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INS1202

Phase 1

Amyotrophic Lateral Sclerosis | Gene therapy | Neurology |Insmed Incorporated|Last Updated: Sep 1, 2026

Target and mechanism

Molecular targetSOD1
Target classGene
ModalityGene therapy

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment23

FDA Designations

No designations recorded

Clinical trial landscape

INS1202 · 1 trial · 1 indication

Phase 1 1
NCT07290062A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral Sclerosis
RECRUITING23 Analytics
PHASE1RECRUITING
A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral SclerosisUnlock trial analytics

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 to 79 Years
SexALL
Healthy VolunteersNo
Study Sites7

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)

LOWSep 1, 2026NCT07290062lastUpdatePostDate: changed
LOWSep 1, 2026NCT07290062lastUpdatePostDate: changed
LOWJul 6, 2026NCT07290062lastUpdatePostDate: changed
LOWJul 6, 2026NCT07290062lastUpdatePostDate: changed

Frequently asked questions about INS1202

What is INS1202 used for?

INS1202 is an investigational gene therapy being developed for the treatment of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease. It is administered as a single intrathecal injection and is currently in Phase 1 clinical development.

What does INS1202 target?

INS1202 targets the SOD1 gene, which is associated with a form of amyotrophic lateral sclerosis. By targeting this gene, the therapy aims to address the underlying genetic cause of the disease. It is delivered via a single intrathecal injection.

Who is developing INS1202?

INS1202 is being developed by Insmed Incorporated, a biopharmaceutical company listed on the NASDAQ under the ticker symbol INSM. The company is conducting a Phase 1 clinical trial to evaluate the safety and pharmacodynamics of the therapy in patients with ALS.

What phase is INS1202 in?

INS1202 is in Phase 1 clinical development. It is an investigational gene therapy that has not yet been approved by regulatory authorities. A Phase 1 trial is currently recruiting participants to assess its safety and pharmacodynamics in individuals with amyotrophic lateral sclerosis.

What clinical trials is INS1202 in?

INS1202 is being studied in a Phase 1 clinical trial with the identifier NCT07290062. This trial is investigating the safety and pharmacodynamics of a single intrathecal injection of INS1202 in participants with amyotrophic lateral sclerosis. The study is recruiting up to 23 participants in the United States.