Akcea Therapeutics provides more pivotal data on rare disease treatment

Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals (Nasdaq: IONS), has presented data from a pivotal study into its rare disease therapy candidate volanesorsen.

The company’s share price fell last month when initial findings from the study raised concerns over the safety profile of the novel biologic.

Akcea says the new data confirms positive results in patients with FCS, a rare genetic disease characterized by the build-up of chylomicrons, large particles that transport dietary fat and cholesterol in the body.