CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Exicure, Inc. (NASDAQ:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, today announced Friedreich’s ataxia (FA) as the therapeutic indication for the company’s first neurology development program. Exicure also announced the expansion of its Scientific Advisory Board.
“Over the past year Exicure has developed extensive preclinical data supporting the development of our SNAs for neurological disorders. We’re eager to evaluate our technology for this important unmet medical need,” said David Giljohann, PhD, Exicure’s chief executive officer. “We are also pleased to add the experience and team from the Friedreich’s Ataxia Research Alliance (FARA),” added Dr. Giljohann.
Patients living with FA experience a devastating and progressive loss of neurological function. There are no approved therapies for FA. Exicure’s FA program will be designed and developed with guidance from and in collaboration with FARA.
"FA is a rare, progressive and life-shortening disease, and there is a critical need for effective treatments," explained FARA's Chief Executive Officer, Jennifer Farmer. "Exicure’s SNA technology is unique in its ability to target the affected gene in FA. FARA has championed collaborative approaches to drug development, and we’re thrilled to work with Exicure as they develop their genetically targeted therapeutic candidate.”
Exicure expects IND-enabling work to begin in 2020. Exicure’s preclinical data in rodents and non-human primates showed distribution of SNAs to all brain regions following intrathecal administration, including those relevant in FA. Additional preclinical rodent data, in a head-to-head comparison with an FDA-approved oligonucleotide, nusinersen, showed improved potency in mice when put into an SNA format.
Exicure also is announcing the expansion of the company’s Scientific Advisory Board to include neurology experts Dr. Susan Perlman, MD, Professor of Neurology at University of California Los Angeles and Medical Director for the National Ataxia Foundation and Dr. Hank Paulson, MD, PhD, Lucile Groff Professor of Neurology for Alzheimer's Disease and Related Disorders in the Department of Neurology at the University of Michigan.
“We are pleased to bring together this fantastic group of patient advocates, medical leaders, and disease experts to join the Exicure team in our mission to launch a program in Friedreich’s ataxia,” Dr. Giljohann concluded.
About Friedreich’s Ataxia (FA)
FA is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use; diminished vision, hearing and speech; scoliosis (curvature of the spine); increased risk of diabetes; and a life-threatening heart condition. There are no FDA-approved treatments. An estimated 5,000 patients in the US and 15,000 patients worldwide are affected by FA.
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia. www.CureFA.org
About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for 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's lead program is in a Phase 1b/2 trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL and in Cambridge, MA.
For more information, visit Exicure’s website at www.exicuretx.com.
Exicure 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 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; and the ability of Exicure to protect its intellectual property rights. Furthermore, data from preclinical studies in mice often fails to be indicative of outcomes in human trials. 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 (including without limitation its cash runway guidance) or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.