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Also known as ISIS 814907, BIIB080
Diranersen · 1 trial · 2 indications
The Clinical Dementia Rating (CDR) scale is a clinician-rated dementia staging system that tracks the progression of cognitive impairment in 6 categories (memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care). Each category is scored on a 5-point scale in which None=0, Questionable=0.5, Mild=1, Moderate=2, and Severe=3. The global CDR score is established by clinical scoring rules and has values of 0 (no dementia), 0.5, (questionable dementia), 1 (mild dementia), 2 (moderate dementia), and 3 (severe dementia). The CDR-SB is obtained by adding the ratings in each of the 6 categories and ranges from 0 to 18 with higher scores indicative of greater impairment.
| Arm | Type | Description |
|---|---|---|
| Placebo Q12W | PLACEBO_COMPARATOR | Participants will receive diranersen-matching placebo, intrathecal (IT) injection, once on Day 1 and then once every 12 weeks (Q12W) for up to 72 weeks, during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will be randomized to receive diranersen high dose, IT injection, either Q12W or once every 24 weeks (Q24W) for an additional 96 weeks. |
| Diranersen Low Dose Q24W | EXPERIMENTAL | Participants will receive a low dose of diranersen, IT injection, Q24W from Week 1 up to 72 weeks and diranersen-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive diranersen low dose, IT injection, Q24W for an additional 96 weeks. |
| Diranersen High Dose Q24W | EXPERIMENTAL | Participants will receive a high dose of diranersen, IT injection, Q24W from Week 1 up to 72 weeks and diranersen-matching placebo, IT injection, once at Weeks 12, 36, and 60 during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive diranersen high dose, IT injection, Q24W for an additional 96 weeks. |
| Diranersen High Dose Q12W | EXPERIMENTAL | Participants will receive a high dose of diranersen, IT injection, once on Day 1 and then Q12W for up to 72 weeks during the placebo-controlled period. Eligible participants will enter the LTE period, during which they will continue to receive diranersen high dose, IT injection, Q12W for an additional 96 weeks. |
| Name | Type | Description |
|---|---|---|
| Diranersen | DRUG | Administered as specified in the treatment arm. |
| Diranersen-matching placebo | DRUG | Administered as specified in the treatment arm. |
Key Inclusion Criteria for Placebo-controlled Period: * Must meet all the clinical staging criteria for MCI due to AD (Stage 3) or mild AD dementia (Stage 4) according to the National Institute on Aging at National Institutes of Health and the Alzheimer's Association (NIA-AA) and must have the foll...
Diranersen is an investigational RNA therapy being developed for mild cognitive impairment due to Alzheimer's disease, as well as mild Alzheimer's disease dementia. It is not yet approved for any indication and remains in clinical development. The Phase 2 study enrolls participants between 50 and 80 years of age with these conditions.
Diranersen is an antisense oligonucleotide, an RNA therapy that targets RNA to reduce production of a specific protein. It belongs to the -sen antisense oligonucleotide class. By targeting RNA rather than a protein directly, it aims to lower levels of the disease-related protein before it accumulates. The specific protein target is not disclosed in the available program information.
Diranersen is being developed by Biogen Inc., which trades on the Nasdaq under the ticker BIIB. Biogen is the sponsor of the ongoing Phase 2 clinical trial evaluating the drug in Alzheimer's disease. The company holds the development program for this investigational RNA therapy.
Diranersen is in Phase 2 clinical development. It has not been approved by the FDA for any indication. The FDA has granted Diranersen Fast Track designation, which is intended to expedite the review of drugs that treat serious conditions and fill an unmet medical need. One Phase 2 trial is currently active.
Diranersen is being studied in one active Phase 2 trial, NCT05399888, titled a study to learn about the safety of Diranersen (BIIB080) injections and whether they can improve symptoms of participants with mild cognitive impairment due to Alzheimer's disease or mild Alzheimer's disease dementia. The trial is randomized, double-blind, placebo-controlled, and has an enrollment of 416 participants across multiple countries.
Yes, Diranersen is also known as BIIB080. The two names refer to the same investigational antisense oligonucleotide developed by Biogen. The Phase 2 trial NCT05399888 uses the BIIB080 designation in its title, while the drug is also referred to as Diranersen.