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AG-946

Phase 1

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment122
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04536792A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell DiseasePHASE1 COMPLETED 122Jul 10, 2020Dec 19, 2023Apr 16, 202410 United States, Denmark +1
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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 — 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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