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KSQ Therapeutics Begins Trial of USP1 Inhibitor for Advanced Solid Tumors

NEW YORK – KSQ Therapeutics said on Tuesday that it has begun treating patients with solid tumors in a Phase I trial of its investigational USP1 inhibitor KSQ-4279.

The trial will enroll roughly 140 patients with advanced solid tumors across five sites in the US, all of whom will receive KSQ-4279 either as a monotherapy or in combination with another agent such as a PARP inhibitor.

Cambridge, Massachusetts-based KSQ will determine KSQ-4279's safety and maximum tolerated dose as primary endpoints in the trial. Investigators are also looking into the agent's pharmacokinetics and preliminary antitumor activity and trying to determine potential biomarkers and other genetic factors that might predict patients' treatment outcomes.

In developing KSQ-4279, the firm used its CRISPRomics platform to home in on USP1 as a potential target to exploit synthetic lethality in cancers with DNA repair defects. Preclinical studies of the agent suggested that cancer cells use different genetic drivers to develop resistance to USP1 and PARP inhibitors, which supports combining KSQ-4279 with PARP inhibitors to mitigate treatment resistance. The firm also intends to explore the synergistic activity of KSQ-4279 with other therapies.