FDA Grants Fast Track Designation to AUTX-703 In R/R AML

FDA granted fast track designation to AUTX-703, an oral KAT2A/B degrader by Auron Therapeutics, for relapsed or refractory acute myelogenous leukemia.

The Food and Drug Administration (FDA) has granted fast track designation to the novel drug AUTX-703 for the treatment of patients with relapsed or refractory (r/r) acute myelogenous leukemia (AML).

The fast track designation was announced in a news release issued by AUTX-703’s manufacturer, clinical-stage biotechnology company Auron Therapeutics, which described AUTX-704 as a novel, first-in-class, oral KAT2A/B degrader. The company also announced that, following the FDA’s clearance of its Investigational New Drug application, the company plans to initiate clinical development of the drug in hematological malignancies in the first quarter of this year.

“We are gratified by the FDA’s recognition of AUTX-703’s potential as an important, novel treatment option for [patients with] relapsed or refractory AML, … a community that remains in desperate need of new therapies,” said Kate Yen, founder and Chief Executive Officer of Auron. “The FDA Fast Track Designation underscores the urgent need for innovative treatments for these patients and this milestone comes at a pivotal moment as we prepare to advance AUTX-703 into the clinic. Our team is energized by the potential to bring a first-in-class therapy to patients who have limited effective options today, while simultaneously exploring the broader applications of KAT2A/B modulation.”

Promising preclinical data on AUTX-703 were presented last year at the 66^th American Society of Hematology Annual Meeting and Exposition, according to a news release issue by Auron in December 2024. The data, the company stated, showed that treatment with AUTX-703 was well-tolerated and effective.

“These are the first data to demonstrate AUTX-703’s potent degradation of KAT2A/B promoting cellular differentiation and leading to a significant survival advantage in a primary AML model,” said Yen in the December 2024 news release.

Fast track designation, according to the FDA’s website, is a process that is designed to facilitate the development and expedite the review of drugs intended to treat serious conditions and fill an unmet medical need, with the purpose being to make important new drugs available for patients sooner.

As explained by the Leukemia & Lymphoma Society, refractory AML is a term usually used to describe patients who have not achieved complete remission after two cycles of induction chemotherapy, while relapsed AML is used to describe patients who reach remission and then have a return of leukemia cells in the bone marrow and a decrease in normal blood cells.

AML, as described in the news release, is an aggressive blood cancer that involves the rapid growth of abnormal cells that build up in a patient’s bone marrow and interfere with normal blood cell production. Patients with r/r AML have a five-year survival rate of approximately 10%, Auron stated.

The American Cancer Society estimates that in 2025, approximately 22,010 people will receive diagnoses of AML, most of whom will be adults, and approximately 11,090 people, also mostly adults, will die from the disease.

AML makes up approximately a third of all leukemias in adults, but only accounts for 1% or so of all cancers, the American Cancer Society stated, nothing that the disease is uncommon in patients under the age of 45 years old and the average age of patients when they are first diagnosed is about 69 years old.

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