CHICAGO, IL & CAMBRIDGE, MA, USA I June 16, 2020 I Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, announced today the dosing of the first patient enrolled in the Phase 2 dose expansion stage of the cavrotolimod (AST-008) Phase 1b/2 clinical trial (ClinicalTrials.gov identifier: NCT03684785).

“We believe this milestone marks an important step toward fulfilling an unmet treatment need for Merkel cell carcinoma patients who have been resistant to checkpoint inhibitor antibody therapy,” said Dr. Douglas Feltner, Chief Medical Officer of Exicure.

The Phase 2 dose expansion stage includes two cohorts of patients with advanced or metastatic cancer: patients with Merkel cell carcinoma (MCC) and patients with cutaneous squamous cell carcinoma (CSCC). MCC is a rare, aggressive skin cancer considered more deadly than melanoma. Risk factors for MCC include sun exposure and a weakened immune system. Advanced or metastatic CSCC is an uncommon form of skin cancer associated with significant morbidity and mortality.

The dose expansion stage is designed to assess the anti-tumor response rate of cavrotolimod plus checkpoint inhibitor treatment, the safety and tolerability of the combination, and drug pharmacodynamics and pharmacokinetics. Patients enrolled in the dose expansion cohorts must have recently documented tumor progression despite anti-PD-1 or anti-PD-L1 antibody monotherapy.

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 patients 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.

About Exicure, Inc.

Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for neurology, immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. Exicure believes that its proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure is in preclinical development of XCUR-FXN, an SNA–based therapeutic candidate for the treatment of Friedreich’s ataxia (FA). Exicure’s drug candidate AST-008 is currently in a Phase 1b/2 clinical trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL and also has an office in Cambridge, MA. For more information, visit Exicure’s website at www.exicuretx.com.

SOURCE: Exicure