Recent Updates
Recently added Catalysts

ENTR-601-44

Phase 1

Duchenne Muscular Dystrophy (DMD) | Small molecule | Neurology |Entrada Therapeutics, Inc.|Last Updated: Mar 9, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07037862A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44PHASE1 RECRUITING 24 — — Jun 30, 2025Mar 28, 2029Mar 9, 202614 Belgium, Italy +2
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and Open Label (OL) Period)
From baseline through End of Study (up to 62 weeks).

Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.

Secondary Endpoints
Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite (Part A and Open Label (OL) Period)
From Baseline through End of Study (up to 62 weeks).
Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A)
Baseline, End of Part A (up to 25 weeks)
Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)
Baseline, End of Part A (up to 25 weeks)
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ENTR-601-44EXPERIMENTALintravenous infusion every 6 weeks
PlaceboPLACEBO_COMPARATORintravenous infusion every 6 weeks
Interventions
NameTypeDescription
ENTR-601-44DRUGintravenous infusion
ENTR-601-44 - matching placeboDRUGintravenous infusion
Unlock Study Design Details
Eligibility Criteria
Age Range4 Years — 20 Years
SexMALE
Healthy VolunteersNo
Study Sites14

Principal inclusion criteria 1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor. 2. Assigned male at birth with clinical signs compatible with Duchenne muscular dys...

Countries:BelgiumItalySpainUnited Kingdom
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07037862primaryCompletionDate: changed
LOWMay 24, 2026NCT07037862studyFirstPostDate: changed