After FDA Accepts Lomitapide NDA, Aegerion Seeks $47.3M

By Marie Powers
Staff Writer

A month after the FDA accepted its new drug application (NDA) for lead compound lomitapide as an adjunct to a low-fat diet and other lipid-lowering therapies to reduce cholesterol in patients with homozygous familial hypercholesteremia (HoFH) and familial chylomicronemia (FC), Aegerion Pharmaceuticals Inc. priced an underwritten public offering of 3.4 million shares of common stock at $14.75 each, seeking to raise approximately $47.3 million.

The Cambridge, Mass.-based company granted the underwriters a 30-day option to purchase up to 510,000 additional shares, potentially generating an additional $7.5 million.

Jefferies & Co. Inc. and J.P. Morgan Securities LLC are acting as joint book-running managers in the offering, with Leerink Swann LLC, Canaccord Genuity Inc. and Needham & Co. LLC acting as co-managers.

The offering, expected to close June 19, priced at a 10.8 percent discount to Wednesday's closing price of $16.53, and shares (NASDAQ:AEGR) quickly dropped to the offering price. The stock lost $1.81 to close at $14.72, with volume topping 2 million shares for the first time since June 24, 2011, when Aegerion filed to raise $50.4 million in a previous public offering. (See BioWorld Today, June 27, 2011.)

The offering represents Aegerion's second follow-on since the company went public in 2010. (See BioWorld Today, Aug. 12, 2010.)

"If you look at our 15-day average, our 30-day average and our 60-day average, I think it was a very appropriate price," explained CEO Marc Beer, noting that the stock's recent uptick came on low volume. Shares traded at a 30-day average of $14.78 at the time of the pricing.

"You have to look through the lens of the investor when you price," Beer told BioWorld Today.

Moreover, pricing was only one factor in the strategy behind the financing, designed to give Aegerion sufficient runway to prepare for launch activities in the U.S. and Europe. More important to the company was the fact that both existing and new investors – 60 institutions – supported the offering.

"We had broad support and did not need any retail," Beer said, adding that the top 10 to 15 are long-play investors.

Aegerion expects to apply the capital quickly. The company is optimistic about a single-cycle approval for lomitapide, a small-molecule microsomal triglyceride transfer protein inhibitor being developed as an oral, once-daily treatment. The compound is designed to work by preventing the liver and intestines from secreting lipids into the bloodstream, thereby reducing those levels.

Lomitapide was granted orphan drug designation by the FDA in HoFH and FC and by the European Medicines Agency (EMA) in FC.

Beer expects lomitapide to go before an FDA advisory committee panel prior to its Dec. 29 PDUFA date, so the company needs to prep for the panel in addition to laying the groundwork to hit the market as early as January 2013.

FDA approval would set a precedent that could enable the company to expand lomitapide into additional countries outside the U.S. and Europe on a named patient basis – perhaps beginning with Brazil, where the company is already communicating with patients and health care providers.

"This capital will allow us to do that in a more robust way," Beer said.

Launching in an ultra-orphan indication gives Aegerion the luxury of targeting lomitapide to key decision-makers. The company plans to hire only 15 clinical specialists in the U.S. – half of whom are already in place, Beer said. The company expects to bring the remaining sales force onboard in the fall, following the AdComm meeting.

The EMA accepted the company's marketing authorization application for lomitapide in March, and Beer said the agency's 80-day letter contained no questions the company cannot address "in a timely way."

Aegerion expects to meet with the EMA in the first quarter of 2013, setting up potential approval in the EU during the second quarter and a launch before mid-year 2013, Beer said.

The company already has country managers on the ground in the five key EU states, with additional hires planned next year as the marketing effort expands.

"We have about 55 employees today, and we'll exit 2012 around 100," Beer said.

Over two years, Aegerion has moved lomitapide from the initiation of Phase III studies to an NDA filing, and Beer said the company has every reason to believe it will maintain that pace – especially since Aegerion is out front in the HoFH space. Last month, Sanofi SA unit Genzyme filed an NDA for Kynamro (mipomersen sodium), a first-in-class apolipoprotein B synthesis inhibitor, in HoFH. The drug was developed by Isis Pharmaceuticals Inc., of Carlsbad, Calif., which partnered with Cambridge, Mass.-based Genzyme in early 2008. (See BioWorld Today, Jan. 9, 2008, and May 30, 2012.)

What keeps Beer awake, he said, are the testimonies of HoFH patients seeking treatment, like a young man "with 70-year-old arteries in a 19-year-old" body.

"We're not in a competitive battle at all," Beer said, "but the patients are waiting."

In other financings news:

• Allon Therapeutics Inc., of Vancouver, British Columbia, said shareholders approved the terms of the $10 million brokered private placement equity offering disclosed May 2. The approval triggers the conversion of the subscription receipts sold as part of the financing, making the full $10 million raised available to the company for development of lead product davunetide. Allon is testing davunetide in a pivotal trial in orphan indication progressive supranuclear palsy under a special protocol assessment with the FDA. Top-line data are expected late this year. (See BioWorld Today, Jan. 5, 2011.)

• Novelos Therapeutics Inc., of Madison, Wisc., said it closed a public offering of about 5.4 million units priced at $1 each for gross proceeds of $5.4 million. Each unit consisted of one share of common stock, a Class A warrant with a five-year term to purchase one-half of one share of common stock at an exercise price of $1.25 each and a Class B warrant with a 90-day term to purchase one share of common stock at an exercise price of $1 each. Proceeds are expected to fund R&D activities and general corporate purposes. Rodman & Renshaw LLC acted as exclusive placement agent. Separately, Novelos reported that a second amended complaint in a putative federal securities fraud class-action suit brought in the District Court for the District of Massachusetts, originally in March 2010, was dismissed without prejudice. The plaintiffs had alleged that Novelos had made materially false and misleading statements and omissions regarding the progress of its Phase III trial before the FDA for oxidized glutathione compound NOV-002, in application to non-small-cell lung cancer. In February 2010, Novelos said the Phase III study had concluded unsuccessfully, and the company's stock price dropped by about 80 percent. In dismissing the second amended complaint, the judge concluded that the plaintiffs failed to allege sufficient facts to permit an inference of scienter and failed to allege loss causation adequately. (See BioWorld Today, Feb. 25, 2010.)