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Good morning, fellow Foolish investors! Let's take a look at three biotech stocks that could loom large in health-care headlines this morning -- Alexion Pharmaceuticals (ALXN), Salix Pharmaceuticals (NASDAQ: SLXP), and Ampio Pharmaceuticals (AMPE).
Alexion's Soliris is granted a new orphan designation in Europe
First and foremost, Alexion announced that the European Commission has granted an orphan drug designation to Soliris for the prevention of delayed graft function (DGF) after solid organ transplantation. This follows the FDA granting an orphan drug designation to Soliris for the same indication last month.
Soliris is approved in the United States, Europe, Japan, and several other countries for the treatment of two ultra-rare diseases -- paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Due to the rarity of both diseases, Soliris is the most expensive drug in the world, with a wholesale price up to $400,000 per patient per year.
DGF occurs when a transplanted organ fails to function normally immediately after a transplant. In the case of a DGF occurring during a kidney transplant, the patient will require immediate dialysis. Since there is currently a shortage of kidney donors, the improved treatment of DGF will result in a more efficient use of donated kidneys. Today, 15% to 20% of donated kidneys are reportedly not used and discarded in the U.S. and Europe, due to a DGF disrupting the transplantation procedure. Peak sales estimates for Soliris are around $3.4 billion. In 2013, sales of Soliris, Alexion's only marketed product, rose 37% year over year to $1.55 billion.
Salix and Pharming receive a new PDUFA date for Ruconest
Meanwhile, Salix and its partner Pharming just announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) Action Date to July 16, 2014 for the company's biologics license application (BLA) for Ruconest, a treatment for acute angioedema attacks in patients with hereditary angioedema (HAE). Salix gained the drug via its acquisition of Santarus last year.
HAE is a rare genetic disorder that causes attacks of uncontrollable swelling, which can be fatal if the respiratory system is affected. There are currently four approved HAE treatments in the U.S. -- Shire's Cinryze and Firazyr, Dyax's Kalbitor, and CSL Behring's Berinert. Of these four drugs, only Cinryze, which Shire acquired through its acquisition of ViroPharma, can be used as a preventative measure, whereas the other three drugs are administered during acute attacks.
Since HAE only affects one in every 10,000 to 50,000 people, the size of the market is fairly limited. Therefore, HAE treatments are very expensive -- Cinryze, for example, costs approximately $350,000 per patient per year. Due to the small size of the market and the existence of four entrenched treatments, Ruconest isn't expected to be a blockbuster. Analysts expect the drug to generate peak sales of $100 million, still providing a slight boost for Salix, which reported $735 million in net product revenues in fiscal 2012.
Ampio announces a new offering of 8 million shares
Last but not least, Ampio just announced that it plans to offer 8 million shares of common stock in a proposed underwritten public offering. The pricing has not yet been announced. That big sale will considerably dilute the 42.1 million outstanding shares of the stock, so investors should expect some price insability.
Ampio's stock has rallied more than 130% over the past 12 months, but the company finished last quarter with only $26.3 million in cash and equivalents. With a negative operating cash flow of $19.1 million, a new offering wasn't a major surprise.
Ampio doesn't have any marketed products, but its two most advanced pipeline candidates -- Ampion for osteoarthritis of the knee (OAK) and Optina for diabetic macular edema (DME) -- show considerable promise. Ampion is currently in phase 3 trials for OAK, and Optina is in phase 2 trials for DME.
Analysts forecast that Ampion and Optina could respectively hit peak sales of $1 billion and $500 million if approved. Optina is notable in the field of DME treatments, since it is an oral drug compared to Novartis' (NYSE: NVS) Lucentis, which is injected into the eye.