Ionis Pharmaceuticals (IONS)

Company Description

Ionis Pharmaceuticals is leading antisense drug discovery and development company with a large pipeline encompassing multiple therapeutics with potential value over a wide-range of disease areas.  The company currently has one marketed product, Kynamro, which is a treatment in familial hypercholesterolemia (HoFH); and the drug is under Phase III study in severe heterozygous familial hypercholesterolemia (HeFH).  Top-line pivotal study result releases of ISIS’s leading pipeline products, ISIS-APOCIIIRx in familial chylomicronemia syndrome (FCS) and severely high triglycerides (>880 mg/dL); ISIS-SMNRx in infant and children SMA patients; and ISIS-TTRRx in familial amyloid polyneuropathy (FAP), could start in 2016 and 2017 with possible approval and product launch in 2017 and beyond.  ISIS has one of the richest pipelines in the industry, with a total of more than 32 clinical programs in development at different clinical stages.