Two Years After Rising from Ashes, Aegerion Preps for Drug Debut

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When Marc Beer joined Cambridge, MA-based Aegerion Pharmaceuticals (NASDAQ:AEGR) as CEO in August 2010, the company was $4 million in debt, nearly insolvent, and “not following a clear path,” he says. By October of that year, Beer, a former Genzyme executive, had worked out a development plan for the Aegerion’s cholesterol-lowering drug and taken the company public, raising $48.8 million.

Since then, Beer and his growing team have been focusing on proving the value of the company’s lead drug, lomitapide, in treating a rare disease called homozygous familial hypercholesterolemia (HoFH). It only affects about 6,000 patients in the U.S. and Europe, but it’s effectively a death sentence: HoFH patients have as much as six times the normal amount of LDL, or “bad” cholesterol, in their blood, putting them at such a high risk for heart attacks most die before age 30. Statins and other cholesterol-lowering drugs don’t really work against the disease, leaving patients with few options.

On Wednesday, Beer told investors during Aegerion’s second-quarter conference call that the drug is on track to win FDA approval by year-end and that the company should be cash-flow positive in 2014. Lomitapide was granted “orphan” status by the FDA, guaranteeing Aegerion seven years of market exclusivity plus other benefits, such as tax credits. (Aegerion, however, isn’t alone in its pursuit of this patient population. Sanofi’s Genzyme unit and Carlsbad, CA-based Isis Pharmaceuticals are seeking FDA clearance of mipomersen as a cholesterol-lowering drug with a different way of working for tough-to-treat populations. They expect a verdict from the FDA in late January.)

Investors are clearly nervous about Aegerion’s near-term challenges: The company’s net loss in the second quarter widened to $13.9 million from $8.6 million in the same quarter last year, and Aegerion’s stock fell 10 percent to $13.37 after the earnings release.

Beer will have some obstacles to overcome if he’s to regain investors’ interest over the coming months. Lomitapide, designed to be taken as a once-daily pill, is the first in a class of drugs called MTP inhibitors. MTP (microsomal triglyceride transfer protein) is responsible for binding lipids and shuttling them around the body. Aegerion’s studies have shown that inhibiting the protein can lower blood levels of LDL in HoFH patients by around 40 percent. “This product has a significant ability to be disease-modifying in a patient population that has seen everything the medical community throws at them not succeed,” Beer says.

Like most companies seeking approval for a drug with a new mechanism of action, however, Aegerion will be facing an advisory panel meeting at the FDA sometime prior to December 29, when the agency is expected to render a verdict on whether to approve lomitapide. During the meeting, a panel of experts will fire questions at the company and then vote on whether or not the FDA should approve the drug. The agency doesn’t have to follow the advice of its advisory panels, but it generally does.

Th advisory panel meeting, which has not yet been scheduled, generated quite a few worries during Aegerion’s analyst call after the second-quarter earnings release. Several analysts pressed Beer and other executives to predict what topics might be discussed during the panel, and to explain how Aegerion was … Next Page »

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