Exicure Announces First Neurological Development Program in Friedreich’s Ataxia and Expands Scientific Advisory Board
December 16 2019 - 7:00AM
Business Wire
- Exicure to collaborate with
Friedreich’s Ataxia Research Alliance (FARA)
- Hank Paulson, MD, PhD and Susan Perlman, MD,
added to Scientific Advisory Board
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.
About FARA
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.
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Media: MacDougall Karen Sharma 781-235-3060
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