One of the most heavily researched pathways by pharmaceutical and biotechnology companies in the area of cancer, immune disorders and metabolic diseases is the phosphoinositide 3-kinase (PI3K) pathway. PI3Ks are a family of related enzymes that have been linked to an extraordinarily diverse group of cellular functions and biological processes such as cell growth and proliferation, adhesion, differentiation, survival, and intracellular transport.

Aquinox’s scientific founders discovered a unique biochemical enzyme called SH2-containing inositol 5′-phosphatase (known as SHIP1) and have shown that it regulates the critical PI3K pathway in blood cells. They have also demonstrated that small molecule drugs can regulate SHIP1. SHIP1 is unique in that it exclusively regulates blood cells and can be harnessed in the treatment of disorders such as multiple myeloma, leukemia, lymphoma, inflammation, allergy and autoimmune diseases while having no, or minimal, effect on non-blood tissues. SHIP1 is an ideal drug target allowing for the development of a number of potential drug candidates that interact with it. From this discovery, the company has created two research and development programs: the SHIP1 activator program and the SHIP1 inhibitor program.

The SHIP1 activator program is Aquinox’s most advanced program for blood and immune disorders and extensive studies are being carried out with AQX-1125, a potent activator of SHIP1, and closely related analogs. AQX-1125 possesses many desirable drug properties including its high selectivity, cell permeability and scalable synthesis. It is also a small molecule, is orally bioavailable, and has demonstrated potent anti-inflammatory activity both in vitro and in vivo.

The SHIP1 inhibitor program is Aquinox’s second program for blood cell recovery and is in the early states of lead evaluation.