I want to add to the excellent and conservative reply by Applejungle. For the next several years Vertex will not see any competition in treating underlying cause of CF unlike other biotech companies. Vertex CEO does not advertise drugs in preclinical development. Gilead's Harvoni sales will decrease by 60% or more when Regulus' RG-101 is launched in a few years. Celgene will face serious competition from several immunoncology drugs which are already marketed. For all drugs Isis develops against liver based proteins Alnylam can create far better ones...more effective and safer.
VRTX has a massive negative ($4,528,794,000) retained earnings on its balance sheet.
I do not understand this company either. "Castles in the air" bubble?
Vertex is completely out of HCV, only does CF. Kalydeco is on market for 294$/yr. Although few patients, but they take the drug for life! Terrific drug. With more countries approved and recent label expansion, Kalydeco can hit 1B revenue alone.
In addition, VX-809/Kalydeco NDA is expected to be approved middile of the year. Although not as efficacious as Kalydeco for certain mutation patients, the population is 10X that of Kalydeco. The price is likely b/w 150K-200K/yr, so peak sale may hit 3-5B/yr. Further more, they have VX-661/Kalydeco in Ph3, which may be better than VX-809 in efficacy, and possibly treat another 20-30% more CF patients. If Ph3 results are good, VX-661/Kalydeco may contribute another 1-3B sales...
Also rumor that GILD may buy VRTX, but just rumor...
I do not own this stock but see it getting a lot of attention. I am trying to understand why it is so expensive; i.e. what is the upside here? Is it in their hep C program or just the CF program?
I own CELG, GILD, ISIS and some smaller biotech companies but this one just befuddles me. VTX has an enormous market cap, 1800 employees (if one believes Yahoo), and a very small pipeline in my estimation. When I compare what ISIS has in the pipeline (and only 365 employees) - what is the valuation based on that I'm missing?
It would be nice to hear positive phase 2 VX 661/770 clinical trial results treating 508d homozygote CF patitents at next Monday's presentation. It would certainly make the citi biotech analyst's report released today look stupid, and push the stock up to new 52 week highs. Even without releasing the 661/770 data, just an upbeat presentation and timeline for 2015 R&D milestones and expected approvals for 809/770 and the progress in the cf and non cf pipeline will likely be a catalyst for the stock.
I have a progressive disease, Parkinson's. I would pay $340k to halt the progression of my disease and to make a small improvement in symptoms to boot.
I think anyone with a progressive disease that becomes more debilitating over time would pay almost anything for such a drug.
Unlike HepC where there are alternative medicines approved and on the market, CF has no alternative meds albeit for a few years if there is no toxicity and equal efficacy.
Apples and oranges. Hep C is one of those diseases people can live with for a long time before their quality of life is seriously impaired. With CF, quality of life is all-important, and that begins with a slight improvement in the ability to breathe.
to all Vertex longs. I am convinced that 2015 will see new highs for VRTX sp,
based on the assumption of approval for combo and positive data on 661 I dare to predict a sp well above $150 at the end of the year.
What's your guess?
Speculation that phase 2 VX 661/770 results will be positive seems to have the stock moving very quickly in the first hour of trading of 2015! Happy New Year to all longs and the CF community!
Specifically, Altshuler and his colleagues have shown that certain mutations in the SLC30A8 gene protects the carriers from contracting type 2 diabetes. This means that deletion of this gene would help people with T2D!!! However, other groups have shown that T2D is caused by mutation in the same gene in mice. This inconsistency can be resolved in favor of Altshuler if Vertex found an inhibitor to “loss of function” zinc transporter ZnT8 (encoded by SLC30A8) mutations. It is possible that Vertex has already found several compounds which exactly treat animal model T2D. This is a good reason for Altshuler to join the Vertex research group.
In real life, a mutation that abolishes the function of a protein useful in normal people, can reduce the risk of human disease in others. Examples include knockdown of antithrombin to treat hemophilia, or inhibition of PCSK9 function to treat hypercholesterolemia to potentially reduce heart attacks. But no target have yet been exhibited for type 2 diabetes (T2D) until this year. Would Vertex have potential drug candidates?
Dr. David Altshuler had been the second man in command at the prestigious Broad Institute in Cambridge, MA. Dr. Altshuler was also an associate professor of Genetics and Medicine at Harvard Medical School. His scientific research has focused on the discovery of causal connections between genetic mutation and health/disease. His most recent work involves genetic basis for the risk of developing type 2 diabetes (=T2D). Hence, he is an academic physician who is devoted for patient care. He is only 49 years young and a former classmate at Harvard Med tells me that he has a dynamic and friendly personality as well. He could have taken a position in any research or educational institution. Vertex is dedicated to bringing revolutionary medicine to the world, but it is not a pure research institution. I could not help but ask; what is he looking for at Vertex and what does Vertex expect to achieve by appointing an academic physician to occupy the position of CSO? As a geneticist Dr. Altshuler would be most gratified by finding out the genetic cause for a particular disease. Vertex as a biotech is interested in what molecule can cure the disease. Could these two separate goals converge?
Those two distinct goals can converge if medicine can be found based on genetic codes. What better way is there that can prove genetic basis of disease than inventing a molecule or a molecular process that can precisely repair the mutation? Altshuler’s hypothesis that certain genetic makeup is protected from T2D. This hypothesis is shown by genetic analysis of 150,000 human data, but other groups have shown earlier that the opposite is true. [read on]
Happy New Year to you Rojo as well as all the patient Vertex longs and the CF community who are finally getting the reward they have waited for. The JP Morgan Healthcare conference starting in 2 weeks in San Francisco will hopefully give the outline of expected progress in both the expanding CF and non CF pipeline from Vertex in 2015. VX 661 Phase 2 data treating homozygotes and the advancement of this drug into Phase 3 clinical trials for treatment of both 508d heterozygotes as well as homozygotes, with or without second gen correctors, in combination with Ivacaftor, will be a focus of analysts, along with the approval timeline and potential pricing for 809/770 in 508d homozygotes in 2015. The non CF pipeline may finally get mention by Vertex at JP Morgan as well including VX 787, in development by Janssen, in light of bad influenza season already in progress in the US, The two Vertex drugs in Phase 1 clinical trials for cancer, VX 803 and 970, may also finally get mentioned. Hopefully Huntington's Disease and/or chronic MS clinical drug trials are also moving forward, after years of pre-clinical investigation. The treatment of the genetic basis for CF, Huntington's etc,, as well as Diabetes type 2 may be the future focus of Vertex research given the expertise of David Altshuler in his new role as VRTX CSO. It should be an exciting 2015 for Vertex longs!