Moleculin Biotech Inc. (NASDAQ:MBRX) has announced that the FDA has approved its Rare Paediatric Disease designation request for the WP1066 drug candidate. The designation entitles the company to receive transferable Priority Review Voucher upon New Drug Approval for medulloblastoma, diffuse intrinsic pontie glioma, and atypical teratoid rhabdoid tumor.
WP1066 approved from three indications
Walter Klemp, the company’s CEO, said that they are witnessing unexpected and encouraging early activity with WP1066. The approval of WP1066 for three Rare Paediatric Diseases is a reminder of how important the company’s efforts are to help children with brain tumors. Klemp added that it is important for investors to understand Priority Review Vouchers implications. He said that the PRVs are issued after drug approval for rare diseases indication from the FDA.
Klemp added that once the FDA has issued the PRVs are transferable to other drug developers. Historically the PRVs have held significant values and most recently were sold for over $100 million. Therefore, For Moleculin to have these three PRVs is a massive achievement for the company.
WP1066 inspired by bee pollen ingredient
The company’s focus is on innovative therapies for high resistant cancers. Usually, the blood-brain barrier can limit cancer drugs from reaching brain tumors this limiting most treatment alternatives. Previous research in brain tumor drugs has not shown significant improvement in overall survival. An active ingredient present in bee pollen inspired Moleculin’s WP1066, which modulates the immune response to tumors as well as aberrant oncogenic transcription factors activity to enable progression and tumor survival. The patient’s immune systems will then fight tumors and, at the same time, reduce cell signaling supporting tumor activity.
As an Immune/Transcription Modulator, WP1066 can inhibit P-STAT3 as well as other oncogenic transcription factors. WP1066 can, at the same time, stimulate a natural immune response targeting brain tumors in children. For Moleculin, the Rare Paediatric Disease designation for WP1066 is a massive milestone. As Moleculin continues to develop WP1066 for the three indications, it will benefit from an array of benefits offered by the regulatory agency.