Agastiya Biotech, a US-based new drug development company delivering innovative pharmaceutical products to the marketplace, and Vopec Pharmaceuticals, an India-based company that is manufacturing Ayurveda and siddha formulations, announced on Tuesday that the US FDA has granted Orphan Drug Designation to AB001, the company's novel small molecule, to treat Pancreatic Cancer and acute myeloid leukaemia (AML).

The product is a small molecule PD-L1 checkpoint inhibitor that also targets aberrant intracellular signalling pathways in the tumour microenvironment, such as Wnt/beta-catenin, PI3K, and RAS. This dual mechanism has been shown in the animal model to work across multiple cancer types, including solid and liquid tumours and, also, on non-PDL1+ cancers. The research offers potential proof for the drug-ability of the so far undruggable beta-catenin and clear advantages of AB001 over Gemcitabine which is the standard of care for pancreatic cancer. Furthermore, AB001 exerts anti-leukaemia activity in preclinical models of acute lymphoblastic leukaemia, likely through inhibiting VEGFR2-mediated signalling pathways, and suggests potential benefits and clinical application of AB001 in treating Leukaemia patients.