FDA Grants Orphan Drug Designation to AUM302 for Neuroblastoma

The FDA has granted an orphan drug designation to AUM302 for the treatment of patients with neuroblastoma.

The FDA has granted an orphan drug designation (ODD) to AUM302 for the treatment of patients with neuroblastoma, according to an announcement from AUM Biosciences.1

AUM302 is a potential first-in-class oral kinase inhibitor that targets PI3K, plus the key resistance mechanisms PIM and mTOR.

“The ODD for AUM302 is the second we have received for our products this year, marking a significant milestone for AUM as we advance our diverse pipeline of precision oncology therapeutics designed to reverse cancer resistance,” Vishal Doshi, founder and chief executive officer of AUM, stated in a press release. “AUM302 has the potential to be the first-in-class multi-kinase inhibitor for treatment of neuroblastoma. The FDA decision reinforces the strength of our drug development strategy and clinical trial design to deliver affordable, safe, and effective oncology treatments.”

AUM302 is designed to uniquely combine pan-PIM kinase, pan-PI3K, and mTOR inhibition in a single agent, using a single molecule to inhibit multiple key pathways.

Preclinical studies have shown that the inhibition of PIM/PI3K/mTOR used to complement conventional chemotherapy has the potential to inhibit cancer cell growth, prevent resistance emergence, and improve clinical outcomes in children with high-risk neuroblastoma.

“Despite progress in understanding drug resistance over the last decade, knowledge gaps remain about the underlying biological causes of drug resistance and the design of cancer treatments to overcome it,” Doshi continued. “Our goal is to reverse cancer resistance with a broad portfolio of cost-effective therapies.”

In early studies, AUM302 has shown promise with good tolerability and favorable drug properties. In a study of patient-derived xenografts from those with neuroblastoma, AUM302 was more effective than single PI3K inhibition in vitro, and treatment of these xenografts induced apoptosis, differentiated tumor cells, and decreased N-Myc protein levels.2

Additionally, AUM302 amplified the effects of cisplatin, doxorubicin, and etoposide. Findings showed that the combination of AUM302 and chemotherapy inhibited patient-derived xenograft neuroblastoma growth.

In a mouse model of mice treated with placebo, AUM302 alone, cisplatin alone, or AUM302 plus cisplatin, the combination group featured a subgroup of partial responders, including 2 mice that substantially responded to treatment by displaying slow or no tumor growth and surviving the study period. There was a significant difference in survival between the four study treatment groups (P = .0014).

Notably, the mice treated with the combination were given low-dose cisplatin plus AUM302 at 50% of the maximum tolerated dose to reduce the concentration of toxic cisplatin. Treatment with the combination did not result in any overt adverse effects in terms of general mouse health or body weight.


  1. AUM Biosciences receives FDA orphan drug designation for AUM302 for the treatment of neuroblastoma. News release. AUM Biosciences. November 30, 2022. Accessed December 2, 2022. https://bit.ly/3H0wBlv
  2. Mohlin S, Hansson K, Radke K, et al. Anti-tumor effects of PIM/PI3K/mTOR triple kinase inhibitor IBL-302 in neuroblastoma. EMBO Mol Med. 2019;11(8):e10058. doi:10.15252/emmm.201810058
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