Phase III data showing statistically significant reduction of triglycerides will bolster the pivotal data expected early next year. Akcea is not concerned by the study’s 20% dropout rate because there were no discontinuations in a subset of patients with FCS.
Awaiting data from its pivotal APPROACH trial during the first quarter of 2017, Akcea Therapeutics was encouraged by safety and efficacy data unveiled Dec. 19 for lead candidate volanesorsen from a separate Phase III study in patients with severe hypertriglyceridemia, including a subset of patients with familial chylomicronemia syndrome (FCS).
Akcea, a subsidiary of antisense-focused Ionis Pharmaceuticals Inc
The therapy has been developed for GM1 and GM2 gangliosidoses and Niemann-Pick disease type C.
The presentation and publication of the Phase IIIb study showing greater efficacy for Zepbound over Wegovy come at a time when competition has been heating up between the two obesity medications.
InSilico will leapfrog Phase IIb to progress its AI-generated candidate for idiopathic pulmonary fibrosis directly into a Phase III trial in China, as it prepares for an IPO in Hong Kong.
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events and approvals announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
