to show any statistical benefit or improvement in FEV-1 in the Parion/ Vertex phase II CF trial alone and with saline. This result is similar to trials done with inhaled Amiloride on CF patients in the past i.e ENaC inhibitors don't work. It is a big blow for this drug development in non CF lung indication like PCD and bronchiectasis which were planned in the second half of 2016. We are paying 1.2 B for a failed drug, just like Virochem and Alios failed programs in the recent years.
VX 150 program in osteoarthritis is going to face cartilage damage issues with FDA just like JNJ/ AMGN osteoarthritis drug due to worsening joint damage after un-inhibited use of knee joint from lack of pain feelings. CRISPR program has no legs, just theory and R&D expanses. This company need to be sold.