Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ALN-SOD · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| Cohort 1 - Dose Level 1 | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 2 - Dose Level 2 | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 3 - Dose Level 3 | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 4 - Dose Level 4 | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 5 (Optional) - ≤ Dose Level 5 | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Selected Dose Expansion Cohort(s) | EXPERIMENTAL | Open Label Treatment (OLT) at selected dose levels. EU Participants can only be included in the Dose Expansion portion of study (Phase 2), not in Dose Escalation (Phase 1) |
| Name | Type | Description |
|---|---|---|
| ALN-SOD | DRUG | Administered per the protocol |
| Diluent | OTHER | Administered per the protocol |
| Placebo (PB) | DRUG | Administered per the protocol |
Key Inclusion Criteria: 1. Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol 2. Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in uprigh...
ALN-SOD is an investigational small molecule being developed for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
ALN-SOD is being developed by Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN). The company is conducting a Phase 1 clinical trial to evaluate the drug in adult participants with SOD1-ALS.
ALN-SOD is in Phase 1 clinical development. It is an investigational drug and has not been approved for any use. A first-in-human study is currently recruiting participants to evaluate its safety and tolerability.
ALN-SOD is being studied in a Phase 1 first-in-human trial with the identifier NCT06351592. The trial is recruiting 54 adult participants with ALS associated with a SOD1 gene mutation across multiple countries, including the United States, Australia, and several European and Asian nations.
ALN-SOD is a distinct investigational drug candidate. No alternative names have been reported for this asset. It is being studied specifically for SOD1-mutation-associated ALS and is not known to be identical to any other approved or investigational therapy.