On February 5, 2019 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Status for its drug candidate WP1066 for the treatment of glioblastoma, the most aggressive form of brain tumor (Press release, Moleculin, FEB 5, 2019, View Source [SID1234533053]).
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"We continue to be encouraged by the progress of the physician-led clinical trial of WP1066," commented Walter Klemp, Moleculin’s Chairman and CEO, "and, now having the FDA grant Orphan Drug status for WP1066 positions us well for potential marketing of this drug. We believe that WP1066 represents a new class of drugs which we call ‘Immune/Transduction Modulators’ because it has demonstrated the ability in preclinical testing in animals to both stimulate a natural immune response to tumors and directly attack tumor cells by inhibiting multiple key oncogenic transcription factors, including STAT3, HIF1-α and c-Myc."
Dr. Sandra Silberman, Chief Medical Officer for New Projects at Moleculin added: "The development of WP1066 is gaining momentum. In addition to the glioblastoma trial at MD Anderson, we have had interest from additional investigators, including Emory University and Mayo Clinic for conducting clinical trials for the treatment of pediatric brain tumors, as well as others interested in treating a range of highly resistant tumors including AML and pancreatic cancer. Because we’ve seen strong anti-tumor activity in a wide range of animal models, we believe this represents an important new approach to treating many types of cancer."
The FDA grants orphan drug designation to drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.