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ALN-AGT01

Phase 2

Hypertension | Small molecule | Cardiovascular |Alnylam Pharmaceuticals, Inc.|Last Updated: Dec 23, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment518

FDA Designations

No designations recorded

Clinical trial landscape

ALN-AGT01 · 2 trials · 1 indication

Phase 2 1Phase 1 1
NCT04936035A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate HypertensionHypertension
COMPLETED394 Analytics
PHASE2COMPLETED
A Study to Evaluate Efficacy and Safety of ALN-AGT01 in Patients With Mild To-Moderate Hypertension
HypertensionUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline at Month 3 in 24-hour Mean SBP Assessed by ABPM
Baseline and Month 3

24-hour ABPM was programmed to take readings every 20 minutes during the day (6 am to 9:59 pm) and every 30 minutes during the night (10 pm to 5:59 am). An ABPM was considered adequate if: 1. the number of successful daytime readings were ≥33, 2. the number of successful nighttime readings were ≥11, 3. no more than 3 hours are not represented (i.e., 3 sections of 60 minutes where 0 valid readings were obtained). To summarize the 24-hour ABPM, the hourly adjusted mean was calculated. Hourly mean was the average of BP by each hour of the day. The 24-hour mean was the average of the hourly means. Least squares (LS) mean and standard error (SE) were calculated using a mixed model repeated measures (MMRM) approach.

Number of Participants with Adverse Events (AEs)
Parts A, B and E: each up to approximately 12 months; Part D: up to approximately 18 months

Secondary Endpoints

Change From Baseline at Month 3 in Mean Sitting Office SBP
Baseline and Month 3
Change From Baseline at Month 6 in 24-hour Mean SBP Assessed by ABPM
Baseline and Month 6
Change From Baseline at Month 6 in Mean Sitting Office SBP
Baseline and Month 6
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PlaceboPLACEBO_COMPARATORParticipants received zilebesiran matching placebo, subcutaneous (SC) injection, once every 3 months (Q3M), with re-randomization at Month 6 to 1 of the initial 4 zilebesiran regimens. Participants will continue their respective zilebesiran regimen up to Month 12 in the DB period and up to 24 additional months in the DB Extension period. Upon implementation of Amendment 6, the DB Extension period was closed.
Zilebesiran 150 Milligrams (mg) Once Every 6 Months (Q6M)EXPERIMENTALParticipants receive zilebesiran, 150 mg, SC injection, Q6M, during the 12-month DB period. Participants will continue receiving the same zilebesiran regimen for up to 24 additional months in the DB Extension period. Participants in this arm will receive a placebo during those dosing visits when they do not receive zilebesiran to maintain the blind. Upon implementation of Amendment 6, the DB Extension period was closed.
Zilebesiran 300 mg Q6MEXPERIMENTALParticipants receive zilebesiran, 300 mg, SC injection, Q6M, during the 12-month DB period. Participants will continue receiving the same zilebesiran regimen for up to 24 additional months in the DB Extension period. Participants in this arm will receive a placebo during those dosing visits when they do not receive zilebesiran to maintain the blind. Upon implementation of Amendment 6, the DB Extension period was closed.
Zilebesiran 300 mg Q3MEXPERIMENTALParticipants receive zilebesiran, 300 mg, SC injection, Q3M, during the 12-month DB period. Participants continue receiving the same zilebesiran regimen for up to 24 additional months in the DB Extension period. Upon implementation of Amendment 6, the DB Extension period was closed.
Zilebesiran 600 mg Q6MEXPERIMENTALParticipants receive zilebesiran, 600 mg, SC injection, Q6M, during the 12-month DB period. Participants will continue receiving the same zilebesiran regimen for up to 24 additional months in the DB Extension period. Participants in this arm will receive a placebo during those dosing visits when they do not receive zilebesiran to maintain the blind. Upon implementation of Amendment 6, the DB Extension period was closed.
Part A: SAD: ALN-AGT01EXPERIMENTALParticipants will be administered a single dose of ALN-AGT01.
Part A: SAD: ALN-AGT01-Matching PlaceboPLACEBO_COMPARATORParticipants will be administered a single dose of ALN-AGT01-matching placebo.
Part B: SD: ALN-AGT01EXPERIMENTALParticipants with controlled salt intake will be administered a single dose of ALN-AGT01.
Part B: SD: ALN-AGT01-Matching PlaceboPLACEBO_COMPARATORParticipants with controlled salt intake will be administered a single dose of ALN-AGT01-matching placebo.
Part D: MD: ALN-AGT01 + Irbesartan-Matching PlaceboEXPERIMENTALParticipants, who are obese, will be administered multiple doses of ALN-AGT01 and irbesartan-matching placebo.
Part D: MD: ALN-AGT01-Matching Placebo + IrbesartanACTIVE_COMPARATORParticipants, who are obese, will be administered multiple doses of ALN-AGT01-matching placebo and irbesartan.
Part E: Open Label: ALN-AGT01 + IrbesartanEXPERIMENTALParticipants will be administered a single dose of ALN-AGT01 and multiple doses of irbesartan.

Interventions

NameTypeDescription
PlaceboDRUGPlacebo administered by SC injection
ALN-AGT01DRUGALN-AGT01 administered by SC injection
ALN-AGT01-Matching PlaceboDRUGNormal saline (0.9% NaCl) matching volume of ALN-AGT01 doses will be administered SC.
IrbesartanDRUGIrbesartan will be administered orally.
Irbesartan-Matching PlaceboDRUGIrbesartan-matching placebo will be administered orally.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites75

Inclusion Criteria: * Daytime mean SBP ≥135 mmHg and ≤160 mmHg by ABPM, without antihypertensive medication Exclusion Criteria: * Secondary hypertension, orthostatic hypotension * Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>2× upper limit of normal (ULN) * Elevated potass...

Countries:United StatesCanadaPuerto RicoUkraineUnited Kingdom
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Frequently asked questions about ALN-AGT01

What is ALN-AGT01 used for?

ALN-AGT01 is an investigational drug being developed for the treatment of hypertension, a cardiovascular condition characterized by elevated blood pressure. It is currently in clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with hypertension, including those with mild to moderate forms of the condition.

What does ALN-AGT01 target?

ALN-AGT01 is a small molecule being developed by Alnylam Pharmaceuticals. While the specific molecular target of ALN-AGT01 has not been disclosed, it is being investigated for its potential to lower blood pressure in patients with hypertension. The drug's mechanism of action is not publicly detailed, but it is being evaluated in clinical trials for its efficacy and safety.

Who makes ALN-AGT01?

ALN-AGT01 is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol ALNY. The company is conducting clinical trials to evaluate the drug's safety and efficacy in treating hypertension. Alnylam is known for its focus on RNA interference therapeutics, though ALN-AGT01 is classified as a small molecule.

What phase is ALN-AGT01 in?

ALN-AGT01 is currently in Phase 2 clinical development. It has completed two clinical trials, including a Phase 1 study and a Phase 2 study. The drug is investigational and has not received FDA approval. It is being studied for the treatment of hypertension, with the Phase 2 trial evaluating its efficacy and safety in patients with mild to moderate hypertension.

What clinical trials is ALN-AGT01 in?

ALN-AGT01 has been studied in two completed clinical trials. The first, NCT03934307, was a Phase 1 study in patients with hypertension conducted in the United Kingdom with 124 participants. The second, NCT04936035, was a Phase 2 study in patients with mild to moderate hypertension conducted in the United States, Canada, Puerto Rico, Ukraine, and the United Kingdom, enrolling 394 participants.

Is ALN-AGT01 the same as any other drug?

ALN-AGT01 is the primary name for this investigational drug, and no alternative names have been disclosed. It is being developed by Alnylam Pharmaceuticals under this identifier. Patients and healthcare providers should refer to the drug by its official name, ALN-AGT01, when discussing clinical trials or potential treatment options.