"These data provide solid support for the therapeutic potential of ANGPTL3 to reduce multiple lipid risk factors in patients with cardiovascular disease and to affect liver fat accumulation in patients with NASH," said Dr. Tsimikas. "Pre-clinical studies in animal models of atherosclerosis and obesity demonstrate that lowering ANGPTL3 levels with antisense oligonucleotides significantly reduces progression of atherosclerosis and liver fat accumulation. The potential of ANGPTL3 reduction to improve cardiovascular risk is also supported by genetic studies in humans with both heterozygous and homozygous loss-of-function mutations in their ANGPTL3 gene showing that individuals with lower or absent plasma levels of ANGPTL3 protein exhibit low plasma levels of triglycerides and LDL cholesterol, which are both risk factors for cardiovascular disease."
Subjects who received multiple doses of 10 mg, 20 mg, 40 mg, or 60 mg of IONIS-ANGPTL3-LRx achieved dose-dependent, statistically significant mean reductions at Day 37 in ANGPTL3 of up to 83 percent (p ≤0.001). These subjects also experienced statistically significant mean reductions in triglycerides of up to 66 percent (p ≤0.001), in LDL-C of up to 35 percent (p ≤0.001), and in total cholesterol of up to 36 percent (p ≤0.001). In this study, IONIS-ANGPTL3-LRx displayed a favorable safety and tolerability profile. There were no discontinuations due to adverse events and no clinically meaningful platelet declines.
"These study results support the potential to develop IONIS-ANGPTL3-LRx to treat multiple diseases, such as severe dyslipidemias and NASH," said
IONIS-ANGPTL3-LRx is a LIgand Conjugated Antisense (LICA) drug designed to reduce the production of the ANGPTL3 protein, a key regulator of plasma lipids, such as triglycerides and LDL-cholesterol. Ionis' LICA technology is designed to enhance the potency of its drugs by increasing efficient drug uptake in target tissues, which provides the potential for improved therapeutic margin, improved tolerability and dosing schedule flexibility.
"In this study, IONIS-ANGPTL3-LRx, demonstrated, consistent with the data we have observed with our other LICA drugs, the ability to robustly lower its intended target at much lower doses than previously used to achieve similar target reduction with our non-LICA drugs," said
ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics is focused on developing and commercializing drugs for patients with serious cardiometabolic diseases caused by lipid disorders. Established as a wholly owned subsidiary of
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 a dozen drugs in mid- to late-stage development. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary, Akcea Therapeutics, to treat patients with either familial chylomicronemia syndrome or familial partial lipodystrophy; IONIS-TTRRx, a drug Ionis is developing with GSK to treat patients with TTR amyloidosis; and SPINRAZA (nusinersen), a drug Ionis is developing with Biogen to treat infants and children with spinal muscular atrophy. Ionis' patents provide strong and extensive protection for its drugs and technology. Additional information about Ionis is available at www.ionispharma.com.
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the development, activity, therapeutic and commercial potential and safety of IONIS-ANGPTL3-LRx. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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