The FDA has approved the request submitted by Moleculin Biotech, Inc. for rare disease designations to be granted their drug candidate WP1066, an agent that appears to directly elicit tumor cell death and rouse the immune system to eliminate tumors.1,2 With this decision, the company can receive a transferrable priority review voucher (PRV) upon new drug approval for each of the following 3 indications: diffuse intrinsic pontine glioma, medulloblastoma, and atypical teratoid rhabdoid tumor.

“The early activity we are seeing with WP1066 is both unexpected and encouraging,” Walter Klemp, chairman and CEO of Moleculin, stated in a press release. “The approval of these 3 rare pediatric disease designations is a reminder of just how important our efforts are to potentially help children with brain tumors.”

The discovery that led to the development of WP1066 was made by Waldemar Priebe, PhD, professor of medicinal chemistry in the Department of Experimental Therapeutics of the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center.2 By examining naturally-occurring compounds in propolis, Priebe was able to examine the ability of caffeic acid benzyl ester (CABE) to inhibit p-STAT3, a key target for anticancer drugs.

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