BMRN - The thing I find most interesting about Biomarin is that they are much more open than almost all other companies - with themselves and with investors.
I have, literally, never seen another biotech show a slide of their pipeline from 3 years ago comparing it to today.
That said, I agree there are significant weaknesses:
a) the PEG-PAL data looks pretty weak. Yes, some fraction of the population seems to do well on it - but in general the protocol looks hellaciously complicated to try to avoid immune system problems. Not sure why they don't go back to the drawing board and try again with a different pegylation scheme or ... .
b) Their cash spend rate is outrageous given their pipeline. They seriously need to curtail their spending. They may have the cash to burn - but burning it doesn't speak well of their management decisions.
As for the GALNS - I need to do more in-depth background research.
PS I am ignoring the PARP program because I consider the oncology space wildly overcrowded at this point so unless a company has a really particular niche I consider it a likely bust just based upon the competition. Too many competitors, who goes first line, who second, ... ?
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