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Akcea retains rights for heart drug as Novartis opts out of developing therapy

Akcea Therapeutics Inc. retained the rights to develop and commercialize heart drug Akcea-APOCIII-LRx after Novartis AG declined to exercise its option to do so.

Boston-based Akcea is developing the therapy for multiple diseases including familial chylomicronemia syndrome, or FCS — a disease which prevents the body from breaking down fats — and cardiovascular disease. The drug works by reducing production of a protein called apoC-III — a process which lowers the levels of triglycerides, a type of fat, and lowers the risk of cardiovascular disease.

Ionis Pharmaceuticals Inc.-unit Akcea, entered into an exclusive, worldwide option and collaboration deal with Switzerland's Novartis in January 2017 to develop and commercialize Akcea-APOCIII-LRx, as well as Akcea-APO(a)-LRx for heart indications.

Ionis and Akcea are evaluating Akcea-APOCIII-LRx in a phase 2 clinical trial to treat patients with hypertriglyceridemia and cardiovascular disease. The companies are expected to release topline results from the trial in early 2020.

In a phase 1/2 clinical study, Akcea-APOCIII-LRx showed significant, dose-dependent reductions in apoC-III protein of up to 84% after six weeks of treatment and a decline in triglyceride levels of up to 71%.