Drug intelligence / Profile preview

autx-703

Development stage
Phase 1
Lead developer
Auron Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

AUTX-703 is a novel, first-in-class, orally bioavailable small molecule degrader targeting the histone acetyltransferases Lysine acetyltransferase 2A (KAT2A) and Lysine acetyltransferase 2B (KAT2B). Developed by Auron Therapeutics using its AURIGIN platform, it is designed to modulate cell-state plasticity in cancer. Preclinical studies have demonstrated that AUTX-703 selectively and potently degrades KAT2A/B proteins, inhibits tumor growth, induces differentiation of cancer cells, and provides significant survival advantages in models of acute myeloid leukemia (AML), small cell lung cancer (SCLC), and neuroendocrine prostate cancer (NEPC). The drug has received FDA Fast Track designation for relapsed or refractory acute myelogenous leukemia (AML) and entered phase 1 clinical trials for hematological malignancies in early 2025[1][2][3][4][5][6].

Other names
AUTX 703AUTX703AUTX-703
02

Targets

KAT2A (Lysine acetyltransferase 2A)KAT2B

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