What a great surprise for the holidays. Monetizing vx 765 would be a huge unexpected revenue stream on top of CF. It may have greater commercial potential to treat HIV worldwide than vx 509 has in autoimmune disease, given the current competition in the RA/autoimmune market. Hopefully Vertex will retain majority control of the commercial potential of this asset as it develops a collaboration agreement.
The difference is that since Vertex owns the molecule for which there is no apparent competitor in this indication, and since VX 765 has been proven safe if prior Phase 1 and 2A clinical trials already run by Vertex (in psoriasis and epilepsy) Vertex could run a small Phase 2A HIV clinical trial with or without a partner. Gladstone Institute scientists' in vitro studies showed the potential of using VX 765 in HIV treatment. Vertex controls the use of their drug. Remember Vertex has experience with HIV drug development with their first commercially approved drug and co-developed it with Glaxo which eventually bought out the rights to that drug. I'm sure Glaxo and others would be interested if a Phase 2A study showed positive results.
I was hoping to hear the opinions of Third regarding the articles in Nature and Science that demonstrate the therapeutic potential to use VX 765 to treat HIV infection. Any comments Third?
Thank you Third. VX 765 may be an important addition to ART therapy. Given it's known safety profile, it would be easy to collaborate with UCSF clinicians and other HIV research clinics worldwide to run phase two clinical trials using VX 765 with ART to determine efficacy in eradicating the 'reservoir' of HIV, reducing the incidence of AIDS related dementia and cancer, as well as the efficacy of VX 765 alone or in combination with ART in eradicating HIV in infected patients who have not yet developed AIDS. There are no guarantees in the outcomes of future clinical research, but the Nature and Science articles are encouraging, and VX 765 may finally have an important contribution to make as a 'breakthrough' improvement in HIV treatment. Initial Phase 2A clinical trials to demonstrate proof of concept would not be too expensive for Vertex to run and would IMHO be a worthwhile investment.
Widespread Influenza reported in four states, with this week's Christmas travel likely to greatly expand the number of cases, Influenza testing should spike and Sofia sales likely to continue to ramp up, which may explain QDEL's upward stock price move on Friday.
QDEL up over $3 after hours on news of second Quidel amplivue molecular test approval by the FDA for Beta Streptococcal infections that could be a significant source of revenue going forward. With over 17% of the outstanding QDEL shares short, the stock could move up quickly, especially after Canacord analyst reiterated a $35 price target and it was mentioned as a 2014 small cap pick by William Blair analysts.
Happy New Year to you Third, Pappa, and all the VERTEX longs. It was an exciting 2013, and will be even more exciting in 2014! .
It's just the start of a big run in 2014 for QDEL as the new products approved start to generate sales on top of the influenza season just getting underway, and the rest of the new products in development get approval, and eventual sales on top of the molecular diagnostics already approved. Great intermediate and long term growth prospects.
It is still possible that a licensing deal will be announced for VX 787, as well as for VX 509 and VX 765, perhaps at the JP Morgan healthcare conference on Jan 14. On the other hand, large pharma companies may not be willing to license drugs until completion of Phase 3 trials. Vertex executives may have changed their strategy and chose to develop these drugs themselves, after the CF (VX 809 and 770) start generating expected large revenue streams sufficient to allow Vertex to develop the rest of it's drug pipeline independently.
No mention of elevated LFTs using the 200 mg dose. FDA likely to reapprove testing VX 135 in 200 mg dose range in the U.S. in Phase 2B trials with Daclatasvir.
Market potential (In the billions per year) will come down to speed in progression to regulatory approval, and ultimately once approved, it will compete based on pricing, efficacy in the various subpopulations of hep C (various genotypes, prior treatment failures, HgIV co-infected, liver transplants) convenience of dosing and length of treatment needed, and of course tolerability/side effects.
First hurdles will be getting FDA to approve testing VX 135 in the US at the 200mg dose once again, and of course BMY willing to partner to license VX 135 for development with Daclatasvir.,
The stock is recovering nicely going into next Tuesday morning's presentation by Jeff Leiden in SF at the JP Morgan healthcare conference. Besides outlining the progress in CF already known and now renewed interest in hep C with VX 135, the catalysts for the stock to test it's highs from last year could include mention of the following:
1. Successful discussion with FDA announcing release of the partial hold on testing VX 135 in the 200mg dose in Phase 2B clinical trials, and planned initiation of those trials in combination with BMY's daclatisvir in the first half of this year.
2. Timeline for release of VX 661/770 data treating CF 508d heterozygotes and homozygotes in the first half of this year, and the projections for starting Phase 3 trials using 661 or 809 and 770 with and without an additional second generation CFTR corrector in treating CF 508d heterozygotes.
3. Reaffirming time of release of VX 809/770 Phase 3 clinical trial data in the spring of this year with projected NDA filing for treatment of CF 508d homozygous patients this summer, and with 'breakthrough drug designation' by the FDA, allowing early FDA action, allowing sales of VX 809/770 to over 50% of the US CF population in the second half of this year, along with the expected filing and approval for the expanded label for Kalydeco monotherapy treatment in 7,000 CF patients whose mutations qualify.
4. Possible licensing deals with large pharma or the decision by Vertex execs to develop it earlier pipeline assets independently, specifically VX 509 for autoimmune disease, VX 787 for influenza, and VX 765 for HIV.
5. Announcement of which pre-clinical drug candidates (e,g, second generation correctors for CF, chronic MS and oncology drugs) may be advanced to clinical trials after projected sales of VX 809/770 in the second half make the company cash flow positive.
Based on today's news release for 2014 business goals, most all resources and focus for Vertex R&D are going to be limited to CF, at least until 809 and 770 gets FDA approval to generate enough revenue to pay for Vertex to develop it's earlier pipeline assets. Probably the right thing to do since trying to develop multiple drugs in several different disease states at once has proven to be slow to bear fruit.
Early release of better than expected 4th qtr revenue bodes well for positive outlook to be presented by QDEL CEO at JP Morgan conference in SF on Wednesday. Increased institutional support likely based on numerous products recently approved for sale and Savannah product coming next year. Buy out potential by a larger diagnostics company a strong possibility as well, as the new products begin to increase top and bottom line estimates by analysts
Vertex execs cannot forecast revenue for K+809 sales or expanded label use of kalydeco revenue outside of approved indications until final Phase 3 data and NDAs are submitted and FDA approval and pricing by health insurance carriers is worked out. But that leaves a lot of upside revenue and earnings potential for the second half of 2014, when positive phase 3 data is reported by June of this year in the various CF clinical trials approaching completion.
Dr. Leiden delivered an effective presentation, well received by investors based on stock price following his appearance at the conference. Clearly the traffic/transport studies are felt by most analysts to be powered to achieve statistically significant improvements in FEV-1, the primary end point, and hopefully statistically significant improvements in the secondary clinical endpoints e.g weight gain, quality of life, hospitalizations etc. The clinical trial results to be released sequentially between now and June, if positive, will continue to build investor enthusiasm for VRTX culminating in the results for TRAFFIC & TRANSPORTthis spring.
Adam Feuerstein, TSC's biotech reporter, predicts VRTX will double it's stock price this year after announcing positive Phase 3 clinical trial results using VX 809 and 770 to treat the most common CFTR mutation causing CF. He's not too worried about current eps and cash flow since revenue and earnings will rapidly grow upon FDA approval of these CF drugs in the second half of this year. The Street seems to agree with this prediction, given the current stock price following the Vertex presentation this past week in SF at the JP Morgan healthcare conference.
The institutional investors who bought these 'insider' shares this week, have since driven the price per share up an additional four dollars since the $78 sales price on January 15 They recognize the growth potential of VRTX from its CF drugs about to get FDA approval later this year. Informed investors would not be worried about executives at Vertex cashing in some of their stock-option based salary to pay their bills and quarterly taxes. Incidentally, the overall core long-term stock holdings of these two executives remains the same after exercising these current options. .
New Vertex CMO, a specialist in infectious disease, has the experience from his work at Merck in early and late stage drug development to advance VX 787 to treat influenza, as well as VX 765 to treat HIV. Maybe the company will announce plans to further develop these assets once Phase 3 VX 809 data is announced and signals positive cash flow for Vertex to fund additional drug development in the second half of this year.