CHICAGO & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, will present a poster at the 2020 Annual Meeting of the American Society of Clinical Oncology (ASCO), taking place virtually from May 29-31.
The ASCO poster, by Dr. Mohammed Milhem of the University of Iowa and coauthors, titled “AST-008: A Novel Approach to TLR9 Agonism with PD-1 Blockade for Anti-PD-1 Refractory Merkel Cell Carcinoma (MCC) and Cutaneous Squamous Cell Carcinoma (CSCC)” will be presented virtually in the Developmental Therapeutics—Immunotherapy Poster Session under abstract number TPS3164.
The poster will present details of the phase Ib/II trial of Exicure’s drug candidate, AST-008, a toll-like receptor 9 agonist oligonucleotide being developed for the treatment of MCC and CSCC in patients progressing on immune checkpoint inhibitor monotherapy.
Key features of the phase Ib/II trial described in the poster include:
- Advanced/metastatic MCC and CSCC are skin cancers with high unmet medical need;
- Data presented earlier suggest that AST-008 elicits a Th1-type cytokine response and immune cell activation;
- The phase II dose escalation study will use the recommended dose of AST-008, administered intratumorally, in combination with pembrolizumab or cemiplimab to treat two cohorts of patients with advanced/metastatic MCC or CSCC respectively;
- The enablement of a dose expansion of AST-008 plus cemiplimab in CSCC without having performed a separate dose escalation with the latter combination;
- Each expansion cohort will enroll up to 29 patients across about 15 US-based sites;
- Patients enrolled into the phase II will have to have progressed on a single-agent checkpoint therapy;
- The key objective of the phase II expansion is to provide an estimate of preliminary efficacy of intratumorally administered AST in the trial subjects. Other endpoints include safety, pharmacokinetic and pharmacodynamic assessments.
AST-008 is an SNA consisting of toll-like receptor 9 agonists designed for immuno-oncology applications. As of January 31, 2020, we have dosed 17 patients in the Phase 1b stage of the clinical trial. We have observed no treatment related serious adverse events, nor have we observed any dose-limiting toxicity among the treated subjects. The most common reported adverse event was injection site reactions. In December 2019, we received preliminary results from the Phase 1b/2 stage of the clinical trial showing potential signs of anti-tumor activity in patients with Merkel cell carcinoma. In the second quarter of 2020, we plan to initiate a Phase 2 dose expansion for intratumoral AST-008 in combination with approved checkpoint inhibitors to treat two cohorts of patients with advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma. Each cohort is expected to enroll up to 29 patients who have failed anti-PD-1/PD-L1, or programmed cell death protein 1/programmed death-ligand 1, therapy. We expect to open a total of up to 15 sites in the United States.
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.