Replies to post #130789 on Biotech Values

[ghmm]

FOLD:

I think in Pompe is the potential for the greatest benefit to patients with ERT+chaperone. Its the ERT (well technically 2) that I am aware of with the poorest uptake into the cells. Unfortunately Amicus's Pompe chaperone has some tox question marks.

In Fabry I think part of the thinking was the incidence may be much higher then thought as many may not be diagnosed till late in life. Reaching these people though may not be easy. I believe it was TKT that also first discovered the incidence in women is much more then was thought (X linked).

Unfortunately, I don't think Fabry is enough to get the company profitable. They announced on their quarterly call a vote to increase the share authorization.

[jq1234]

FOLD - Market size depends on pricing and efficacy/safety data. ERT is well established therapy, so it won't be as easy to take market share as it seems. If oral monotherapy has similar efficacy as ERT, priced similarly, it can get a hundred million or more a year in US. The upper bound for number of Fabry patients in US probably is 4000. There are Fabry patients who can't tolerate ERT, and patients who have really mild version of the disease, not on ERT yet. These are good starting points.

ERT has really changed patients' life, you can see from the problem caused by shortage in the last a couple of years. Thus there wasn't as much outcry of ERT pricing north of $200,000/year as you would think. Any drug used in combination with ERT has to dramatically improve efficacy to have added value. To add another expensive drug to already very expensive ERT with marginal efficacy improvement has no market place today IMO.