Drugs / Mivelsiran
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05231785 | Feb 2022 → Apr 2029 expected | Alzheimer disease | Alnylam Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT07636811 | Jul 2026 → May 2030 expected | Alzheimer disease | Alnylam Pharmaceuticals | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT06393712 | May 2024 → Aug 2027 expected | cerebral amyloid angiopathy | Alnylam Pharmaceuticals | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 12
| Date | Issuer | Release |
|---|---|---|
| 2026-07-15 | Alnylam Pharmaceuticals | Results Alnylam Highlights Progress with Neuroscience Programs at AAIC 2026, Showcasing Potential of RNAi in Neurological Disease alnylam.com ↗
Presents Updated Phase 1 Data on Mivelsiran in Early Onset Alzheimer’s Disease (EOAD) Showing Low Incidence of Amyloid-Related Imaging Abnormalities (ARIA) |
| 2025-02-25 | Alnylam Pharmaceuticals | Results Alnylam Highlights Significant Pipeline Progress and Platform Innovation at R&D Day alnylam.com ↗
Alnylam scientists will share additional data from both the single-ascending dose and multiple dose parts of its Phase 1 study of mivelsiran in early-onset Alzheimer’s disease, which further demonstrate potent and durable reduction of soluble APPβ – a key marker of target engagement – in the cerebrospinal fluid as well as an encouraging safety profile. |
| 2024-10-31 | Alnylam Pharmaceuticals | Results Alnylam Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights Recent Period Activity alnylam.com ↗
We also made great strides with our pipeline, sharing new results today from the Phase 1 study of mivelsiran in patients with Alzheimer’s disease and initiating a Phase 1 study for ALN-HTT02 in adult patients with Huntington’s disease earlier in the third quarter. |
| 2024-02-15 | Alnylam Pharmaceuticals | Regulatory Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Period Activity alnylam.com ↗
The FDA has confirmed that multiple-dosing in the Phase 1 study may proceed at doses up to 180 mg given every six months, which covers all dose regimens planned to be explored in Part B. |
| 2023-10-25 | Alnylam Pharmaceuticals | Results Alnylam Reports Additional Positive Interim Phase 1 Results for ALN-APP, in Development for Alzheimer’s Disease and Cerebral Amyloid Angiopathy alnylam.com ↗ |
| 2023-08-03 | Alnylam Pharmaceuticals | Results Alnylam Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Presented Updated Positive Interim Results from Phase 1 Study of ALN-APP in Patients with Early-Onset Alzheimer’s Disease |
| 2023-07-17 | Alnylam Pharmaceuticals | Results Alnylam Reports Updated Positive Interim Phase 1 Results for ALN-APP, in Development for Alzheimer’s Disease and Cerebral Amyloid Angiopathy alnylam.com ↗
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced today updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). |
| 2023-05-04 | Alnylam Pharmaceuticals | Results Alnylam Pharmaceuticals Reports First Quarter 2023 Financial Results and Highlights Recent Period Activity alnylam.com ↗
Reported positive interim results from the ongoing single ascending dose (SAD) part of the Phase 1 study of ALN-APP in patients with early-onset Alzheimer’s disease (EOAD). |
| 2023-04-26 | Alnylam Pharmaceuticals | Results Alnylam and Regeneron Report Positive Interim Phase 1 Clinical Data on ALN-APP, an Investigational RNAi Therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy alnylam.com ↗
Patients treated with ALN-APP experienced dose-dependent, rapid and sustained reduction in cerebrospinal fluid of both soluble APPα (sAPPα) and APPβ (sAPPβ), biomarkers of target engagement, with maximum reduction of 84% and 90%, respectively. |
| 2022-06-02 | Alnylam Pharmaceuticals | Results Alnylam Announces Publication of Preclinical Results Based on Novel Conjugate Technology That Facilitates Delivery of Short Interfering RNA to the Central Nervous System and Other Extrahepatic Tissues alnylam.com ↗
In a Mouse Model of Alzheimer’s Disease, C16-siRNA Targeting the Amyloid Beta Precursor Protein Gene Led to Potent and Durable Knockdown in the CNS and Ameliorated Physiological and Behavioral Deficits |
| 2021-12-22 | Alnylam Pharmaceuticals | Regulatory Alnylam Submits CTA Application for ALN-APP, an Investigational RNAi Therapeutic for the Treatment of Alzheimer’s Disease and Cerebral Amyloid Angiopathy alnylam.com ↗ |
| 2021-09-29 | Alnylam Pharmaceuticals | Results Alnylam Continues Scientific Leadership in Advancement of RNAi Therapeutics at the 17th Annual Meeting of the Oligonucleotides Therapeutics Society alnylam.com ↗
An APP siRNA was shown in vitro to cause greater intracellular reductions in soluble APP beta (sAPPβ) fragments compared to β-site APP cleaving enzyme (BACE) inhibitors. |
All press releases naming this drug 12 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Mivelsiran | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07636811 ↗ |
| Known as | ALN-APP | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06393712 ↗ |
| Route | Intrathecal | “administered intrathecally (IT)” NCT05231785 ↗ |