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Exicure Presents Cavrotolimod (AST-008) Clinical Data at AACR 2020 Virtual Meeting


Poster presentation of pharmacodynamic and safety data of cavrotolimod (AST-008) from Exicure’s ongoing Phase 1b/2 clinical trial in solid tumor patients

CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, is presenting updated pharmacodynamic and safety data at the American Association of Cancer Research (AACR) Virtual Annual Meeting II, occurring June 22 – 24, 2020.

The AACR poster, titled “Phase 1b/2 Study of an Intratumoral TLR9 Agonist Spherical Nucleic Acid (AST-008) and Pembrolizumab: Evidence of Immune Activation,” is presenting new preliminary pharmacodynamic and safety data of cavrotolimod (AST-008), alone and in combination with pembrolizumab, from Exicure’s ongoing Phase 1b/2 clinical trial ( identifier: NCT03684785). Cavrotolimod (AST-008) is a novel SNA configuration of a toll-like receptor 9 (TLR9) agonist oligonucleotide, designed to trigger anti-tumor immune responses.

Gene expression analysis data from patient tumor biopsies demonstrated increases in leukocytes in injected tumors after intratumoral (IT) cavrotolimod (AST-008) alone and in combination with pembrolizumab versus baseline. Uninjected tumors also showed increased immune cell levels after patients received cavrotolimod (AST-008) and pembrolizumab, suggesting immune cell trafficking.

Dose-dependent activation of key immune cells, including cytotoxic T cells and natural killer cells, as well as increases in cytokine/chemokine levels were observed in patient blood after IT cavrotolimod (AST-008) treatment alone, and cavrotolimod (AST-008) plus pembrolizumab treatment. We expect that activation of these cell types and expression of immune system signaling proteins may help produce anti-tumor effects.

Cavrotolimod (AST-008) was well-tolerated, with a safety profile consisting primarily of injection site reactions and flu-like symptoms, which is believed to reflect local and systemic immune activation. No cavrotolimod (AST-008)-related serious adverse events or dose limiting toxicity have been reported.

Using these data, a recommended Phase 2 dose of 32 mg cavrotolimod (AST-008) has been identified for the Phase 2 portion of the clinical trial now underway, where cavrotolimod (AST-008) will be given in combination with pembrolizumab or cemiplimab for the treatment of locally advanced or metastatic Merkel cell carcinoma or cutaneous squamous cell carcinoma, respectively, in patients with progression despite approved anti-PD-(L)1 therapy.

This poster is being presented during the AACR Virtual Meeting II in the session Late-Breaking Research: Clinical Research 1 / Endocrinology under abstract number LB-140. The poster will be available for viewing from June 22 – 24.

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 cavrotolimod (AST-008) is currently in a Phase 1b/2 clinical trial in patients with Merkel cell carcinoma or cutaneous squamous cell carcinoma in the advanced/metastatic stage. Exicure is based in Chicago, IL and also has an office in Cambridge, MA. For more information, visit Exicure’s website at

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release other than statements of historical fact could be deemed forward looking including, but not limited to, the company’s expectations regarding potential benefits, of cavrotolimod (AST-008), including the ability of cavrotolimod (AST-008) to trigger anti-tumor immune responses, and the safety and tolerability of cavrotolimod (AST-008). The forward-looking statements in this press release speak only as of the date of this press release, and the company undertakes no obligation to update these forward-looking statements. Forward-looking statements are based on management’s current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the risks that the ongoing COVID-19 pandemic may disrupt the company’s business and/or the global healthcare system more severely than anticipated; the cost, timing and results of clinical trials; the ability to enroll and maintain patients in clinical trials; and possible safety and efficacy concerns. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the section titled “Risk Factors” in the company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2020, as updated by the company’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and the company undertakes no duty to update this information, except as required by law.

Media Contact
Karen Sharma

Source: Exicure, Inc.

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