FDA Clears IND for Accent Therapeutics' Oral DHX9 Inhibitor ATX-559

Accent Therapeutics, a biopharmaceutical innovator in cancer treatments, has announced the clearance of its Investigational New Drug (IND) application for ATX-559 by the U.S. Food and Drug Administration (FDA). ATX-559, a unique DHX9 inhibitor, is set to begin a Phase 1/2 clinical trial targeting patients with BRCA1/2-deficient breast cancer and various solid tumors with microsatellite instability-high (MSI-H) or deficient mismatch repair (dMMR). The trial, registered as NCT06625515, is expected to start dosing patients in late 2024. This study will assess the drug's safety, tolerability, pharmacokinetics, pharmacodynamics, and early efficacy.

Jason Sager, M.D., Chief Medical Officer of Accent Therapeutics, highlighted the significance of the FDA's IND clearance, emphasizing that it validates their meticulous approach to discovering new therapeutic targets. He expressed optimism about ATX-559's potential, based on promising preclinical outcomes, to improve the prognosis for patients with challenging cancers.

In preparation for the clinical trial, Accent Therapeutics has formed a Clinical Advisory Board (CAB) comprised of esteemed professionals in oncology drug development. The board will provide strategic direction to advance Accent's clinical programs. The CAB includes notable members such as Dr. Lillian L. Siu, Dr. Josep Tabernero, and Dr. Sam Blackman, all of whom bring a wealth of expertise and leadership in cancer research and treatment.

Shakti Narayan, Ph.D., J.D., the CEO of Accent Therapeutics, expressed gratitude for the involvement of these distinguished experts, whose guidance will be crucial as the company progresses its clinical initiatives. The establishment of the CAB marks a strategic step as Accent moves forward in its mission to develop groundbreaking therapies for cancer patients.

In addition to ATX-559, Accent is advancing its second program targeting KIF18A, a mitotic kinesin motor protein essential for cell division in certain tumors. This program is on track to enter clinical trials in early 2025. KIF18A inhibitors show potential for treating cancers with chromosomal instability, such as ovarian and triple-negative breast cancer. Preclinical studies have demonstrated that KIF18A inhibition induces rapid cell death in aneuploid tumor cells, while sparing normal cells.

ATX-559 and KIF18A are part of Accent's broader strategy to create small molecule cancer therapies that address critical intracellular dependencies across diverse cancer types. ATX-559 targets DHX9, a DNA/RNA helicase involved in vital cellular processes such as replication and transcription. By inhibiting DHX9, ATX-559 exploits tumor-specific vulnerabilities, potentially leading to selective cancer cell death.

Accent Therapeutics is at the forefront of developing precision cancer therapies. Leveraging its expertise in RNA-modifying proteins (RMPs) and related high-value areas for drug discovery, the company is committed to producing transformative treatments for large patient populations with unmet medical needs. Accent's flexible approach aims to address both novel and inadequately managed oncology targets, with a focus on creating biomarker-driven cancer medicines that improve patient outcomes.

As Accent Therapeutics continues to build on its foundational platform technology, the company remains dedicated to translating advanced scientific research into life-changing therapies for cancer patients. The upcoming clinical trials for ATX-559 and the KIF18A program represent significant milestones in their journey to innovate cancer treatment.