Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ALN-APP · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| ALN-APP | EXPERIMENTAL | Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period. |
| Placebo/ALN-APP | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo during the double-blind treatment period. Participants who continue into the optional open-label extension period will be administered multiple doses of ALN-APP. |
| Part A: ALN-APP | EXPERIMENTAL | Participants will be administered a single dose of ALN-APP. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be administered a single dose of placebo. |
| Part B: | EXPERIMENTAL | Participants will be administered multiple doses of ALN-APP. |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Placebo will be administered intrathecally |
| ALN-APP | DRUG | ALN-APP will be administered intrathecally |
Inclusion Criteria (sporadic CAA patients): * Is 50 years or older * Has probable CAA per the Boston Criteria Version 2.0 Inclusion Criteria (Dutch-type CAA patients): * Is 30 years or older * Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA Exclusion Criteria: *...
ALN-APP is an investigational small molecule being developed by Alnylam Pharmaceuticals for early-onset Alzheimer disease and cerebral amyloid angiopathy. It is currently in Phase 2 clinical development for cerebral amyloid angiopathy and has been studied in Phase 1 for early-onset Alzheimer disease.
ALN-APP is an investigational small molecule designed to treat neurological conditions. While its specific molecular target is not publicly detailed, it is being studied in early-onset Alzheimer disease and cerebral amyloid angiopathy, both of which involve amyloid protein accumulation in the brain.
ALN-APP is developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ALNY. The company is conducting clinical trials of ALN-APP in multiple countries including the United States, Canada, Netherlands, and United Kingdom.
ALN-APP is in Phase 2 clinical development for cerebral amyloid angiopathy, with an active Phase 2 trial that is not yet recruiting participants. It has also completed Phase 1 development for early-onset Alzheimer disease, and is not yet approved by regulatory authorities.
ALN-APP has two clinical trials. NCT05231785 is a Phase 1 study in early-onset Alzheimer disease, currently recruiting 60 participants in the US, Canada, Netherlands, and UK. NCT06393712 is a Phase 2 trial in cerebral amyloid angiopathy, enrolling 210 participants across the US, Australia, Canada, Netherlands, Switzerland, and UK.
ALN-APP is the investigational name used by Alnylam Pharmaceuticals for this drug candidate. No alternative names have been reported in clinical trial registries, and it is distinct from other amyloid-targeting therapies in development.