US FDA Grants Moleculin Orphan Drug Designation of WP1122 to Treat Glioblastoma Multiforme


The US Food and Drug Administration (FDA) has granted Orphan Drug Designation of WP1122 to Moleculin Biotech, Inc., a clinical-stage pharmaceutical company, for the treatment of Glioblastoma Multiforme (GBM).

Walter Klemp, chairman and CEO of Moleculin Biotech, Inc., said the receipt of Orphan Drug Designation is represents an important milestone for their promising WP1122 development program.

He also said they would give the progress of their Phase 1 clinical trial in healthy volunteers, with the string of preclinical data supporting GBM as one of the several potential indications. They also believe that this designation further supports the potential of WP1122, and is another step forward in validating their deep pipeline.

WP1122 was developed as a 2-DG prodrug to provide a more favourable pharmacological profile, and was found to have higher potency than 2-DG alone in the preclinical models where the tumor cells require higher glycolytic activity than the normal cells.

Based on the preclinical data that indicates the potential for WP1122 as a treatment for GBM, the company received Investigational New Drug status and it is evaluating opportunities for partnership in the clinical development.

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The Orphan Drug Designation also provides Moleculin Biotech, Inc., certain benefits, including financial incentives to support the clinical development of the drug in the United States.