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Aegerion brings new hope to HoFH patients

Authored by Karl Simpson

As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are active in rare disease research. In the third of the series we look at US based company Aegerion.

Patients suffering from homozygous familial hypercholesterolemia (HoFH) were given a boost last year when Aegerion got regulatory approval for new therapy, Juxtapid. A rare genetic disease, HoFH prevents cholesterol from being cleared from the body and leads to patients building up fatty deposits in their tissues and blood, often resulting in fatal heart attacks.

At the beginning of 2013, Isis Pharmaceuticals and their partner, Sanofi /Genzyme were given FDA approval for the product Kynamro, also indicated for HoFH. The Isis product, Kynamro, is an injectable and Aegerion ‘s Juxtapid is a pill. Since the approval of the Kynamro product, much has been made of the difference in the price being set for the respective products by the two companies, something we’ll return to later.

The estimated patient population for HoFH in the US is approximately 3000 and Aegerion already claims to have identified around 1500 patients in the US. As with almost all rare diseases, it is incredibly difficult to understand the prevalence of the disease and only once you get deeper into the commercial launch of the product do you get a true sense for the market potential.

So what for Juxtapid in Europe? – Well, in a recent interview with the The Street , Aegerion’s CEO Mark Beer forecasts that the company is likely to receive EMA approval by mid-year 2013, with the reimbursement and market access process taking several months following this approval. Patient prevalence in Europe is more difficult to project at this time despite some very good data in countries like Germany who have excellent tracking information for patients on lipid apheresis. It seems that the total patient pool might be something equal to that of the US and hence Aegerion is ramping up its leadership presence in the region with the appointment of Genzyme executive Massimo Boriero as President EMEA. This is one of a number of key executive appointments to help scale the business and also the addition of former Chairman and CEO of Celgene, Sol Barer, to the board.

Aegerion also joined a rather elite club in 2012, for it became one of the rare examples in recent years of a biotechnology company managing to go public with an IPO, which it ultimately priced at $9.50 per share. Aegerion had tried to go public before in both 2007 and 2008 but owing to the severe market conditions at that time, they withdrew. On writing this article, Aegerion were trading a touch shy of $30 per share which values the company in excess of $800m.  This upside perhaps emphasising the current sentiment towards companies operating in the rare diseases market, but no doubt also reflecting the commercial expectation for Juxtapid.

How does the commercial potential of Aegerion look?  Well, the company got a nice pop in the share price back in October when the FDA gave the nod to Juxtapid. This was a catalyst for the share price to continue rising, alongside a run up in a range of other equity classes over recent months. Then came the Isis / Genzyme approval for Kynamro which has been priced at $176,000 versus the $235,000 for Juxtapid. The impact of the competition on Aegerion is very difficult to determine at this stage as rare diseases differ from many traditional drug sales and marketing models. It demands close work with patients and patient groups as well as close work with physicians and Aegerion has already spoken about what it is doing to educate and train physicians in its risk management programme.

Clearly, debate will continue over whether patients and physicians will opt for the cheaper Kynamro injection or the Aegerion pill, which carries a higher cost. Aegerion knew and expected this competition, albeit perhaps not quite so soon. It has therefore priced its drug with this competition in mind. The pill therapy will still need an escalating dosing regime and we understand patients will be required to undergo liver tests at each dosing increase. Meanwhile, the clinical data has suggested that Aegerion’s product is perhaps a more effective therapy.

Whichever therapy wins through, the news is good for patients of this rare genetic disease, HoFH. They have now got a modifying therapy to this disease and ultimately they worry not about the price but the quality and extent of their lives. Aegerion has been able to bring an innovative therapy for this disease to market and this rare disease company will hopefully be able to find commercial success for their endeavours and take their experiences for this development and commercialisation process to help other drugs to market.

Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter

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