AUTX-703 Clinical Trial Begins, Auron Doses First Patient In Phase 1 Study For Advanced Hematologic Malignancies

Auron Therapeutics, a clinical-stage biotechnology company focused on targeting cell-state plasticity to improve outcomes in cancer and inflammatory diseases, has announced that the first patient has been dosed in a Phase 1 clinical trial of AUTX-703. This marks the beginning of the first-in-human study of investigational therapy. AUTX-703 is a novel, first-in-class, orally available KAT2A/B degrader that was discovered and developed using Auron’s proprietary AURIGIN™ Platform. 

Kate Yen, Chief Executive Officer of Auron Therapeutics, said in a statement,“The initiation of our first Phase 1 trial marks a significant milestone for Auron in our transition to a clinical-stage company. This achievement reflects the strength of our scientific platform and the dedication of our team in advancing a novel therapeutic approach targeting KAT2A/B – the epigenetic regulators that play a central role in driving cellular plasticity. By modulating this key mechanism, we aim to overcome one of the fundamental barriers to durable cancer treatment. We believe this strategy has the potential to deliver transformative benefits to patients while creating long-term value for our stakeholders.”‍

Dr. Stephen Strickland, Director of Leukemia Research for Sarah Cannon Research Institute (SCRI), mentioned, “We are excited to initiate this Phase 1 clinical trial of AUTX-703 in advanced hematologic malignancies. There remains a critical unmet need for novel targeted therapies in acute myeloid leukemia, particularly one with the potential to benefit a broad patient population. With its differentiated mechanism of action and promising preclinical data, AUTX-703 represents a compelling opportunity to advance treatment options in AML, and we look forward to evaluating its potential in the clinic.”

In preclinical studies, AUTX-703 demonstrated a dose-dependent survival benefit in a primary model of acute myeloid leukemia (AML), indicating its potential effectiveness in treating hematologic malignancies. The ongoing clinical trial is a Phase 1, open-label, dose-escalation and dose-optimization study. It is designed to evaluate the safety, tolerability, pharmacokinetics (how the drug moves through the body), pharmacodynamics (how the drug affects the body), and preliminary signs of clinical activity of AUTX-703. The trial is enrolling patients with relapsed or refractory AML or myelodysplastic syndromes (MDS), both of which are difficult-to-treat forms of blood cancer.