If you want a picture of what can be possible in biotech look no further than Genzyme. Bell Potter, in their October 2012 Research Report on Neuren, discuss how orphan drugs (and the right business strategy) turned Genzyme from a US$100m IPO into a $20bn company. Orphan drugs open up powerful opportunities for junior biotech companies; the combination of factors including FDA fast track and priority review, venture philanthropy, a highly educated and committed target market and reimbursement programs are discussed in the Report.
What is the right business strategy? Henri Termeer, the CEO of Genzyme believed that one of the guiding principles was "self marketing the drug to better control its destiny".
Partnership is not an end in itself. There is a growing confidence that Neuren have the resources to take NNZ-2566 all the way to commercial development.
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