Moleculin Awarded New Rare Pediatric Disease Designation

 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPD) to its p-STAT3 inhibitor, WP1066, for the treatment of ependymoma.

Ependymoma is a rare type of tumor that can form in the brain or spinal cord. Ependymoma begins in the ependymal cells in the brain and spinal cord that line the passageways where the fluid (cerebrospinal fluid) that nourishes your brain flows. Ependymoma can occur at any age, but most often occurs in young children.

"The FDA's recognition of the high prevalence and unmet needs in the treatment landscape for ependymoma, especially in pediatric patients is a significant milestone as we continue to advance and expand the WP1066 development program. We currently have Orphan Drug Designation for WP1066 for the treatment of brain tumors, as well as RPD designation for three other pediatric indications, and believe that ependymoma represents another important rare indication. We continue to be encouraged by the data WP1066 has demonstrated to date and believe it has the potential to be an effective therapy for pediatric patients with ependymoma," commented Walter Klemp, Chairman and CEO of Moleculin.

https://finance.yahoo.com/news/moleculin-awarded-rare-pediatric-disease-113500720.html