Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS

Eligibility Criteria

• 1. Participant must be ≥18 years of age
• 2. Participant must have confirmed diagnosis as follows:
• 3. Participant must be willing and able to comply with scheduled study visits and treatment plans.
• 4. Participant must be willing to undergo all study procedures unless contraindicated due to medical risk.
• 5. Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of ≤2
• 6. Participant must have adequate hepatic function
• 7. Participant must have adequate renal function
• 8. Participant must have adequate cardiovascular function
• 9. Participant must have a white blood cell (WBC) count ≤20 × 10⁹/L (with stable hydroxyurea use allowed)
• 10. Participant must meet timing requirements with respect to prior therapy and surgery
• 11. Participant must agree to use effective contraception during the study and for the required post-treatment period: Males: Use condoms (even if vasectomized) during the study and for 90 days post-treatment. Females of childbearing potential: Use a combination of 1 highly effective and 1 effective method of contraception during the study and for 180 days post-treatment.
• 1. Participant is unable to provide informed consent and/or to follow protocol requirements.
• 2. Participant has undergone chimeric antigen receptor T cell therapy or HSCT within 60 days of the first dose of study treatment or has active clinically significant graft-versus-host disease (GVHD)
• 3. Participant has another malignancy that may interfere with diagnosis and treatment of R/R AML or R/R MDS.
• 4. Participant has an active severe infection that requires anti-infective therapy or has an unexplained temperature of \>38.5°C during screening visits or on their first day of study treatment.
• 5. Participant has a known sensitivity to AUTX-703 or any of its components.
• 6. Participant is taking systemic strong CYP3A4 inhibitors or inducers within 14 days of the first dose of study treatment.
• 7. Participant who are taking proton pump inhibitors should be switched to another acid-reducing agent such as an antacid or H2 blocker
• 8. Participant is taking P-gp and breast cancer resistance protein (BCRP) inhibitors or inducers within 14 days of first dose of study treatment.
• 9. Participant has active hepatitis B virus (HBV) or hepatitis C virus (HCV) infections with detectable viral load
• 10. Participant has experienced AIDS related illness within the past 6 months or have detectable HIV viral load.
• 11. Participant has an uncontrolled intercurrent illness
• 12. Participant has active Class III or IV cardiovascular disease within 6 months prior to the start of study treatment
• 13. Participant is unable to tolerate the administration of oral medication or has GI dysfunction that would preclude adequate absorption, distribution, metabolism, or excretion of an oral medication
• 14. Participant is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment