Market Assessment
Akcea Therapeutics (AKCA), a subsidiary of Ionis Pharmaceuticals (IONS), is a commercial-stage small-cap ($1.98B) that was founded in 2014. Akcea's scientific forte is developing innovative therapeutics to treat rare and life-ending hereditary diseases associated with dysregulated lipid metabolism. AKCA has a diverse pipeline with drug candidates at different stages of clinical development.
Its two commercialized products, Tegsedi and Waylivra (formerly volanesorsen), are for the treatment of hereditary transthyretin-mediated amyloidosis and familial chylomicronemia syndrome ((FCS)), respectively. AKCA is also developing therapeutics for cardiometabolic lipid disorders including FCS, familial partial lipodystrophy