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Spherical Nucleic Acid Technology Shows Promising Results in Phase 0 Trial in Patients With Glioblastoma at Northwestern University


CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, is pleased to share that researchers at Northwestern University have utilized Exicure’s licensed first generation gold-nanoparticle SNA technology in an investigator-initiated trial for the treatment of glioblastoma (GBM), a deadly form of brain cancer.

The results were published in an article in Science Translational Medicine. DOI:10.1126/scitranslmed.abb3945

The researchers that led the study, Drs. Priya Kumthekar and Alexander Stegh, conducted a single-arm, open-label phase 0 first-in-human clinical trial (NCT03020017) to determine safety, pharmacokinetics, intratumoral accumulation and gene-suppressive activity of systemically administered SNAs comprised of siRNA specific for the GBM oncogene Bcl2L12. Patients with recurrent GBM were treated with intravenous administration of siBcl2L12-SNAs (NU-0129).

The paper reports first-in-human evidence that siRNA-based SNAs can be administered intravenously, cross the blood-brain-barrier in glioblastoma patients, accumulate in GBM tumor cells, and engage with their target gene.

“We congratulate Drs. Kumthekar and Stegh on these results,” said Dr. David Giljohann, the CEO of Exicure. He added, “We are excited about the impact to patients and its implications in treating other diseases, as Exicure continues to build and grow our pipeline in neurological disorders.”

Exicure anticipates filing an IND for its lead program in Friedreich’s ataxia by the end of 2021.

About Glioblastoma

Glioblastoma (GBM) is the most common and most aggressive type of primary malignant tumor of the central nervous system. The global incidence of glioblastoma is less than 10 per 100,000 people. Standard treatment for patients with newly diagnosed glioblastoma can include surgery followed by radiation and chemotherapy, but treatment options are limited. The last investigational medicine to improve survival for patients with newly diagnosed glioblastoma was approved by the U.S. Food and Drug Administration in 2005. The five-year survival rate of patients with GBM is less than five percent.

About Friedreich’s Ataxia

Friedreich’s ataxia (FA) is the most commonly inherited ataxia, a degenerative neuromuscular disease leading to progressive loss of coordination, causing severe childhood disability and early mortality, in most cases before age 40. It is a monogenic disorder caused by mutations in the FXN gene resulting in reduced levels of frataxin protein. FA affects about 13,500 people in the US, Europe, Canada and Australia combined. There are currently no approved therapies for Friedreich’s ataxia patients.

About Exicure, Inc.

Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for neurology, immuno-oncology, inflammatory diseases and other 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 a lipid-nanoparticle SNA–based therapeutic candidate, for the intrathecal treatment of Friedreich’s ataxia (FA). Exicure's therapeutic candidate cavrotolimod (AST-008) is in a Phase 1b/2 clinical trial in patients with advanced solid tumors. Exicure is based in Chicago, IL and 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 initiation, timing and results of the Company’s preclinical studies and clinical trials, including its lead program in Friedreich’s ataxia; and the potential of the Company’s SNA technology to provide therapeutic benefit to target diseases, including its ability to address the genetic challenges posed by Friedreich’s ataxia and other neurological conditions. 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 coronavirus disease 2019, or COVID-19, pandemic or its impact or effects may disrupt the Company’s business and/or the global healthcare system (including its supply chain) more severely than it has to date or more severely than anticipated; unexpected costs, charges or expenses that reduce the Company’s capital resources; the Company’s preclinical or clinical programs do not advance or result in approved products on a timely or cost effective basis or at all; the cost, timing and results of clinical trials; that many drug candidates do not become approved drugs on a timely or cost effective basis or at all; the ability to enroll patients in clinical trials; possible safety and efficacy concerns; regulatory developments; risks that preliminary results from clinical trials are not necessarily predictive of future clinical trial results; and the ability of the Company to protect its intellectual property rights. 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 Annual Report on Form 10-K for the year ended December 31, 2020, which is expected to be filed on or approximately on the same date hereof, 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. In addition, the COVID-19 pandemic and the associated containment efforts have had and continue to have a serious adverse impact on the economy, the severity and duration of which are uncertain. Government stabilization efforts have only partially mitigated the consequences. The extent and duration of the impact on the Company’s business and operations is highly uncertain, and that impact includes effects on its clinical trial operations, timelines and supply chain. Factors that will influence the impact on the Company’s business and operations include the duration and extent of the pandemic, the extent of imposed or recommended containment and mitigation measures, and the general economic consequences of the pandemic. The COVID-19 pandemic or its impact or effects could have a material adverse impact on the Company’s business, operations and financial results for an extended period of time.

Karen Sharma

Source: Exicure, Inc.

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