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The study, Targeting ANGPTL3 with an antisense oligonucleotide in adults with dyslipidemia (TRANSLATE-TIMI 70), will evaluate various doses of vupanorsen to inform potential future development.
In the Phase 2a study, vupanorsen met the primary endpoint of significant reductions in TG levels and multiple secondary endpoints compared to placebo, with a favorable safety and tolerability profile.
'Results from the Phase 2a study recently presented at the
The first patient has been treated in the multicenter, double-blind, placebo-controlled, dose-ranging Phase 2b study. TRANSLATE-TIMI 70 has an estimated total enrollment of 260 participants (40 years old) with elevated non-HDL-C (100 mg/dL) and triglycerides (150-500 mg/dL) who are receiving a stable dose of a statin. The study will explore different doses and dose regimens versus placebo, with patients receiving either 80 mg, 120 mg or 160 mg every 4 weeks, or 60 mg, 80 mg, 120 mg or 160 mg every two weeks via subcutaneous injection. The study (NCT04516291) will assess the efficacy, safety, tolerability and pharmacokinetics of vupanorsen, and the primary endpoint is percent change from baseline in non-HDL-C at week 24.
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ABOUT VUPANORSEN
Vupanorsen is an investigational antisense therapy being developed for potential indications in cardiovascular risk reduction and severe hypertriglyceridemia. Vupanorsen is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. This antisense therapy was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform. The potential therapeutic benefits of ANGPTL3 reduction are supported by the discovery that people with a genetic deficiency in ANGPTL3 have reduced levels of low-density lipoprotein cholesterol (LDL-C) and TG, and a decreased risk of diabetes and cardiovascular disease.1 In a Phase 1 study, subjects treated with vupanorsen achieved robust, dose-dependent reductions in ANGPTL3, TG, LDL-C, non-HDL-C and total cholesterol with a favorable safety and tolerability profile.2 In a Phase 2a study, vupanorsen met the primary endpoint of significant reductions in TG levels and multiple secondary endpoints compared to placebo, with a favorable safety and tolerability profile.
Vupanorsen was discovered by Ionis and has been co-developed by Akcea and Ionis. In
About
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for all patients, children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to potentially treat a broad range of disease, including neurological, cardio-renal, metabolic, infectious, and pulmonary diseases.
ABOUT
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis' business, financial guidance and the therapeutic and commercial potential of vupanorsen (AKCEA-ANGPTL3-LRx) and Ionis' technologies and products in development, including the business of
Contact:
Tel: 760-603-2681
Email: rpatterson@ionisph.com
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