FDA Grants Orphan Drug Designation to AUM302 for Neuroblastoma


The multi-kinase inhibitor AUM302 received orphan drug designation from FDA for the treatment of neuroblastoma.

AUM302, a first-in-class multi-kinase inhibitor targeting PI3K, PIM, and mTOR, has received orphan drug designation from the FDA for patients with neuroblastoma, according to a press release from AUM Biosciences.1

Preclinical findings have determined that treatment with chemotherapy plus PIM, PI3K, and mTOR inhibition could yield better outcomes, inhibit cancer cell growth, and prevent resistance in a pediatric population diagnosed with high-risk neuroblastoma. Early data have also indicated that AUM302 demonstrated positive tolerability and favorable drug properties.

“AUM302 has the potential to be the first-in-class multi-kinase inhibitor for treatment of neuroblastoma,” Vishal Doshi, founder and CEO of AUM Biosciences, said in the release. “The FDA decision reinforces the strength of our drug development strategy and clinical trial design to deliver affordable, safe, and effective oncology treatments.”

Orphan drug designation is granted to drug and biologic candidates created to treat, diagnose, and prevent rare diseases that impact fewer than 200,000 patients. The designation can help to offer certain benefits that support agents' clinical development up to 7 years of market exclusivity following the agent's approval in its intended indication.

AUM302 was designed to combine pan-PIM kinase, pan-PI3K, and mTOR inhibition in 1 oral agent. The multi-targeted strategy was engineered as 1 molecule with the capability of inhibiting multiple key intracellular pathways while also inhibiting growth and resistance.

The agent was found to be effective vs single PI3K inhibition in vitro, according to early research.2 The goal of the study was to examine possible avenues of overcoming PI3K resistance. AUM302 was assessed in 700 cell lines that represented 47 tumor types that were determined to be neuroblastoma.Moreover, in a neuroblastoma patient–driven xenograft model, AUM302 resulted in apoptosis, differentiated tumor cells, and a decrease in N-Myc protein levels. The agent's mechanism also improved the effect of standard cytotoxic chemotherapy agents, including cisplatin, doxorubicin, and etoposide.


  1. AUM Biosciences Receives FDA Orphan Drug Designation for AUM302 for the Treatment of Neuroblastoma. News release. AUM Biosciences. November 30, 2022. Accessed December 5, 2022. https://bit.ly/3UCIhho
  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
Related Videos