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

Rhea-AI Summary

Exicure, Inc. (NASDAQ: XCUR) will host a virtual R&D Day on July 15, 2021, highlighting its neuroscience pipeline, particularly XCUR-FXN for Friedreich's Ataxia (FA). This program targets the disease's molecular cause and is on track for IND filing in late Q4 2021. The event will also cover advancements in therapies for SCN9A-related neuropathic pain and CLN3 Batten Disease. CEO Dr. David Giljohann emphasizes the potential of their SNA platform to address neurological disorders with promising data that may benefit patients.

Positive

• XCUR-FXN program is advancing towards IND filing in Q4 2021.
• New preclinical data will be presented, showing progress in XCUR-FXN development.
• Expansion into rare neurological diseases with high unmet need.

Negative

• None.

Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory drugs utilizing spherical nucleic acid (SNA™) technology, will host a virtual R&D Day on Thursday, July 15, 2021 from 10:00 am to 11:00 am ET. The event will showcase Exicure’s neuroscience pipeline, including its lead program for Friedreich’s Ataxia (FA), XCUR-FXN, which is designed to address the underlying molecular cause of FA.

Exicure will present new and previously unreleased preclinical data and discuss progress with XCUR-FXN, which is on track for IND filing in late Q4 2021. Additionally, Exicure will discuss its perspective on XCUR-FXN’s competitive differentiation in FA and the program’s path to clinical validation.

Also, Exicure will provide an update on its expanding pipeline across several rare neurodegenerative diseases of high unmet need and highlight progress with two preclinical programs targeting SCN9A (Na_v1.7) for neuropathic pain and CLN3 for Batten Disease.

“We believe that our SNA platform is ideally suited to address neurological disorders based on the demonstrated ability of SNAs in nonclinical studies to achieve higher cellular uptake in all key cell types and broad biodistribution in the CNS, including deep brain regions,” says Dr. David Giljohann, Exicure’s CEO. “We are excited to translate our platform’s capabilities to potential benefits for patients as demonstrated by our bi-specific SNA, XCUR-FXN for Friedreich’s Ataxia, anticipated to enter the clinic in the first half of 2022.”

A live webcast will be available in the Events and Presentations section of Exicure’s website on July 15, 2021 at 10:00 am ET. An archived version will be available on the company website following the event. Additional information can be found here: https://event.on24.com/wcc/r/3305687/FA6C43097DA849A64659B1FB63A8C259

About Friedreich’s Ataxia

Friedreich’s Ataxia is the most commonly inherited ataxia, a degenerative neuromuscular disease leading to progressive loss of coordination and 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 Na_v1.7, a trans-membrane sodium channel, that plays a critical role in pain signal signaling. Na_v1.7 is strongly expressed in dorsal root ganglion (DRG) neurons, which mediate transmission of peripheral pain signals to the brain. Na_v1.7’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. Na_v1.7-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 batten 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 greater than 1,800 individuals in the United States and Europe. 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 cavrotolimod (AST-008) is currently in a Phase 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.

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 developmental and regulatory submission timelines and anticipated data read-outs; and the timing and success of 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, 2020, 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.

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FAQ

What is the focus of Exicure's upcoming virtual R&D Day?

The virtual R&D Day on July 15, 2021, will focus on Exicure's neuroscience pipeline, particularly the XCUR-FXN program for Friedreich's Ataxia.

When will Exicure file for IND for XCUR-FXN?

Exicure plans to file for IND for XCUR-FXN in late Q4 2021.

What diseases is Exicure targeting in its expanding pipeline?

Exicure is targeting rare neurodegenerative diseases, including Friedreich's Ataxia, neuropathic pain associated with SCN9A, and CLN3 Batten Disease.

What is Friedreich's Ataxia and its impact?

Friedreich's Ataxia is a degenerative neuromuscular disease caused by FXN gene mutations, affecting approximately 13,500 people across the US, Europe, Canada, and Australia.

How does the SNA technology benefit neurological disorders?

Exicure's SNA platform shows high cellular uptake and broad biodistribution in the CNS, potentially providing effective treatments for neurological disorders.