I have done a little investigation into VRTX Multiple Sclerosis regenerative medicine program in collaboration with Cleveland Clinic. Program has been going on since 2009 and it is an exciting medicine to stimulate cells to produce mylin sheath. Biogen is obviously has its finger into the similar research. Lets hope Traffic and Transport show absolute FEV1 of at least 6%. After that we could go full speed on the highway. Thank you for the AIDS project update.
Great news. Thank you for calling Gladstone and finding out Vertex' intentions. In conference presentations speakers did not give a hint of their plans on the HIV collaboration. It would look foolish not to participate in the endeavor. The work done by Greene et al. look so solid that the FDA would certainly act positively. I'd buy more shares when they announce the deal.
Rojospan, I thought you might enjoy this news release from Gladstone Institute from last December. Today I called the spokesperson from Gladstone, responsible for this press release, and she confirmed that Vertex and Gladstone will be undertaking a Phase 2A clinical trial,after receiving FDA regulatory approval, to test the efficacy of VX 765 in the treatment of HIV infected patients. VX 765 could become a transformative treatment that revolutionizes HIV treatment in 34 million patients.
SAN FRANCISCO, CA—December 19, 2013—Research led by scientists at the Gladstone Institutes has identified the precise chain of molecular events in the human body that drives the death of most of the immune system’s CD4 T cells as an HIV infection leads to AIDS. Further, they have identified an existing anti-inflammatory drug that in laboratory tests blocks the death of these cells—and now are planning a Phase 2 clinical trial to determine if this drug or a similar drug can prevent HIV-infected people from developing AIDS and related conditions.
The Phase 2 trial—which will test an existing anti-inflammatory’s ability to block inflammation and pyroptosis in HIV-infected people—promises to validate a variety of expected advantages to this therapy. For example, by targeting the human body, or host, instead of the virus, the drug is likely to avoid the rapid emergence of drug resistance that often plagues the use of ARVs. The anti-inflammatory may also provide a bridge therapy for the millions without access to ARVs, while also reducing persistent inflammation in HIV-infected people already on ARVs. Many suspect this inflammation drives the early onset of aging-related conditions such as dementia and cardiovascular disease. By reducing inflammation, the drug might also prevent expansion of a reservoir of latent virus that hides in the body where it thwarts a cure for HIV/AIDS
Apparently you are not well informed about treatment provisions for rare diseases. Are you aware that Genzyme, now owned by Sanofi, charges about $200,000 per year for its drug to treat Gaucher disease? Insurance companies have been paying that every year since, I believe, early 90's. For those who can not afford or do not qualify for insurance, drug companies try to assist the patients to tap into other sources of funds and then provide it even free if there are no such funding sources.
Where in the world will patients get $250,000 per YEAR to pay for the drug(s) if approved? Insurance does not have to pay absurd prices, or some bizarre price VRTX thinks it deserves. I think I read that the average American family has $22,000 saved. It's their life savings. And what about all the people in the world who can't afford $250 per year, let alone $250,000 per year. And remember, supposedly that $250,000 per year is REAL money, i.e. money actually collected by VRTX. We all know that the label price on a drug may be $1000 for example, but after insurance companies and the government negotiate, the price they actually pay might only be $125. I might add that I thing Adam F. has made a fool of himself on many occasions. That being said, so have millions of investors. So who knows how high the stock could go with approval. Also, haven't we all seen biotechnology stocks that had so much hype built into them, that they fizzled after approval. It seems people are treating that $250,000 as if people can just waltz down to the drug store and buy the drugs with their balance of their weekly paycheck. What am I missing????
This might encourage your sense of the potential value of VX 809 in combination with Kalydeco (excerpted from a blog written in October of last year by a pediatric cf patient's parent:
"From the Trenches
What follows is a first hand account of someone who is enrolled in the phase 3 vx-809 trials. He’s ddf508. I suspect this blog will get taken down at some point so I thought I’d copy and paste it here just in case. I don’t know this guy, can’t vouch for the accuracy of any of it. But it makes me hope…
“Quick disclaimer, this is just a personal journal of what I have been experancing on the 24 week VX 809/ VX770 study for CFers that have the double delta F 508 gene type. Please just remember that we all respond differently to treatments……
My lungs haven’t felt this good in 10 years. I’ve now been in the vertex drug trial for 3 weeks. My PFT’s have gone from 53% to 64%. I’m a lucky man to be getting the goods"
"I have now been on the drugs for the past 3 weeks. Things have settled down, I feel like I’m at my new normal. PFT’s feel like they are in the low to mid 60′s, I feel like I’m moving a lot more air in and out of my lungs, my mucus is still greenish but down in the amount I’m brining up, I’m guessing 1/3 of what it was. I feel very lucky and a little guilty for getting these drugs. It’s just kind of weird. It seems to be the closest thing to a “cure” I have ever heard about."
Adam would not overestimate the market potential. Let's calculate the minimum share price assuming that the FEV1 absolute change is around 6%.
The market value would be higher than 200 k which 1 year Kalydeco dosing alone costs. Assume that the combination pill would cost 250 k for taking a year. There are 28,000 homozygous 508ers in NA and EU.
Assume again only 1/2 of these candidates takes in the first year of launch.
Then, the revenue from the sales of the combo will be 250 k x 14 k = 3.5 B in 2015. Add to this the revenue from Kalydeco monotherapy responsive CFers. The sum is about 4.2 B in the first year. The expenditure for the entire year would be 1.5 B. The profit before tax would be 2.7 B. For this year the tax would be about 35%, then you get 1.75 B for after tax earnings. If you divide this figure with the number of outstanding shares, which may approach 250 M, you get $7 for the per-share earnings. The minimum PE is 35 for Vertex because of its pipeline. Multiplying 7 by 35, you get 147. But this is 18 months away, so you have to discount by 40%. The final estimate is about $150 per shr.. Because of short-squeeze the share may go over 200. Look at what happened to Alnylam on January 13 this year.
What would the PPS be if VX 809 shows promise in Delta 508 mutation patients?
Adam Fuerstein thinks that the price would double considering the market that would open up ( estimated to be around 7 Billion)
Sentiment: Strong Buy