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SRP-1005

Phase 1

Huntington's Disease | Small molecule | Rare Disease |Sarepta Therapeutics, Inc.|Last Updated: Jun 4, 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
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07536061A First-in-human Study of the Effects of SRP-1005 in Participants With Huntington's DiseasePHASE1 RECRUITING 32May 12, 2026Nov 30, 2027Jun 4, 20262 New Zealand
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Study Endpoints
Primary Endpoints
Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
Baseline through Week 24
Secondary Endpoints
Plasma Concentration of SRP-1005
Baseline through Day 17
Urine Concentration of SRP-1005
Baseline through Day 17
Cerebrospinal Fluid Concentration of SRP-1005
Baseline through Week 24
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1: SRP-1005 Dose 1EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 2: SRP-1005 Dose 2EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 3: SRP-1005 Dose 3EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Cohort 4: SRP-1005 Dose 4EXPERIMENTALParticipants will receive SRP-1005 or placebo.
Interventions
NameTypeDescription
SRP-1005DRUGSubcutaneous injection
PlaceboDRUGSubcutaneous injection
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Eligibility Criteria
Age Range21 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites2

Key Inclusion Criteria: * Genetically confirmed diagnosis of huntingtin (HTT) cytosine-adenine-guanine repeat length ≥40. * Participant has HD Integrated Staging System (HD-ISS) Stage 2 or Mild Stage 3. At screening, the participant must be classified with the Enroll-HD HD-ISS Modified Calculator a...

Countries:New Zealand
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Competitive Landscape -Huntington's Disease 9 trials
Recent Changes (Last 90 Days)
LOWJun 4, 2026NCT07536061Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 4, 2026NCT07536061Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 4, 2026NCT07536061Status: NOT_YET_RECRUITING → RECRUITING
LOWJun 4, 2026NCT07536061Status: NOT_YET_RECRUITING → RECRUITING
LOWMay 26, 2026NCT07536061primaryCompletionDate: changed
LOWMay 24, 2026NCT07536061studyFirstPostDate: changed
LOWMay 21, 2026NCT07536061NEW_TRIAL: changed
LOWMay 21, 2026NCT07536061NEW_TRIAL: changed