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Regeneron's Drug To Treat Genetic Form Of Bad Cholesterol Aces Late-Stage Trial

Shanthi Rexaline

Regeneron Pharmaceuticals Inc (NASDAQ: REGN) has received ample support from multiple catalysts this week.

On Tuesday, the company announced FDA approval for its prefilled Eylea syringe. The good news continued Wednesday with a positive clinical readout.

What Happened

Regeneron said positive top-line results from a pivotal Phase 3 trial that is evaluating evinacumab in patients with homozygous familial hypercholesterolemia, or HoFH - a severe inherited form of high cholesterol.

Evinacumab, which has a Breakthrough Therapy designation from the FDA, is the company's investigational angiopoietin-like 3 antibody.

See Also: Regeneron's Eylea Prefilled Syringe Clears FDA Hurdle

The investigational therapy when administered along with other lipid-lowering theraies to patients, who had an average LDL level of 255 mg/dL despite being on other lipid-lowering therapies, including maximally-tolerated statins, PCSK9 inhibitors, ezetimibe, LDL apheresis and lomitapide, lowered LDL cholesterol by 49%, on average, compared to lipid-lowering therapies alone. This compares to a 2% increase for placebo.

The absolute change in LDL cholesterol from the baseline was 132 mg/dL for evinacumab compared to a 3mg/dL reduction for placebo.

Why It's Important

HoFH patients have limited choices for reducing their LDL cholesterol, and therefore there is a significant unmet need for an effective and safe therapeutic option.

Patients with HoFH are prone to atherosclerotic disease, sometimes suffering cardiac events as early as their teenage years.

"These results raise the potential that evinacumab may have value for other patients with severe, refractory hypercholesterolemia, where we have a trial ongoing," said George Yancopoulos, Chief Scientific Officer of Regeneron.

Regeneron's traded around $304.19 per share at time of publication.

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