The most recent Form 10-Q mentions pipeline including oncology, MS, and flu drug VX-787 and its backup VX-353. 10-Q also states that the IVA+LUM combo addressable group is greater than 22K excluding young people of 11 year old or younger.
I speculate that the reason they don't mention VX-765 in relation to AIDS is that they want to make sure that it works in alive human patients. The preclinical results are so good that it takes equally good clinical results to convert doubters. Also, they don't forget the demise of VX-222 and -135.
As Rojospan found out, Vertex is running a clinical trial of VX-970 which potentiates tumor destruction by inhibiting tumor DNA damage repair. The targeted patients have advanced squamous non-small-cell lung carcinoma, or small-cell lung carcinoma. Surgery or radiation is out of question for these patients although for localized solid tumors VX-970 would be an ideal drug to potentiate radiotherapy.
VX-970 is an inhibitor of ATR, which along with ATM and DNA PK is a signaling molecule to trigger DNA damage repair and also to stop cell-cycling. Etoposide, gemcitabine, and/or cisplatin are DNA damaging agents to be given along with VX-970. The scientific basis of this trial is highly impressive, which was published a month ago. The management will probably disclose the cancer program along with VX-765 based AIDS treatment trial, and MS program.
There is a good indicator to predict the rate of uptake of IVA and LUMA post-launch. We know that there are 22000 12 year or older 508del homozygotes in major markets. But not all of 22000 will be taking the combo in the first full year of marketing. Safety and efficacy of the combo would build compliance and loyalty to drug regimen. But those alone cannot tell directly what the treatment participation will be.
On June 24 the PR reported that more than 1000 out of 1100 who had taken part in the Phase III have decided to participate in the rollover study. The is greater than 90% participation rate. This indicates a very high loyalty. This indicates to me that a large percentage of 508del homozygotes will choose the option of taking the combo when the combo becomes available. One of reasons why the drug combo is so popular might be related to the decrease in the number of pulmonary exacerbations; the decreases are 30% and 39% for the 600 mg Qd and 400 mg Q12h regimens, respectively.
My calculation shows that from the sales of Kalydeco and VX-809 alone the share price will reach 200 in two years on the basis of very conservative numerical assumptions. Today's share price is really low.
Glad, how are you doing in this ridiculous assault on the share price? I have been buying yesterday and today. As for the conf call, there is a real need of appointing a sharp Chief Scientific Officer who can quickly learn every detail of drugs in development. A CMO alone cannot handle it all.
Stay in two years. You can more than double your investment. Gilead, Celgene, or Regeneron would not double in two years.
The articles by Veit et al. (McGill U. group) and Cholon et al. (UNC group) in the July 23 issue of Science Translational Medicine reported results of their independently obtained cell culture work into the effects of Ivacaftor on F508del CFTR that has been transported to, and localized to the plasma membrane by the action of Lumacaftor. A preliminary results of the UNC group had been presented in the European CF conference last year. Analysts asked about those at the conference. Van Goor responded to the questions. So, Vertex is well aware of their work. What is new is that their results in the two papers are complementary in a number of observations and support the conclusion that Ivacaftor rolls back, to a degree when dosed chronically, the very rescue action engineered by Lumacaftor. All in cell culture. However, the two reports contradict each other in a major way on the effect of Ivacaftor on wild type (normal) CFTR. The work of Veit et al. reports that there is no effect of chronically delivered Ivacaftor on the function of wild type CFTR, but the work of the UNC group reports a large effect. This latter work, of course, contradicts the superb clinical efficacy observed with Ivacaftor, which have been dosed for three years continuously.
Nevertheless, their work appear to support the notion that the putative antagonism within the synergy between Iva and Luma is the root cause of rather modest efficacy of the combo in treating homozygous F508del CFers.
Papa, the company succeeded in transferring genes to red blood cells is bluebird bio. I agree that gene therapy for CF would be a big challenge and it may take more than 10 years, but transplanting normal CFTR to the lung cell nucleus are being attempted in UK as I said before.
Barron's had the following:
Expect Gilead Sciences (GILD) to post a “blowout” quarter, says Bernstein’s Geoffrey Porges and Wen Shi:
Based on our analysis of domestic product sales trends, likely effects of currency, shipping days, weather and other seasonal effects we are quite positive about the outlook for the approaching earnings season for the biotech stocks in our coverage. It is worth remembering that Q2 is typically the best quarter of the year for biotech companies across the board – Q1 headwinds from co-payments, deductibles and “donut holes”
are over, shipping days usually peak, weather effects are minimal and management teams are inclined to signal more of their full and realistic earnings potential for the year, lest they be accused of “sandbagging” for the year after a decent first half. Clearly the most likely company to surprise to the upside is Gilead…
We increased our forecast for Truvada, Complera, and Stribild, and trimmed our forecast for Viread and Atripla. Our analysis points to US Sovaldi revenue above $3bn, well above recent consensus of $2.6bn; we are also expecting $700mm in OUS revenue for Sovaldi in the quarter. Our total revenue forecast for Q2 is now over $7bn, or 24% above recent consensus. Our EPS estimate is $2.24, or 35% above recent consensus.
I saw a Market Talk headline yesterday, but I did not know the source. Will they pay a 50% premium? It should not be sold below 150/shr.
I consider Kalydeco is a more secure drug than Sovaldi in the sense that Kalydeco opened up a new life to a lot of people and there are no other alternative to treat at the very basic level.
If you had sold shares at 60's, buy them before they go over 100. You can recover the loss in a year. You can also make money by selling put options for the future months. Put premiums are still quite high... to buy 1 Contract of Jan put at sp of 85 cost you around $570, but if you SELL the same put, you can collect 570 per Contract. If the VRTX share price ever goes below 85, then you have to buy them at 85 in January.
Verity, the Traffic trial yielded the absolute change of 3.6% for 600mg Lum within the group, and 4% relative to placebo. The Transport yielded a lower value of 2.6%. These compared with the Phase II 4+4 wk data of 3.4% within group and 6.7% relative to the placebo. All numbers quoted above were absolute ones. The Ph II data were noisy. As you stated, Ph II data of 661/Iva combo gave slightly better results. After 4 wks' of the 100mg 661 dosing, FEV1 changed by 4.8% absolute (9% relative) within group as well as relative to placebo.
The most important results from the Ph III data are not FEV1 changes, but the significant reduction in the frequency of pulmonary exacerbations that participants experienced, and the high number(90%) of participants in the rollover study.
Vertex is borrowing $300M at an interest rate of 7.2%. The purpose for this loan is said to be related to strengthening financial position:
[[ The credit agreement is intended to strengthen our financial position as we invest in our key development programs in cystic fibrosis and in research for future medicines, including potential collaborations to support priority programs. ]]
Vertex may need funds for supporting Phase 2 trials of reversing, and blocking AIDS using caspase-1 inhibitor, VX-765.
Agree 100%. Selection of a 2nd gen corrector and start of clinical trials of triple combo will be another sp moving events just as the FDA marketing approval of 508 homozygote treatment. An optimistic disclosure of the collaboration with Greene and co. for HIV treatment using VX-765 would be a lifting event also.
I think it was Q2 earnings call, not Q1, when Mueller mention gene therapy. In a year or two Vertex will have money to partner with, or acquire a promising CF drug companies.
In response to analyst's query into future drug research in the Q1 E CC, Peter Mueller mentioned that he is looking into gene therapy. He didn't specify what disease the gene therapy targeted at the time, but I suspected it was CF therapy by the context. A UK consortium is running a clinical trial since 2012. Bluemoon appeared to have succeeded in treating a rare disease using lentivirus recently, and Bayer also has committed to work with Dimension Th. for hemophilia A. I am certain that Vertex is intensely watching the development. As you suggested, Vertex learned an important lesson from Incivek debacle.
I liked the phase 2 result of their JAK1 inhibitor for RA. They still have not started phase 3. I would think that even if their potentiator is effective and safe it won't be out for at least 3 years and their corrector for 6 years. Their potentiator has to compete against the VX-661+Kalydeco combo for 551/508 heterozygotes.
Geoffrey Porges also changed his mind.
Vertex Pharm tgt raised to $107 from $65 at Bernstein
Vertex Pharm tgt raised to $115 from $93 at UBS; Buy
Vertex Pharm target raised to $125 from $100 at Leerink Partners; Outperform
Needham raises their VRTX tgt to $115 from $95 given favorable outcome and
substantial commercial opportunity. Co announced positive results today from
two Phase 3 trials of ivacaftor/ lumacaftor (TRAFFIC/TRANSPORT) in Cystic
Fibrosis patients with two copies of the F508del mutation. Both trials met the
primary endpoint as well as key secondary endpoints. A trend toward
improvement with respect to a quality of life metric was also observed.
Although impact on lung function was moderate, overall data suggest a
clinically meaningful and commercially viable drug regimen; maintain Buy.
Vertex Pharm: Hearing Tier 1 firm raising tgt to $130 from $100 following data
Maxim Group raises their VRTX tgt to $107 from $92; their valuation is driven
by the potential of a Kalydeco/corrector combination expanding the franchise
in CF. As they further understand the totality of the Vertex data we will
review their market size calculations which may show our valuation is