SKOKIE, Ill.--(BUSINESS WIRE)--Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA™) constructs, announced today that Exicure and its collaborators at The Ohio State University Wexner Medical Center will show preclinical data demonstrating the performance of Exicure’s SNA compound designed for use in spinal muscular atrophy (SMA). These data will be presented at the Cure SMA Annual Conference in Dallas, Texas on June 14, 2018.
“Exicure’s spherical nucleic acid version of nusinersen demonstrates increased survival and decreased toxicity in the translationally-relevant SMA mouse model,” said David Giljohann, PhD, CEO of Exicure. “We believe these results are important for developing improved treatments for patients with SMA. These data also suggest that Exicure’s technology platform could potentially create more potent therapies for other disorders of the central nervous system, including Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.”
At the meeting, Arthur Burghes, PhD, from Ohio State’s Wexner Medical Center, and Exicure will present data from preclinical studies in a SMA mouse model. The poster is titled “Nusinersen in spherical nucleic acid (SNA) format improves efficacy both in vitro in SMA patient fibroblasts and in Δ7 SMA mice and reduces toxicity in mice.” The presentation will highlight that Exicure’s proprietary technology:
- Prolonged survival by four-fold (maximal survival of 115 days compared to 28 days for nusinersen-treated mice);
- Doubled the levels of healthy full-length SMN2 mRNA and protein in SMA patient fibroblasts when compared to nusinersen;
- Doubled the quantity of healthy full-length SMN mRNA levels in spinal cord tissue compared to untreated mice;
- Mitigated toxicity of nusinersen at the highest dose tested in mice.
In August 2017, Exicure and The Ohio State University established a collaboration to further validate and characterize the pharmacology of Exicure’s nusinersen-SNA compound in mouse models. This collaboration’s ongoing in vivo research is conducted by Dr. Burghes, an internationally known researcher, leading basic and clinical research on SMA and other genetic neuromuscular disorders.
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 (SNA™) architecture unlocks the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address oncology, inflammatory diseases and genetic disorders. Exicure is based outside of Chicago, IL. For more information, please visit www.exicuretx.com.
About Spinal Muscular Atrophy (SMA)
SMA is the most common genetic cause of death for infants. SMA results from the loss of the SMN1 gene and an inability of SMN2 to produce sufficient full-length protein to make up for the loss of SMN1. The SMN1 gene, in a healthy person, produces a full-length protein that is essential to the function of the nerves that control muscles. Without sufficient SMN protein, the nerve cells cannot properly function and eventually die. This leads to debilitating and even fatal muscle weakness.
Nusinersen, marketed as Spinraza® by Biogen, is a modified antisense oligonucleotide. In December of 2016, nusinersen was approved by the US FDA for the treatment of SMA in pediatric and adult patients.
Forward Looking Statements
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