Testing AUTX-703 for advanced blood cancers

A Phase 1 Study of AUTX-703 in Participants With Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

This study is testing a new drug called AUTX-703 to see if it can safely help people with advanced blood cancers like acute myeloid leukemia and myelodysplastic syndromes.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 69 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Auron Therapeutics, Inc. (Industry-sponsored)
• Drugs / interventions: chimeric antigen receptor
• Locations: 9 sites (Duarte, California and 8 other locations)
• Trial ID: NCT06846606 on ClinicalTrials.gov

What this trial studies

This Phase 1, multicenter, open-label study evaluates the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of AUTX-703 in patients with relapsed or refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The study is divided into two parts: the first part focuses on dose escalation to determine the maximum tolerated dose, while the second part optimizes the dose to further assess safety and efficacy. Participants will receive AUTX-703 orally as part of the treatment regimen.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with relapsed or refractory AML or MDS who have not responded to or cannot tolerate existing therapies.

Not a fit: Patients who have not been diagnosed with relapsed or refractory AML or MDS, or those who have not exhausted other treatment options, may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced blood cancers who have limited treatment alternatives.

How similar studies have performed: While this approach is novel, similar studies targeting hematologic malignancies have shown promise in the past.

Eligibility criteria

Key Inclusion Criteria:

1. Participant must be ≥18 years of age
2. Participant must have confirmed diagnosis as follows:

   R/R AML and has not achieved adequate response to, cannot tolerate, or refused all approved therapies known to be active for treatment of their disease OR R/R MDS with over 10% blasts in the bone marrow and has not achieved an adequate response to at least 4 cycles of a hypomethylating agent (HMA)- containing regimen or other treatment known to be active for their disease OR R/R AML or R/R MDS that has relapsed after a hematopoietic stem cell transplant (HSCT)
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.

Key Exclusion Criteria:

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

Where this trial is running

Duarte, California and 8 other locations

• City of Hope National Medical Center — Duarte, California, United States (Not_yet_recruiting)
• H Lee Moffitt Cancer Center and Research Institute — Tampa, Florida, United States (Not_yet_recruiting)
• Roswell Park Comprehensive Cancer Center — Buffalo, New York, United States (Recruiting)
• Memorial Sloan Kettering Cancer Center — New York, New York, United States (Not_yet_recruiting)
• University of North Carolina at Chapel Hill — Chapel Hill, North Carolina, United States (Not_yet_recruiting)
• Ohio State University, The James Comprehensive Cancer — Columbus, Ohio, United States (Not_yet_recruiting)
• UPENN Perelman Center for Advanced Medicine — Philadelphia, Pennsylvania, United States (Not_yet_recruiting)
• Sarah Cannon Center for Blood Cancer at TriStar Centennia — Nashville, Tennessee, United States (Recruiting)
• University of Texas MD Anderson Cancer Center — Houston, Texas, United States (Not_yet_recruiting)

Study contacts

• Study coordinator: Auron Clinical Trials

Conditions Relapsed Acute Myeloid Leukemia; Refractory Acute Myeloid Leukemia; Relapsed/Refractory AML; Relapsed Myelodysplastic Syndromes; Refractory Myelodysplastic Syndromes; Relapsed/Refractory Acute Myeloid Leukemia; AUTX-703; Acute Myeloid Leukemia