Curis Announces FDA Acceptance of Investigational New Drug Application for CA-170, the First Orally Available Small Molecule to Target and Inhibit Immune Checkpoints

On June 01, 2016 Curis, Inc. (Nasdaq:CRIS), a biotechnology company focused on the development and commercialization of innovative and effective therapeutics for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has accepted the company’s Investigational New Drug (IND) application for CA-170 (Press release, Curis, JUN 1, 2016, View Source [SID:1234512911]). CA-170 is a first-in-class orally available small molecule that has been designed to target and inhibit the immune checkpoints, Programmed Death Ligand-1 (PD-L1) and V-domain Immunoglobulin Suppressor of T-cell Activation (VISTA).

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"The acceptance of CA-170’s IND by the FDA marks an important milestone in the advancement of immuno-oncology therapy," said Ali Fattaey, Ph.D., Curis’s president and CEO. "The last few years have seen the successful development and commercial launch of multiple checkpoint inhibitors to treat a broad range of human cancers. However, all checkpoint inhibitors developed thus far have been monoclonal antibodies, with similar pharmacokinetic properties, and can only be administered by IV infusion. Today, the FDA has cleared us to test the first small molecule checkpoint inhibitor, CA-170, that will be taken orally by cancer patients. We envision that the pharmacokinetic properties of a small molecule will likely provide an advantage in dosing flexibility of a checkpoint inhibitor, either as a monotherapy or in combination with other cancer treatment regimens. We believe that, if successful, CA-170 can provide a compelling treatment alternative for patients and physicians."

CA-170 is an orally available, small molecule designed to selectively target and inhibit both PD-L1 and VISTA checkpoint regulators of immune activation. Preclinical data have demonstrated that CA-170 can induce effective proliferation and cytokine production by T cells in culture that are specifically suppressed by PD-L1 or VISTA. In addition, CA-170 demonstrated in vivo anti-tumor activity similar to anti-PD-1 or anti-VISTA antibodies in multiple mouse tumor models and appeared safe to administer based on toxicology studies.