In a significant breakthrough for 280Bio, Inc., a clinical-stage biotechnology company, the US Food and Drug Administration (FDA) has given the green light to its Investigational New Drug (IND) application for YL-17231.

This investigational drug, a small molecule inhibitor of RAS signaling, will now proceed to Phase 1 clinical study in the U.S.

The RAS genes, namely KRAS, NRAS, and HRAS, often undergo mutations in various tumor indications. These oncogenic mutations propel tumor cell growth. In pre-clinical studies, YL-17231 exhibited strong inhibitory effects on tumor cell proliferation in vitro and in vivo. Notably, it showed activity against a wide range of RAS mutations, including tumors resistant to KRAS G12C inhibitors.

Following FDA approval, 280Bio will initiate a Phase 1 study for the treatment of advanced cancer patients whose tumors have RAS mutations. This U.S.-based Phase 1 clinical study will take place at several clinical cancer research centers, including the MD Anderson Cancer Center in Houston, Texas. The company aims to commence patient enrollment for the dose escalation component of Phase 1 in the final quarter of 2023.

Michael Hui — 280Bio Inc. CEO said: “The IND approval by the FDA enables 280Bio to initiate the Phase 1 clinical trial of our novel KRAS inhibitor marking a major milestone for the company’s clinical development program.

“We are excited to explore the therapeutic potential of YL-17231 targeting a broad patient population with RAS mutations to potentially create a transformative therapy option.”

YL-17231 is the fruit of a co-development effort between 280Bio and Yingli Pharma. This joint venture forms part of 280Bio’s strategic collaboration with The University of Texas MD Anderson Cancer Center, where further preclinical research on this investigational drug is ongoing.

  280Bio, cancer, MD Anderson Cancer Center, Michael Hui, US FDA, US Food and Drug Administration, USA, Yingli Pharma, YL-17231