Auron Therapeutics, a biotechnology firm specializing in advanced targeted therapies, has unveiled promising data on its leading compound,
AUTX-703, at major scientific conferences in Barcelona and Carlsbad. The new data showcases the compound's effectiveness in degrading
KAT2A/B, a key histone acetyltransferase implicated in various
cancers, including
small cell lung cancer (SCLC),
neuroendocrine prostate cancer (NEPC),
castration-resistant prostate cancer (CRPC), and acute myeloid leukemia (AML).
AUTX-703 is an orally bioavailable heterobifunctional degrader designed to target and eliminate KAT2A/B, thereby disrupting the growth and survival of cancer cells. The compound leverages Auron's proprietary AURIGIN platform, which employs artificial intelligence and machine learning to identify oncogenic cell states and develop precise cancer therapies.
Research presented at the EORTC-NCI-AACR (ENA) Symposium and the Prostate Cancer Foundation (PCF) Annual Scientific Retreat highlighted several key findings. In preclinical studies, AUTX-703 demonstrated potent degradation of KAT2A/B and induced significant tumor growth inhibition and cell differentiation in SCLC and NEPC models. In laboratory settings, AUTX-703 effectively degraded KAT2A/B at very low concentrations, resulting in notable inhibition of cell growth and promotion of a more differentiated cell state.
Further, the compound showed impressive results in patient-derived organoids. In 12 out of 16 SCLC-derived organoids treated with AUTX-703 for 10 to 18 days, there was a marked inhibition of tumor growth coupled with cell state differentiation. Additionally, in vivo experiments using SCLC patient-derived xenograft models revealed that oral administration of AUTX-703 led to significant, dose-dependent tumor growth inhibition and cell differentiation.
In NEPC studies, AUTX-703 maintained its potency, with 2 out of 5 patient-derived organoids demonstrating substantial growth inhibition and cell state differentiation after treatment. The compound also exhibited effectiveness in a prostate cancer cell line resistant to Enzalutamide, a commonly used therapy. Treatment with AUTX-703, both alone and in combination with Enzalutamide, reduced cell growth and diminished key signaling pathways involved in cancer cell survival.
Kate Yen, Ph.D., Founder and Chief Executive Officer of Auron Therapeutics, expressed enthusiasm about the potential of AUTX-703. She emphasized the compound's ability to selectively target and degrade KAT2A/B, resulting in significant tumor inhibition across various cancer models. Dr. Yen also highlighted the role of the AURIGIN platform in identifying new therapeutic targets and developing advanced cancer treatments.
Auron Therapeutics plans to submit an Investigational New Drug (IND) application for AUTX-703 by late 2024, with the aim of initiating clinical trials in early 2025. The company's broader mission focuses on leveraging its AURIGIN platform to pioneer the next generation of targeted cancer therapies, addressing both solid tumors and hematologic malignancies.
With these promising preclinical results, AUTX-703 stands out as a potential breakthrough in targeted cancer therapy, offering new hope for patients with challenging cancer types. The upcoming clinical trials will further evaluate its safety and efficacy, potentially paving the way for new, effective treatments for cancers driven by KAT2A/B.
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