Akcea, Ionis Phase II hypertriglyceridemia data hint at improved safety for conjugated antisense therapy

Akcea and Ionis announced plans to develop AKCEA-APOCIII-LRx for familial chylomicronemia syndrome based on Phase II safety and efficacy results from hypertriglyceridemia patients. It gives the companies a second shot at marketing an APOC-III antisense oligonucleotide in the U.S. for FCS.

Akcea Therapeutics Inc. (NASDAQ:AKCA) already markets Waylivra volanesorsen, an unconjugated APOCIII antisense inhibitor co-developed with Ionis Pharmaceuticals Inc. (NASDAQ:IONS), in Europe for FCS. However, the drug received a complete response letter from FDA in August 2018. The companies have not disclosed FDA’s reasoning, but the decision followed a February 2018 disclosure of four cases of grade 4 thrombocytopenia in an open-label extension study (see “Delay for Akcea’s Volanesorsen”)...