So Third, based on this logical reasoning, what is your prediction for the mean percentage increase in absolute FEV-1 in TRAFFIC and TRANSPORT?
Vertex Pharmaceuticals Incorporated is looking to hire a Senior Director to join the Global Talent Acquisition Team in Boston, MA. The Senior Director, Global Talent Acquisition will provide strategic and operational leadership to Vertex as the company continues its rapid growth. With their business mindset this HR leader will bring a proactive planning focus to Talent Acquisition at Vertex. He/she will focus on Talent Acquisition Operations, the quality of candidates to ensure they fit not only technically but culturally and to drive workforce planning.
This weekend Vertex employees are participating across the country in the annual 'Great Strides' fund raising walk-a-thon benefiting the CF foundation, as part of May being national CF awareness month. It's wishful thinking on my part, but It would be fitting and timely to announce positive Transport and Traffic Phase 3 trial 809/770 results on Monday on this occasion of national CF awareness being publicized this weekend by the CF community. It would also make the UBS investor conference presentation this Tuesday a lot more exciting. If no announcement is made this week, I believe the next logical time for the announcement of this Phase 3 CF trial results would be around the June 11 annual European CF meeting in Sweden, when CF clinicians and scientists from Europe and North America present their current research.
Here's the opinion of the biotech analyst from JP Morgan from an interview on May 15, 2014:
GM: We also like Vertex Pharmaceuticals Inc. (VRTX:NASDAQ)—a much smaller large cap than Gilead. This name has caused worry in the current biotech equity environment, with the recent shift in sentiment. I think a lot of investors are unwilling to take on a binary event in this environment—the appetite for that is very low now.
Vertex has two Phase 3 trials, called TRAFFIC and TRANSPORT, for a form of CF where patients are homozygous (have the same gene from both parents) for a mutation called delta F508. The data are due out literally any day now, with the latest date in July of this year. Some investors say this trial could fail. Well, yes, that’s true. I would say that the probability of the combination of Kalydeco (ivacaftor) + the experimental drug VX-809 (lumacaftor) not showing any signal of activity at all is less than 5%. Three different Phase 2 trials prove that Kalydeco plus a corrector, whether it’s VX-661 or VX-809, is efficacious. That’s why we like the risk/reward on Vertex at these current levels.
Another sign that positive results from VX 809/770 Phase 3 clinical trials will soon be released
“Mr. Young is a recognized leader within the biotech industry and brings a wealth of scientific and operational experience to our Board,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “Importantly, Mr. Young has been part of multiple life sciences companies at times of rapid growth and transition. His insights will be invaluable to Vertex as we continue to focus our business on advancing transformative medicines for people with cystic fibrosis.”
FWIW, Terrence Flynn ate crow when the 661/770 data was released in April 2013, when he had a sell rating on VRTX and a price target of $35. Now he is still somewhat 'cautious' ascribing only a50% chance of success to TRAFFIC and TRANSPORT, He can'tsee the .likelihood of success of 809/770 based on the proof of concept studies confirming efficacy in the in vitro & the Phase 2 trials involving both 809/770 and 661/770 both of which work by the same mechanism. I'm confident he will be eating a second helping of crow soon.
Third, thank you for the above analysis. What is your opinion regarding the fact that Vertex management chose not to disclose the response data of the four patient placebo group in the just announced 508/551 heterozygote trial using 661/770? Clearly the analysts were unhappy with the lack of full disclosure of that information, and the usual analysts negative about the likelihood of success for TRAFFIC and TRANSPORT (e.g. Terrence Flynn from GS) wrote in their post conference call notes to clients yesterday that the 508/551 heterozygote trial did not provide insight into the likelihood of success of TRAFFIC and TRANSPORT.
IMO, the management team at VRTX should have provided the placebo groups response data even though it was so small in number. They could have pointed out any placebo patient with a higher than expected FEV-1 was an outlier, given the statistically/clinically significant 'off and on effect' in the treatment group of 14 patients. This was just a proof of concept study, demonstrating the potential to further incrementally improve the response rate of508/551 heterozygotes using 661 with 770 to show once again the correlation of positive in vitro studies using CFTR correctors and potentiators reverse the basic phenotypic defect in the most common CF mutations in clinical trials as well.
Papa and Glad, I like both of you, cannot predict which pre-clinical candidates might offer more value than 787 and 509 i.e. which ones will be a success?. Strategic focus of the company's assets is critical to a rational and systematic development of drugs on a long term basis. CF clearly has the pressing need for breakthrough treatments and an immediate market for Vertex drugs that has no competition., One big multi- 'billion dollar molecule' success story like the CF franchise will certainly pay for more molecules to be tested, or alternatively acquired from another company for further growth.
Existing products already dominating the influenza market might change quickly with the emergence Tamiflu resistant strains of pandemic influenza (probably only a matter of time, given the prevalence of most all strains of influenza are now resistant to amantidine and flumadine).
VX 509 has only been tested in RA and given the diverse autoimmune diseases it would have potential to treat, and the fact these chronic diseases usually become refractory to existing immunosuppressive treatments in a significant percentage of cases, an alternative oral once a day treatment like 509, that could benefit such patients would have a market..... whether the costs to develop this drug would be worth the payoff for a piece of this market is less clear.
Just when we thought VX 765 was another drug that was not promising in psoriasis and epilepsy, we find out in the last six months that it may be the biggest breakthrough in the treatment of HIV in decades, and could potentially represent a cure for HIV infection, or at least a further benefit to HIV patients worldwide, Do not assume drugs that are currently not the focus of current development have no future potential, they may just be waiting their turn for development when the time is right.
Papa ... I think you mean VX 787 and VX 509 have not been partnered yet. I would not assume they are 'dead' I would rather describe their status as 'dormant'. They could still be developed internally .once VX 809/770 is approved and cash flow at VRTX permits expanding the R&D budget to further develop their potential. Likewise no mention of VX 765.for the treatment of HIV. Management at VRTX wants the focus of Wall Street to be strictly on CF;, and I agree that VX 809 Phase 3 studies will be positive and that will be the catalyst for the stock move to new highs, and the trigger to announce the development of the non CF assets in the VRTX pipeline.
I believe that after experiencing the Phase 2 809 data interpretation bungle by the outside consultants, Vertex management is actively involved in the analysis of the raw data from the Phase 3 809/770 trial, and is not passively signing off on analysis of Phase 3 data reviewed by outside vendors. Tomorrow's conference call should give us some sense of when they intend to reveal the final 'scrubbed' data from TRAFFIC and TRANSPORT. But I believe they are recruiting these latest marketing positions because they know the raw data supports pulling the trigger to hire highly paid and experienced in-house legal personnel who would only leave their current jobs if they had assurances that 809/770 regulatory approval is coming soon with a compensation package with a future upside that would make it worth their while to join Vertex. I suspect the recruitment of Jeff Chodewitz from Merck to be CMO last January was based similarly on known data from Warner Greene's lab making his career move to develop VX 765 as a HIV treatment at Vertex, an incentive he could not resist.
Final raw data from TRAFFIC and TRANSPORT has been in for two weeks. Why would just this week, Vertex management start to recruit experienced in house counsel to establish the marketing of a new orphan drug in six countries around the world, if the raw data available for their review did not support the hiring of such highly paid professionals to establish a successful commercial launch. Wouldn't one wait to hire such expensive professionals until one was certain of their imminent need?
Six separate postings just listed recruiting for in house corporate counsel to support launch of new orphan drug in separate international markets: Here's one example in Germany:
Functional Area: General Business / Operations
Seeking in-house attorney to provide legal support for the international operations of a US-based pharmaceutical organization.
Duties include advising Country Managers and functional teams on EU and national law matters, advertising and promotion of pharmaceuticals, contracting, competition law, data privacy, transparency, supply and distribution and compliance with internal policies and applicable regulations and industry codes.
The company has recently set up commercial operations in countries, including Germany, France, UK, Spain, Italy and Australia. Candidates must demonstrate ability to manage multiple clients and provide clear advice in a fast paced work environment.
The successful candidate will upon hire be supporting one or more regions with all commercial legal matters. Excellent communication skills, good judgment, ability to work independently, and strong business acumen are essential. The role will report to the Legal Director, International in Switzerland, but can be based in any of the European offices. Travel is required (approximately 25%) mainly in Europe
European Law Degree
Previous professional experience
Experience in a life sciences or pharmaceutical company, preferably supporting an orphan drug
Good knowledge of commercial law and competition law
Experience with privacy and regulatory issues
I totally agree that the hep C market and lifecycle of telaprevir was totally misjudged by VRTX execs. But VRTX will not be a single drug company for much longer, and with VX 770, 809, 661 approved to treat CF, the company's revenues will skyrocket. Add the potential of VX 765 to treat HIV and the growth potential of VRTX is far greater. VX 787 and 509 have not yet been written off, (unlike the VRTX hep C drugs), although their market potential is presently more uncertain and limited by competitive markets dominated by existing treatments. They still could have competitive advantage if tamiflu resistant strains of influenza increase in number creating a greater need for VX 787, or new studies using VX 509 show improved efficacy and safety in treating refractory cases of autoimmune disease. The rest of the preclinical pipeline is yet to be proven, but new approaches to cancer, Huntington's disease etc represent future growth possibilities in the years to come.
It's probably not unreasonable to wonder if the lack of any public discussion by Vertex management about their plans for their development of the non CF drug pipeline assets is a 'lack of any enthusiasm on the part of VRTX management' for the application of VX 765 to treat HIV. But sometimes you need to read between the lines of what has transpired since December's announcement by Warner Greene's lab and their discovery of the effect of VX 765 on HIV infected tissues in vitro. Vertex announced it had hired in early January 2014, Jeffrey Chadakewitz as it's new CMO. His expertise and academic career at Yale and subsequently at Merck was devoted in large part to trying to develop a cure for HIV infection. The timing of his hiring at Vertex is hardly a coincidence and represents anything but a lack of enthusiasm on the part of VRTX management for the development of VX 765 to treat HIV. The only reason it has not yet been discussed publicly by VRTX management IMO, is that management does not want to distract Wall Street from the focus Jeff Leiden has made to the company's commitment to CF. The need to timely execute the NDA submission of 809/770 is a business priority to monetize the company sufficiently to allow it to self finance the development of it's earlier stage non CF assets. I suspect if no mention of VX 765's application to HIV is made by management on this coming Thursday's May 1. 2014 first quarter conference call, it will certainly be announced sometime soon following the release of the clinical trial data from TRAFFIC and TRANSPORT. And this will likely raise the price of the stock and any theoretical projected value of any Vertex buyout offer by a larger drug company. VX 765 could be worth more than all the CF drugs combined.
A Vertex buyout will be expensive and may have a number of bidders. A Poison pill plan has been in effect for years in the Vertex corporate by-laws and management has until now resisted the idea of being taken over, and new management still seems to carry on Joshua Boger's vision of Vertex becoming a large independent pharma with it's own drugs being discovered through it's own preclinical R&D pipeline. The lack of development of the non-CF assets tp date, does not necessarily represent their failure for future approval and meaningful sales. I agree that the hep C assets (VX 135, Incivek) are not likely to go anywhere given the success of Sovaldi and the looming all oral treatment competition in hep C that even Gilead will have to compete with on a price basis soon from Abbot, Merck, BMY, JNJ etc.. However, the other non CF assets that already have proven safety records through the completed Phase 2A clinical trials, also have potential for significant treatment efficacy in multiple serious diseases, that provide new therapeutic options for patients not responding sufficiently to existing approved treatments for influenza (VX 787) , autoimmune disease (VX 509), and HIV (VX 765).
Jeff Leiden has made the strategic focus of the company purely CF for now, looking to license the development of the other non-CF assets to larger pharma partners due to the need conserve the cash reserves of the company. However, once positive results are announced for TRAFFIC and TRANSPORT, the FDA's break though drug designation given to 809/770 will likely result in prompt approval of treatment for 28.000 CF patients and the cash flow from CF sales will no longer require 'giving away' the non CF assets for lower than desired licensing royalties that were likely offered by potential partners. with whom Vertex had likely been in discussions. By becoming cash flow positive and profitable next year, they can develop these non-CF assets themselves.
Vertex Pharmaceuticals Incorporated (VRTX) has been struggling lately, but the selling pressure may be coming to an end soon. That is because VRTX recently saw a Hammer Chart Pattern which can signal that the stock is nearing a bottom.
Plus, earnings estimates have been rising for this company, even despite the sluggish trading lately. Estimates have actually risen so much that the stock now has a Zacks Rank #1 (Strong Buy) suggesting this relatively unloved stock could be due for a breakout soon.
$100 billion take over price reportedly being offered by PFE for Astra Zeneca despite the fact several blockbuster Astra drugs have gone or are about to go generic (e.g. Nexium, Crestor), and it's R&D pipeline while interesting, remains unproven. When 809/770 results prove positive in the next few weeks, VRTX will be high on the list of potential takeover targets for large pharma needing to bolster falling revenues due to generics, with Vertex R&D pipeline having much more short-term and long term payoff potential for a less costly acquisition price.
On May 1, the Vertex 2014 first quarter conference call to analysts will be informative regarding the status of the entire CF pipeline, and perhaps be the occasion to release at least some of the highly anticipated clinical trial data. While TRAFFIC and TRANSPORT have just completed their clinical data collection, I expect this Phase 3 809/770 data analysis about to be conducted by Vertex will be a careful and extended process with release of final data more likely to be later in May at the earliest, or even early June prior to the annual European CF meeting in Sweden.
Phase 2 VX 661 data, testing VX 661 and 770 in G551/d508 heterozygotes for 28 days could be released at any time since the final data collection was likely completed in the past month. The status of meetings with the FDA to get approval for extended label use of IVACAFTOR in Non G551 gating mutations and R117H mutation patients 18 years of age and older should also be made public very soon as well. The earlier R&D programs for early pipeline assets may also get mentioned, but more likely just get referred to as programs to be announced in the second half of 2014. Likewise, the status of approval to get payment for IVACAFTOR in treating G551 patients in Canada and Australia will probably be announced in the second half of this year..
At this point, the analysts' main focus will be on wanting to know the 809/770 Phase 3 data release date so that they can plan when they will receive the data for their pronouncements on the near term commercial success of Vertex (the so-called 'binary event'), Based on the Phase 2 clinical trial results testing 809/770 in 508d homozygotes, I expect the Phase 3 data to be positive and the stock price will quickly jump to new highs after it's release..
Amazing novel approach to treating cancer if it works. I'm more excited about VX 765 and it's more immediate prospects for clinical use if proof of concept Phase 2 clinical trials prove positive in treating HIV.(I suspect Vertex new CMO who is an HIV expert, and is taking over for retiring Bob Kaufman this summer, is working with Warner Greene to plan this Phase 2A trial).
Wall Street needs to see the TRAFFIC and TRANSPORT clinical trial data before rewarding VRTX, but when positive 809/770 data supporting the NDA filing is announced some time in the next 8 weeks, Vertex will likely start to be valued for the expected revenue growth for treating the majority of CF patients. IMO after that, management will reveal it's plans for R&D going forward for all it's early pipeline assets (in addition to CF treatments for 508 heterozygotes), including VX 765 to treat HIV, it's chronic MS remyelination drug, and this stem cell oncology program. (Management knows the cash flow from CF is what Wall Street is fixated on, and they do not want to distract analysts with new programs until they reward Vertex for the likely success of 809/770 suggested by management's new manufacturing facility for continuous production of 809/770 tablets in Boston, the hiring currently underway of the expanded international commercial team to market 809/770, and the work underway in the development of these preclinical pipeline assets, which could only be funded by the huge cash flow expected from the imminent approval of 809/770 and the expanded indications for Ivacaftor monotherapy.
The Biotech sector will likely come back to life very quickly with the announcement of positive TRAFFIC and TRANSPORT data and the NDA filings for 809/770 treatment of CF in 508d homozygotes.
Third, what's your impressions of this new position being recruited on the career page of the Vertex web site? I find this new interest in developing a research center in Shanghhai for translational research in Stem Cell/ regenerative medicine by Vertex,a sign of confidence in the near term for commercial success of the CF drugs to foot the cost of this new long term research commitment.
"We're looking for a seasoned Research Fellow to help establish a new stem cell based discovery platform in its new R&D center in Shanghai, China. This exciting opportunity calls for someone who has deep and broad expertise in the area of stem cell biology, iPS differentiation and disease modeling, with at least 7-10 years of post-doctoral experience with demonstrated success in project leadership and advancing key scientific understanding of the stem cell field. Superior skills in organization and communication are required.
Describe the essential, day-to-day job duties; include specifics around the project, deliverables, accountabilities, and timelines with which each job responsibility is tied and highlight the different contacts, both internal and external, with which these duties will require interaction.
You will be responsible for building a stem cell discovery platform, and leading a group of biologists targeting cancer stem cells and screening for compounds promoting tissue regeneration. Reporting to the SVP and General Manager of China, you would be responsible for project and external collaboration leadership and you would be a key contributor to the development of our regenerative medicine strategy.
- Provides the scientific and technical lead role for stem cell platform at the Vertex Pharmaceuticals China site
- Hires, manages, mentors and develops Ph.D. and M.Sc./B.Sc. level staff, initially through a partnership with a CRO
- Proposes, implements and leads new and innovative conceptual/seeds projects and lead optimization projects"