Quick notes from the Isis Pharma $ISIS conference call after tepid endorsement of KYNAMRO (mipomersen) by the FDA metabolic and endocrine products advisory committee. You can find more of my commentary on this event (as well as competitor Aegerion Pharma $AEGR and their drug lomitapide) on the Chimera Research Group blog.
- US - 200-300 pts on apheresis out of 12000-15000 eligible (don't have specific # that are hoFH - is believed that about 10% of them can access and tolerate the procedure). Therefore most on apheresis are actually heFH
- FOCUS trial - SPA negotiated with understanding that outcome trial not possible in severe HeFH either. Would you consider adding atherosclerosis measurement etc? We considered imaging, but not acceptable endpoint to regulators. [context for this is a dissatisfaction expressed by the AdComm regarding the LDL endpoint, particularly for any population broader - and lower risk - than homozygous familial hypercholesterolemia]
- refused to compare/contrast US/EU regulatory process. "it is proceeding" [context: it was clear form the panel that the broader label in the EU sought by ISIS/Sanofi was in grave jeopardy - a fact that was finally admitted much later on Isis's 3q-2012 earnings conference call]
- nothing about the panel causes us to change our estimates of # of pts and # of accessible pts [complete BS of course...]