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Exicure Announces Positive Phase 1 Results for its Lead Immuno-Oncology Asset


-- First in human results demonstrate potent immune system activation with no serious adverse events or dose limiting toxicity --

SKOKIE, Ill.--(BUSINESS WIRE)--Sep. 20, 2018-- Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional spherical nucleic acid (SNA™) constructs, announced today Phase 1 results for AST-008, an SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications.

“Phase 1 results for AST-008 demonstrated our desired highly potent immune system activation without serious adverse events or dose limiting toxicity. We believe this molecule could lead to better combination therapies for patients with cancer and, to that end, we expect to initiate a Phase 1b/2 trial in patients before year end,” said Dr. David Giljohann, Chief Executive Officer of Exicure. “These data support the potential of our SNA platform for immuno-oncology and set the stage for ongoing development of the SNA platform into indications well beyond cancer.”

Exicure’s Phase 1 Trial Results

The Phase 1 trial of AST-008 was a single ascending subcutaneous dose trial comprised of 16 healthy volunteers. AST-008 was shown to be safe and tolerable in all subjects, with no serious adverse events and no dose limiting toxicity. AST-008 was well tolerated and all AST-008-related adverse events were of short duration, reversible and consistent with TLR9 activation.

In addition to the principle safety and tolerability endpoint, the trial screened for levels of select cytokines and markers of immune cell activation. AST-008 was shown to elicit high levels of certain cytokines as well as activate important effector cells of the immune system including T cells and natural killer cells, the main drivers of anti-tumor response.

For the four subjects receiving the trial’s top dose of about 20 µg/kg of AST-008, initial analyses suggest that the average fold-increase above baseline for these cytokines is approximately as follows: IFN-gamma: 3 fold; IL-6: 57 fold; IL-12: 2 fold; IP-10: 32 fold; and MCP-1: 4 fold. At this dose, AST-008 also elicited 9.5 fold and 3.5 fold increases in the fraction of activated T cells and natural killer cells, respectively, compared to baseline.

Exicure’s Planned Phase 1b/2 Patient Trial

Exicure intends to begin an open-label Phase 1b/2 trial of intra-tumorally dosed AST-008 in combination with a checkpoint inhibitor before year end. The trial will begin with an AST-008 dose finding Phase 1b stage, followed by a Phase 2 expansion stage. In the Phase 1b, Exicure will enroll patients with superficial injectable tumors and will prioritize those with Merkel cell carcinoma, cutaneous squamous cell carcinoma, melanoma, and squamous cell carcinoma of the head and neck. Preliminary data from the Phase 1b stage are expected in late 2019.

Historical TLR9 Agonist Healthy Volunteer Data

In 2015, Mologen AG published results (European Journal of Cancer, 2015, volume 51, supplement 1, page S12) from a healthy volunteer trial. In a single cohort, 13 subjects each received one 60 mg dose (equivalent to 923 µg/kg for a 65 kg subject) of lefitolimod subcutaneously. On average, across the cohort, there was a 7 fold-increase in IP-10 expression above baseline. No cell activation data were reported. Lefitolimod is currently in a Phase 3 clinical trial.

In 2004, Coley Pharmaceutical Group (now Pfizer, Inc.) published results (Journal of Immunotherapy, 2004, Volume 27, pages 460–471) from a single ascending dose healthy volunteer trial. In that trial, their TLR9 agonist, PF-03512676, was administered subcutaneously to six subjects per dose level. For the 20 µg/kg dose level, the average fold-increase above baseline for these cytokines is as follows: IFN-gamma: no change from baseline; IL-6: 8 fold; IL-12: no change from baseline; IP-10: 9 fold; and MCP-1: 3 fold.

Upcoming Presentations

Exicure management expects to be presenting and available for meetings at a number of fall investor conferences including:

  • October 2 – Ladenburg Thalmann 2018 Healthcare Conference at the Sofitel Hotel New York, NY;
  • October 3 – Leerink Partners Roundtable Series: Rare Disease & Oncology at the Lotte New York Palace, New York, NY;
  • October 9 – Chardan’s 2nd Annual Genetic Medicines Conference at the Westin Grand Central, New York, NY;
  • October 25BTIG Biotech Conference at the Langham Hotel, New York, NY.

Exicure also intends to present at a variety of scientific conferences including:

  • September 27 – CEO Dr. David Giljohann at the SMi Immuno-Oncology conference at the Copthorne Tara Hotel, London;
  • September 30 – CSO Dr. Ekambar Kandimalla will present a poster at the Fourth CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference at the Marriott Marquis, New York, NY;
  • October 1 – Poster at the Oligonucleotide Therapeutics Society meeting at the Marriott Waterfront, Seattle, WA;
  • November 9-10 – Poster at the Society for Immunotherapy of Cancer (SITC) conference, Washington DC.

About Exicure, Inc.

Exicure, Inc. is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure's proprietary 3-dimensional, spherical nucleic acid spherical nucleic acid (SNA™) architecture is designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address inflammatory diseases, genetic disorders and oncology. Exicure is based outside of Chicago,

Forward Looking Statements

This press release contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning the Company, the Company’s technology, potential therapies and other matters. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “plan,” “believe,” “intend,” “look forward,” and other similar expressions among others. Statements that are not historical facts are forward-looking statements. Forward-looking statements are based on 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: unexpected costs, charges or expenses that reduce cash runway; that Exicure’s pre-clinical programs do not advance into the clinic 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 that have completed Phase 1 trials do not become approved drugs on a timely or cost effective basis or at all; possible safety and efficacy concerns; regulatory developments; and the ability of Exicure to protect its intellectual property rights. Exicure’s pipeline programs are in various stages of pre-clinical and clinical development, and the process by which such pre-clinical or clinical therapeutic candidates could potentially lead to an approved therapeutic is long and subject to significant risks and uncertainties. Risks facing the Company and its programs are set forth in the Company’s filings with the SEC. Except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Source: Exicure, Inc.

MacDougall Biomedical Communications
Karen Sharma, 781-235-3060
The Del Mar Consulting Group, Inc.
Robert Prag, 858-794-9500

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