Aegerion Pharmaceuticals, Inc.’s (AEGR - Analyst Report) shares gained 5.7% immediately after the commencement of a phase III study in Japan to evaluate Juxtapid as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in homozygous familial hypercholesterolemia (HoFH) patients. However, shares went down by 4.8% in the next trading session. Overall shares were up 0.6% on the news. Aegerion is currently enrolling patients in this study.
The open-label phase III study is designed to mirror the completed phase III study conducted in the U.S. and other countries. The enrolled patients will be given concomitant lipid-lowering therapies. Following a six-week run-in period, patients will receive Juxtapid starting at 5 mg/day and rising to a maximum of 60 mg/day depending on tolerability for 26 weeks. After 26 weeks, patients will enter a 30-week safety phase.
The percentage changes in LDL-C levels from baseline to week 26 constitute the primary endpoint of the study. Secondary endpoints of the study include percentage change in lipid parameters, long-term safety, and changes in hepatic fat from baseline to week 56.
Juxtapid was granted orphan drug designation in Japan last year. As indicated by the Japanese regulatory authorities, new drug application (NDA) can be filed following completion of the 26-week efficacy period. Aegerion expects to file for approval in mid-2015.
Aegerion’s sole marketed drug Juxtapid for the HoFH indication was launched in Apr 2012 in the U.S. and demand has been rising ever since. Moreover, Aegerion gained EU approval for the drug in Jul 2013 under the brand name Lojuxta. Net Juxtapid revenues in 2013 were $48.5 million.
Juxtapid targets the highly competitive and crowded cardiovascular disease market. We note that Isis Pharmaceuticals Inc./Sanofi’s (ISIS - Analyst Report)/(SNY - Analyst Report) Kynamro is also available in the U.S. HoFH market since the first quarter of 2013.
Aegerion carries a Zacks Rank #3 (Hold). Investors looking for better-ranked stocks in the biopharma sector may consider a company like Shire (SHPG - Analyst Report), which carries a Zacks Rank #2 (Buy)
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