Approval Probability
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BIIB078 · 1 trial · 1 indication
An AE is any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. An SAE is an AE resulting in any of the following outcomes or deemed significant for any other reason: death, places participant at immediate risk of death, requires initial or prolonged inpatient hospitalization, results in persistent or significant disability/incapacity, results in congenital anomaly, is a medically important event.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: BIIB078 First Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 2: BIIB078 Second Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 3: BIIB078 Third Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days. |
| Cohort 4: BIIB078 Fourth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohort 5: BIIB078 Fifth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohort 6: BIIB078 Sixth Dosage | EXPERIMENTAL | BIIB078 will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by five maintenance doses on five later days. |
| Cohorts 1-6: Placebo | PLACEBO_COMPARATOR | Matching placebo will be administered as a loading regimen of 3 doses, on Day 1 and two later days, followed by two maintenance doses on two later days (Cohorts 1 through 3) and five maintenance doses on five later days (Cohorts 4 through 6). |
| Name | Type | Description |
|---|---|---|
| BIIB078 | DRUG | Administered as specified in the treatment arm. |
| Placebo | DRUG | Administered as specified in the treatment arm. |
Key Inclusion Criteria: * Ability of the participant to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize the use of confidential health information in accordance with national and local participant privacy regulations; or, in the event of...
BIIB078 is an investigational small molecule being developed for the treatment of amyotrophic lateral sclerosis (ALS). It is being studied in adults with C9ORF72-associated ALS, a genetic form of the disease. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
BIIB078 is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. Biogen is conducting clinical research on this investigational therapy for amyotrophic lateral sclerosis.
BIIB078 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or other regulatory agencies. The Phase 1 trial has been completed, and the drug remains in early-stage clinical research for amyotrophic lateral sclerosis.
BIIB078 has one completed Phase 1 clinical trial registered as NCT03626012. This study assessed the safety, tolerability, and pharmacokinetics of BIIB078 in adults with C9ORF72-associated amyotrophic lateral sclerosis. The trial enrolled 106 participants across the United States, Canada, Ireland, Netherlands, Switzerland, and the United Kingdom.
BIIB078 is the primary name for this investigational drug. No alternative names have been reported for this compound in the available clinical trial information. It is being studied specifically for C9ORF72-associated amyotrophic lateral sclerosis.