Vertex Pharmaceuticals Incorporated Message Board

What's next continued....

5. Final results of the positive VX 661/770 Phase 2 data at the fall national CF meeting, along with the announcement of second degneration correctors being initiated in clinical trials for use in combination with VX 661 or 983 to treat CF 508d heterozygotes. Also announcement of completion of enrollment of Phase 3 clinical tirilas using VX 809 and 770 in 508d homozygotes with completion of those results and submission for NDA approval to treat another 50% of the worldside CF population in the first half of 2014.

Excellent presentation by Quidel's CEO, Douglas Bryant. Well worth listening to. Rest of this year will bring lots of of new molecular tests to the company's growing portfolio of rapid diagnostic tests. Early reception by the customers has been impressive, and I suspect second and third quarter revenues and earnings will be higher than current analyst estimates. This company has an ambitious growth plan, and appears to be executing it well. As it grows revenues and market share, it may become a takeover target by a larger diagnostics company looking to grow their molecular diagnostic product line.

VX 135 Daclatasvir combo trial posted on clinicaltrials.gov, not yet recruiting, but likely to start enrollment soon. 12 week interim EOT reuslts, and 4 week SVR data shoud be out before AASLD this fall.

IMHO the mutual funds who bought over 25 million shares of VRTX at over $80 a share after the 661/770 data was released last month, will easily buy up the 8.2 million shares of Vertex that may be sold by current bond holders after VRTX calls in the bonds and converts them into shares. The price of those shares IMO will probably be right where the shares have been trading the past week, if not higher,, and are likely to move up from the current price as the shorts, who have been betting on a further price drop, scramble to cover when that price drop fails to materialize. In the next two weeks we'll see how this works out. Intermediate, to long term investors will be glad to have bought shares at current prices.

Also a site in Florida is actively recruting Phase 3 809/770 patients.

Red, Best of luck with your child's results in the Phase 3 study. The clinicaltrials.gov web site states 3 sites are actively recruiting patients in South Dakota, Oklahoma, and Minnesota, with the other 107 sites 'not yet recruiting' so I suspect the IRB approvals are slowy coming along at each site causing the frustrating postponement of enrollment for some families like yourself, eager to start treatment. Please let us know how things are progressing for your child's enrollment and particpation in the trial, as we are all rooting for him or her to get well!
Best,
Q

Third, my understanding is that the Phase 3 trial is up and running with the only bottleneck in enrolling patients at each specific site being the approval required by IRB committees/or comparable local regulatory bodies at sites outside of the U.S. Antibiotic dosing is not specified in the protocol on the clinical trial.gov site, but one can be sure all factors relevant to patient response to treatment is being included in the data to be submitted to the FDA. Once IRB approval is obtained at each site, CRO personnel are geared up to rapidly activate each site for enrolling patients. Given the huge interest of the patients and caregivers in advancing this desparately needed treatment, a rapid enrollment is expected with interim six month data likely to be out in the first quarter of 2014. Postive results would likely make the 'breakthrough drug designation' given to 809/770 a green light for rapid NDA filing and approval in a few months, with treatment approved for use in the US in the second half of 2014.

Both 'transport' and 'traffic' K+809 Phase 3 studies started enrollment.. Check clinicaltrial.gov and type in VX 809 in the search bar and you can monitor the initiation of the enrollment progress at the various sites as they start dosing patients.

Bootpart, I would not worry too much about the time it's taking to announce partnerships to develop VX 787 and 509. The terms of the partnership, specifically milestone payments, royalties for future sales, and division of any shared research expense and regulatory submissions may be more favorable to Vertex with potential large pharma partners, if the management at Vertex waits for additional positive VX 509 clinical trial results due in the second half of this year, and for results of testing VX 787 sesitivity in H7N9 influenza vial isolates recently provided last month to the CDC by Chinese and WHO researchers. Obviously negotiating the best possible deal for these assests owned by Vertex will be more likely by demonstrating as much efficacy and safety in all the trials still pending both in the clinic as well as the lab where in vitro or in vivo animal studies of efficacy and toxicology studies are likely ongoing. The development of these assests going forward, and the regualtory approval process for their eventual sale worldwide will be costly and time demanding. The sales of these drugs worldwide will require both a large sales force and a large marketing budgetary committment. Negotiating these terms is best done with patience and the strongest data available to value the deals being made.

...and next year Vertex will be able to provide 'breakthrough' treatment for the majority of CF patients:

S&P analyst's remarks on May 1: ".We remain
confident in VRTX's cystic fibrosis program, with key F508del mutation sub-group
on track for '14 FDA filing. "

Count on expdited approval by the FDA in 2014 to treat close to twenty thousand CF patients in the US alone. That's multibillion dollar a year market. so 300 million of lost Incivek revenue won't be a huge problem going forward, and VX 135 will likely generate far more in the years to come. Then factor in the poetential for VX 509, 787, 661 983, and the second gneration correctors for treating the 508d heterozygotes with CF, and the future for Vertex is amazing.

High SVR rates from past clinical trials combining 7977 with BMY's daclatasvir, makes the upcoming VX 135 and daclatasvir combo the Phase 2 trial of most interest among the hep C program at Vertex, and the fact that it is the first hep C collaborative trial getting underway this quarater by Vertex, makes it among the most important clinical data to be announced in the the second half of the year A twelve week trial would likely have results available for AASLD in the fall. That combined with Kalydeco Phase 3 trial results for non 55lD mutations due this summer and fall, and VX 509 data due in the second half, will create plenty of catalysts for VRTX stock over the remainder of the year. And of course announcements of any licensing or partnerships with VX 787 and 509 will likely provide milestone payments to Vertex which will enhance it's financial postiion while allowing more rapid development of these assests as well. Certainly news flow about the completion of enrollment of all the CF studies including Phase 3 809/770 as well as the remaining Kalydeco monotherapy trials will be of great intsserest as well, along with announcements of further trials of VX661 and 983 and newer second generation correctors to be combined for use in ther CF 508 heterozygotes in clinical trials next year. Lots of things to look forward to for the reaminder of the year!

Rojospan. We'll get a clearer read from Vertex management of how 809/770 enrollment is proceeding at the CC on Tues. The clinical trial.gov website lists 110 international sites enrolling about 1000 patients. Not a small logistical feat for any pharma studying so many parameters in a very sick patient population. I suspect however enrollment will be complete before the end of June, given the time elapsed since announcement ot a registration path, and the high interest in CF patients for being in studies that could halt the progression of their otherwise terminal disease. Verity might be able to give us information from her perspective as to how quickly this study is being enrolled from the CF patient family's point of view.
I hope we'll also get some information on Tuesday regarding the timing of initiation of clinical trials using VX 135 with GSK, JNJ, and BMY's compounds, in pan-genotypic populations of hepatitis C. Gilead will have maybe one year before serious competition in next generation all oral treatment of hep C evolves from Vertex and it's potential partners, with potentially greater efficacy in all genotypes and hard to treat subsets of hep C.
Also, hope for news on Tuesday about the partnerships being negotiated using VX 509 for treatment of multiple autoimmune diseases, and VX 787 for treatment of both seasonal and pandemic strains of influenza. This latter drug as well as VX 509 has been largely ignored by analysts in their valuation of the Vertex pipeline, and any announced deal will give color to the potential revenue associated with each drug. Both compounds may have significant improvements for treating their respective patient populations over existing treaments in either efficacy and or tolerability. The ongoing evolution of mutaions of avian H7N9 influenza currently being monitored in China makes development of VX 787 particularly important if person to person transmission develops in this potentially deadly virus.

Instead of a buyout , long term VRTX investors would do better for the company to stay independent, with the stock likely to spike higher in the short to medium term with the eventual announcement of lucrative partnerships with larger pharmas to devleop VX 787 and VX 509. Then watch the stock fly as clinical trials start to be reported through the remainder of the year. Three Kalydeco monotherapy Phase 3 trials in non G551 mutations will be out this summer, and VX 509 Phase 2 trial in RA this fall, along with VX 135 all oral hep C treatments being tested with BMY, JNJ and GSK compounds. And of course, Phase 3 809/770 six month interim data due either at the end of this year or start of 2014, to allow NDA submission. With a nearly $18 billion market cap, expect addition of VRTX to the S&P 500, with the subsequent required purchase of VRTX by all the index mutual funds. Enjoy the ride.......

Hi Verity. Just wanted to share my good wishes and congratulations on the implications for the 661/770 Phase 2 trial results for the CF population. All the analysts are finally acknowleging the clinical effectivenessl of combining corrector small molecule drugs and 770 to improve CFTR function in the majority of CF patients Not only is 661 validated as a future treatment in 508 homozygotes but now, as you have pointed out the combination of multiple correctors with 770 will be explored in the heterozygotes as well, and optimism about postive results in Phase 3 809/770 clinical trials currently underway, suggests approvalble treatment for the majority of CF patients by next year, given the 'breakthrough' drug designation given to comibination therapy with 809 and 770 by the FDA, and the liklihood of rapid regulatory approval follwing submission early next year.