Aegerion Shares Soar on New Numbers For Rare Disease Drug

Investors appear to be pretty happy with Aegerion Pharmaceuticals’ (NASDAQ: AEGR) latest results for its rare disease drug, lomitapide (Juxtapid).

Shares of the Cambridge, MA-based biotech jumped about 14 percent, to $88 apiece, in pre-market trading as Aegerion released its second-quarter earnings and showed that sales of lomitapide are tracking ahead of its estimates. Aegerion reported that 215 patients are currently on the drug, which puts it on track to beat its expectations of 250 to 300 patients on lomitapide by the end of the year.

Leerink Swann analyst Joseph Schwartz said in a recent research note that he expected 125 patients on therapy, and that “most investors” thought Aegerion would hike its projections.

Aegerion proved up to the task. On Tuesday, the company raised its projected revenue numbers for 2013 from between $15 million and $25 million to between $30 million and $35 million. Aegerion has $6.5 million in sales from the drug this quarter and $7.7 million from lomitapide so far this year. The FDA approved lomitapide in December. It costs about $295,000 per patient for a year of treatment.

Lomitapide treats homozygous familial hypercholesterolemia, an ultra-rare inherited disease that causes people to have four to ten times the normal amount of LDL, or “bad” cholesterol. This can lead to heart attacks or death at an early age. Isis Pharmaceuticals’ and Sanofi/Genzyme’s mipomersen sodium (Kynamro) was approved by the FDA in January to treat the same condition. Aegerion’s therapy, however, is a once-daily pill—the Isis and Sanofi drug is an injectable treatment.

Aegerion CEO Marc Beer estimated on a conference call Tuesday that there are about 15,000 patients eligible for treatment worldwide, including about 3,000 in the U.S. Aegerion expects European regulators to approve the drug in August and to begin selling the drug overseas on a “limited” basis late in the third quarter, Beer said.

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