The mTOR / PI3K pathway plays a major role in multiple critical cellular processes. Aberrant mTOR pathway activation is associated with a broad range of diseases and frequently driven by mutations in mTOR or upstream mTOR pathway genes (e.g., TSC2 and PTEN).
Currently approved mTOR inhibitors do not address a significant proportion of mTOR-driven diseases, nor do they target specific biomarker driven populations. Furthermore, we have observed in preclinical models that current mTOR inhibitors have significant limitations (e.g., poor PK/PD and incomplete target suppression) that result in inferior outcomes vs. nab-sirolimus. Our mission is to realize the full potential of mTOR inhibition by addressing the limitations of existing mTOR inhibitors and targeting biomarker driven populations with known mTOR pathway activation.