhaseTrials

Mexitil

Boehringer Ingelheim

Approved

1 2

Tideglusib

AAMO Pharma Limited

Phase 3

4 3

VX-670

Vertex Pharmaceuticals

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1/2Recruiting

Myotonic Dystrophy

NCT06185764

Clinical

Trials by Indication[1]

Myotonic Dystrophy1 trial

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Recruiting· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1/2·NCT06185764·44 enrolled

Myotonic Dystrophy Type 1 (DM1)1 trial

An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type I

Enrolling by invitation· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT06926621·44 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for VX-670. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — VX-670 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for VX-670. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Vertex Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: Vertex Pharmaceuticals

VX-670

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Trials by Indication[1]

Myotonic Dystrophy1 trial

Myotonic Dystrophy Type 1 (DM1)1 trial

References

Drug Info

Quick Facts

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