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Also known as mivelsiran
ALN-APP · 3 trials · 3 indications
| Arm | Type | Description |
|---|---|---|
| Mivelsiran (Treatment Group A) | EXPERIMENTAL | Participants will be administered multiple low doses of mivelsiran. |
| Mivelsiran (Treatment Group B) | EXPERIMENTAL | Participants will be administered multiple high doses of mivelsiran. |
| Placebo (Treatment Group C) | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo. |
| 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 |
|---|---|---|
| Mivelsiran | DRUG | Mivelsiran will be administered intrathecally |
| Placebo | DRUG | Placebo will be administered intrathecally |
| ALN-APP | DRUG | ALN-APP will be administered intrathecally |
Inclusion Criteria: * Clinical diagnosis of Down-Syndrome (DS) associated with trisomy 21 * Positive amyloid PET scan * Cognitively stable in the opinion of the investigator * Seizures must be well controlled with no occurrence of seizures in the 6 months prior to screening Exclusion Criteria: * ...
ALN-APP is an investigational RNA therapy being developed for cerebral amyloid angiopathy and early-onset Alzheimer disease. It is not approved for any indication and remains in clinical development. Alnylam Pharmaceuticals is evaluating it in a Phase 2 trial in cerebral amyloid angiopathy and a Phase 1 study in early-onset Alzheimer disease.
ALN-APP is an RNA therapy designed to target amyloid precursor protein, or APP, production. By reducing APP synthesis, the therapy aims to lower amyloid beta accumulation associated with cerebral amyloid angiopathy and early-onset Alzheimer disease. It is administered as an investigational treatment and has not been approved by any regulatory agency.
ALN-APP is being developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. The company is running clinical trials of ALN-APP in cerebral amyloid angiopathy and early-onset Alzheimer disease. Alnylam is a biopharmaceutical company focused on RNA interference therapeutics.
ALN-APP is in Phase 2 clinical development. The Phase 2 trial is evaluating ALN-APP in patients with cerebral amyloid angiopathy. A separate Phase 1 study is evaluating the therapy in patients with early-onset Alzheimer disease. ALN-APP is investigational and has not received FDA approval.
ALN-APP is being studied in two clinical trials. NCT06393712 is a Phase 2 trial in patients with cerebral amyloid angiopathy, with 210 participants across the United States, Australia, Canada, Netherlands, Switzerland, and the United Kingdom. NCT05231785 is a Phase 1 study in early-onset Alzheimer disease, with 60 participants across the United States, Canada, Netherlands, and the United Kingdom.