Aegerion CEO Speaks On Juxtapid Launch, Orphan Drug Pricing And Competition

Aegerion does not anticipate any changes to its launch strategy for Juxtapid as FDA approval of Kynamro was expected. We believe physicians will prescribe the medicine that they determine has the best benefit/risk profile for their HoFH patients.

You say Aegerion has 1,500 U.S. HoFH patients in your patient-tracker database and that there are as many as 3,000 HoFH patients overall in the U.S., yet the FDA and other experts put the number of patients at several hundred. What accounts for this difference?

Prevalence in most genetic diseases is an evolution... We've conducted primary market research. We've asked a lot of KOLs who've performed different analyses on large patient populations which shows the prevalence may be four or five times what was previously published. The FDA has cited some publications that are respected but dated. We don't claim to know for sure but we'll be smarter in 12-24 months as we look at the patients put on Juxtapid compared to the patients in our database. Right now, we feel comfortable with our data sources that there are approximately 3,000 patients. I don't think the real number will be a lot more or a lot less but we'll definitely be smarter about it in 12-24 months.

What about patient numbers in Europe?

In Europe, our ability to identify patients depends on the country. In Germany, for instance, there are 1,500 patients on lipid apheresis, which is significantly more patients than in the U.S on apheresis. Germany also tracks the LDL-C levels of patients so we have a very defined patient pool there. It's a well-characterized market. In other countries, we don't the same tracking data so we're going KOL by KOL [key opinion leader] and polling physician practices. In total, we believe there are approximately 3,000 HoFH patients in Europe but we don't have them well defined yet.

When will Juxtapid receive European approval?

I believe we will get EMA [European] approval mid year but as you know, it will take another three to 12 months to get reimbursement approved. We've cautioned investors that our European business will not build as quickly or as efficiently as the U.S. because reimbursement in Europe will be slower.

Can you give an update on the work being done to expand Juxtapid approval into the pediatric HoFH population?

Pediatric is one of the more important things to me. I would have liked to have already started the pediatric study but U.S. and European regulators asked us to do a preclinical toxicity study first. That study is already started and will take about 12 months, so we expect to start the pediatric clinical trial early in 2014.

Do you expect to see significant pediatric off-label sales of Juxtapid?

Physicians have told us that they are anxious to get their pediatric patients on Juxtapid. We cannot market it there but insurance carriers will likely allow patients under 18 to go on drug, our research indicates. I don't know how many pediatric patients will use Juxtapid.

To this point, you've focused on developing Juxtapid for the small HoFH population, but do you see a place for the drug in the larger hypercholesterolemia market? Is this a growth opportunity for Aegerion?

I came into Aegerion two and half years ago after the prior CEO was trying to take Juxtapid to a broader patient population. The board and investors, however, had lost faith in that strategy. It was clear to me after doing research that Juxtapid was perfectly positioned for the HoFH patient population, so that's the direction I took the company.

With that said, we do need to look carefully at a clinical development program for the broader, severe [HeFH] patient population. I think we will pursue that area because the drug deserves to go there and patients deserve the drug to go there. The big unanswered question will be coming into alignment with the FDA over what they want to see. We need to have a discussion with FDA about whether there is a reasonable clinical program to expand the drug's label. We will have that conversation with FDA in the next 12-18 months.

Are you concerned about competition from PCSK9 drugs?

PCKS9 is a promising platform and will help a lot of patients, especially those who are statin intolerant. In patients with no receptors, PCKS9s may work some but they won't get anywhere near the efficacy of Juxtapid. Certain HeFH patients, where there is a lot of receptor defects, PCKS9s will not work, so we believe Juxtapid is a protectable franchise.

Right now, Aegerion is a single drug, single indication company. What will Aegerion look like in five years?

Our first goal is to make sure we get the infrastructure in place and the Juxtapid launch right. That will take a couple of years. In five years, I'd like to see us leverage our regulatory and commercial expertise, our people, to bring in another compound that is like Juxtapid -- one that is addressing a needy patient population. I'd like to stick with the ultra orphan indications. But not for a couple of years, right now, we really need to focus on Juxtapid. We have plans to take this drug into 40 countries, so we need to stay focused on the launch.

-- Reported by Adam Feuerstein in Boston.

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Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

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