The U.S. Food and Drug Administration (FDA) has granted Checkpoint Therapeutics, Inc. (“Checkpoint”) (CKPT)'s Orphan Drug Designation to CK-101 (also known as RX518), the Company’s third-generation epidermal growth-factor receptor (EGFR) inhibitor, for the treatment of EGFR mutation-positive non-small cell lung cancer (NSCLC).

CK-101 is currently being studied in the Phase 1 dose-escalation portion of a Phase 1/2 clinical study. The Phase 1 portion of the study is evaluating the safety and tolerability of ascending doses of CK‐101 in patients with advanced solid tumors to determine the maximum tolerated dose and / or recommended dose for the Phase 2 portion of the study. The Phase 2 portion will evaluate the safety and efficacy of CK-101 in patients with EGFR T790M mutation-positive NSCLC.

“Securing Orphan Drug Designation is a significant milestone that will support the clinical development of our lead targeted therapy, CK-101, in EGFR mutation-positive NSCLC,” said James F. Oliviero, President and Chief Executive Officer of Checkpoint. “This Orphan Drug Designation builds upon the issued U.S. composition of matter patent for CK-101, and could provide the benefit of additional market exclusivity.”

Orphan Drug Designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases / disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives that may include tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.