Exicure Announces Agenda for Upcoming Neuroscience Pipeline Update at Virtual R&D Day

CHICAGO & CAMBRIDGE, Mass.–([1])–Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) technology, will host a virtual R&D Day on Thursday, January 7, 2021 from 09:00 am to 10:30 am ET to discuss Exicure’s neuroscience pipeline, including its lead program for Friedreich’s Ataxia (FA).

Exicure will discuss its progress with XCUR-FXN, which advanced into IND-enabling studies in Q4 2020, and provide its perspective on the path for the product candidate to clinical validation for the treatment of Friedreich’s Ataxia. In addition, Exicure will provide an update on its clinical pipeline across a number of rare neurodegenerative diseases of high unmet need, and highlight the advancement of two preclinical programs: one targeting SCN9A for neuropathic pain and the other targeting CLN3 Batten Disease.

Underscoring the differentiating nature of Exicure’s SNA platform for discovery of oligonucleotide-based therapeutics for neurodegenerative diseases, Exicure will present results of CNS biodistribution studies of SNA therapeutics in both rodent and non-human primates.

Exicure’s Scientific Advisory Board member Dr. Susan Perlman and the CEO of the Friedreich’s Ataxia Research Alliance (FARA) Jennifer Farmer, will join Exicure’s leadership team in discussing Exicure’s progress in the treatment of Friedreich’s Ataxia as well as discuss its neuroscience pipeline.

Susan L. Perlman, MD, is a Clinical Professor in the Department of Neurology at the David Geffen School of Medicine at UCLA, where she is also the Director of Clinical Trials and Director, Ataxia Clinic for UCLA’s Neurogenetics program. She is a member of the Medical Research Advisory Board for the National Ataxia Foundation and has been a primary investigator for several Friedreich’s Ataxia trials.

Jennifer Farmer, MS is the CEO of the Friedreich’s Ataxia Research Alliance (FARA). Since joining FARA in 2006, she has led FARA’s efforts to establish clinical research infrastructure and clinical trial readiness, grown the research grant program, and led efforts to engage bio-pharmaceutical companies in drug discovery and development for Friedreich’s Ataxia.

A live webcast will be available in the Events and Presentations section of Exicure’s website on January 7, 2021 at 09:00 am ET. An archived version will be available on the company website following the event. Additional information can be found here: https://troutaccess.com/index.php/c/ExicureResearchDay2021[2]

About Friedreich’s Ataxia

Friedreich’s Ataxia 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 SCN9A

SCN9A is the gene encoding NaV1.7, a trans-membrane sodium channel, that plays a critical role in pain signal signaling. NaV1.7 is strongly expressed in dorsal root ganglion (DRG) neurons, which mediate transmission of peripheral pain signals to the brain. NaV1.7’s critical role for pain signaling has been biologically validated by human gain-of-function mutations leading to severe pain conditions such as Inherited Erythromelalgia and Small Fiber Neuropathy and human loss-of-function mutations lead to congenital insensitivity to pain. NaV1.7-targeting therapies could provide a novel treatment option for neuropathic pain conditions in which currently available therapies are largely ineffective.

About CLN3 Batten Disease

CLN3 Batten Disease is a monogenic, autosomal recessive, lysosomal storage disorder caused by mutations in the CLN3 gene resulting in battenin protein deficiency. Affected individuals experience childhood blindness, pediatric dementia syndrome, seizures, and early death between age 20 and 30. CLN3 Batten is estimated to affect about 700 individuals in the United States. There are currently no approved therapies for these 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 an SNA–based therapeutic candidate, for the treatment of Friedreich’s ataxia (FA). Exicure’s drug candidate AST-008 is currently in a Phase 1b/2 clinical trial in patients with advanced solid tumors. Exicure is in Chicago, IL and has an office in Cambridge, MA.

For more information, visit Exicure’s website at www.exicuretx.com[3].

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, statements regarding the advancement of the Company’s clinical programs and its expansion into neuroscience; its clinical development of XCUR-FXN including timelines and anticipated data read-outs; and its preclinical programs including a program targeting SCN9A for neuropathic pain and a program targeting CLN3 Batten Disease. 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 it has to date or more severely than anticipated, which may have the effect of impacting or delaying the company’s ongoing Phase 1b/2 clinical trial; 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 results of early clinical trials are not always being predictive of future results; 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 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, 2019, 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.


  1. ^ (www.businesswire.com)
  2. ^ https://troutaccess.com/index.php/c/ExicureResearchDay2021 (cts.businesswire.com)
  3. ^ www.exicuretx.com (cts.businesswire.com)

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