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news.The US Food and Drug Administration (FDA) has granted orphan drug designation for US-based biopharmaceutical firm RestorGenex's RES-529, a first-in-class inhibitor of the PI3K/Akt/mTOR pathway, to treat glioblastoma multiforme.
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The company said that signaling components of the PI3K pathway are central regulators of cell proliferation, growth, differentiation, survival and angiogenesis.
About 80% of tumor types have been shown to have an aberrant up-regulation of the PI3K pathway and activation of this pathway has been observed in glioblastoma patients thus making PI3K pathway inhibition a validated target for therapeutic intervention in glioblastoma multiforme.
RestorGenex chief executive officer Stephen Simes said: "We are pleased that the FDA has granted RES-529 orphan drug status to treat glioblastoma multiforme as this disease is a tremendous unmet medical need, and thus far has been challenging for drug development. This represents a significant regulatory milestone for RestorGenex.
"RES-529 is an inhibitor of both TORC1 and TORC2 mechanistically differentiated from other PI3K pathway inhibitors currently in development.
"We have shown activity in both in vitro and in vivo glioblastoma animal models, and we plan to complete necessary work to start a Phase I/II glioblastoma human clinical trial in 2016."
The company has developed RES-529 from a non-steroidal, small molecule drug library through computational design, synthetic and medicinal chemistry.