On March 11, 2021 Exicure, Inc. (NASDAQ: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) technology,reported to share that researchers at Northwestern University have utilized Exicure’s licensed first generation gold-nanoparticle SNA technology in an investigator-initiated trial for the treatment of glioblastoma (GBM), a deadly form of brain cancer (Press release, Exicure, MAR 11, 2021, View Source [SID1234576542]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
The researchers that led the study, Drs. Priya Kumthekar and Alexander Stegh, conducted a single-arm, open-label phase 0 first-in-human clinical trial (NCT03020017) to determine safety, pharmacokinetics, intratumoral accumulation and gene-suppressive activity of systemically administered SNAs comprised of siRNA specific for the GBM oncogene Bcl2L12. Patients with recurrent GBM were treated with intravenous administration of siBcl2L12-SNAs (NU-0129).
The paper reports first-in-human evidence that siRNA-based SNAs can be administered intravenously, cross the blood-brain-barrier in glioblastoma patients, accumulate in GBM tumor cells, and engage with their target gene.
"We congratulate Drs. Kumthekar and Stegh on these results," said Dr. David Giljohann, the CEO of Exicure. He added, "We are excited about the impact to patients and its implications in treating other diseases, as Exicure continues to build and grow our pipeline in neurological disorders."
Exicure anticipates filing an IND for its lead program in Friedreich’s ataxia by the end of 2021.
About Glioblastoma
Glioblastoma (GBM) is the most common and most aggressive type of primary malignant tumor of the central nervous system. The global incidence of glioblastoma is less than 10 per 100,000 people. Standard treatment for patients with newly diagnosed glioblastoma can include surgery followed by radiation and chemotherapy, but treatment options are limited. The last investigational medicine to improve survival for patients with newly diagnosed glioblastoma was approved by the U.S. Food and Drug Administration in 2005. The five-year survival rate of patients with GBM is less than five percent.
About Friedreich’s Ataxia
Friedreich’s ataxia (FA) 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.