Auron Announces First Patient Dosed in Phase 1 Clinical Trial of AUTX-703 in Advanced Hematologic Malignancies

Novel approach targeting KAT2A/B with preclinical validation across multiple cancer models and tumor types

/EIN News/ -- NEWTON, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Auron Therapeutics, a clinical-stage biotechnology company targeting cell-state plasticity to improve patient outcomes in oncology and inflammatory disease, today announced that the first patient has been dosed with AUTX-703 in a first-in-human, Phase 1 clinical trial for patients with advanced hematologic malignancies (NCT06846606).

AUTX-703 is a novel, first-in-class, orally available KAT2A/B degrader discovered and developed by Auron using its AURIGIN™ Platform. The Company has previously presented preclinical data demonstrating a dose dependent survival advantage with AUTX-703 in a primary acute myeloid leukemia (AML) model.

“The initiation of our first Phase 1 trial marks a significant milestone for Auron in our transition to a clinical-stage company,” said Kate Yen, Chief Executive Officer of Auron Therapeutics. “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.”

“We are excited to initiate this Phase 1 clinical trial of AUTX-703 in advanced hematologic malignancies,” said Dr. Stephen Strickland, Director of Leukemia Research for Sarah Cannon Research Institute (SCRI). “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.”

The trial is a Phase 1, open label, dose escalation and dose optimization study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 in patients with relapsed/refractory AML or myelodysplastic syndromes (MDS).

About AUTX-703

AUTX-703 is a first-in-class, oral KAT2A/B degrader, being developed for the treatment of multiple tumor types. Leveraging its AURIGIN™ platform, the Company identified KAT2A/B as a key driver of cell plasticity and disease. KAT2A/B are lysine acetyltransferases that epigenetically reprogram cells to a more proliferative and plastic cell state to drive disease. Auron has validated the target in several disease models including acute myeloid leukemia (AML), small cell lung cancer (SCLC) and neuroendocrine prostate cancer (NEPC). AUTX-703 has cleared two INDs and is in a Phase 1 study in relapsed / refractory (r/r) AML and myelodysplastic syndromes (MDS). In addition, the FDA granted Fast Track designation to AUTX-703 for the treatment of r/r AML.

About Auron Therapeutics

Auron Therapeutics is a platform-powered, product-driven company targeting cell-state plasticity to improve patient outcomes in oncology and inflammatory disease. Auron pioneered its AURIGIN platform, which uses AI and machine learning to compare cell states and identify novel drug targets, optimal development models, and biomarkers to facilitate proper patient selection, ultimately accelerating the development of effective and durable therapies. Using AURIGIN, the Company is building a pipeline of targeted therapies, led by a first-in-class, oral KAT2A/B degrader, AUTX-703, which is being developed for the treatment of both hematologic malignancies and solid tumors. For more information, please visit aurontx.com and follow us on LinkedIn.

Investor Contact:

Renee Leck
THRUST Strategic Communications
renee@thrustsc.com

Media Contact:

Carly Scaduto
Carly Scaduto Consulting
Carly@carlyscadutoconsulting.com