FDA Grants Fast Track Designation to AUTX-703 for Relapsed, Refractory AML

The FDA granted Fast Track Designation to AUTX-703 for the treatment of relapsed or refractory acute myeloid leukemia (AML), according to a press release from Auron Therapeutics, the developer of the drug.¹

The clinical-stage biotechnology company plans to begin clinical development of AUTX-703, a novel, first-in-class, oral KAT2A and KAT2B degrader, in the first quarter of 2025 following recent clearance of its Investigational New Drug application.

“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,” said Kate Yen, PhD, Founder and Chief Executive Officer of Auron, in the press release. “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.”

An abstract presented at the 66th American Society of Hematology Annual Meeting and Exposition evaluated AUTX-703 in a primary human AML xenograft model. Ex-vivo AUTX-703 treatment induced monocytic differentiation and inhibited the growth of CD34+ blasts from multiple patients with AML, therefore offering a survival advantage.²

“These data provide evidence that degradation of KAT2A and KAT2B can reverse the block in cellular differentiation in primary patient AML cells, which could provide therapeutic benefit to patients,” the authors wrote. “A phase 1 clinical trial testing the safety and tolerability of AUTX-703 is planned in AML.”²

References

1. Auron Therapeutics Announces AUTX-703 Granted Fast Track Designation by the FDA for Relapsed or Refractory Acute Myelogenous Leukemia. News release. Auron. February 24, 2025. Accessed February 26, 2025. https://www.aurontx.com/news/auron-therapeutics-announces-autx-703-granted-fast-track-designation-by-the-fda-for-relapsed-or-refractory-acute-myelogenous-leukemia
2. Duparc H, Neef J, Kandiah J, et al. AUTX703, a novel and potent KAT2A and KAT2B protein degrader, induces differentiation and offers survival advantage in a primary human AML xenograft model. Abstract presented at: 66th American Society of Hematology Annual Meeting and Exposition; December 7-10, 2024; San Diego, CA.