ALN-SOD / Alnylam, Regeneron - LARVOL DELTA

First-in-human study design to evaluate ALN-SOD in patients with ALS and SOD1 variants (JSNE 2025) - No abstract available

Clinical • P1 data • Amyotrophic Lateral Sclerosis

First-in-human study design to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ascending doses of ALN-SOD in patients with amyotrophic lateral sclerosis and SOD1 variants (ALS-MND 2024) - "Tofersen, a monthly intrathecally (IT) administered antisense oligonucleotide targeting SOD1, led to reductions in key disease-related biomarkers (SOD1 protein in cerebro-spinal fluid [CSF] and neurofilament light chain) (1). This FIH study will provide information about the safety, tolerability, pharmacokinetics, and pharmaco-dynamics of ALN-SOD in participants with SOD1-ALS. The results will guide selection of dosing regimens to assess in the subsequent clinical development program."

Clinical • P1 data • PK/PD data • Amyotrophic Lateral Sclerosis • CNS Disorders • NEFL • SOD1

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS) (clinicaltrials.gov) - P1 | N=42 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Amyotrophic Lateral Sclerosis • CNS Disorders

First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS) (clinicaltrials.gov) - P1 | N=42 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals

New P1 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2021 Financial Results and Highlights Recent Period Activity (BioSpace) - “Submitted a CTA for ALN-APP, an investigational RNAi therapeutic for the treatment of Alzheimer's disease and cerebral amyloid angiopathy. The Company announces today that it has initiated a Phase 1 study of ALN-APP in patients with early-onset Alzheimer’s disease….Disclosed new programs, including ALN-SOD targeting SOD-1, in development for the treatment of SOD-1-Specific Amyotrophic Lateral Sclerosis (ALS)…”

New trial • Trial status • Alzheimer's Disease • Amyotrophic Lateral Sclerosis • CNS Disorders