your post is confusing, you obviously refer to this Vertex paper
Journal of Medicinal Chemistry, 2014;57(8):3382-3400.
which describes a classical medicinal chemistry effort, which has no relation to stem cell therapy
when was it that VRTX last presented an overview on their complete pipeline? I fully appreciate the focus on CF, but that should not exclude timely updates for investors what else is going on in the company. Maybe the announced departure of the cso Mueller is an indication that there are problems with the pipeline outside cf.
some time ago JPM had an estimate on VRTX share price after positive combo results. If I remember correctly it was about $100 on 3% FEV1 and about $150 on 6% FEV1. Now that we know that the combo FEV! is about 3% the current share price supports the JPM prediction. However, a correlation of FEV1 and share price would suggest that sales of combo is dependent on FEV1. But does the number of patients using the combo really depend on FEV1? I guess not, once approved CF patients will use the combo, no matter what the FEV1 number is. Having said that, what is a realistic share price to be expected on approval?
this is a huge opportunity! has the potential to outshine sales from cf franchise,
absolutely not priced-in in the current share price.
at this point in time gene therapy to treat cf is "blue sky research", various allele involved, a lot of basic problems have to be solved, will take +10 years to have a product in clinical testing.
The good news for cf patients is that the combo strategy - corrector plus potentiator - pioneered by Vertex really works. Kaleydeco und Lumacaftor are just the beginning, as Galapagos notes "there is room for improvement". Actually, I had hoped for much better data for the 809 combo. But already 661 will show better results and the preclinical data from Galapagos compounds indicate even better improvements. Vertex ought to look very carefully at these data and not rely on new approaches but to protect its cf franchise by best-in-class combos.
I agree on your timeline, but I just hope that VRTX has learned the HCV lesson and will proactively look to acquire potential CF competitors.
VRTX is far ahead of competition, but midterm Galapagos/AbbVie may be a strong competitor, has promising preclinical candidates, Galapagos (GLPG) has an informative presentation on its website, worth reading
a potential competitor is Galapagos which intends to start a phase I study in 2016
has an informative presentation on its website
now I am curious how the market will react.
in my interpretation this is a great day for cf patients, the overall improvements reported ought to make a big difference, hospitalizations will be reduced dramatically and lead to better quality of life.
Porges' argumentation is rather contradictory, on the one hand he claims that Vertex has not sufficient preclinical data and rushed prematurely into pivotal trials with the combo, on the other hand he claims that cell culture experiments indicate failure of the trials.
I can't take this guy seriously, he might have non-disclosed reasons for his assertions.
"By not saying anything at all, investors are assuming that the results must be bad "
most likely quite some guys will go short on today's analyst report, will make the VRTX stock even more explosive when positive results are announced..
keep your faith and relax
does it make sense to present a rehash of "old stuff" on tuesday and announce "break-through" results on wednesday morning?
according to a new report cited by Forbes VRTX is ranked #3 in R&D performance! The ranking of the top 22 firms by R&D productivity has been created by Sector & Sovereign Health and its lead analyst, Richard Evans, a former Roche executive and longtime Wall Street analyst. For details google "Who's The Best In Drug Research?"
Ian Smith indicated some interesting numbers:
2 k patients on Kalydeco - $400 mio per year
7k patients from label expansion - 1.5 bil per year
28 k patients from Kalydeco + 809 - 6 bil per year
17 k patients from heterocygotes patients - 4 bil per year
so if all adds up VRTX has the chance to generate about $12 bil per year from CF.
now keep your fingers crossed that the clinical results support that target ;-)
about a year ago Adam Feuerstein published a concise article on Vertex road to success in CF, worth reading again, just google "Vertex Motors Down Cystic Fibrosis Highway"