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 being developed for amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene, a form known as SOD1-ALS. It is an investigational small molecule and has not been approved for any use. The Phase 1 study enrolls adults with this genetic form of ALS.
ALN-SOD is being developed by Regeneron Pharmaceuticals, Inc., which trades on Nasdaq under the ticker REGN. Regeneron is the sponsor of the clinical program evaluating the small molecule in SOD1-ALS.
ALN-SOD is in Phase 1 development. The first-in-human study is currently recruiting participants and has not been completed. As an investigational therapy, ALN-SOD has not received FDA approval and remains in clinical testing for SOD1-ALS.
ALN-SOD is being evaluated in the first-in-human study NCT06351592, titled a First in Human Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene. The Phase 1 trial is recruiting, is controlled and randomized, and plans to enroll 54 participants across Australia, Belgium, Canada, Germany, Japan, Poland, South Korea, Sweden, and Taiwan.
The NCT06351592 study enrolls adults 18 years and older of any sex who have amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene. Healthy volunteers are not eligible. The trial is biomarker selected, meaning participants are identified by the SOD1 mutation, and it is recruiting across nine countries.