Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BIIB147 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| MAD: Cohort A | EXPERIMENTAL | Participants will either receive Dose A of BIIB147 or a placebo for 12 weeks. |
| MAD: Cohort B | EXPERIMENTAL | Participants will either receive Dose B of BIIB147 or a placebo for 12 weeks. |
| MAD: Cohort C | EXPERIMENTAL | Participants will either receive Dose C of BIIB147 or a placebo for 12 weeks. |
| MAD: Cohort D | EXPERIMENTAL | Participants will either receive Dose D of BIIB147 or a placebo for 20 weeks. |
| OLE | EXPERIMENTAL | Participants from Cohorts A-D who complete the MAD period will enter a 4-week blinded loading-dose period. During this period, participants randomized to BIIB147 in the MAD period will receive placebo, followed by BIIB147, while participants randomized to placebo during the MAD period will receive BIIB147. Upon completion of the blinded loading-dose period, all participants will enter the open-label maintenance dose period and receive BIIB147 for up to 340 weeks. |
| Name | Type | Description |
|---|---|---|
| BIIB147 | DRUG | Administered intrathecally (IT) |
| Placebo | DRUG | Administered IT |
Key Inclusion Criteria: MAD Period * Participants must have a diagnosis of ALS consistent with the Gold Coast criteria. * No pathogenic variants, likely pathogenic variants, or variants of uncertain significance in the superoxide dismutase 1 (SOD1) or fused in sarcoma (FUS) genes (confirmed by cen...
BIIB147 is an investigational small molecule being studied for the treatment of amyotrophic lateral sclerosis (ALS). It is not approved for any indication and remains in clinical development. The only trial listed for BIIB147 is a Phase 1 study in adults with ALS, so its use is currently limited to research settings.
BIIB147 is being developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen is the sponsor of the Phase 1 study evaluating BIIB147 in adults with amyotrophic lateral sclerosis. No other developer or partner is identified for this asset.
BIIB147 is in Phase 1 clinical development. The single registered trial for BIIB147 is a Phase 1 study in adults with amyotrophic lateral sclerosis. Because it is still in Phase 1, BIIB147 is investigational and has not been approved by the FDA for any indication.
BIIB147 is being evaluated in one clinical trial, NCT07855211, titled A Study to Learn About the Safety of BIIB147 and Its Effects in Adults With Amyotrophic Lateral Sclerosis (ALS). The Phase 1 trial is not yet recruiting, plans to enroll 69 participants, is randomized, double blind, and controlled, and includes adults 18 years and older.
No, BIIB147 is not FDA approved. It is an investigational small molecule in Phase 1 clinical development for amyotrophic lateral sclerosis. The only registered study, NCT07855211, is a Phase 1 safety and effects trial that has not yet begun recruiting participants.