Alnylam Pharmaceuticals

Open enrollment with an early-phase pipeline tilt

According to ClinicalTrials.gov-derived indexes, Alnylam Pharmaceuticals sits in the top decile of US sponsors by registered trial count (#380 of 15,742 sponsors by registered trial count).

49 total trials 14 currently recruiting 25 completed early-phase tilt top decile by trial count

Alnylam Pharmaceuticals: 49 sponsored US clinical trials, 14 recruiting.

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.

Top-decile sponsor footprint

Alnylam Pharmaceuticals ranks in the top decile of sponsors by registered trial count (#380 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.

Trial completion reliability score

F 48/100

25 of 30 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (83.3%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.

Open and recent trials

49 total, page 1 of 1

RECRUITING Phase 2

A Study to Evaluate ALN-AGT01 RVR in Adult Patients With Mild to Moderate Hypertension Pretreated With Zilebesiran

NCT07553442

RECRUITING Phase 3

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

NCT07223203

RECRUITING Phase 2

A Study to Evaluate ALN-4324 on Insulin Sensitivity in Adults With Type 2 Diabetes Mellitus

NCT07465224

RECRUITING

DemonsTTRate: A Global, Observational, Multicenter, Long-term Study of Patients With ATTR-CM in a Real-World Setting

NCT07358078

RECRUITING Phase 3

Zilebesiran in Patients With Hypertension Not Adequately Controlled and With Either Established Cardiovascular Disease or High Risk for Cardiovascular Disease

NCT07181109

RECRUITING Phase 3

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT07052903

RECRUITING Phase 1

A Study to Evaluate ALN-4324 in Overweight to Obese Healthy Volunteers and in Overweight to Obese Patients With T2DM

NCT06845202

RECRUITING Phase 1

A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma

NCT06600321

RECRUITING Phase 1

A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)

NCT06659640

RECRUITING Phase 2

A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

NCT06393712

RECRUITING Phase 1

A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

NCT05231785

RECRUITING

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

NCT04883905

RECRUITING

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

NCT04561518

RECRUITING

Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program

NCT05040373

ACTIVE NOT RECRUITING

BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)

NCT04982393

ACTIVE NOT RECRUITING Phase 3

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

NCT04153149

ACTIVE NOT RECRUITING Phase 3

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

NCT03997383

NOT YET RECRUITING Phase 2

A Study to Evaluate Mivelsiran in Adult Participants With Early-Stage Down Syndrome-Associated Alzheimer's Disease (DS-AD)

NCT07636811

NOT YET RECRUITING Phase 2

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

NCT07575308

COMPLETED Phase 2

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

TERMINATED Phase 1

A Phase 1/2 Study to Evaluate ALN-KHK in Overweight to Obese Healthy Volunteers and Obese Patients With T2DM

NCT05761301

TERMINATED Phase 2

A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels

NCT05161936

COMPLETED Phase 2

Zilebesiran as Add-on Therapy in Patients With Hypertension Not Adequately Controlled by a Standard of Care Antihypertensive Medication (KARDIA-2)

NCT05103332

COMPLETED Phase 2

A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate Hypertension

NCT04936035

TERMINATED Phase 1

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)

NCT04565717

COMPLETED Phase 3

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

NCT04152200

COMPLETED

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418

COMPLETED Phase 3

A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

NCT03905694

COMPLETED Phase 3

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

NCT03759379

TERMINATED

A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease

NCT03774784

COMPLETED Phase 3

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

NCT03681184

TERMINATED

INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP

NCT03547297

COMPLETED Phase 3

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

NCT03338816

COMPLETED Phase 1

A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

NCT02949830

COMPLETED Phase 3

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

COMPLETED Phase 1

A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

NCT02452372

COMPLETED Phase 3

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

NCT02319005

COMPLETED Phase 2

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

NCT02292186

COMPLETED

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

NCT02240784

COMPLETED

DISCOVERY: A Study Examining the Prevalence of TTR Mutations in Subjects Suspected of Having Cardiac Amyloidosis

NCT02252653

COMPLETED Phase 2

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

NCT01981837

COMPLETED Phase 3

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

NCT01960348

COMPLETED Phase 2

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

COMPLETED Phase 2

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

NCT01617967

COMPLETED Phase 1

Multi-center, Open Label, Extension Study of ALN-VSP02 in Cancer Patients Who Have Responded to ALN-VSP02 Treatment

NCT01158079

COMPLETED Phase 2

Phase 2b Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)

NCT01065935

COMPLETED Phase 1

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

COMPLETED Phase 2

Phase 2 Study of ALN-RSV01 in Lung Transplant Patients Infected With Respiratory Syncytial Virus (RSV)

NCT00658086

COMPLETED Phase 2

Intranasal ALN-RSV01 Administered to Adult Volunteers Experimentally Inoculated With Respiratory Syncytial Virus

NCT00496821

What is open in the Alnylam Pharmaceuticals pipeline

14 recruiting of 49 indexed (29% open); 25 completed (51%).

Phase mix: 13 late (III/IV) vs 26 early (I/II) (early-phase heavy).

Top condition: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy (2 trials), 9 others listed.

How open-pipeline share is computed

Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.

Alnylam Pharmaceuticals: open-pipeline peers nationwide

Peers matched on 49 registered trials and 29% open enrollment, not the therapeutic-area sidebar.

Alnylam Pharmaceuticals sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot). 2×2 strategic matrix plotting 9 entities by Total trials (X) and Recruiting share (Y), with a crosshair dividing the plot into four quadrants. Higher volume & more openFocused & more openHigher volume, mostly closedSmaller & mostly closed 0102030405060 0%10%20%30%40%50% Total trials Recruiting share Alnylam PharmaceuticalsargenxBraintree LaboratoriesCynosureMirati Therapeutics89bioAlabama Physical Therapy & AcupunctureAlloSourceAnnovis Bio
Alnylam Pharmaceuticals sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together. Source: ClinicalTrials.gov (2026-08-08 snapshot).

Alnylam Pharmaceuticals sits at or above the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.

Sponsors near 49 trials (± recruiting share)

Sponsors near 29% open enrollment (± trial volume)

What to do with this Alnylam Pharmaceuticals page

49 registered trials is a research-attention snapshot, not an endorsement of Alnylam Pharmaceuticals or any single study.

Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.

Alnylam Pharmaceuticals pipeline card: 49 registered trials from official public datasets.Report a data error.