Just to keep FGFR potential in perspective...
Although Pona is a best-in-class pan-FGFR inhibitor, soon to be validated in vivo in numerous trials and studies, Ariad is keeping its near-term potential focused on the largest markets via ISTs.
FGFR1 in NSCLC occurs in 2% of WW pts., roughly 20,000 patients each year, similar to populations for either RET and ROS1.
FGFR1 in SCC occurs in 21% WW pts., over 100,000 per year.
FGFR2 in endometrial occurs in 30% WW pts., or 160,000 patients per year.
FGFR3 in Bladder occurs in 50% WW pts., or 190,000 patients.
FGFR1/2 in Breast occurs in 14% WW pts., but this yields almost 200,000 per year.
(Note: assume US values at about 40% of WW total for all of the above)
Phase 2 IST trials in SCC and Endo are underway and I expect we will see a trial in Bladder by year end, given huge unmet need. Although Breast may have the largest FGFR population the market is very competitive, with many types of treatments and established early detection eligible for surgery. I think ASCO 2014 will be big for Pona, especially in FGFR cancers (no offense to its activity KIT, RET, & FLT3 driven cancers).
