On March 3, 2021 Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its clinical product candidate, cavrotolimod (AST-008), for the treatment of patients with Merkel cell carcinoma (MCC) (Press release, Exicure, MAR 3, 2021, View Source [SID1234576008]).
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"We are excited to have been granted Orphan Drug Designation by the FDA for cavrotolimod for MCC," said Dr. Douglas Feltner, Chief Medical Officer of Exicure. "This designation marks significant progress toward our goal of fulfilling the unmet medical need and finding a treatment for Merkel cell carcinoma patients."
Exicure previously announced that the FDA granted two Fast Track designations for cavrotolimod (AST-008) for MCC and cutaneous squamous cell carcinoma (CSCC), both in the advanced/metastatic setting after progression on anti-PD-1/PD-L1 antibodies.
About Orphan Drug Designation
Orphan Drug Designation is a designation granted by the FDA which provides orphan status to drugs or biologics which are intended to treat rare diseases or disorders that affect fewer than 200,000 people in the United States. This designation provides the sponsor of the drug certain incentives, including tax credits for qualified clinical trials and fee waivers. Orphan Drug Designation confers eligibility for seven years of market exclusivity to an orphan drug post-approval, subject to a receipt by the FDA of marketing approval.
About Cavrotolimod (AST-008)
Cavrotolimod (AST-008) is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications. In December 2019, Exicure announced preliminary results from the Phase 1b stage of the clinical trial including potential signs of anti-tumor activity with cavrotolimod in combination with pembrolizumab in cancer patients, including those with MCC. To date, 20 patients in the Phase 1b stage of the clinical trial have been dosed, and no cavrotolimod-related serious adverse event or dose-limiting toxicity has been reported. The most commonly reported adverse events were injection site reactions and flu-like symptoms. In the second quarter of 2020, Exicure initiated Phase 2 dose expansion cohorts of intratumoral cavrotolimod in combination with approved checkpoint inhibitors to treat two cohorts of patients with advanced or metastatic MCC or CSCC. Each cohort is expected to enroll up to 29 patients whose tumors have progressed on anti-PD-1/PD-L1, or programmed cell death protein 1/programmed death-ligand 1, antibody monotherapy.