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Amgen Drug for HoFH Meets Primary Endpoint in Phase 3 Trial

MARCH 17, 2014
James Radke

Patients with homozygous familial hypercholesterolemia (HoFH) may have a third treatment option to choose from soon (in addition to Juxtapid and Kynamro).  Today, Amgen announced that the Phase 3 TESLA (Trial Evaluating PCSK9 Antibody in Subjects with LDL Receptor Abnormalities) trial evaluating evolocumab met its primary endpoint of the percent reduction from baseline at week 12 in low-density lipoprotein cholesterol (LDL-C).

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.

The Phase 3 TESLA trial evaluated the safety, tolerability and efficacy of evolocumab (420 mg subcutaneous monthly injections) compared to placebo in 49 adult and adolescent patients with HoFH who were on a stable dose of statin therapy and other lipid-lowering medication.

Details of the efficacy and safety results were not provided but it was noted that:
  1. Primary endpoint was met (percent reduction from baseline at week 12 in LDL-C)
  2. The most common adverse events in the evolocumab group were upper respiratory tract infection, influenza, gastroenteritis and nasopharyngitis.
In the press release, Sean E. Harper, M.D., executive vice president of Research and Development at Amgen said “Homozygous familial hypercholesterolemia is a rare and devastating disease characterized by extremely high LDL-C levels that increase cardiovascular risk in these patients, many of whom are affected at an early age," adding, "We are encouraged by the results of the TESLA trial, the first Phase 3 data of a PCSK9 inhibitor in homozygous familial hypercholesterolemia patients, which suggest evolocumab may offer a new treatment option for these patients who currently have significant unmet medical needs."

No further details of the study results are known at this time but Amgen noted the Phase 3 TESLA trial will be presented at a future medical conference and submitted for publication. 

Homozygous familial hypercholesterolemia (HoFH)

HoFH is a rare genetic lipid disorder resulting in an accumulation of low-density lipoprotein (LDL-C) in the blood. Patients with untreated HoFH have extremely high LDL-C levels, typically between 400 mg/dL and 1,000 mg/dL. These patients are at severely high risk of premature cardiovascular events, such as heart attack or stroke.

There are currently two orphan approved drugs for HoFH -  Juxtapid (once daily tablet) and Kynamro (weekly subcutaneous injection).  Evolocumab (monthly subcutaneous injection) is not listed with the FDA as an orphan designated drug.

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