ISIS Pharmaceuticals, Inc. (ISIS), Aegerion Pharmaceuticals, Inc. (AEGR): Two Drugs Competing for an One-In-A-Million Disease

Both ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) and Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR) have a single drug in the market that help heart patients deal with what is called bad cholesterol, or LDL. The particular disease they both target is called HoFH, and it is a very rare genetic disease. Both companies have similar levels of market capitalization, and the FDA approved their respective drug candidates within months of one another. There was considerable euphoria in the market when Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)’s Juxtapid came up for approval and an even larger euphoria when Kynamro from ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) was approved a month later.

Caveat: This is a very small market where these companies can hardly recover the development costs. I think investors need to understand that.

What is HoFH?

HoFH or homozygous familial hypercholesterolaemia is a rare genetic condition marked by high cholesterol, particularly LDL, as the body is unable to remove LDL from the body, resulting in early cardiovascular disease. The most common issue with HoFH is occurrence in a much younger age than otherwise expected in the general population.

Isis’ Kynamro for HoFH

Kynamro (mipmersen sodium) is an inhibitor of apolipoprotein B. Apolipoproteins are proteins that bind lipids to form lipoproteins. Kynamro has been approved as a subcutaneous injection (once weekly) to be given along with medication for lowering lipids and diet to reduce LDl, apolipoprotein B and total cholesterol in HoFH patients.

Launched in partnership with Sanofi, Kynamro carries a warning that it may cause liver toxicity. Kynamro is yet to be approved by the EMA but stands a good chance if the risk of liver toxicity is mitigated.

Aegerion’s Juxtapid

Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR)’s Juxtapid (lomitapide) is a selective inhibitor of a protein which increases LDL levels in HoFH patients. After FDA, the EMA also approved it for sale in Europe where it is marketed as Lojuxta. The drug is available only through a restricted program under JUXTAPID REMS.

Juxtapid is the company’s only marketed product and as of today, according to their website, the company is engaged ONLY in the development activities related to marketing approval of lomitapide in Japan and its clinical study for non-adult patients. It reported revenue of $1.23 million for the first time for the quarter ended March 30, 2013.

What about Isis?

Engaged in discovery of drugs based on its antisense drug discovery platform, ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS) follows a royalty-based business model. After initial discovery, the company finds a partner from whom it receives royalty once the drug is marketed. Besides some smaller companies like OncoGenex, its partners include Sanofi (for Kynamro), Pfizer, AstraZeneca, Biogen Idec and Teva. The company reported Q1 FY 2013 revenue of $43.36 million and a loss of $1.67 million.

ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS)’s experience with the antisense technology has been much more pleasant than for Gilead Sciences, Inc. (NASDAQ:GILD). Gilead Sciences, Inc. (NASDAQ:GILD), formed in 1989, gave up on the antisense technology after a few years and sold the patents it did not need to ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS). Gilead Sciences, Inc. (NASDAQ:GILD) is now valued at $93 billion and reported annual revenue of nearly $1 billion for FY 2012. For the most recent quarter, ended March 31, 2013, the company reported an 8% increase in product sales ($2.39 billion) and net income of $722.19 million on revenue of $2.53 billion.