AQX-300

AQX-300 is a series of small molecule activators of the Src homology 2 domain-containing inositol 5-phosphatase 1 (SHIP1) enzyme, originally developed by Aquinox Pharmaceuticals. SHIP1 is a lipid phosphatase that acts as a key negative regulator of the PI3K/AKT/mTOR signaling pathway by converting PI(3,4,5)P3 to PI(3,4)P2. In many hematologic malignancies, this pathway is constitutively active, driving uncontrolled cell proliferation and survival. By pharmacologically activating SHIP1, the AQX-300 series aims to reduce pro-survival signaling and induce apoptosis in malignant blood cells. The program was identified in Aquinox's pipeline for the treatment of various heme malignancies but remained in the preclinical stage prior to the company's merger with Neoleukin Therapeutics.