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A Phase 1 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ARO-SOD1 in Adult Patients With Amyotrophic Lateral Sclerosis Harboring a Superoxide Dismutase-1 Mutation Considered to be Causative of Amyotrophic Lateral Sclerosis
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jul 28, 2026
Report code
NCT05949294-Jul 28, 2026
NCT ID
NCT05949294
Status
WITHDRAWN
Phase
Phase 1
Sponsor
Arrowhead Pharmaceuticals
Executive brief
Investment-Ready Snapshot
ARO-SOD1, developed by Arrowhead Pharmaceuticals, targets a specific genetic mutation (SOD1) implicated in ALS, a progressive neurodegenerative disease with significant unmet medical need. The market for ALS therapies is expanding, particularly for genetic forms of the disease, with increasing interest from both patients and investors. However, the trial has been withdrawn due to a company business decision, which may indicate strategic realignment or resource allocation issues. This withdrawal could impact Arrowhead's competitive positioning in the ALS space, especially against other emerging therapies targeting SOD1 and other genetic mutations. Diligence implications include a need to assess the company's pipeline and financial health post-withdrawal.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT05949294
Indication
Amyotrophic Lateral Sclerosis
Modality
small molecule
Target
Superoxide Dismutase-1 (SOD1) mutation associated with Amyotrophic Lateral Sclerosis (ALS)
Intervention
ARO-SOD1 Injection
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.