FDA grants Orphan Drug Designation Status for tasquinimod to treat MM

On 12th April 2017, tasquinimod from Active Biotech AB (Nasdaq Stockholm: ACTI) was granted Orphan Drug Designation status by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma (MM)¹.  

Tasquinimod is a small molecule (second-generation quinoline-3-carboxamide variant) immunomodulatory drug that targets the tumor microenvironment by controlling metastatic and angiogenic function of myeloid derived suppressor cells (MDSCs). Specifically, it binds to the pro-inflammatory protein S100A9 (also known as migration inhibitory factor-related protein 14, MRP14), blocking its interaction with receptors TLR4, RAGE, and CD147². 

Previous development of tasquinimod focused on prostate cancer, with promising outcomes in phase 2 and 3 trials. Recently, pre-clinical studies using MM models have shown promising antitumor effects, including a significant reduction in angiogenesis in the bone marrow.