Today's announced invitation of Vertex Regeneron, and Merck execs to the White House this Friday certainly is a sign that the President's personalized medicine initiative mentioned in his SOTU speech is moving forward with these companies likely to be the first recipients of any tax dollars earmarked for R&D into the White House 'pet' research preferences. (Much the same thing occurred in the 1960's when then President Richard Nixon declared his famous war on cancer and earmarked federal funds for cancer research,)
A more important opportunity on Friday is for these execs to remind the president that drugs like lumcaftor/ivacaftor, which already have 'breakthrough drug status' and have completed all required clinical trials, still require another 6 months or more of review (before the PDUFA date of July 5, 2015 in lumacaftor/ivacaftor's case). Why does a 'breakthrough drug' monitored in it's development at every step for the last 5 years by the FDA need six more months to get approval, after all the data has been submitted? The inefficiencies of the federal regulatory process need to be reviewed by the White House as part of his 'accountable care'. CF patients lives are depending on this.
If 398 has such long odds for success then why did Baxter partner with Mack? I suspect Baxter's analysis of the potential of 398 was quite different from yours (and likely a lot more accurate) since they put up a lot of money to co-develop and seek regulatory approval and market 398 ex-US. Baxter also has shown considerable interest in Mack when Baxter closed their San Diego and European research labs and moved them to Cambridge to be literally 'next door neighbors' to Mack in Kendall Square. The Mack drug discovery platform and drug pipeline has tremendous potential to change the treatment of cancer and the coming year is more than likely to result in commercial success of 398 which will finance the development of the earlier stage assets in the Mack R&D pipeline.
Bill Elder, a CF patient (who is also a medical student in Ohio) with G551 mutation and is on treatment with Kalydeco, and is also an active CF advocacy group spokesperson, was a guest of Michelle Obama at the SOTU speech last night. Articles in the WSJ and Bloomberg this morning are appearing mentioning him and the 'personalized medicine' initiative that Obama proposed in his speech, using Bill Elder as an example of the success using this approach. Hopefully this means the White House is listening to the importance of the rapid approval of these drugs for the CF community. From a broader perspective Obama mentioned the use of the genetic basis of disease for creating personalized medicine to treat diabetes and cancer to benefit larger segments of the population. This fits nicely with Vertex new appointment of geneticist/endocrinologist David Altschuler MD as CSO, whose career at Harvard and the Broad Institute has been focused on this approach to treating diabetes and other diseases.
Now lets see the FDA act swiftly to allow early approval of this breakthrough treatment for this deadly disease. CF support groups should grab this opportunity to press the White House to have the FDA move to quickly to approve 809/770, a drug which already has been given the FDA's 'breakthrough drug designation' to slow the progression of CF in the majority of CF patients.
VX 661/770 interim phase 2 data. Hopefully out in the next couple of weeks around the EOY/4thQTR conference call just prior to the announced earlier than expected start of pivotal Phase 3 clinical trials using VX 661 in both 508dd homozygotes and selected subgroups of 508 heterozygotes. That plus announcement of results for new oncology drugs already in clinical trials and the start of clinical trials in CF with second gen correctors in combination with 661/770 and drugs to treat chronic MS and Huntington's.
Any guesses as to the market's reaction tomorrow? I suspect big move up and new 52 week highs as the optimism implied in the ambitious 2015 R&D timetable to be presented tomorrow will make analysts raise their future revenue and earning estimates for Vertex.
It would be nice to hear positive phase 2 VX 661/770 clinical trial results treating 508d homozygote CF patitents at next Monday's presentation. It would certainly make the citi biotech analyst's report released today look stupid, and push the stock up to new 52 week highs. Even without releasing the 661/770 data, just an upbeat presentation and timeline for 2015 R&D milestones and expected approvals for 809/770 and the progress in the cf and non cf pipeline will likely be a catalyst for the stock.
Speculation that phase 2 VX 661/770 results will be positive seems to have the stock moving very quickly in the first hour of trading of 2015! Happy New Year to all longs and the CF community!
QDEL share price moving u[p on volume as severe influenza outbreak gets more press. The last quarter of 2014 and first quarter of 2015 will likely beat estimates given the uptick in visits of patients to ERs and urgent care clinics and pediatricians all screening patients for influenza, RSV strep with QUIDEL POC diagnostic tests. Sofia and Amplivue sales should pick up with the recent FDA approvals of a wider number of tests available for testing on these platforms. Positive Savanah clinical trial results will be the next catalyst for a huge rise in potential revenue for making a low cost POC HIV viral load test used to diagnose and determine response to treatment.
Happy New Year to you Rojo as well as all the patient Vertex longs and the CF community who are finally getting the reward they have waited for. The JP Morgan Healthcare conference starting in 2 weeks in San Francisco will hopefully give the outline of expected progress in both the expanding CF and non CF pipeline from Vertex in 2015. VX 661 Phase 2 data treating homozygotes and the advancement of this drug into Phase 3 clinical trials for treatment of both 508d heterozygotes as well as homozygotes, with or without second gen correctors, in combination with Ivacaftor, will be a focus of analysts, along with the approval timeline and potential pricing for 809/770 in 508d homozygotes in 2015. The non CF pipeline may finally get mention by Vertex at JP Morgan as well including VX 787, in development by Janssen, in light of bad influenza season already in progress in the US, The two Vertex drugs in Phase 1 clinical trials for cancer, VX 803 and 970, may also finally get mentioned. Hopefully Huntington's Disease and/or chronic MS clinical drug trials are also moving forward, after years of pre-clinical investigation. The treatment of the genetic basis for CF, Huntington's etc,, as well as Diabetes type 2 may be the future focus of Vertex research given the expertise of David Altshuler in his new role as VRTX CSO. It should be an exciting 2015 for Vertex longs!
Newly approved amplivue pertusiss test and clia waived strep tests on sofia platform will boost sales in the coming year. Fidelity (FMR) recently filed 13-G form with SEC (they increased their position to owning 7.5% of QDEL shares). Savanah clinical trials should be out in the first half of 2015..... approval likely in 2015, This company is about to dramatically grow it's sales and could easily be a buyout target.
Replacement for Peter Mueller is David Altschuler, already on the Vertex BOD and scientific advisory committee, and one of the pre-imminent medical scientists at Harvard med school. A geneticist, he has the background to address the underlying genetic basis of many diseases, which include his past focus which was diabetes, as well as the genetics basis of Vertex current pipeline e,g, CF, cancer, neurologic diseases etc.
Both an M.D. and Phd, Altshuler will bring both clinical and research experience to the CSO job. He knows Vertex well having been on it's BOD for the past few years, and will likely bring fresh ideas to the Vertex R&D efforts.
Terry Gregg, Dexcom CEO announced FDA approval of Animus vibe pump and the fact an additional one hundred people from the Animus/JNJ sales force will be selling Dexcom's sensor with their Animus pump.
Ian Smith's DB investor conference presentation worth listening to. Highlights include not only positive spin on rapid adoption and high value for 809/770 in the U.S., but finally a discussion of non-CF pipeline development programs including cancer and neurologic disease. Market reacting positively with stock making new 52 week highs every day this week.
Michael Yee, analyst from RBC, quoted in Barron's this week:
'Vertex should be a long into early ’15 Phase II VX-661 3-month homozygous data which should look better than first-gen ’809 combo. We want to own into potential ’809 strong pricing (we think possibly higher than $225,000 gross price) and USA launch for mid-15'
DB investor conference presentation tomorrow
Continued raising of PT of stock by analysts e.g. Jason Kolbert at Maxim raising his PT to $148 today citing underestimate of Vertex future CF revenue by the street.
FDA approval of sNDA for use of Ivacaftor in treatment of R117 mutation and EU approval for the same to follow.
Anticipation of VX 661/770 Phase 2 clinical trial data treating 508d homozygotes to be released in1Q 2015, possibly before JP Morgan biotech investor conference in early January.
S&P analyst predicts peak sales of VRTX CF meds of $6-7 Billion/year. I think that is a very conservative estimate. My prediction after VX 661 is approved is $8-10 billion/year.
Andrew Fein always sees the worst scenarios possible for VRTX to justify his opinion and price target on the stock. With FDA approvals and huge sales imminent his next thesis is potential competition and a crowded CF space that is untested, and not even close to representing true competition. He and Geoff Porges are propping up short positions in the stock.