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.
Adam Feuerstein recently posted this sales estimates:
"Based on this math, admittedly imprecise, in order to spend $3.3 billion for royalties today, Royalty Pharma is betting Vertex can generate $6 billion annually in cystic fibrosis drug sales at peak."
Right. Both Santaris and Regulus have been working on this miRNA-122 targeting drugs for several years. Santaris tested Telaprevir with Miravirsen. However, both development face a formidable challenge of all-oral drugs. Santaris has to come up with a drug better than 3 log reduction at 5 injections of 7 mg/kg dose. They better use the GalNAc delivery system of Alnylam as Regulus does. It will be difficult to compete against Harvoni if it takes 5 injections. Even 2 injections will be too many. The CEO of Regulus said the company may report the results from 2nd cohort, who takes a single 4 mg/kg dose, in January. I would not be surprised if the dose brings down viral load by 5 log.
Santaris Pharma published ( NEJM) their drug results in 05/2013 i.e Miravirsen and my understanding is that project has Roche backing. They are doing phase II now in liver transplant patients. JNJ has killed Incivek market with a better partner drug and have taken over 787 along with Alios. Conducting VX 135 in Maldives was a mistake. Lot of corruption there. RGLS is at least 3 year behind Santaris.
A month ago, Regulus, a biotech specializing in drugs targeting micro-RNA, announced a surprising clinical results. Their second drug in development RG-101 knocked down HCV by 4.1 log with a single 1 ml subcu injection of a low 2mg/kg dose. Four weeks after this single dose, 6 out of 14 treatment naive and experienced patients treated with this low dose did not have any detectable virus in their blood. Several years ago Jopling and Sarnow showed that a human factor called mir-122 is essential for the survival of HCV genome in human body. Mir-122 is abundant only in hepatocytes and HCV can survive only in the liver for this reason. Very recently this year Sarnow showed that mir-122 forms a protective shield for HCV RNA against the host ribonuclease called XRN2. Without mir-122 HCV RNA will be degraded by the nuclease. Regulus is testing this drug with J&J's Simeprevir (OLYSIO) to study the effectiveness of the combination. The idea underlying the combination treatment is that 2 - 4 week OLYSIO regimen sandwiched between only two injections of RG-101 could destroy any genotype of HCV in patients.
So, Regulus has opened the potential to revolutionize hep C treatment. It has also opened the possibility that Vertex can once again market Telaprevir in combination with RG-101. The side effects plaguing Telaprevir+IFN+RIBA do not appear in the first 2-4 weeks of dosing in the majority of hep C patients and Telaprevir is as effective as Simeprevir. RG-101 is very very safe. Vertex management should consider testing Telaprevir in combination with RG-101. Simeprevir (OLYSIO) is absolutely useless by itself because of Gilead's Harvoni and Abbott's combo, and RG-101 may turn out to be its savior.
If 10% of the company revenues ( say company) can be sold for 3.3 B plus another 400 million form an unknown source i.e total 3.7 B, the entire company revenue ( say company) can be sold/ worth close to 37 B and NOT 27 B.
Andrew Fein always sees the worst scenarios possible for VRTX to justify his opinion and price target on the stock. With FDA approvals and huge sales imminent his next thesis is potential competition and a crowded CF space that is untested, and not even close to representing true competition. He and Geoff Porges are propping up short positions in the stock.