Moleculin Biotech Inc. (NASDAQ:MBRX) has received approval from the FDA for the Rare Paediatric Disease designation application for its WP1066 drug candidate. With the approval, the company now has a transferable Priority Review Voucher after the FDA granted New Drug Approval for three indications. The indications include diffuse intrinsic pontine glioma, atypical teratoid rhabdoid tumor, and medulloblastoma.
Moleculin to transfer PRVs to other companies
The company’s CEO Walter Klemp stated that Moleculin sees unanticipated and positive preliminary activity with WP1066. Following WP1066’s approval for three Rare Paediatric Diseases, it is evident how committed the company is to develop a treatment for children with brain cancers. Walter added that investors should be aware of the Priority Review Vouchers’ implications, stating that they are useable after FDA drug approval for rare diseases in children.
He further indicated that after the issue of the PRVs from the FDA, they could transfer them to other drug manufacturers. The PRVs have a history of holding massive value, and in recent times they were sold for more than $100 million. For that reason, the three PRVs are a massive milestone for Moleculin to potentially monetize them.
Moleculin developing treatment for drug-resistant tumors
Moleculin focuses on delivering novel treatments for therapy-resistant types of cancers. These forms of cancers are resistant to treatment because the blood-brain barrier limits drugs from reaching cancers in the brain. Therefore this restricts available treatment alternatives. According to past studies, brain tumor treatments have not had significant enhancement in patients’ overall survival. However, Moleculin’s WP1066 is an immune response modulator to treatment-resistant tumors and aberrant cancer transcription factors, enabling tumor and progression survival. The modulator bolsters the immune system to fight the tumors and reduces cell signaling that supports tumor activity.
WP1066 is a transcription/immune modulator that inhibits P-STA3 and other oncogenic transcription aspects. On the other hand, WP1066 can also stimulate a natural immune response in children to target brain cancers. WP1066’s grant of Rare Paediatric designation will benefit from the FDA’s perks to support drug development.