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Re: mcbio post# 184762

Tuesday, 12/16/2014 9:39:45 PM

Tuesday, December 16, 2014 9:39:45 PM

Post# of 252505
Its very early stage so I would guess on the low side if they license it this early (which they seem to want to do). A rough ballpark analog is to look at what BMRN did when they were desperately struggling to survive and licensed to Merck Serono x-US rights (here is the filing).
http://www.secinfo.com/duvJ5.z2f4.htm
At that time it was thought it could work in multiple diseases and they had (still have) an option on PEG-PAL.
Kuvan was at an early stage too but I believe in the clinc already and BH4 had a lot of data on it to make Kuvan approval probably one of the fastest approvals from start to finish.

Its not clear (to me) if this would compete with PEG-PAL (i.e. allow for pfe lowering to normal/near normal without dietary restrictions). If they can get it to normal levels I'd wonder why BMRN wouldn't pay up for this. On the surface it would avoid a lot of the headaches of PEG-PAL. I believe Altus Pharma (no longer in existence) had an oral candidate as well which like Codexis thought they could overcome one of the key obstacles of the enzyme surviving in the pH of the stomach. Incidental if they can do this it may open the doors for other drugs too.

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