Alnylam Pharmaceuticals
Alnylam Pharmaceuticals sponsors 49 registered US clinical trials on ClinicalTrials.gov, 14 of them currently recruiting, and 25 completed. 13 of these trials are in Phase 3-4 (later-stage) and 26 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Transthyretin Amyloidosis (ATTR) With Cardiomyopathy, with 2 trials.
Trial Pipeline
49 total, page 1 of 1
A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran
NCT07553442
TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
NCT07223203
A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus
NCT07465224
DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting
NCT07358078
Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease
NCT07181109
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT07052903
A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM
NCT06845202
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
NCT06600321
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
NCT06659640
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
NCT06393712
A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD
NCT05231785
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
NCT04883905
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
NCT04561518
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
NCT05040373
BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)
NCT04982393
HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT04153149
APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
NCT03997383
A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)
NCT07636811
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
NCT07575308
Zilebesiran as Add-on Therapy in Patients With High Cardiovascular Risk and Hypertension Not Adequately Controlled by Standard of Care Antihypertensive Medications (KARDIA-3)
NCT06272487
A Phase 1/2 Study to Evaluate ALN-KHK in Overweight to Obese Healthy Volunteers and Obese Patients With T2DM
NCT05761301
A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
NCT05161936
Zilebesiran as Add-on Therapy in Patients With Hypertension Not Adequately Controlled by a Standard of Care Antihypertensive Medication (KARDIA-2)
NCT05103332
A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate Hypertension
NCT04936035
A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)
NCT04565717
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
NCT04152200
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
NCT04201418
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
NCT03905694
HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
NCT03759379
A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
NCT03774784
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
NCT03681184
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
NCT03547297
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
NCT03338816
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
NCT02949830
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
NCT02510261
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
NCT02452372
ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
NCT02319005
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
NCT02292186
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
NCT02240784
DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis
NCT02252653
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
NCT01981837
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
NCT01960348
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
NCT01961921
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
NCT01617967
Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment
NCT01158079
Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)
NCT01065935
Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous ALN-VSP02 In Patients With Advanced Solid Tumors With Liver Involvement
NCT00882180
Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)
NCT00658086
Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial Virus
NCT00496821
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 10 |
| Phase 2 | 16 |
| Phase 3 | 13 |
Therapeutic Areas
What the Pipeline for Alnylam Pharmaceuticals Shows
According to the ClinicalTrials.gov registry, Alnylam Pharmaceuticals is linked to 49 US clinical trials across every stage of research activity. Of those, 14 studies are currently recruiting, about 29% of the sponsor's indexed portfolio, and 25 are already marked complete, representing roughly 51% of the total.
The phase mix for Alnylam Pharmaceuticals reports 13 late-stage studies (Phase 3 and Phase 4 combined) and 26 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for Alnylam Pharmaceuticals is Transthyretin Amyloidosis (ATTR) With Cardiomyopathy with 2 linked trials, and 9 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.