The bigger question is will VX 661, which reportedly does not have an interaction with Ivacaftor, have a significantly better improvement in absolute FEV-1 (compared to the phase 3 results in TRAFFIC/TRANSPORT) in the ongoing 12 week phase 2 clinical trial testing the two drugs in combination in 508d homozygotes? How much added benefit would a second corrector with a different mechanism contribute when combined with 661 and ivacaftor in either 508d homozygotes or heterozygotes? These studies may be discussed during tomorrow's Q&A portion of the second quarter conference call with analysts.
Tuesday's conference call may address Vertex plans in more detail regarding it's own internal development of next generation correctors to be used with 661 and Kalydeco which had been previously mentioned as potential treatment for the vast majority of CF patients including 508 heterozygotes as well as homozygotes. The first clinical trials have been mentioned in past conference calls to be started at the end of this year or in early 2015. From a more positive perspective, the number one biotech analyst at ISI group, Mark Shoenebaum, in recent CNBC interviews and in his reports, has been quite positive about the potential for Vertex plans to treat the vast majority of the CF population with this combination of drugs, and sees further upward valuation in the company in the mid term from this triple drug combination, as positive clinical trial data is read out next year Given the competition chasing Vertex and the lesson learned from telaprevir being replaced so quickly, there likely will be questions during the Q&A during Tuesday's conference call from analysts like Geoff Porges or Andrew Fein from HC Wainwight whose reports have been focused on the future commercial competiitve risks facing Vertex in it's CF franchise. A confident response from Vertex executives to these inquiries will help convince analysts that Vertex is aggressively developing more effective next generation treatment to help most all patients with CF and that Vertex will remain the leader in the CF field.
Even the most bearish analyst from Wedbush is slowly moving up his QDEL price target and
upgrading the stock to a neutral rating. High short interest likely resulting in short squeeze today as savannah is being debuted in Chicago next week and solana makes it's debut this year and sofia placements and amplivue and Lyra sales start to ramp up and eventualy become acretive to earnings that up until now have been from legacy POC diagnostic tests.
Thank you Rojo for this update on VX 509. You may be right about the decision to do the phase 3 trials for VX 509 in RA in house, but given the competition for existing biologics already FDA approved and being used widely to treat the autoimmune diseases 509 could treat, is the cost of the lengthy trials in the various disease indications likely to result in a significant market for an oral small molecule alternative with the efficacy described above? Is it safer/ more effective than existing biologics, or is it simply going to marketed as more convenient but just equally effective/safe as existing parenteral treatments which are already widely adopted ? Would the latter advantage alone be likely to be worth the cost of development, by Vertex or is this better to license, like VX 787 to a larger pharma (if one was interested) which is better funded to absorb the development costs?
The cost of drug research is not unique to Vertex. It is largely mandated by the federal law enforced by the FDA. The difference between the Vertex of 15 years ago (the time of the publication of Werth's first book prior to the approval of any Vertex drug) and now is like night and day. Business people are at the helm of the company and are prioritizing development resources which has resulted in financial stability of the company, Look at the street's analyst's average revenue and eps projections for VRTX in 2015 and you can see the science is finally going to generate a sustained profit with 809/770 being approved by the FDA. The R&D focus of the company will continue to be pursued with a reasonable allocation of the company's assets to allow for future growth. The initial vision of the founder will finally be realized but within the context of responsible financial stewardship for shareholders, That is why the stock is quite stable at the current stock price, and with each press release detailing further progress in the approval process for it's pipeline assets, the share price is only likely to rise further given the growth potential of these breakthrough treatments.
Thanks Rojo, great news for future growth of Vertex R&D pipeline and it sounds promising. Any further word on new or planned clinical trials testing VX 765 in the treatment of HIV?
The calls from certain members of Congress to explain high drug prices has no teeth in terms of laws. Both Gilead and Vertex continue to get their negotiated current rates for their breakthrough drugs world wide, often with discounts. Regulation on pricing would stopfurther drug research and development unless they eliminated the legally required and extremely costly regulatory development process which will never happen. If there is no reward for the high costs of development and the high risk of failure and potential competition, there would be no advances in treatments for these dreadful diseases by the pharm industry. Society eventually gets the opportunity to get the drugs at a severe discount after the patents expire and the drugs are generic (80% of all drugs sold in the US are now generic). In the meantime the investors who take all the financial risk for new drug development are entitled to the reward if they succeed after the average wait of 10 to 15 years in takes in the US from pre-clinical testing to approval. It's a high risk, but high reward investment, The investment process is part of capitalism. It is the reason HIV, Cancer, hep C and diseases like CF have finally received breakthroughs in treatment and patients are benefiting, along with those investors who took the financial risk . Also remember, government taxes the profits made on these drugs both from the drug companies and it's investors and employees, and thereby benefits as well from the tax revenues and the productivity of patients who are now able to work instead of being ill and disabled by their disease when these drugs are approved and getting prescribed.
Drs. Leiden, Chodakewitz, Boger and the Vertex scientific advisory committee will recruit someone comparable to Peter Mueller to lead the Vertex R&D program. I suspect they will have an easy time finding such a candidate given the growth trajectory and revenue stream Vertex is now expecting with the approval of 809/0770 just months away and a pipeline of preclinical drug candidates for cancer, neurologic disease, autoimmune disease as well as second generation correctors for CF. Their experience and contacts in the pharmaceutical and academic research worlds will certainly give them plenty of candidates to consider for the position. At this time, Vertex is a great career move for the right person wanting to lead the growth of the R&D arm of this growing company.
Since release of TRAFFIC/TRANSPORT data, analysts have been modeling a discount on the 809/770 pricing and still predicting peak annual sales averaging 7 billion worldwide (and it could be more since these are hypothetical projections). Long term efficacy data regarding decreased rates of infection and hospitalization, as well as slowing the progressive loss of pulmonary function will be a important factors in getting better pricing from payers. If data coming from VX 661/770 Phase 2 trials currently underway shows better improvement in absolute FEV-1 compared to TRAFFIC/TRANSPORT, the stock will get higher valuations seeing the likelihood of better pricing for the eventual use of 661/770 in 508d homozygotes and eventually in the 508d heterozygotes when combined with second generation correctors.
Consider the possibilities: At a minimum the above article suggests the possibility of using VX 765 in every HIV infected patient. VX 765 would prevent the the infected patient's self destruction of it's own CD4 T cells that seems to naturally occur in response to HIV infection. Furthermore, it would prevent the emergence of resistance to ARV drugs, and thereby enhance the effects of ARV therapy. Lastly, it may prevent the inflammatory complications of long term ARV therapy which include premature aging and death due to heart disease and dementia. This would be an incredible step forward in the treatment of HIV worldwide. Another first to market opportunity for Vertex with incredible benefit to a large and suffering population worldwide.. It could reduce health care costs overall by preventing the emergence of HIV resistance requiring changes in ARV treatment, prevent the development and need for treatment of the long term complications of ARV drugs (heart disease and dementia) and could be part of a treatment that actually eradicates the latent HIV reservoir in patients being treated for a potential cure for HIV infection.
From an article posted on the Gladstone Institue web site:
Once the scientists discovered this key process, as described in Nature, they began to investigate how the body senses the fragments of HIV’s DNA in the first place, before alerting the enzyme caspase-1 to launch an immune response in the CD4 T cells. To identify the so-called DNA sensor, the scientists found a way to genetically manipulate CD4 T cells in spleen and tonsil tissue. In doing so, they discovered that reducing the activity of a protein known as IFI16 inhibited pyroptosis, explained Zhiyuan Yang, PhD, a Gladstone postdoctoral fellow who is one of the paper’s two lead authors.
“This identified IFI16 as the DNA sensor, which then sends signals to caspase-1 and triggers pyroptosis,” says Kathryn M. Monroe, PhD, the Science paper’s other lead author, who completed the research while a postdoctoral fellow at Gladstone. “We can’t block a process until we understand all of its steps—so this discovery is critical to devising ways to inhibit the body’s own destructive response to HIV. We have high hopes for the upcoming clinical trial.”
The Phase 2 trial—which will test an existing anti-inflammatory’s ability to block inflammation and pyroptosis in HIV-infected people—promises to validate a variety of expected advantages to this therapy. For example, by targeting the human body, or host, instead of the virus, the drug is likely to avoid the rapid emergence of drug resistance that often plagues the use of ARVs. The anti-inflammatory may also provide a bridge therapy for the millions without access to ARVs, while also reducing persistent inflammation in HIV-infected people already on ARVs. Many suspect this inflammation drives the early onset of aging-related conditions such as dementia and cardiovascular disease. By reducing inflammation, the drug might also prevent expansion of a reservoir of latent virus that hides in the body where it thwarts a cure for HIV/AIDS
Hopefully clinical trials using VX 765 treating HIV infected patients will be announced this summer. I think Jeff Chodakewitz (an authority on HIV) being hired as CMO at Vertex at the time Warner Green published his work testing VX 765 in HIV infected human tissue was no coincidence. Now that thephase 3 809/770 CF data has been released, and is positive, Vertex will likely start advancing some of it's non-CF R&D pipeline assets as well, and VX 765 is certainly advanced far enough from a safety standpoint in humans to resume it's testing in this new potentially breakthrough clinical application. Warner Green said in interviews last December that Vertex was negotiating with his Gladstone Institute at UCSF to start clinical trials and estimated those trials could start in a matter of months. That suggests an announcement for those clinical trials may be announced very soon.
As the second quarter wraps up tomorrow, any sense of how well Dexcom is competing with Medtronics for the CGM market, and whether they will beat street revenue/eps estimates for the quarter? The Type I diabetic community seems to like the Dexcom product better based on recent posting on blogs and social media posts. Is that trend translating into increasing sales? Are pediatric endocrinologists starting to prescribe CGN for their patients since Dexcom's product got approval in kids ages 2 and older earlier this year?
The major risk is currently out of the stock after the 'binary event' . Those who sold their shares on the advise of Geoff Porges before the release of the binary event TRAFFIC/TRANSPORT data last Tuesday have sellers remorse. Those who bought expensive put options or shorted Vertex before Tuesday lost a bundle. Those who bought shares this past week and have some patience will IMO at least double their money over the next 18 to 24 months. Currently, it's relatively safe to be long Vertex and not worry too much about day to day price fluctuations. Each positive data release on 770 monotherapy and 661/770 and 809/770 ongoing trials, as well as news about the initiation of addition clinical trials for both CF second gen correctors and non CF assets, and news of sNDA filing and NDA filings for 770 and 809 will bring increasing price per share going forward from now until 809/770 receives approval next year. Then the market will reward the stock on a ten fold increase in revenue over the next two years. Enjoy the ride.
The facts are that preclinical tests in theoretical competitors are years away from proving efficacy and safety. The facts are that since 809/770 has sustained benefit in slowing the loss of lung function in 508dd XF patients, it is the first and only approvable treatment that can make an impact on prolonging life in this otherwise fatal disease. In younger patients, it will prevents progression of irreversible lung damage. Sure competition is on the horizon, and the most advanced competitor is coming from Vertex itself, in the form of VX 661 and second gen competitors already in, or about to enter clinical trials (not preclinical testing years behind Vertex). Vertex owns this space for the next few years and long term, (after telaprevir's rise and fall) VRTX will certainly be aggressive in developing and/or acquiring next generation treatments to improve outcomes and expand the CF treatment population. .
The conference call this morning was excellent and spells out the success of TRAFFIC/TRANSPORT, Verity, Third, Pappa, Glad, Harvey, and all longs;;;;;;; Congratulations for your patience and faith in this long journey, which is just starting to deliver on it's promise. VX 661 and second generation correctors will likely improve outcomes even greater for the CF community and the revenue from 809/770 will allow vertex to progress those assets plus further develop the non-CF pipeline of drugs to treat autoimmune diseases, cancer, neurologic diseases, and HIV.
The WF investor conference presentation by Quidel's CEO in Boston yesterday, was very upbeat about the future potential growth from all the new molecular diagnostic products..Mutual fund analysts were likely present and liked what they heard, resulting in the further bump in stock price this week. Good to know Fidelity was taking such a large position, which will likely be for the long term to reap the growth anticipated from all the new products being launched.
Boomer Esiason is a major fund raiser for the CFF. Listen to the above interview but especially the 6:00 to 6:30 minute mark in this interview, where Boomer answers the question "do you expect a cure?" He talks about the amazing beneficial effects his 23 yo son, Gunnar, who has CF, is experiencing on a study drug with results of study soon to be released. Sounds like VX 809/770 to me, which is going to have a profound effect on CF patients. Its going to allow patients like Gunnar Esiason the chance to outlive their parents.
I think giving the BOD the rights to purchase shares at $72 just before the release of the combo data sounds like good news is coming for all shareholders, and especially for the cf community!