Hypothesis I: f508d defects vary & this explains great combo results
Wow. That was a pretty technical post. I liked the club analogy.
Wasn't the purpose of the Phase II trails to measure a great improvement in SC? This was not accomplished. Vertex does not want the focus on the primary goal of the research which is where it was focused as a large improvement in SC would be in the right direction.
Highlighting the FEV ( I can breathe better ) improvement of ~ 5% seems weak to me. I would argue that a good steriod-based inhaler would increase the FEV more, but the test was not measured against all known forms of treatment ( antibiotics, inhalers, etc. ), it was measured against a placebo ( a control created to do absolutely nothing on purpose).
I am as hopeful as the next person in medical advancements and cures, but these tests are just not there yet. I think you can complicate the argument with deep-lingo science terms that only another geneticist would appreciate, but in the end, you need to create something that is orders of magnitude better than current treatment options. Vertex has not gotten there yet, maybe they will someday.
I can't buy into the idea that just because we learned how to create a telescope today, tomorrow we will all be traveling the galaxy. I can imagine it, I can hope for it, I can dream about it. But is a telescope really going to help me create a interstellar spaceship or will it just motivate me to want one.
I too will give you the benefit of the doubt that you really don't understand what you are saying as oppose to trying to mislead others. Dr. White has explained SC and FEV. Let me just add re the "value" of the drug other currently available drugs. Our 2 year old spend 40 minutes a day doing hyper-sal for that 5-7% improvement. And another 10 minutes daily doing Pulmyzyme for another 5-7% improvement. And we spend another 15 minutes cleaning and sterlizing multiple nebulizers. These drugs work to fix the underlying defect AND are 3 pills which take what 1 minute total to take? The value to Cfers is truly amazing.
Re my scientific lingo. As I’ve often said here, I haven’t had a science class since I was 15 or 16. Anyone who has followed Vertex and the science of CF understands the basics. I wasn't complicating anything, but merely putting it all together and seeking educated insights, which I’ve been blessed to get by several others on this Board who truly are interested in understanding the drugs and how they work, etc., and who have taught me immeasurably in this area.
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!)