1. Phase 3 Kalydeco monotherapy clinical trials currently underway in G551 mutaion age 2 to 5 and non-G55l gating mutations, and those with residual CFTR function, will become availiable throught the summer and early fall. With breakthrough durg designation for VX 770, the expanded drug labell to treat this group of patients comprising an additional 10 percent of the CF population in the US, should be FDA approved by year end if not soooner.
2. Vx 509 Phase 2B clinical trial results in RA reconfirmed to be released in the second half of this year. I would expect a deal with a larger Pharma to be announced following release of positive results in this trial to allow rapid co-development of this asset in multiple autoimmune diseases to expedite it's development and monetize it in the short to intermedicate term with milestone payments, and in the longer term with royalties upon approval and sales in multiple autoimmune disease indications e.g. RA, psoriasis/sporiatic arthritis,IBD (Crohns/Ulcerative colitis etc.)
3. Announcement of a partnership to develop VX 787 for treatment of influenza with a novel mechanism of action, having clear advantages over Tamiflu and Releanza. (effective up to four days after onset of symptoms, superior efficacy in shortening duration of symptoms in seasonal influenza, and effective in tamiflu resistant strains of influenza). Once again short to intermedicate term monetization possisble with milestone payments and long term monetization for Vertex with royalties upon FDA approval, without the need for Vertex to spend money on development and reuglatory submission or a huge sales and marketing force after approval.
4. VX 135 Phase 2 studies with riba, Daclatsvir, and the drugs from GSK and JNJ/Medivir. Some resutls likely to be out in time for AASLD, and the drugs demonstrated to work best will be likely partner with VX 135 to co-formualte as a single tablet to be taken once a day in the for the shortest time period possible.
5. Final results of the positive VX 661/770 Phase 2 data at the fall national CF meeting, along with the announcement of second degneration correctors being initiated in clinical trials for use in combination with VX 661 or 983 to treat CF 508d heterozygotes. Also announcement of completion of enrollment of Phase 3 clinical tirilas using VX 809 and 770 in 508d homozygotes with completion of those results and submission for NDA approval to treat another 50% of the worldside CF population in the first half of 2014.
Well put together Qdelfan. I would emphasis that the movers would be the last part of item 5, and item 4. I think we could see SVR4 data for the VX-135 / Rib study soon. Items 2 and 3 could come out of nowhere... so sitting.
Going to move some more capital back into Vertex over the next three months. Last week didn't pan out so well and still nursing my 4% hit. I am glad I diversify because there are some people hurting a lot worse. Hope all is good with you.