A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the Body in Healthy Participants

NCT07075640 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: AG236-C-001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The primary purpose of this study is to assess the safety and tolerability of a single dose of AG-236 administered subcutaneously in healthy participants.

Conditions

Healthy Participants

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to Study Drug

  Up to Day 57

Secondary Outcomes (15)

• Area Under the Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of AG-236

  Predose and multiple time points postdose from Day 1 to Day 57

• Area Under the Concentration-time Curve From Time Zero to Last Quantifiable Concentration (AUC0-t) of AG-236

  Predose and multiple time points postdose from Day 1 to Day 57

• Maximum Observed Plasma Concentration (Cmax) of AG-236

  Predose and multiple time points postdose from Day 1 to Day 57

• Percentage of Area Under the Concentration-Time Curve due to Extrapolation From the Last Quantifiable Concentration to Infinity (AUC%extrap) of AG-236

  Predose and multiple time points postdose from Day 1 to Day 57

• Time to Last Measurable Concentration (tlast) of AG-236

  Predose and multiple time points postdose from Day 1 to Day 57

Study Arms (3)

Treatment Group A

EXPERIMENTAL

Participants will receive a single dose of AG-236 at dose level 1 or placebo on Day 1 under fasted conditions.

Drug: AG-236; Drug: Placebo

Treatment Group B

EXPERIMENTAL

Participants will receive a single dose of AG-236 at dose level 2 or placebo on Day 1 under fasted conditions.

Drug: AG-236; Drug: Placebo

Treatment Group C

EXPERIMENTAL

Participants will receive a single dose of AG-236 at dose level 3 or placebo on Day 1 under fasted conditions.

Drug: AG-236; Drug: Placebo

Interventions

AG-236 DRUG

Subcutaneous (SC) Injection

Treatment Group A; Treatment Group B; Treatment Group C

Placebo DRUG

SC Injection

Treatment Group A; Treatment Group B; Treatment Group C

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Male or female, of any race, between 18 and 55 years of age, inclusive.
• Females must be of nonchildbearing potential.
• Males must agree to use contraception.
• Males must agree not to donate sperm during the study and for 90 days or 5-half-lives of AG-236 in plasma, whichever is longer, after dose administration.
• Body mass index between 18.0 and 32.0 kilograms per square meter (kg/m2), inclusive.
• Body weight between 50 and 100 kg, inclusive.
• In good health, as determined by no clinically significant findings from medical history, 12-lead electrocardiogram (ECG) and vital sign measurements, and clinical laboratory evaluations (congenital nonhemolytic hyperbilirubinemia \[eg, suspicion of Gilbert's syndrome based on total and direct bilirubin\] is not acceptable) at screening and check-in, and from the physical examination at screening, as assessed by the investigator or designee.
• Able to comprehend and willing to sign the informed consent form (ICF) and abide by the study restrictions.

You may not qualify if:

• Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, neurological, respiratory, endocrine, or psychiatric disorder, as determined by the investigator or designee.
• History of malignancy, with the exception of adequately treated non-melanomatous skin carcinoma.
• History of significant hypersensitivity, intolerance, or allergy to any drug compound, food, or other substance, as determined by the investigator or designee.
• History or presence of iron deficiency or iron deficiency anemia and/or currently receiving oral or parenteral iron supplementation as treatment for those conditions or deemed high risk of iron deficiency as determined by the investigator.
• Males: ferritin \<30 nanogram per millilitres (ng/mL) and/or transferrin saturation (TSAT): ≤25%.
• Females: ferritin \<30 ng/mL and/or TSAT: ≤20%.
• Fever (oral body temperature \>38°C) or symptomatic viral or bacterial infection within 2 weeks prior to screening; evidence of intestinal infection (such as a participant-reported history of gastrointestinal symptoms \[eg, nausea, vomiting, diarrhea\] that are consistent, in the opinion of the investigator, with an acute viral or bacterial process) within 30 days prior to screening.
• Confirmed systolic blood pressure \>140 or \<90 millilitres of mercury (mmHg), diastolic blood pressure \>90 or \<50 mmHg, or pulse rate \>100 or \<40 beats per minute. If any parameter is out of range, measurements should be repeated twice. Participants will be excluded if the average of the 3 measurements are outside of the corresponding reference range.
• Clinically significant abnormality, as determined by the investigator, on ECG performed at screening or check-in or any of the following:
• QT interval corrected for heart rate using Fridericia's formula (QTcF) \>450 milliseconds (ms) in males or \>470 ms in females, confirmed by calculating the mean of the triplicate values obtained
• history of additional risk factors for torsades de pointes (eg, heart failure, hypokalemia, or family history of long QT syndrome).
• Clinical laboratory values that are outside the normal reference range and are considered clinically significant, as determined by the investigator.
• Clinical laboratory values for hemoglobin (Hb) for males \<13.8 grams per decilitres g/dL and females \<12.1 g/dL.
• Clinical laboratory values for aspartate aminotransferase (AST), alanine aminotransferase (ALT) and/or total bilirubin \>1 × upper limit of normal (ULN).
• Participants will be excluded if a single repeat confirms the result.

Sponsors & Collaborators

• Agios Pharmaceuticals, Inc.: lead

Study Sites (1)

Fortrea Clinical Research Unit Inc.

Madison, Wisconsin, 53704, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 11, 2025
• First Posted: July 20, 2025
• Study Start: July 7, 2025
• Primary Completion: February 11, 2026
• Study Completion (Estimated): August 31, 2026
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)