Biotech Values

[ghmm]

BMRN:

Perhaps a bit of a delayed reaction to them regain rights to their PKU franchise. I had been thinking that PEG-PAL could be a 1B product for some time though some set backs made me think I overshot but today JJ said the franchise (both Kuvan and PEG-PAL) could be 1B. I doubt the street had forecast such high peek sales.

I think Serono was in an awkward position especially as PEG-PAL nears the market. I hadn't realize no registration plan was even started yet for EU. On call said that 5 years ago tried to reacquire but couldn't agree on terms now with them not focusing on neurology were able to come to terms.

To me the keys for better adoption are:
1-Can BMRN show neurocognitive benefit
2-Can they eliminate the diet for a great percentage of patients
3-An easier way to monitor PFE level (their attempts at a home unit failed)
4-Easier dosing schedule regimin. THe induction dosing seemed awkward and I would have liked to see them have a less frequent dosing schedule as originally thought.
They said Kuvan is commanding around 180k on average per patient. So that is likely a floor for PEG-PAL.

Their 190 program (for a form of Batten disease) could turn out to be a very big product too in the nearer term. They've said about getting positive feedback from regulators and seems like 1 trial will be what they file on. From some tweets there was a father outside the Leerink conference trying to get attention to get drug for his daughter. It is really a terrible disease which seems like (though still very rare) is more common then originally thought and the drug has a real chance to slow the disease (extend life meaningfully).