Akcea and Ionis Report Top-line Results from the BROADEN Study with WAYLIVRA® (volanesorsen) in Patients with Familial Partial Lipodystrophy
BOSTON, Mass. and CARLSBAD, Calif., Aug. 06, 2019 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today topline results from the BROADEN study in patients with familial partial lipodystrophy (FPL). The study met its primary endpoint demonstrating a statistically significant reduction in triglyceride levels in patients with FPL treated with WAYLIVRA compared to placebo-treated patients. In addition to achieving the primary endpoint, the study achieved an important secondary endpoint of a statistically significant reduction in liver fat.
“We are encouraged by the substantial reduction in triglycerides and hepatic fat fraction achieved with WAYLIVRA in patients with FPL and we will be reviewing the totality of the data internally and with experts in the field to determine the most appropriate next steps for this program,” said Louis O’Dea, chief medical officer at Akcea Therapeutics. “We are grateful to the FPL patient community for their input and participation, and we understand their desperate need for a treatment option.”
“We are pleased by the safety and efficacy profile demonstrated by WAYLIVRA in patients with FPL, adding to the already established profile in patients with FCS,” said Brett P. Monia, Ph.D., chief operating officer of Ionis Pharmaceuticals. “These results represent another important outcome of our antisense platform for patients desperate for new treatment options.”
Akcea and Ionis received marketing authorization in Europe for WAYLIVRA in May 2019 as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. The Companies’ efforts are focused on the commercial launch of WAYLIVRA in the EU and working with regulatory agencies to determine a path forward so that patients with FCS in the U.S. and Canada may have access to this treatment.
BROADEN Study Background and Results:
The most common adverse events observed in WAYLIVRA-treated patients were mild or moderate in severity and included injection site reactions, nasopharyngitis, urinary tract infection and reductions in platelet levels. There were no serious or severe decreases in platelets.
About WAYLIVRA® (volanesorsen) for the treatment of FCS
WAYLIVRA, a product of Ionis’ proprietary antisense technology, is designed to reduce the production of ApoC-III, a protein that regulates plasma triglycerides and may also affect other metabolic parameters.
The European Commission’s marketing authorization of WAYLIVRA is based on results from the Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension (OLE) study and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with WAYLIVRA reduced triglycerides 77% (-94% when compared to placebo). All patients in the trial maintained a low-fat diet.
WAYLIVRA is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. The most frequently observed adverse reactions (more than 10%) during treatment with WAYLIVRA were events associated with injection site reactions and reduction in platelet levels/thrombocytopenia.
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In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics®, TEGSEDI® and WAYLIVRA® are trademarks of Akcea Therapeutics, Inc.
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