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!
More likely JNJ would buy out DXCM, since it's finger stick blood testing franchise is threatened by this technology and it's insulin pump sensing capability is being co-developed with DXCM.
Needham & Company analyst Alan Carr weighed in on Vertex (NASDAQ: VRTX) following top-line results from a Phase 2 trial of ivacaftor in cystic fibrosis patients with residual function CFTR mutations.
"We note statistically significant, but modest absolute changes in FEV1 in the two-part trial (2.8% vs. 0.6% placebo [p=0.004] in 2-wk blinded Part 1 and 4.7% [p=0.0001] in 8-wk open-label Part 2)," Carr said. "Based on these results, the company plans to move forward with a Phase 3 trial after discussions with regulators around trial design. Results from the pivotal TRAFFIC and TRANSPORT trials are expected soon. These trials are testing ivacaftor/lumacaftor in homozygous F508del pts and are at the core of the company's CF strategy. We continue to expect a positive outcome and reiterate our BUY rating and $95 target."
Verity, I agree this is great news. By the way, what happened regarding the FDA's decision to approve Kalydeco monotherapy to treat r117 residual function patients age 18 and older? I thought Vertex had been in discussions with the FDA over the past six months to apply for this expanded use of Kalydeco based on the data from the phase 3 trial announced back in December?
Phase 3 study to be started which would allow 3000 more patients to be approved to use kalydeco monotherapy.
And the date of the presentation at the GS investor conference is on the day preceding the start of the european annual CFF research meeting in Sweden....... that would certainly be an appropriate time for the TRAFFIC/TRANSPORT data release.
This position is for eventually marketing both Kalydeco as a monotherapy for all approved genotypes and VX 809/770 combination therapy when approved for use in 508d homozygotes CF patiaents as well.
Key Account Manager - NWS & QLD
Functional Area: General Business / Operations
Vertex Pharmaceuticals Inc. is seeking a Therapeutic
Specialist (Key Account Manager) to be a part of the exciting launch effort of an innovative
breakthrough treatment for Cystic Fibrosis (CF). The Therapeutic Specialist
will be responsible for ensuring that all barriers to usage of this new orphan
product are addressed and that all patients who could benefit from the product
have access to it. This goal will be achieved by the development and execution
of specific account level business plans at all CF centers; communicating
disease and product knowledge to healthcare professional customers and ensuring
that the full long term value of the product is fully understood by all
relevant stakeholders This elite individual must thrive in a fast-paced, highly
collaborative environment, drive for results, understand market dynamics,
demonstrate excellent judgment, and be motivated by the desire to improve
patients' lives. The ability to see the world through the eyes of the varied
customers and stakeholders with which this role will interact will be a
necessary and differentiating quality.
Associate Director, Technical Operations
Functional Area: Pharmaceutical Development, Operations and Quality
Location: Boston, MA
Vertex Pharmaceuticals creates new possibilities in medicine to cure diseases and improve people’s lives. The Associate Director, Technical Operations - Analytical is an exciting opportunity to join Vertex’s growing Technical Operations team and contribute to the rapid commercialization of new life- changing products. If you are looking for an entrepreneurial, non-hierarchical, hands-on, team-work oriented environment where you can have a big impact, then this is the perfect place for you!
The Associate Director will partner with late-stage product development teams in order to ensure the robustness of analytical methodology in a QC commercial setting by applying Quality by Design principles and will take a leadership role in its implementation at several contract manufacturing facilities. The Associate Director is expected to lead continuous improvement opportunities throughout method life-cycle and have a solid understanding of the regulatory and business impact of making changes to existing analytical technology in order to help define the appropriate regulatory and validation strategies to support post-approval changes.
The Associate Director will lead high-impact cross-functional initiatives, the resolution of complex analytical / manufacturing issues and provide strategic input to the commercialization of new products and the direction of the Technical Operations - Analytical group.
.... and do you think there will be a clinically significant difference between the two doses of VX 809 being tested in these pivotal phase three clinical trials??