Hypothesis I: f508d defects vary & this explains great combo results
I am not sure if you are sincere with the questions you raise or if you are just trying to confuse less sophisticated investors. I will give you the benefit of the doubt and I will assume you are sincere. First, SC is just a marker and it does not necessarily correlate perfectly with improvements in the condition of CF patients. The main purpose of treatments for CF is to make the patient breathe better, whether or not their sweat is less salty. And that is exactly what the new study shows: significant FEV1 improvements which is what really matters. Why are these new CF drugs so much better than what is already on the market? Because Vertex's drugs treat the underlying cause of the disease, not the manifestations. Other treatments are aimed at dealing with the thick mucus after it is already in the lungs while Vertex's drugs should prevent the thick mucus from forming in the first place. These are revolutionary new drugs for the treatment of CF!
These 'first drugs', Kalydeco, 809, 661 are just that...first drugs that attempt to 'correct the root cause of CF". NOTE: they WILL NOT be the end all ~ be all. These wonderful and ‘pioneering’ drugs will be ADDED to the CF patients therapeutic arsenal which, if all goes well, will get smaller as certain drugs become unnecessary since the lungs should become less hospitable to the plethora of issues that plague CF lungs. Use of antibiotics, antifungals, steroids, mucus thinners, the list goes on, should decrease and perhaps someday even stop. Bottom line, Kalydeco (K) or 809+K or 661 should make the lungs a happier & healthy place…that’s a good thing! That’s progress but keep in mind the following…
(God willing) each of Vertex's CF drugs WILL get superseded by a BETTER corrector/potentiator or ‘other method’ of ‘CF correction’ or who knows what down the road (maybe even gene therapy). I bet by 2027 (that’s the patent life, I think) CF patients won't be using ANY of these Vertex drugs because a company like Pfizer, Genzyme, or even a small biotech will have gotten something better approved. In many ways, Vertex is doing the ‘heavy lifting’ for these other companies – several companies are HOT on their path and want this blockbuster market too…that’s not so good for investors…but darn good for CF-ers! Perhaps I’ll find some of you on these other threads along the way?
Here’s a few random thoughts on CF meds/treatments and their respective futures…(to name a few obviously)
- Pulmozyme sales (Roche/Genentech) will plummet 3-5 years after a drug like 809 gets approved – they only way they will maintain revenues with Pulmozyme will be by reformulating to a dry inhaled version, maybe adding something else to make it even better.
- HTS treatments will continue for the long haul – but –the delivery may change to a dry inhaled type…much faster and better than nebulization…there’s a company I think in Boston working on this I think.
-TOBI/inhaled antibiotics – sales will plummet in the next 5-7 years…won’t be needed nearly as much.
-Allergy/Mold sufferers will likely have to remain vigilant even after a corrector/potentiator gets approved…the world needs better drugs to deal with aspergillus it’s a bigger problem than people know.
- Pancreatic enzyme makers are pretty safe. CF patients shouldn’t have as many issues keeping their weight on because they’re not fighting lung infections. But, “gut issues” will persist for years though CF patients should get some relief from these correctors/potentiators. It would be great to see Eli Lilly to get their synthetic pancreatic enzyme resubmitted to the FDA…it blows away Creon and others and doesn’t come from pigs! (no plastic in the capsules either!)
Agree with nearly everything you say, but I think Vertex will be in the game longer. CFF did $75 M of which only $25M was for 661 so they have much more in the early pipeline for CF. I haven't followed the other companies but will as more details come out! I knew CFF had also invested in Pfizzer (sp?), and I thought one other company for correctors, but right now so need to get something to us and then I'll look longer term. But you're right this is first generation. By the time you kiss your dd off to college she'll just have a small medical bag with some CF pills!!!!