Companies / Alnylam Pharmaceuticals
Alnylam Pharmaceuticals Pipeline
CompanyAlnylam Pharmaceuticals is an American biopharmaceutical company focused on discovering, developing, and commercializing RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
Continuously tracked·last revised
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Recruiting Phase 3
Recently started
| Trial | Phase | Status | Sponsor | Started |
|---|---|---|---|---|
| A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD) NCT07636811 | Phase 2 | Recruiting | Alnylam Pharmaceuticals | Started 2026-08 |
| HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB NCT07575308 | Phase 2 | Recruiting | Alnylam Pharmaceuticals | Started 2026-07 |
| A Study to Evaluate ALN-6222 in Participants With Obesity NCT07624071 | Phase 1 | Recruiting | Alnylam Pharmaceuticals | Started 2026-06 |
| A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran NCT07553442 | Phase 2 | Recruiting | Alnylam Pharmaceuticals | Started 2026-04 |
| A Study to Evaluate ALN-4915 in Adult Healthy Volunteers NCT07535606 | Phase 1 | Recruiting | Alnylam Pharmaceuticals | Started 2026-03 |
Results recently posted
Pipeline
Phase by status
| Phase | Recruiting | By invitation | Active | Completed | Terminated | Withdrawn | Approved for marketing | No longer available | Total |
|---|---|---|---|---|---|---|---|---|---|
| Phase 1 | 7 | — | — | 14 | 3 | — | — | — | 24 |
| Phase 1/2 | 1 | — | 2 | 4 | 3 | — | — | — | 10 |
| Phase 2 | 4 | — | 1 | 12 | 2 | 1 | — | — | 20 |
| Phase 3 | 3 | 1 | 1 | 10 | — | — | — | — | 15 |
| Unknown | 4 | — | 2 | 3 | 2 | — | 3 | 1 | 15 |
Trials started per year
Conditions studied, by phase
| Condition | Phase 1 | Phase 1/2 | Phase 2 | Phase 3 | Unknown | Total |
|---|---|---|---|---|---|---|
| Amyloidosis | — | — | — | 7 | 7 | 14 |
| Amyloidosis, Hereditary, Transthyretin-Related | 1 | — | — | 7 | 4 | 12 |
| Amyloidosis, Familial | 1 | — | — | 5 | 4 | 10 |
| Amyloid Neuropathies, Familial | 1 | — | 1 | 3 | 4 | 9 |
| Cardiomyopathies | — | — | 1 | 5 | 3 | 9 |
| Hypertension | 1 | 1 | 4 | 1 | — | 7 |
| Amyloid Neuropathies | 1 | — | — | 2 | 3 | 6 |
| Coproporphyria, Hereditary | 1 | — | — | 1 | 4 | 6 |
| Hyperoxaluria, Primary | — | 1 | 1 | 3 | 1 | 6 |
| Porphyria, Acute Intermittent | 2 | 1 | — | 1 | 2 | 6 |
Most active sponsors
| # | Sponsor | Trials | Phase 3 |
|---|---|---|---|
| 1 | Alnylam Pharmaceuticals | 84 | 15 |
Frequent collaborators
| # | Collaborator | Trials |
|---|---|---|
| 1 | Hoffmann-La Roche | 2 |
| 2 | Regeneron Pharmaceuticals | 1 |
| 3 | The Medicines Company | 1 |
Most studied interventions
| # | Intervention | Trials |
|---|---|---|
| 1 | patisiran | 12 |
| 2 | lumasiran | 7 |
| 3 | givosiran | 5 |
| 4 | revusiran | 5 |
| 5 | Saline Solution | 2 |
| 6 | ALN-PCS | 1 |
| 7 | Amlodipine | 1 |
| 8 | Indapamide | 1 |
| 9 | Irbesartan | 1 |
| 10 | olmesartan | 1 |
Terminations
Stated reasons
Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.
Recently stopped
| Trial | Status | Stated reason | Stopped |
|---|---|---|---|
| NCT05761301 Terminated | Terminated | Business reasons (not safety related) | 2025-04-03 |
| NCT04565717 Terminated | Terminated | Study ALN-HSD-001 was terminated due to business reasons and changes in the development strategy of the product. No safety issues were found. | 2023-12-21 |
| NCT03841448 Terminated | Terminated | Study was terminated early having identified clinically meaningful magnitude of proteinuria reduction due to cemdisiran (study goal). Participants completed DBT period\&were in OLE period. Sponsor had no concerns with safety\&integrity of participants. | 2023-06-27 |
| NCT05256810 Terminated | Terminated | The Sponsor elected not to continue with the study | 2023-01-25 |
| NCT05161936 Terminated | Terminated | Study terminated early due to low enrollment | 2022-11-01 |
| NCT03767829 Terminated | Terminated | The sponsor decided not to proceed with Part B for business reasons. | 2020-06-25 |
| NCT03774784 Terminated | Terminated | The study was terminated due to the impacts of COVID-19 on patient safety and enrollment. | 2020-05-18 |
| NCT03547297 Terminated | Terminated | Sponsor elected not to continue with study | 2019-01-15 |
| NCT03303313 Withdrawn | Withdrawn | Study terminated early due to lack of enrollment. | 2018-09-12 |
| NCT02503683 Terminated | Terminated | Observation of low incidence of asymptomatic, transiently elevated liver enzymes in a subset of study subjects | 2018-01-03 |
Evidence
- Results reported
- 55.8%24 of 43 completed
- Published
- 2.3%1 of 43 completed
- Primary endpoint met
- 92.3%12 of 13 analyses
- Median months to report
- 12
Documents disclosed
Trial profile
- Median enrollment
- 61
- Median duration
- 29.5months
- Countries
- 63
- Study sites
- 1317
Who is enrolled
Median enrollment by phase
- Phase 1
- 43
- Phase 1/2
- 51
- Phase 2
- 28
- Phase 3
- 206
- Unknown
- 207
Where trials run
Busiest cities
| # | City | Country | Trials |
|---|---|---|---|
| 1 | London | United Kingdom | 49 |
| 2 | Boston | United States | 30 |
| 3 | New York | United States | 27 |
| 4 | Barcelona | Spain | 20 |
| 5 | Rochester | United States | 20 |
| 6 | Chicago | United States | 19 |
| 7 | Houston | United States | 19 |
| 8 | Dallas | United States | 17 |
| 9 | Philadelphia | United States | 17 |
| 10 | Baltimore | United States | 16 |