Amgen on GAUSS-3: Evolocumab Meets LDL-Based Primary End Points in Statin-Intolerant Patients

February 05, 2016

THOUSAND OAKS, CA — Reflecting shorter-term results in a prior trial, the phase 3 Goal Achievement After Utilizing an Anti-PCSK9 Antibody in Statin Intolerant Subjects-3 (GAUSS-3) trial "met its co–primary end points," Amgen said in a statement aimed at the investment community[1].

It means that one of its highest-profile products, evolocumab (Repatha), already on the market, cut mean LDL-C levels significantly further than treatment with ezetimibe over 24 weeks in patients who had been intolerant of statin therapy due to "muscle-related side effects," which usually means pain or spasms.

"There were no new safety findings," the company said.

As is customary in such announcements, relative outcomes implying a significant difference were provided without specific numbers. GAUSS-3, randomized and with double-blinding, is reported to have enrolled 519 patients.

Evolocumab, a human monoclonal antibody for proprotein convertase subtilisin-kexin type 9 (PCSK9), lowers LDL-C via an entirely different mechanism compared with statins or ezetimibe.

The approvals of evolocumab and a competing PCSK9 inhibitor alirocumab (Praluent, Sanofi/Regeneron) indicated them for LDL-C lowering in conjunction with maximally tolerated statins. Approval in the statin-intolerant population tested in GAUSS-2 and GAUSS-3 would expand the indication to a modestly broader population.

Amgen is sponsoring a large cardiovascular-outcomes trial it hopes will settle the big PCSK9-inhibitor question in the medical community, whether the LDL-C-lowering shown by these drugs—evolocumab in this case—can translate to fewer CV events. Top-line results from such a trial, called FOURIER, which randomized about 27,500 patients with clinical cardiovascular disease, "are anticipated in the second half of 2016," according to the company.


Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.