CancerFree KIDS Awards Nationwide Children’s Hospital Monies to Initiate Pediatric Trial

FOR IMMEDIATE RELEASE Covington, KY, June 19, 2018

Sometimes things come full circle. Fourteen years ago, CancerFree KIDS awarded its very first research grant to a young investigator at Cincinnati Children’s Hospital Medical Center (CCHMC) to study a long shot idea that no one else would fund.  This year CancerFree KIDS announced it will fund the first pediatric clinical trial for the drug that resulted from that long shot.

Bexion Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on rare brain tumors, announced today a $50,000 grant awarded by CancerFree KIDS to Nationwide Children’s Hospital in Columbus, Ohio. The monies will be used to initiate a Phase I Pediatric Trial in Solid and Rare Tumors using Bexion’s first in class drug, BXQ-350.

The precursor of the clinical formulation of BXQ-350, called SapC-DOPS, was an accidental discovery at CCHMC by a rare disease researcher, Dr. Xiaoyang Qi. Bexion was founded in 2006 when the Company licensed this technology from CCHMC.

“The very first grant CancerFree KIDS ever awarded was to Dr. Qi in 2004, which happened to be Dr. Qi’s very first grant, as well,” explained Ellen Flannery, Founder and Executive Director of CancerFree KIDS. “We have followed this technology and Bexion since that time, waiting for this pivotal moment of the initiation of a Pediatric Phase I trial. It is rare for a pediatric trial to happen so early in the development of a new treatment, but because of CancerFree KIDS’ involvement from the beginning, Bexion has kept its promise to accelerate pediatric trials. This means we have a chance to get this extremely promising potential new treatment approved for children who need it as quickly as possible, for which we are so grateful.”

Bexion is the Sponsor of the trial to be conducted at several sites, with the first site opening at Nationwide Children’s under the direction of Dr. Tim Cripe, division chief of Hematology/Oncology/Bone Marrow Transplant. The second clinical site will be Cincinnati Children’s Medical Center (CCHMC).

“Before Nationwide Children’s, I was conducting research at CCHMC in collaboration with Dr. Qi including early preclinical studies with SapC-DOPS in Pediatric tumors, particularly Neuroblastoma,” stated Dr. Cripe. “To have the opportunity now to initiate Bexion’s first Pediatric Phase I trial in collaboration with my Nationwide Children’s colleagues Drs. Bhuvana Setty and Mohamed AbdelBaki and with the team at CCHMC is extremely rewarding and exciting.”

“In the Phase Ia portion of our Phase I Safety Study, BXQ-350 illustrated a tolerable safety profile with no significant toxicities at the highest planned dose, supporting continued development. Currently in the expansion portion of our Phase 1 study, BXQ-350 has shown promising clinical activity in heavily pre-treated patients with advanced solid and brain tumors,” stated Dr. Ray Takigiku, Founder and CEO of Bexion.

About BXQ-350

BXQ-350 is a unique formulation of a synthetically produced, human lysosomal protein, Saposin C (sphingolipid activator protein, or SapC), and the phospholipid dioleoylphosphatidylserine (DOPS).

About Bexion Pharmaceuticals

Bexion Pharmaceuticals is a privately-held biotech company focused on the development and commercialization of innovative cures for cancer. Bexion’s first-in-class biologic, BXQ-350, has demonstrated selective tumor targeting with the potential for clinical efficacy in a broad range of cancers.  In 2013 the NCI awarded Bexion a prestigious “Bridge Award” of $3MM to support testing of BXQ-350 in the clinic. In February 2015, the FDA granted Bexion Orphan Drug status for Saposin C, the active ingredient in its proprietary drug BXQ-350, for the potential treatment of glioblastoma multiforme (GBM), a type of brain cancer. In June 2015, Bexion won a Tibbett’s Award by the Small Business Administration for exemplifying the very best in innovation. Currently Bexion is in a Series B fund raise. For more information, visit www.bexionpharma.com

About CancerFree KIDS

The mission of CancerFree KIDS is to eradicate cancer as a life-threatening disease in children by funding promising research that might otherwise go unfunded. CancerFree KIDS was founded by Ellen and Sam Flannery after their baby daughter was diagnosed with cancer in both of her eyes at 5 months of age. Today, thanks to cancer research, that baby is a healthy college student. CancerFree KIDS funds childhood cancer research.

About Nationwide Children’s Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report’s 2017-18 list of “America’s Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric healthcare systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of nearly 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.

About Dr. Tim Cripe

Dr. Cripe is chief of the Division of Hematology/Oncology/BMT at Nationwide Children’s Hospital with an expertise in caring for children with sarcomas. He is also a principal investigator in the Research Institute at Nationwide Children’s Hospital.

About Dr Xiaoyang Qi

Dr. Qi is a Professor of Medicine, Co-Division Chief for the Basic Science Research, and Director of the Translational Medicine Laboratory in the Division of Hematology-Oncology, Department of Internal Medicine, University of Cincinnati College of Medicine. With joint appointments, he is a professor in the Biomedical Engineering Program of the College of Engineering, University of Cincinnati, and in the Division of Human Genetics at Cincinnati Children’s Hospital Medical Center. Dr. Qi is also a director of the Brain Tumor Modeling Program in Gardner Neuroscience Institute, University of Cincinnati College of Medicine.

Media Contact: Margaret van Gilse ●859.757.1652 [email protected]

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