Sanofi and Regeneron push positive results for PCSK9 inhibitor

Sanofi and Regeneron have announced positive results from trials of novel drug alirocumab, which targets PCSK9, in people with hypercholesterolemia.

The results of the four phase III ODYSSEY trials were showcased at the ESC congress in Barcelona and follow hot on the heels of Amgen’s Biologics Licence Application to the US FDA last week for its rival PCSK9 inhibitor drug evolocumab.

One of the four, the ODYSSEY Long Term trial, is evaluating the long-term safety and efficacy of alirocumab in patients with hypercholesterolemia who are at high or very-high cardiovascular (CV) risk, including patients with heterozygous familial hypercholesterolemia (HeFH). Both study groups are treated with statins and some receive additional lipid-lowering therapies. At 24 weeks, there was a significant 61 per cent reduction from baseline in LDL-C levels in the alirocumab group compared to a 1 per cent increase in the placebo group.

At 52 weeks the trend continued, with a 57 per cent reduction from baseline in LDL-C levels in the alirocumab group compared to a 4 per cent increase in the placebo group.

In a post hoc safety analysis, there was a lower rate of adjudicated major CV events in the alirocumab group compared to placebo (1.4 per cent compared to 3.0 per cent), which points to the CV benefit of alirocumab that will be the focus of regulators at the approval stage.

In the three other trials (ODYSSEY COMBO II, FH I and FH II), alirocumab-treated patients receive an initial dose of alirocumab 75 mg every two weeks, increasing to 150 mg if needed to reach pre-specified LDL-C levels. According to Dr Christopher Cannon, Professor of Medicine at Harvard Medical School, most patients who were started at the 75mg dose achieved their target LDL-C goals on the initial dose.

The four ODYSSEY trials reported, along with results from six other phase III studies, encompass more than 5,000 patients studied in double-blind trials for 24-104 weeks and are ongoing.

Regulatory submissions are anticipated by the end of 2014 in the EU and US, with priority review status likely to be sought in the US.

Alirocumab represents one of Sanofi’s best drug prospects, after several setbacks in recent years. However, it is a crowded arena. Also following Amgen’s evolocumab are Pfizer’s bococizumab, Roche’Genentech’s RG7652 and Lilly’s LY3015014.


Amgen submits BLA to FDA for novel cholesterol drug

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