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Aegerion Pharmaceuticals, Inc. (AEGR) Message Board

  • hankmanoo hankmanoo Nov 27, 2013 12:38 PM Flag

    Beacon on Orphan Drugs and PLI

    From Beacon Securities:

    Event: • Speculation that BioMarin Pharmaceutical Inc (BMRN – US) will be acquired by large pharma to gain access to its new drug Vimizim, which is anticipated to be approved in Q1/FY14. • BMRN has a current market capitalization of US$10 billion (@ US$71 per share). Shares are up ~42% YTD and up 150% over the last 3 years • Analysts believe it could be worth over $13+ billion (US$93+ per share) Analysis: • Vimizim, which is anticipated to receive FDA approval in Q1/FY14, treats the rare disease Morquio A syndrome. • The level of incidence is 1 in every 175,000-200,000 births or ~20 new cases in the US every year • Sales of the drug in 2017 are expected to hit ~$400 million. • BioMarin also has four drugs currently no the market, but we do not believe there can be too much excitement surrounding them as its Q3/FY13 revenue showed a y-o-y growth rate of 7% • Large pharma continues to be very interested in “Orphan” drugs. With regulatory anticipated shortly, this would take a lot of the risk out of the drug Conclusion: • This is the third orphan drug company that we have recently noticed receiving take-over speculation. • Viropharma (VPHM – US), a protein-based drug (Cinryze) for the treatment of HAE, is recently bought by Shire for ~8x FY14 revenue forecasts • Alexion Pharmaceuticals (ALXN – US) , is an ultra-rare disease company with its drug Soliris for the treatment of a rare blood disorder (1-2 cases per million). It currently trades at ~12x FY14 revenue forecast • We believe ProMetic could have significantly more value given we anticipate it to have 5 protein-based orphan drugs in clinical trials by the end of the 2014. • From a catalyst perspective, as PLI starts to file INDs and has the ability to manufacture the drugs itself (through the soon to be commissioned plant in Laval), investors (and potentially suitors) should start to see its large inherent value • In addition, PLI also has its therapeutic division (PBI 4050), which we beli

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