RARE Daily

FDA Grants Rare Pediatric Disease Designation to AUM Biosciences’ Treatment for Neuroblastoma

January 5, 2023

The U.S. Food and Drug Administration has granted a Rare Pediatric Disease Designation for AUM Biosciences’ experimental candidate, AUM302, a potential first-in-class oral kinase inhibitor that targets not only PI3K, but also key resistance mechanisms such as PIM and mTOR, for the treatment of neuroblastoma.

Neuroblastoma is the most common extracranial solid tumor in childhood. More than 650 cases are diagnosed each year in the United States. The prevalence is about 1 case per 7,000 live births. The incidence is 10.54 cases per 1 million per year in children younger than 15 years. About 37 percent of patients are diagnosed as infants, and 90 percent are younger than 5 years at diagnosis, with a median age at diagnosis of 19 months.

AUM302 is a potential first-in-class oral kinase inhibitor rationally designed to uniquely combine pan-PIM kinase, pan-PI3K and mTOR inhibition in a single agent. This multi-targeting approach has been engineered into a single molecule that has the potential to inhibit multiple key intracellular pathways, increasing its ability to inhibit cancer cell growth and prevent resistance emergence. In early studies to date, AUM302 has shown promise with good tolerability and favorable drug properties.

“Neuroblastoma is the third most common pediatric cancer, and more than 90 percent of children diagnosed are under five years old. The RPD highlights the urgency for treatment of this devastating disease where AUM302 could potentially emerge as a breakthrough therapy for children,” said Vishal Doshi, chairman and CEO of AUM Biosciences. “This timely addition to our recent orphan drug designation granted by the FDA reinforces our commitment to developing therapeutic candidates that are accessible, safe, effective, and affordable for patients.”

The company expect to file an application with the FDA to begin human clinical trials in mid-2023.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect individuals from birth to 18 years old and fewer than 200,000 persons in the United States. If a New Drug Application for AUM302 is approved, AUM Biosciences is potentially eligible to receive a rare pediatric disease priority review voucher, which is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. This voucher can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application. Most recently, Bluebird Bio in November sold its priority review voucher to Argenx for $102 million.

Author: Rare Daily Staff

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