Conference call next week will be giving guidance reflecting strong backlog of orders for G5 platform extending well into 2016 with world wide sales ramping up as Type 1 diabetics in the EU and US upgrade to this system. Analysts will raise PT and stock price will rise accordingly, not on just the earnings and revenue beat of the past quarter, but the from this increase in projected sales worldwide in the coming quarters. Takeover speculation from GOOGLE health sciences or JNJ or ROCHE could also make the stock price move up very quickly. (The stock trading at a 20% discount from it's high since the biotech bubble burst might make a take over offer a possibility)
Kalydeco's mechainism of action (CFTR modulation) was ignored by big pharma until VRTX successfully proved it's effectiveness and as a result, will own the market for treating the majority of the mutations causing CF for years, as various companies like GLPG chase the VRTX platform with their variiations of the same treatment mechanism (which they still have not proven their clinical efficiacy or safety and are years behind VRTX in entering the market). IMHO, VRTX with David Altshuler's and CRISPER personnel collaborating will likely have a series of drug candidates to effect a genetic cure for CF and Sickle cell anemia within the next three years, which if successful will make all the existing treatments for CF obsolete. The same platform will likely be extended to other genetic disease. This technology is in it's infancy but the timely development of it will be much quicker than you think. Thank you Third for reminding us of your prediction for this technology revolutionizing the treatment of genetic disease since early this year. Vertex has not be idle to pursuing this opportunity. Third-- what is your sense of the hurdles ahead and the timeline to development of this technology in entering clinical trials in the future?
Excellent Q&A, CF and Non-CF pipeline drugs in oncology, pain, Crisper gene editing program, and influenza got mention in either the 3rd quarter earnings press release or the investor conference call discussion. Spinal cord injury drug mentioned at MS investor conference was not mentioned todat, but will surface in presentation at JP Morgan investor conference in January, along with possible mention of Huntington's and chronic MS. Orkambi launch impressed all analysts, and should along with the pipeline update generate some upgrades and/or price target increases among some of the analysts. Should be a fun day for VRTX bulls tomorrow.
Yes Rojo, you called this one to our attention for a while now, and if it's potential is the same or better as Revlimid the value to cancer patients and VRTX will be huge.
Abstract reviewed by Jeffreys analyst (just released for upcoming oncology meeting):
"First look at VRTX's VX-970, which surprises with a CR noted: The drug was tested
in several advanced/difficult-to-treat solid tumor types as monotherapy or in combination
with carboplatin. Impressively, in an advanced colorectal cancer patient, VX-970 generated
a CR as monotherapy. The patient remains in the trial at 59+ weeks. Additionally, 4 patients
treated with monotherapy demonstrated SDs and 7 patients with VX-970+carboplatin
demonstrated SDs that are still ongoing. Recall, VX-970 is a first-in-class ataxia telangiectasiamutated
and Rad3-realted (ATR) inhibitor; we do not believe VRTX receives any credit for its
early stage non-CF programs, but this could begin to attract some attention and potential
Baird reiterated an Outperform rating and $160.00 price target on Vertex (NASDAQ: VRTX) ahead of the company's 3Q15 earnings results and Orkambi launch. Baird's interaction with physicians at the NACF conference earlier this month reaffirmed views that Orkambi will have a strong launch. VRTX is scheduled to report 3Q results on 10/28.
Analyst Brian Skorney commented, "We anticipate a strong Orkambi launch and continued strength in Kalydeco sales when the company reports earnings on Wednesday. Yesterday's CRISPR deal, while early, demonstrates Vertex's commitment to cystic fibrosis and the correction of other genetic diseases. VRTX remains our top pick in large-cap biotech."
Gene editing technology will likely cure CF and Sickle cell anemia and may be applicable to many other genetic diseases. Today VRTX announced it's new CSO's focus (which has been his research interest at Harvard prior to becoming the VRTX CSO). On it's career web page, VRTX has been recruiting scientists with experience in this area of research for the past several months. This collaboration and exclusive licensing and investment in this gene editing company confirms VRTX commitment to developing potential cures for genetic based disease and will keep it in the leadership position in the race to find the most effective treatments for this illnesses..
Medicaid guidelines for approval of Orkambi, which are likely the most restrictive over commercial plans, allow renewal at six months if prescribers certify any one of the following: stability of FEV-1 over the prior 6 months; weight gain, or improvement in QOL ( which includes reduced rate of infections/hospitalizations). Commercial plans would likely be inclined to not be more restrictive for approval or face legal challenge from legal firms that handle class action consumer insurance denials and bad PR as they report record windfall profits from Obamacare enrollment.
Steve, this and the prior 3 posts are probably the same panicked short desperately trying to induce investors to sell shares based on false rumors. He may have just covered his short position as VRTX is picking up steam as the stock price rises going into it's third quarter earnings next Wednesday Judging from today's market's reaction to better than expected earning in the tech space, and from GILD yesterday. A better than expected revenue and earnings number next week from VRTX first quarter of sales of Orkambi since getting FDA approval (predicted already by the MS analyst) will likely give the stock a boost from current depressed levels. Analysts may also finally give additional projected value to the company's non- CF pipeline to be discussed at the 3rd QTR conference call next week. VRTX share price has a lot of reasons to run higher (current earnings growth from Orkambi and expanded sales of kalydeco monotherapy in the U.S. and EU), no serious competition in the CF space for years, and lots of potential revenue from ongoing clinical trials in CF using VX 661/770, Pharion EnAc inhibitor, triple drug CFTR modulators (second gen correctors in combination with Orkambi and 661/770) and all the non-CF assets in oncology, neurology, influenza which will be detailed next week.
UBS' Biotech analyst, Matthew Roden, recently attended the North American Cystic Fibrosis Conference (NACFC), and returned more confident about Vertex's (NASDAQ: VRTX) market position and with incremental information on several other programs.
Vertex presented data on its ENaC program and info on the forthcoming triple combo. Due diligence, both at the conference and though independent research disclosed that although there are there are early stage and preclinical CF programs that appear promising, they are several years behind Vertex. Perhaps more importantly, the analyst could not identify any competing combos that represent a credible threat to the established Vertex position.
From Clinicaltrials.gov website, it looks like data collection completes in May of next year with analysis of data a release possible in time for EU annual CF meeting in Basel Switzerland June 8 - 11 2016. Assuming positive data, the NDA for 661/770 would be submitted in second half of 2016 and with accelerated review for orphan disease and 'breakthrough drug' designation, possible PDUFA date before year end 2016.
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation, and a Second Allele With a CFTR Mutation Predicted to Have Residual Function
Estimated Enrollment: 300
Study Start Date: March 2015
Estimated Study Completion Date: May 2016
Estimated Primary Completion Date: May 2016 (Final data collection date for primary outcome measure
Harvey, I agree that VRTX will remain a long term winner in the CF space because it's 'quad' treatment will own the space with very few CF patients left, who are not already benefiting from VRTX treatments. The limited ability for potential competitors to recruit treatment naïve CF patients for advanced stage clinical trials since the great majority of CF patients will already be benefiting from on effective VRTX treatments and not want to risk worsening their clinical condition by experimenting with untested/unapproved alternatives. This will create high hurdles for potential competitors like GLPG and CNCE to overcome because their CF drugs will not likely offer significant incremental clinical advantages in safety and efficacy (as opposed to theoretical test tube benefits they are claiming in test tube assays ). And the potential for VRTX in it's pipeline of non CF drug candidates in influenza, oncology, neurologic disease (spinal cord injury, chronic MS and Huntington's) and in the treatment of genetic diseases with gene editing technologies (yet to be announced) under CSO David Altshuler's leadership will represent growth prospects for VRTX not yet taken into account by Wall Street analysts.
GLPG data is based on it's own HBE assay to test it's triple combo for 508DD homozygotes, and comparing it to Orkambi pre-clinical data published by VRTX. VRTX says it's own triple combo will be tested with VX 661/770 and one of the two selected second gen correctors noted below in the press release from VRTX last week at the NACFC. (Note that VRTX triple combination treatment has demonstrated a three fold improvement in the company's own HBE assay in cells from 'het-min' CF patients who are 508D heterozygotes for which no treatment is yet available. The six fold increase cited by GLPG is not specified to be in the het min 508 heterozygotes (which are harder to treat) and is reported using it's own HBE assay and comparing it to results from separate HBE studies done by VRTX presumably on 508dd homozygotes. Not comparing the effects of Orkambi using the same HBE assay in a double blinded study in the same mutations makes comparisons between the two drugs impossible in terms of clinical superiority.
From VRTX press release at NACFC meeting last week:
"In human bronchial epithelial (HBE) cells with two copies of the F508del mutation and in HBE cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function, the triple combinations (VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor) resulted in chloride transport (percent of normal) that was approximately three-fold greater than the use of the lumacaftor/ivacaftor combination in these cells. A significant increase in cilia beat frequency was also observed with triple combination therapy as compared to the use of the lumacaftor/ivacaftor combination in these cells. These in vitro data suggest that a triple combination of a next-generation corrector with VX-661/ivacaftor may improve CFTR function in cells with two copies of the F508del mutation and cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function.
RBC Capital analyst Michael Yee reiterated an Outperform rating and $145 price target on Vertex Pharma. (NASDAQ: VRTX) saying the stock remains their top idea and they'd be buying here on the pullback.
"Based on channel checks at the NACF conference we're buyers and believe VRTX will post a solid Q3 result. VRTX is executing on multiple fronts: likely good launch of Orkambi, 2 new correctors into the clinic that should be in Phase II as a "triple pill" next year, introducing pipeline they haven't talked about yet, and likely becoming very profitable..."
UBS reiterated a Buy rating and $160.00 price target on Vertex (NASDAQ: VRTX) following the North American Cystic Fibrosis Conference. VRTX presented preclinical data showing enhanced CFTR function with the two triple combo candidates over Orkambi.
Analyst Matthew Roden commented, "We attended the North American Cystic Fibrosis Conference, where Vertex provided an update to their cystic fibrosis (CF) programs. Of note, the company presented preclinical data showing enhanced CFTR function with the two triple combo candidates over Orkambi. In addition, they highlighted early data showing the benefit of combining Orkambi with ENaC blocker VX-371 (P-1037). Although the focus of the meeting was understandably on CF, management plans to discuss programs outside CF on the 3Q call and at a competitor conference in January. We continue to expect VRTX shares to be a good performer in the fall and into 2016 with what could be an explosive launch, a turn to profitability in 4Q, and several catalysts in 2016
Preclinical triple combo data measuringCFTR function and cilia beat show major improvements likely in the treatment of 508 dd homozygotes and heterozygotes, especially with the addition of EnAc inhibitors from Parion. Expect co-formulationof the triple drug regimen in a single tablet to ease compliance by reducing pill burden. Automated continuous manufacturing of these drugs by VRTX will make supply of drugs smooth and predictable, and will likely given the increased number of patients to be treated make the cost of these meds actually go down. I suspect the benefit of treatment of the youngest children will be seen in the lack of progression of lung disease (they won't be as sick) eliminating need for treatments 2 hrs daily using nebs, vests, feeding tubes as welll as thetreatments for CF when the disease advances e.g. hospitalizations for recurrent infections and lung transplants. That in the long run will mitigate the overall cost of care for CF over the lifetime of the youngest CF patients.
Should have a positive impact on VRTX stock price . Parion drug and new second gen CFTR correctors entering VRTX clinical trials next month should allow VRTX to continue to expand and improve it's CF treatments that address the underlying cause of the disease in 90% of the CF population. Improvements in it's existing treatment with VX661/770 and Parion's 1037 will and will allow it to maintain it's leadership position, and addition of 2nd gen correctors will offer hope for more effective treatment of both 508 homozygotes and heterozygotes.
More than 15 abstracts related to Vertex's CF development program were accepted for presentation at NACFC. Vertex today provided the following updates to its development program in CF and highlighted select presentations from the conference:
Two Next-Generation Correctors To Enter Clinical Development
Vertex today announced that it is advancing two next-generation correctors from its research program into clinical development. Known as VX-152 and VX-440, these next-generation correctors will be evaluated alone and in combination with VX-661/ivacaftor in Phase 1 studies in healthy volunteers beginning in November 2015. Pending results of these studies, Vertex plans to initiate Phase 2 studies in people with CF evaluating VX-440 or VX-152 in combination with VX-661/ivacaftor in the second half of 2016. The studies of a triple combination (VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor) planned for the second half of 2016 are expected to enroll people with CF who have two copies of the F508del mutation and people who have one copy of the F508del mutation and a second mutation that results in minimal CFTR function. VX-152 and VX-440 are designed to further improve processing and trafficking of the CFTR protein to the cell surface, beyond that observed with a single corrector combined with ivacaftor, which may enable increased CFTR chloride transport, a measure of the function of the CFTR protein at the cell surface.
In human bronchial epithelial (HBE) cells with two copies of the F508del mutation and in HBE cells with one copy of the F508del mutation and one copy of a mutation known to result in minimal CFTR function, the triple combinations (VX-152/VX-661/ivacaftor and VX-440/VX-661/ivacaftor) resulted in chloride transport (percent of normal) that was approximately three-fold greater than the use of the lumacaftor/ivacaftor combination in these cells.
Orkambi launch must be tracking well. Looking forward to CF clinical trial data release from VRTX and Pharion at NACF meeting next week and third quarter conference call the week of Oct 26.