In pre-clinical studies, BXQ-350 targets and induces cell death preferentially in cancer cells

Business Wire – In pre-clinical studies, BXQ-350 targets and induces cell death preferentially in cancer cells. BXQ-350 targets an ubiquitous surface marker on tumor cells, and while representing an entirely new and novel approach, capitalizes on proven mechanisms for killing tumors.

Technology Overview

Bexion is developing a new biologic with a novel mechanism of action for targeting and eliminating tumors, including glioblastoma multiforme. The SapC-DOPS proteolipid nanovesicles (BXQ-350) specifically target and kill tumor cells in orthotopic and  xenograft models of cancer,  having a high affinity for aberrant phosphatidylserine (PS)-rich membrane domains that occur on the surfaces of tumor cells and cells of tumor neovasculature, but not normal cells.  The prolongation of life of tumor-bearing mice following BXQ-350 treatment was dramatic and statistically significant.

Orphan Drug Designation

The FDA has granted Bexion Orphan Drug status for Saposin C, the active ingredient in its proprietary formulation BXQ-350 for the potential treatment of glioblastoma multiforme (GBM).

Awards from the National Cancer Institute (NCI) and Small Business Administration (SBA)

In 2015, Bexion was awarded a “Tibbetts Award” from the SBA for representing the very best in developing innovative technologies that address national priorities.  In 2013, Bexion was the only therapeutic recipient of the prestigious “Bridge Award” of nearly $3MM to support testing of BXQ-350 in the clinic.  Since 2009, Bexion has been granted over $5MM in NCI awards for advancement in prostate, pancreatic and glioblastoma cancers.

Competitive Advantage

Multiple lines of evidence suggest that PS is potentially a universal tumor target, to which BXQ-350 is targeted. Alternative PS-targeted therapeutics have been proposed but none have the ability to bypass the blood brain barrier (BBB).  Current radio- and chemotherapies have low therapeutic indices, incur severe side effects, and in the case of brain tumors, also display neurotoxicity. BXQ-350 is expected to avoid the most debilitating side effects because its mechanism of action is significantly different. The efficient targeting of brain tumors by BXQ-350 was confirmed in a recent MRI study in which brain tumors were visualized using paramagnetic Gd-DTPA-BSA/SapC-DOPS vesicles.

Clinical Trials 

In July 2016 the FDA cleared Bexion to initiate Phase I Clinical Trials.  Patients are being dosed at the University of Kentucky, University of Cincinnati, Ohio State University, and the University of New Mexico.


Bexion Pharmaceuticals is seeking strategic partnerships for expanded development of therapeutic areas and eventual commercialization.

Unmet Medical Need & Market Potential

Cancer is one of the top three leading causes of death in the world today. According to the American Cancer Society, in the United States alone, an estimated 1.66 million people would be diagnosed with cancer in 2015 and approximately 590,000 will die of the disease. The incidence of malignant glioma (brain cancers) has been estimated at 20,000 cases per year. Glioblastoma multiforme (GBM), also known as Grade 4 glioma, has one of the worst prognosis of any cancer, with an average survival time of 12 months. The five-year survival rate of GBM is less than 3%, and has remained unchanged over the past 30 years.

In addition to GBM, treatment with BXQ-350 is being evaluated in other rare solid tumors as well, which may also qualify for orphan drug exclusivity.

According to the National Cancer Institute, in 2017, approximately 15,270 children and adolescents ages 0-19 years of age will be diagnosed with cancer and 1,790 will die of the disease in the United States. Clinical studies for pediatric patients are being planned by Bexion in 2018.

Intellectual Property

Bexion has exclusive licenses obtained from CCHMC, and a portfolio of patents and patent applications.

Selected Publications

Wojton J, et al., Systemic Delivery of SapC-DOPS Has Antiangiogenic and Antitumor Effects Against Glioblastoma. Mol Ther. 2013 Aug;21(8):1517-25.

Chu Z, et al., Targeting and Cytotoxicity of SapC-DOPS Nanovesicles in Pancreatic Cancer. PLoS One. 2013 Oct 4;8(10):e75507. doi: 10.1371/journal.pone.0075507.

Qi X et al., Cancer-selective targeting and cytotoxicity by liposomal-coupled lysosomal saposin C protein. Clin Cancer Res. 2009 Sep 15;15(18):5840-51

Management Team:

  • Ray Takigiku, PhD – President and CEO. At Procter & Gamble Pharmaceuticals, Dr. Takigiku was part of the leadership team that developed and marketed the blockbuster osteoporosis drug Actonel, and the market leader for ulcerative colitis, Asacol. He was also interim Co-Director of the Genome Research Institute (now UC Metabolic Disease Institute), University of Cincinnati, College of Medicine where he led development of an academic center for drug discovery.
  • Mike Gazda, PhD – VP, CMC and Development
  • Sandra Nelson, PhD- VP, CMC and Research
  • Christy Rothwell, PhD, JD – VP, Legal and Intellectual Property
  • Margaret van Gilse, MBA – VP, Business Development
  • Tom Wei, PhD – VP, CMC

Board of Directors:

  • Charles R. Scheper – Chairman. Mr. Chuck Scheper was recent Co-Chair of the Cancer Support Community.
  • William P. Butler Mr. Butler is founder and Chairman of the Corporex Companies.
  • Lester M. Crawford, DVM, PhD Dr. Crawford spent over ten years of his career at the Food and Drug Administration (FDA) serving in a variety of pivotal roles, culminating in his appointment as FDA Commissioner.
  • Cameron Durrant, MD, MBA Dr. Durrant is Founder and CEO, PediatRx Inc. and Executive Chairman of Anavex Life Sciences Corp and Pressure Point Inc.
  • Timothy Schroeder CEO and Founder of CTI, a multi-national clinical trial and consulting research firm

This document has been compiled for informational use only. Bexion makes no representation or warranty as to the accuracy or completeness of the information provided.

2018-01-23T15:37:49+00:00 September 6th, 2017|