Recent Updates
Recently added Catalysts

VX-670

Phase 2

Myotonic Dystrophy Type 1 (DM1) | Small molecule | Rare Disease |Vertex Pharmaceuticals Incorporated|Last Updated: Aug 20, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment91

FDA Designations

No designations recorded

Clinical trial landscape

VX-670 · 2 trials · 1 indication

Phase 2 1Phase 1 1
NCT06926621A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type IMyotonic Dystrophy Type 1 (DM1)
ENROLLING BY_INVITATION44 Analytics
PHASE2ENROLLING BY_INVITATION
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
Myotonic Dystrophy Type 1 (DM1)Unlock trial analytics

Study Endpoints

Primary Endpoints

Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 108
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168

Secondary Endpoints

Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
From Day 1 up to Week 96
Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma
From Day 1 up to Week 96
Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma
From Day 1 up to Day 42
Unlock Study Endpoints

Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
VX-670EXPERIMENTALParticipants will receive multiple doses of VX-670.
Part A: Single Ascending DoseEXPERIMENTALParticipants will be randomized to receive a single dose of different dose levels of VX-670.
Part A: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive single dose of placebo matched to VX-670.
Part B: Single and Multiple Ascending DoseEXPERIMENTALParticipants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A.
Part B: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive single or multiple doses of placebo matched to VX-670.

Interventions

NameTypeDescription
VX-670DRUGSolution for intravenous administration.
PlaceboDRUGSolution for intravenous administration.
Unlock Study Design Details

Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites15

Key Inclusion Criteria: • Completed study drug treatment in parent study VX23-670-001 (NCT06185764) Key Exclusion Criteria: • History of any illness or any clinical condition as pre-specified in the protocol Other protocol defined Inclusion/Exclusion criteria may apply.

Countries:United StatesAustraliaBelgiumCanadaGermanyNetherlandsSpainUnited KingdomFranceItaly
Unlock Eligibility Criteria

Recent Changes (Last 90 Days)

MEDIUMAug 20, 2026NCT06185764Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 20, 2026NCT06185764Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWAug 11, 2026NCT06926621lastUpdatePostDate: changed
LOWAug 11, 2026NCT06926621lastUpdatePostDate: changed
LOWAug 11, 2026NCT06926621lastUpdatePostDate: changed
LOWJun 30, 2026NCT06926621lastUpdatePostDate: changed
LOWJun 30, 2026NCT06926621lastUpdatePostDate: changed
LOWJun 30, 2026NCT06926621lastUpdatePostDate: changed
LOWJun 8, 2026NCT06185764Enrollment: 44 → 52
LOWJun 8, 2026NCT06185764Enrollment: 44 → 52
LOWJun 8, 2026NCT06185764Enrollment: 44 → 52

Frequently asked questions about VX-670

What is VX-670 used for in Myotonic Dystrophy Type 1 (DM1)?

VX-670 is an investigational small molecule being developed for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare genetic disorder. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities. The drug is being studied in adult participants with DM1.

Who makes VX-670?

VX-670 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting clinical trials to evaluate the safety and efficacy of VX-670 in patients with Myotonic Dystrophy Type 1.

What phase is VX-670 in?

VX-670 is in Phase 2 clinical development. It is being evaluated in two active clinical trials, including a Phase 1/2 study and a Phase 2 long-term safety and efficacy study. The drug is investigational and has not been approved by the FDA or other regulatory agencies.

What clinical trials is VX-670 in?

VX-670 is being studied in two active clinical trials. NCT06185764 is a Phase 1/2 study in adult participants with Myotonic Dystrophy Type 1, enrolling 47 participants across multiple countries. NCT06926621 is a Phase 2 long-term safety and efficacy study with 44 participants. Both trials are placebo-controlled and double-blinded.

Is VX-670 FDA approved?

VX-670 is not FDA approved. It is an investigational drug currently in Phase 2 clinical trials for Myotonic Dystrophy Type 1. The drug is being studied for safety and efficacy, and regulatory approval has not been granted.