Amgen's ($AMGN) in-development cardio drug came through in two more late-stage trials, significantly lowering bad cholesterol in patients with genetic disorders that put them at serious risk.
In one Phase III trial, Amgen's evolocumab (formerly AMG 145) reduced LDL cholesterol 31% from baseline levels at 12 weeks in patients with homozygous familial hypercholesterolemia, a rare disorder characterized by dangerously high blood plaque. Separately, in a Phase II/III study on 5 patients with the rare and serious gene mutation familial hypercholesterolemia, the drug lowered mean LDL-C by 67% over 12 weeks.
The latest studies, dubbed TESLA and TAUSSIG, are part of Amgen's sweeping late-stage program for evolocumab, which includes 20 Phase III trials. The drug, which blocks the protein PCSK9 to help the body clear LDL cholesterol from the blood, is part of a new class of statin-beating injectables that promise to bring in more than $10 billion at their peak, and Amgen is expected to file for FDA approval this year, giving it a lead on similar offerings from rival tandem Sanofi ($SNY) and Regeneron ($REGN) and third-place Pfizer ($PFE).
Evolocumab had already demonstrated its efficacy in patients with milder levels of elevated cholesterol, and the drug's strong showing among those with genetic disorders only further affirms its promise, lead investigator Frederick Raal said.
"These results are especially exciting as it's the first time we've seen Phase III data for a PCSK9 inhibitor in patients with homozygous familial hypercholesterolemia," Raal said in a statement. "These patients are the most difficult to treat as many of them have fewer functioning LDL receptors than patients studied in previously reported Phase III trials with evolocumab."
And, importantly, missing from the list of adverse events in each study was neurocognitive impairment. Earlier this year, after Sanofi revealed that the FDA asked it to keep tabs on "fuzzy thinking" side effects in its trials, investors worried that the agency would take a dim view of PCSK9 blockers' safety when review time came along.
- read the results
Special Report: Tomorrow's cardio blockbusters: Inside 'the next big leap' in controlling cholesterol