SCL30A8 is a drug target for treatment of DM II, just like PCSK9 for new hypercholestrolemia drugs from REGN and AMGN, The selection of a new CSO make me believe that VRTX is about to enter a new era of gene therapy and genetic targeting of diseases especially Diabetes. CFO has mention gene therapy in his last two conference calls. VRTX is a screaming buy in my opinion.
VERTEX PHARMACEUTICALS INCORPORATED
VERTEX PHARMACEUTICALS INCORPORATED Patent applications
Patent application number Title Published
20140343098 MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS - The present invention relates to modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators. 11-20-2014
20140323521 PHARMACEUTICAL COMPOSITIONS AND ADMINISTRATIONS THEREOF - The present invention relates to the use of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide, solids forms, and pharmaceutical compositions thereof for the treatment of CFTR mediated diseases, particularly cystic fibrosis, in patients possessing specific genetic mutations. 10-30-2014
20140315931 DEAZAPURINES USEFUL AS INHIBITORS OF JANUS KINASES - The present invention relates to compounds useful as inhibitors of protein kinases, particularly of JAK family kinases. The invention also provides pharmaceutically acceptable compositions comprising said compounds and methods of using the compositions in the treatment of various disease, conditions, or disorders. 10-23-2014
20140309413 METHODS OF STEREOSELECTIVE SYNTHESIS OF SUBSTITUTED NUCLEOSIDE ANALOGS - The present invention relates to the novel diastereoselective syntheses for generating phosphorothioate compounds. Examples include nucleoside phosphorothioate analogs that are useful in treating diseases and/or conditions such as viral infections. 10-16-2014
20140308242 BIOMARKERS FOR HCV INFECTED PATIENTS - The invention relates to biomarkers measurable in a human subject that have prognostic value with respect to efficacy of therapeutic treatments for Hepatitis C viral infection. The markers also are believed to have value for diagnosis liver health/liver damage. 10-16-2
If I were not fully invested I would certainly be buying at these prices.
Given the space VRTX finds itself in, it makes little sense that the stock should get hit so hard.
Classic buying opportunity then?
All biotechs got hit badly yesterday. btk, nbi, ibb were all down a bit shy of 3%--of course not as bad as VRTX. I think lots of institutions and mutual funds are taking profits before the year end to show they have done well for the 4th quarter.
Replacement for Peter Mueller is David Altschuler, already on the Vertex BOD and scientific advisory committee, and one of the pre-imminent medical scientists at Harvard med school. A geneticist, he has the background to address the underlying genetic basis of many diseases, which include his past focus which was diabetes, as well as the genetics basis of Vertex current pipeline e,g, CF, cancer, neurologic diseases etc.
Both an M.D. and Phd, Altshuler will bring both clinical and research experience to the CSO job. He knows Vertex well having been on it's BOD for the past few years, and will likely bring fresh ideas to the Vertex R&D efforts.
verity - I think ryderts was referring to FDA's pending decision regarding approval of KALYDECO for people with CF ages 6 and older who have the R117H mutation in the CFTR gene. this decision is expected by December 30th. this will be a nice addition to the KALYDECO label... and a nice way to end 2014!
I have feeling that a partnership is coming. Decernotinib data in Rheumatoid arthritis is roburst and drug is not inferior to Tofacitinib. VRTX should not ignore a future market of 18 B. VRTX is doing an extension safety study that will end in Oct 2015. Patient deaths in rheumatoid arthritis is not an unexpected event and CHF is expected in this age group. Old saying is that if you run a hair growing tonic trial, you will find white hair patients in addition to bald ones. Lets see what companies will shine out from ASH. I like ATR inhibitor and it seems to be very very potent in killing cancer cells.
Ian Smith's DB investor conference presentation worth listening to. Highlights include not only positive spin on rapid adoption and high value for 809/770 in the U.S., but finally a discussion of non-CF pipeline development programs including cancer and neurologic disease. Market reacting positively with stock making new 52 week highs every day this week.
Michael Yee, analyst from RBC, quoted in Barron's this week:
'Vertex should be a long into early ’15 Phase II VX-661 3-month homozygous data which should look better than first-gen ’809 combo. We want to own into potential ’809 strong pricing (we think possibly higher than $225,000 gross price) and USA launch for mid-15'
DB investor conference presentation tomorrow
Continued raising of PT of stock by analysts e.g. Jason Kolbert at Maxim raising his PT to $148 today citing underestimate of Vertex future CF revenue by the street.
FDA approval of sNDA for use of Ivacaftor in treatment of R117 mutation and EU approval for the same to follow.
Anticipation of VX 661/770 Phase 2 clinical trial data treating 508d homozygotes to be released in1Q 2015, possibly before JP Morgan biotech investor conference in early January.
S&P analyst predicts peak sales of VRTX CF meds of $6-7 Billion/year. I think that is a very conservative estimate. My prediction after VX 661 is approved is $8-10 billion/year.