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SAR446268

Phase 1

Myotonic Dystrophy | Monoclonal antibody | Other |Sanofi|Last Updated: Jun 1, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06844214A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1PHASE1 RECRUITING 32 — — Jul 23, 2025Apr 27, 2032Jun 1, 20269 United States, Argentina +4
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Study Endpoints
Primary Endpoints
Part A and Part B: Incidence of treatment-emergent adverse events (TEAEs) following SAR446268 administration
Baseline to Week 52

Number of TEAEs post-SAR446268 administration

Part B: Proportion of participants with at least 40% DMPK mRNA knockdown in muscle biopsy at Weeks 12 and 52 following SAR446268 administration
Weeks 12 and 52
Secondary Endpoints
Part A: Change in 10-meter walk-run test from baseline to Weeks 26 and 52 following SAR446268 administration
Baseline to Week 26 and 52
Part A: Change in myotonia from baseline to Weeks 26 and 52 following SAR446268 administration as measured by the hand opening time (middle finger)
Baseline to Week 26 and 52
Part A: Change in bilateral hand grip test from baseline to Weeks 26 and 52 following SAR446268 administration
Baseline to Week 26 and 52
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SAR446268EXPERIMENTALParticipants will receive a single dose of SAR446268 on Day 1
Interventions
NameTypeDescription
SAR446268BIOLOGICALPharmaceutical form: Solution for infusion; Route of administration: IV infusion
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Eligibility Criteria
Age Range10 Years — 55 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: Participants are eligible to be included in the study only if all of the following criteria apply: * For Part A, participants must be 18 to 55 years of age inclusive, at the time of signing the informed consent. * For Part B, participants must be as follows: * 10 to 17 years...

Countries:United StatesArgentinaAustraliaCanadaIsraelUnited Kingdom
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Recent Changes (Last 90 Days)
LOWJun 2, 2026NCT06844214lastUpdatePostDate: changed
LOWJun 2, 2026NCT06844214lastUpdatePostDate: changed
LOWJun 2, 2026NCT06844214lastUpdatePostDate: changed
MEDIUMMay 26, 2026NCT06844214primaryCompletionDate: changed
LOWMay 24, 2026NCT06844214studyFirstPostDate: changed