Wednesday, September 07, 2022
Moleculin Biotech, Inc., a clinical-stage pharmaceutical company focused on developing drug candidates for highly resistant tumors and viruses, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to WP1122 for the treatment of Glioblastoma Multiforme (GBM).
Walter Klemp, Chairman and CEO of Moleculin, expressed the significance of receiving Orphan Drug Designation for the WP1122 development program. He highlighted the progress of the Phase 1 clinical trial in healthy volunteers, strong preclinical data supporting GBM as a potential indication, and the recent FDA clearance of Investigational New Drug (IND) status for WP1122 in GBM. Klemp believes that this designation further validates the potential of WP1122 and represents a positive step forward for the company's pipeline.
WP1122 is a 2-DG prodrug that was developed to improve its pharmacological profile. It has demonstrated greater potency than 2-DG alone in preclinical models, particularly in tumor cells that exhibit higher glycolytic activity. Moleculin received IND status for WP1122 in GBM based on promising preclinical data and is actively exploring collaboration opportunities for its clinical development.
GBM is an aggressive malignant primary brain tumor with a median survival of only 15 months. It constitutes the majority of malignant primary brain tumors, accounting for 54% of all gliomas and 16% of all primary brain tumors. Despite advancements, survival rates for GBM patients have not significantly improved in the past three decades. The annual age-adjusted incidence rate of GBM in the United States is 3.19 per 100,000 persons.
The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. This designation provides Moleculin with certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug in the U.S. for the designated orphan indication if it receives ultimate approval