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INS1202

Phase 1

Amyotrophic Lateral Sclerosis | Gene therapy | Neurology |Insmed Incorporated|Last Updated: May 20, 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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment23
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07290062A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)PHASE1 RECRUITING 23Jan 9, 2026Mar 31, 2030May 20, 20265 United States
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Study Endpoints
Primary Endpoints
Incidence and Severity of Treatment Emergent Adverse Events (TEAEs)
Up to 48 weeks
Secondary Endpoints
Recommended Phase 2 Dose (RP2D) of INS1202-101
Up to 48 weeks
Viral Vector Shedding Following the IT Administration of INS1202 by Droplet Digital Polymerase Chain Reaction (ddPCR)
Baseline, Week 1, Week 2, Week 4, Week 16 and Week 32
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTALParticipants with sporadic amyotrophic lateral sclerosis (sALS) will be administered INS1202, IT injection at dose level 1 on Day 1.
Cohort 2EXPERIMENTALParticipants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 2 on Day 1.
Cohort 3EXPERIMENTALParticipants with either sALS or SOD1-ALS, will be administered INS1202 via IT injection at dose level 3 on Day 1.
Interventions
NameTypeDescription
INS1202GENETICSuspension for injection.
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Eligibility Criteria
Age Range18 Years — 79 Years
SexALL
Healthy VolunteersNo
Study Sites5

Key Inclusion Criteria: - * Participant with body mass index (BMI) ≥18 kilograms per square meter (kg/m\^2). * Participant with symptomatic ALS as diagnosed by Gold Coast diagnostic criteria. * Sporadic ALS cohorts: Negative testing for known monogenic mutations associated with familial ALS. * SOD1...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07290062primaryCompletionDate: changed
LOWMay 24, 2026NCT07290062studyFirstPostDate: changed