Press Releases
Akcea Initiates AKCEA-ANGPTL3-LRx Phase 2 Program in Patients with Rare Hyperlipidemias
CAMBRIDGE, Mass.,
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AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like 3, or ANGPTL3. The absence of ANGPTL3 has been shown to be cardioprotective and associated with reduced risk of insulin resistance and diabetes mellitus.
The AKCEA-ANGPTL3-LRx Phase 2 program is designed to include three clinical studies in patients with one of three rare hyperlipidemias, including familial chylomicronemia syndrome (FCS), familial partial lipodystrophy (FPL), and homozygous familial hypercholesterolemia (HoFH). The objectives of these studies are to determine the pharmacodynamics, pharmacokinetics, safety and tolerability of AKCEA-ANGPTL3-LRx, and to further characterize the drug’s potential in these patient populations.
“ANGPTL3 is a key regulator of a number of lipid and metabolic pathways and as such is an important target for the therapeutic management of rare hyperlipidemias, such as FCS, FPL and HoFH. In FCS, severe elevation of triglycerides results in a high risk of acute and chronic pancreatitis and other metabolic complications. Patients with HoFH are subject to early cardiovascular disease and premature death due to high LDL cholesterol levels. Patients with FPL often have both high triglycerides and high LDL cholesterol levels as well as other complications related to insulin resistance and fatty liver, which can lead to accelerated atherogenesis and liver failure,” said Louis O’Dea, chief medical officer at Akcea Therapeutics. “We are committed to developing additional therapeutic options for these patient populations whose significant medical needs are currently greatly underserved.”
Consistent with Akcea’s commitment to the FCS community, the first study is designed to evaluate the pharmacodynamics, pharmacokinetics, safety and tolerability of AKCEA-ANGPTL3-LRx in patients with FCS. Study participants will receive AKCEA-ANGPTL3-LRx by subcutaneous injection once weekly for 13 weeks. Akcea plans to report top-line data from this study in 2018. Akcea plans to initiate similar pilot studies shortly in patients with FPL and patients with HoFH. For further study information, please visit www.clinicaltrials.gov and search for AKCEA-ANGPTL3-LRx.
ABOUT AKCEA-ANGPTL3-LRx
AKCEA-ANGPTL3-LRx is a ligand conjugated antisense (LICA) drug designed to reduce angiopoietin-like 3 protein, or ANGPTL3. ANGPTL3 is a key regulator of triglycerides, cholesterol, glucose and energy metabolism. People with lower levels of ANGPTL3 have lower LDL-C and triglyceride levels and lower risk of heart attacks. Ionis and Akcea are developing AKCEA-ANGPTL3-LRx to treat multiple lipid disorders including rare hyperlipidemias and NAFLD with metabolic complications.
In a Phase 1/2 clinical study in volunteers with elevated triglycerides, published in
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, an affiliate of
ABOUT IONIS PHARMACEUTICALS, INC.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Biogen is responsible for commercializing SPINRAZA. Drugs that have successfully completed Phase 3 studies include inotersen, an antisense drug Ionis is developing to treat patients with hereditary TTR amyloidosis (hATTR), and volanesorsen, an antisense drug discovered by Ionis and co-developed by Ionis and Akcea Therapeutics to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy. Akcea, an affiliate of Ionis, is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. If approved, volanesorsen will be commercialized through Ionis' affiliate, Akcea. Inotersen filings for marketing approval have been submitted in the U.S. and EU. Volanesorsen filings for marketing approval have been submitted in the U.S., EU, and Canada. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
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IONIS FORWARD-LOOKING STATEMENT
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Media and Investor Contact:
Kathleen Gallagher
Head of Communications and Investor Relations, Akcea Therapeutics
(617)-207-8509
kgallagher@akceatx.com
Source: Akcea Therapeutics, Inc.
Source: Ionis Pharmaceuticals, Inc.