AEGR to be best-in-class, standard-of-care for HoFH. It will be cycled on and off w/ apheresis so the liver can rid itself of excess lipids. Heavy institutional ownership here. Look for this orphan drug player to be scooped up. Big pharma let ALXN get away w/ their orphan drug, now they're too expensive to be acquired. AEGR has potential (heterozygous and/or pediatric expansion) to generate excess of 1/2 billion/year.
The good news is that the revenue potential is likely to double if ISIS's drug fails review, while AEGR's is very likely to be approved. I owned both but sold ISIS today at a loss. AEGR's drub has a better safety profile, nd they had 78 weeks under their belt whereas ISIS only has 24 or 26 weeks. The fact that 23 people developed tumors will kill approval of ISIS's drug until they do an extended (1-2 year) trial to measure safety over a longer timeline.
I don't think the current market cap reflects the potential yet, especially not after the cancer bombshell from ISIS. AEGR will be first to market. It has better efficacy and safety. Why would these patients then want to try something inferior from ISIS?
NPSP was trash, that's why I didn't bother with it. I don't think AEGR is priced in. I didn't think SRPT was priced in either when it was 8 or 16, then boom it hit 44.