Martina Sersch, M.D., Ph.D., Chief Medical Officer of Mustang, said, "We are very pleased to receive Orphan Drug Designation for MB-108, as it provides Mustang with additional market exclusivity and financial incentives to advance the oncolytic virus. MB-108 has demonstrated the potential to address an area of high unmet medical need, and we believe it is an important new treatment for patients with malignant glioma. Mustang intends to combine MB-108 with MB-101 (IL13Rα2-specific CAR) to potentially enhance efficacy in treating glioblastoma multiforme."
A Phase 1 clinical trial evaluating MB-108 in recurrent glioblastoma multiforme is being conducted at the University of Alabama at Birmingham ("UAB") and is led by James Markert, M.D., Chairman of the Department of Neurosurgery at UAB, who developed MB-108 (C134) in collaboration with Kevin Cassady, M.D., principal investigator in the Center for Childhood Cancer and Blood Diseases at The Research Institute at Nationwide Children's. MB-108 (C134) is a second-generation attenuated herpes simplex virus type 1 (HSV-1) oncolytic virus that has improved replication in tumors in murine models, but with the same toxicity profile as its first-generation predecessors. In these preclinical studies, it not only demonstrates direct anti-tumor activity, but also elicits an immune response that can reverse tumor-associated immunosuppression.
"MB-108 has demonstrated promise in preclinical studies, and we are delighted that it has received Orphan Drug Designation in acknowledgement of its potential to help those living with malignant glioma," said Dr. Cassady, also a physician in Infectious Diseases at Nationwide Children's. "We will continue to learn more about MB-108 in our Phase 1 clinical trial and are excited about developing it in collaboration with UAB and Mustang."
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials and prescription drug user fee waivers. If a product holding Orphan Drug Designation receives the first FDA approval for the disease in which it has such designation, the product is entitled to seven years of market exclusivity, which is independent from intellectual property protection.
About Mustang Bio
Mustang Bio, Inc. ("Mustang") is a clinical-stage biopharmaceutical company focused on translating today's medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.mustangbio.com .
About Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report's 2018-19 list of "Best Children's Hospitals," Nationwide Children's Hospital is one of America's largest not-for-profit freestanding pediatric health care 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 more than 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.
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