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AG-946

Phase 1

Healthy Volunteers | Small molecule | Hematology |Agios Pharmaceuticals, Inc.|Last Updated: Apr 16, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment122

FDA Designations

No designations recorded

Clinical trial landscape

AG-946 · 1 trial · 2 indications

Phase 1 1
NCT04536792A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell DiseaseHealthy Volunteers
COMPLETED122 Analytics
PHASE1COMPLETED
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease
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Study Endpoints

Primary Endpoints

SAD and MAD: Number of Participants Who Experienced at Least One Adverse Event (AE), by Severity
SAD: Up to Day 13; MAD: Up to Day 49
SAD and MAD: Number of Participants Who Experienced at Least One AE Related to the Study Drug
SAD: Up to Day 13; MAD: Up to Day 49
SAD and MAD: Number of Participants Who Experienced at Least One Serious Adverse Event (SAE)
SAD: Up to Day 13; MAD: Up to Day 49
SAD and MAD: Number of Participants With Clinically Significant Laboratory Abnormalities, by Severity
SAD: Up to Day 13; MAD: Up to Day 49
SAD and MAD: Number of Participants With Clinically Significant Changes in Vital Signs and in Electrocardiogram (ECG) Parameters
SAD: Up to Day 13; MAD: Up to Day 49
SCD: Number of Participants Who Experienced at Least One AE, by Severity
Up to Day 56
SCD: Number of Participants Who Experienced at Least One AE Related to the Study Drug
Up to Day 56
SCD: Number of Participants Who Experienced at Least One SAE
Up to Day 56
SCD: Number of Participants With Clinically Significant Laboratory Abnormalities, by Severity
Up to Day 56
SCD: Number of Participants With Clinically Significant Changes in Vital Signs and in ECG Parameters
Up to Day 56

Secondary Endpoints

Maximum Observed Concentration (Cmax) of AG-946
SAD: Up to Day 8; MAD: Up to Day 49; SCD: Up to Day 56
Time to Reach Maximum Observed Concentration (Tmax) of AG-946
SAD: Up to Day 8; MAD: Up to Day 49; SCD: Up to Day 56
Area Under the Plasma Concentration-Time Curve from Time Zero to Last Quantifiable Concentration (AUC0-t), Time Curve from Time Zero to Infinity (AUC0-∞), and Time Curve from Time Zero to Time tau Over the Dosing Interval (AUC0-τ) for AG-946
SAD: Up to Day 8; MAD: Up to Day 49; SCD: Up to Day 56
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1: Single Ascending Dose (SAD) PhaseEXPERIMENTALParticipants will receive a range of doses of AG-946 or placebo, orally, once on Day 1. AG-946 will be given under fasted or fed conditions.
Part 2: Multiple Ascending Dose (MAD) PhaseEXPERIMENTALParticipants will receive a range of doses of AG-946 or placebo, orally, once daily (QD) for 14 days or using an alternative dosing regimen for up to 28 days under fasted conditions.
Part 3: Sickle Cell Disease (SCD) PhaseEXPERIMENTALParticipants will receive a range of selected ascending doses of AG-946, orally, QD or using an alternative dosing regimen for 28 days.

Interventions

NameTypeDescription
AG-946DRUGAG-946, oral encapsulated coated-granules or tablets.
AG-946 Matched PlaceboDRUGAG-946 matched-placebo, oral encapsulated sugar spheres or tablets.
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Eligibility Criteria

Age Range18 Years to 70 Years
SexALL
Healthy VolunteersYes
Study Sites10

Inclusion Criteria: SAD and MAD * Participant is a healthy male or a female of nonchildbearing potential aged 18 to 55 years inclusive at the time of consent; * Participant is able to understand and provide informed consent before the conduct of any protocol-specific screening procedures; * Partic...

Countries:United StatesDenmarkSpain
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Frequently asked questions about AG-946

What is AG-946 used for?

AG-946 is an investigational small molecule being studied in hematology. It is being evaluated in healthy volunteers and in participants with sickle cell disease and anemia. The drug is currently in Phase 1 clinical development and is not approved for any use.

Who makes AG-946?

AG-946 is being developed by Agios Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker AGIO. The company is conducting clinical research on this investigational small molecule in the field of hematology.

What phase is AG-946 in?

AG-946 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 1 study has been completed, and the drug remains under investigation for potential therapeutic use.

What clinical trials is AG-946 in?

AG-946 has one completed Phase 1 clinical trial, identified as NCT04536792. This study assessed the safety, tolerability, pharmacokinetics, and pharmacodynamics of AG-946 in healthy volunteers and in participants with sickle cell disease. The trial enrolled 122 participants across the United States, Denmark, and Spain.

Is AG-946 the same as any other drug?

AG-946 is the investigational name for this drug candidate. No alternative names have been reported for AG-946 in the available clinical trial information. It is a distinct small molecule being developed by Agios Pharmaceuticals.