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24 Jan 2014

Amgen Announces Positive Top-Line Results from Phase III GAUSS-2 Trial of Evolocumab (AMG 145) in Statin Intolerant Patients with High Cholestero?l

Amgen has announced that the Phase III GAUSS-2 (Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-2) trial evaluating evolocumab in patients with high cholesterol who cannot tolerate statins met its co-primary endpoints: the percent reduction from baseline in low-density lipoprotein cholesterol (LDL-C) at week 12 and the mean percent reduction from baseline in LDL-C at weeks 10 and 12. The mean percent reductions in LDL-C, or "bad" cholesterol, compared with ezetimibe were consistent with results observed in the Phase II GAUSS study.


Evolocumab is an investigational fully human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver's ability to remove LDL-C from the blood.1


The GAUSS-2 trial evaluated safety, tolerability and efficacy of evolocumab in 307 patients with high cholesterol who could not tolerate effective doses of at least two different statins due to muscle-related side effects. Patients were randomized to one of four treatment groups: subcutaneous evolocumab 140 mg every 2 weeks and oral placebo daily; subcutaneous evolocumab 420 mg monthly and oral placebo daily; subcutaneous placebo every 2 weeks and oral ezetimibe 10 mg daily; or subcutaneous placebo monthly and oral ezetimibe 10 mg daily.


Safety was generally balanced across treatment groups. The most common adverse events (> 5% in evolocumab combined group) were headache (7.8% evolocumab; 8.8% ezetimibe), myalgia (7.8% evolocumab; 17.6% ezetimibe), pain in extremity (6.8% evolocumab; 1.0% ezetimibe), and muscle spasms (6.3% evolocumab; 3.9% ezetimibe).


"We are pleased to continue to see promising results from the Phase III studies in our comprehensive development programme for evolocumab," said Sean E. Harper, MD, executive vice president of Research and Development at Amgen. "The positive GAUSS-2 results suggest that evolocumab may offer a new lipid-lowering treatment to meet an important medical need for high-risk patients who cannot tolerate effective doses of statins."


Data from the Phase III GAUSS-2 trial will be submitted to a future medical conference and for publication.


According to the Centers for Disease Control and Prevention, more than 71 million American adults have high LDL-C.2 Elevated LDL-C is recognized as a major risk factor for cardiovascular disease.3-4 Although statins are effective, it is estimated that 5–15% of patients cannot tolerate statins, primarily due to muscle-related side effects.5



1. Amgen Data on File, Investigator Brochure.
2. Accessed November 2013.
4. World Health Organization. Global status report on noncommunicable diseases 2010. Geneva, 2011.
5. Fernandez G, Spatz ES, Jablecki C, Phillips PS. "Statin myopathy: a common dilemma not reflected in clinical trials." Cleve Clin J Med. 2011;78:393-403.

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