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Diranersen · 2 trials · 3 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.
CNS biodistribution of \[89Zr\]Zr-DFO-Diranersen will be measured using the Positron Emission tomography (PET) imaging. The mean radioactive counts per voxel will be measured. These counts will be converted to the percentage of dose that is taken up by each ROI (% of injected tracer dose per gram of tissue) to quantify the biodistribution in measurable tissues and CSF spaces at relative distances from the lumbar injection using quantitative scaling algorithms.
| 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. |
| [89Zr]Zr-DFO-Diranersen+ Diranersen | EXPERIMENTAL | Participants will receive a single injection of a small dose of radiolabeled diranersen \[89Zr\]Zr-DFO-diranersen) along with a dose of diranersen together via an intrathecal (IT) injection. |
| Name | Type | Description |
|---|---|---|
| Diranersen | DRUG | Administered as specified in the treatment arm. |
| Diranersen-matching placebo | DRUG | Administered as specified in the treatment arm. |
| [89Zr]Zr-DFO-Diranersen | 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 antisense oligonucleotide being studied for mild cognitive impairment due to Alzheimer's disease and mild Alzheimer's disease dementia. It is also being evaluated in healthy volunteers for biodistribution and safety studies. The drug is in clinical development and has not been approved by the FDA.
Diranersen is an antisense oligonucleotide (ASO), a class of drugs that work by targeting messenger RNA to modulate protein production. Its specific molecular target has not been disclosed in available information. The drug is being investigated for its potential effects on Alzheimer's disease pathology.
Diranersen is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. Biogen is conducting clinical trials to evaluate the safety and efficacy of Diranersen for Alzheimer's disease and related conditions.
Diranersen is in Phase 1 clinical development. One Phase 1 study has been completed, and a Phase 2 study is active but not recruiting participants. The drug is investigational and has not received FDA approval for any indication.
Diranersen is being studied in two clinical trials. NCT05399888 is a Phase 2 study in patients with mild cognitive impairment due to Alzheimer's disease or mild Alzheimer's dementia, with 416 participants across multiple countries. NCT06454721 is a completed Phase 1 microdose study in healthy adults.
Yes, Diranersen is also known as BIIB080. Clinical trial records refer to the drug as Diranersen (BIIB080), confirming that these names identify the same investigational compound being developed by Biogen for Alzheimer's disease.