DFN-529 is a novel, allosteric P13K/Akt/ mTOR (dual TORC1/TORC2) pathway inhibitor — a key pathway involved in the control of cell-cycle signaling, and directly related to cellular proliferation and cancer.

DFN-529 has been studied in two completed Phase 1 clinical trials in patients with age-related macular degeneration.

The compound has also undergone preclinical evaluation for use in treating glioblastoma multiforme (GBM), the most aggressive primary brain tumor.

DFN-529 has received Orphan Drug Designation from the US Food and Drug Administration for treatment of GBM.

Preclinical Experience

DFN-529 inhibits cell growth leading to death of various cancer cell types grown in vitro. DFN-529 has also shown activity in in vivo GBM animal tumor models: where the PI3K/Akt/mTOR pathway is abnormally activated, DFN-529 reduces tumor growth and its mechanism is synergistic with radiation therapy, chemotherapy, and hormonal therapy through inhibition of the PI3K/Akt/mTOR pathway.

Furthermore, the compound has shown antiangiogenic activity in several animal models, including GBM, and has been demonstrated to be orally bioavailable and capable of crossing the blood-brain barrier to penetrate the brain.

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