KSQ Therapeutics, Inc., a clinical-stage biotechnology company developing cancer therapies using its CRISPRomics discovery platform, has entered into a worldwide license and collaboration agreement with Roche for the development and commercialization of KSQ-4279. KSQ-4279 is a first-in-class, potent, and selective small molecule inhibitor of USP1, a protein that regulates DNA damage response (DDR) in a manner distinct from other approaches, including PARP inhibitors.

The molecule is currently in a Phase 1 clinical trial for the treatment of advanced solid tumors. Under the collaboration, Roche will assume development responsibilities for KSQ-4279, which has the potential to treat a variety of cancers. KSQ will receive an upfront payment and will be eligible to receive additional milestone and royalty payments.

USP1 was identified by KSQ’s CRISPRomics platform as an attractive cancer target with established roles in DNA damage repair processes that are distinct from PARP inhibitors and other approaches currently being tested in the clinic. 

“We believe KSQ-4279, which was discovered through our proprietary CRISPRomics platform, has incredible potential to help patients with a variety of solid tumors based on the strong preclinical data we have seen to date,” said Qasim Rizvi, CEO of KSQ. We are confident Roche is the right partner to further the development of KSQ-4279 and maximize its benefits for patients. For KSQ, this agreement enables us to fully focus our attention on advancing our own immunotherapy programs and to continue leveraging our successful platform to discover novel targets.”

James Sabry, global head of pharma partnering, Roche said, “DDR is a promising and emerging area of research in oncology. We are excited to collaborate with KSQ on their novel inhibitor of USP1 as a potential new treatment option for patients with significant unmet medical needs across a range of cancers. This partnership reflects our strategic focus of fitting treatments to patients and delivering personalized healthcare.”