Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BIIB100 · 1 trial · 1 indication
An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, or is a medically important event.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: BIIB100 Dose 1 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 2: BIIB100 Dose 2 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 3: BIIB100 Dose 3 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 4: BIIB100 Dose 4 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 5: BIIB100 Dose 5 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 6: BIIB100 Dose 6 | EXPERIMENTAL | Participants will receive single oral dose of BIIB100 on Day 1. |
| Cohort 1-6: Matching Placebo | PLACEBO_COMPARATOR | Participants will receive single oral dose of matching placebo on Day 1. |
| Name | Type | Description |
|---|---|---|
| BIIB100 | DRUG | Administered as specified in the treatment arm. |
| Placebo | DRUG | Administered as specified in the treatment arm. |
Key Inclusion Criteria: * Must meet the laboratory-supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology El Escorial criteria. * Participants taking concomitant riluzole at study entry must be on a stable dose for greater than or equals...
BIIB100 is an investigational small molecule being studied for the treatment of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease. It is administered orally and is currently in clinical development for this condition.
BIIB100 is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. The company is conducting clinical research to evaluate the safety and efficacy of this oral small molecule in patients with ALS.
BIIB100 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. A Phase 1 study has been completed to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in adults with ALS.
BIIB100 has one completed Phase 1 clinical trial, identified as NCT03945279. This randomized, double-blind, placebo-controlled study enrolled 49 adults with ALS in the United States to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of orally administered BIIB100.
BIIB100 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for amyotrophic lateral sclerosis. The completed Phase 1 trial was designed to evaluate safety and tolerability, and further studies would be needed to support any regulatory approval.