EU CF meeting next month should give insight into efficacy of VX 661/770 as Phase 3 studies from VRTX are reported at this meeting. It may soon replace lumacaftor in 508dd cf with better results.as well as become standard treatment of gating mutations with a 508 allele if it demonstrates incremental benefit over kalydeco treatment alone. It will open the door for Vertex to treat the 508d heterozygote'het min'CF population with a second corrector,
Rojo, ENaC Monotherapy was not expected to yield positive clinical results in this initial Phase 2 trial, and the important outcome was 137 had a clean safety profile using it as monotherapy. The theory of clinical efficacy is based on improvements in ciliary beat frequency in combining this ENaC inhibitor with VRTX potentiator/corrector combinations in vitro. The clinical trials combining VX 137 with Orkambi or 661/kalydeco in the various subpopulations of CF mutations will proceed since that is where the positive clinical results based on the in vitro tests are most likely to show synergistic/additive clinical benefit. Obviously not every investment in acquired drug candidates pay off, but remember the Virochem acquisition, while failing to get viable hep C treatments, has succeeded in at this Canadian VRTX research lab in developing new drugs to treat IBD which are entering clinical trails this year as announced last week with a French privately held company. The cervical spinal cord injury drug candidate is another acquired drug candidate entering clinical trials this year representing a potential break through drug. Effective safe non-opiate painkillers would be medical advance to minimize the problem of addiction and dependence on opiates for chronic orthopedic pain patients suffering from failed back and neck treatments in addition to patients with pain from OA. With appropriate labelling on these drugs about avoiding overuse of OA joints when taking these drugs to prevent accelerating joint damage from uninhibited activity, in many patents, the risks of NSAIDS and opiates would certainly make VX 150 a preferable option. Lastly CRISPR program is a long shot, but a lot of companies are now pursuing gene editing technology to treat genetic disease and David Altschuler, VRTX CSO, has the expertise to collaborate with VRTX chosen partner to develop this technology as a possible cure for CF and Sickle cell disease. IMHO, its an investment worth pursing
ENTEROME Bioscience SA, a pioneer in the development of pharmaceuticals and diagnostics based on the gut microbiome, has signed an exclusive worldwide license agreement with Vertex Pharmaceuticals Inc. (VRTX) to research, develop and commercialize novel small molecule FimH antagonists for use in the treatment of inflammatory bowel diseases (IBD).
The first clinical trials to start this year. These are the drugs being developed at VRTX Canadian research site in Laval Quebec.
More important is the reason for the breakout. Investors may be betting on better than expected sales of orkambi in first quarter and perhaps positive guidance from vertex management at the first quarter conference call regarding the rest of the year's sales and clinical trial data release timeline from the R&D pipeline. Also takeover speculation in the sector may include Vertex as the PfizerAllergandeal unravels and these two companies start looking for alternativeacquistion candidates whose shares are currently greatly discounted and have a rich R&D pipeline of potential blockbuster drugs.
The remaining minority of 508dd homozygous CF patients not yet taking Orkambi this year will eventually get treated either with Orkambi (as word gets out from long term data that it provides sustained benefit to improving the health and longevity of patients taking it) or perhaps with the improved potentiator/corrector combination from Vertex (661/770) currently in phase 3 trials, after it gets approved and everyone with this mutation switches to the next generation of corrector treatments with even better results.
This equates to most all of the 508dd CF homozygotes being treated with Vertex drugs by 2017. The EU countries, Canada and Australia always take much longer to negotiate a discounted price on these breakthrough drugs, but as we have seen with Kalydeco, in the next year or two, most everyone with CF who qualifies will have access to these life saving drugs. Peak sales at this time are purely speculative, but will surely grow steadily to the 4-6 billion range as the patients gain access to treatment by the end of next year.
Add in the possibility of the residual function mutations gaining approval for 66l/kalydeco treatment (also in phase 3 clinical trials) and eventually the "het min" CF population (508d heterozygotes) getting access to 'triple therapy' (two correctors with 770) plus the benefits from VX 371 (which can be used in all CF mutations), to be tested in clinical trials started this year, and the CF revenue potential goes even higher. And then consider the potential of the non- CF pipeline currently in multiple clinical trials in oncology, spinal cord injury, pain, and influenza. This stock is undervalued. Some of these buy side analysts are very short sighted.
I agree the Morningstar article is way better than usual stuff from buy side analysts. It does not mention near term catalysts coming from Vertex specifically advanced stage clinical trial results due over the next 18 months in next generation CF treatments and non CF pipeline assets. Positive results in these clinical trials will raise analyst price targets and protect Vertex from competitors developing CF treatments who are years behind Vertex in their R&D efforts. Vertex non-CF pipeline assets include multiple drugs being tested in oncology, spinal cord injury, pain, and influenza, and they diversify Vertex potential revenue into multiple disease markets with novel breakthrough treatments, many with orphan drug pricing potential.. With near term growth in the approved and soon to be approved CF treatments, IMHO Vertex is greatly undervalued and may be a takeover target from larger pharma looking for both near term revenue growth from CF and the potential from a large diverse pipeline of breaktrhough drugs in multiple diseases. .
Go to clinical trials.gov website and type 'VX' in searchbar. and count the number of clinical trials either recruiting patients or about to start. Management probably not crowing about the possiblities until the results of trials are in but many late stage cf trials with vx661 and 371 and influenza vx 787 and pain (a trial underway to treat chronic pain from OA) with interim or final results due this year and early next year. Spinal injury and numerous oncology trials underway as well. They are clearly quietly building a large number of potential blockbuster breakthrough treatments and not sitting idly by. The stock market is in correction this year and analysts are lowering price targets on all pharma ( look at GILD PE ratio). As the analysts see progress in the clinical trials, the same analysts will raise PT as they give potential value to drugs in development. This means once again being patient as a long term investor.
NHS is paying for Kalydeco in the CF gating mutations, as is the rest of the EU, and Canada and Australia, after each country's equivalent of NICE successfully negotiated a discounted price. The same will eventually happen with Orkambi and it's successors currently in development (e.g. 661/770 and future combinations of correctors and potentiators) that represent breakthrough treatment in the underlying cause of CF in the various mutations. The MP's can respond to their voting constituents by asking NHS officials accept the CF Trust's proposal to allow access of 508dd CF patients in the UK to being prescribed Orkambi while NHS officials continue negotiations with Vertex on a compromise price to save lives of the young CF population in the UK and get more data on the long term benefits of Orkambi as it is used on patients in the UK from the CF registry. The UK is not a third world country like Bangladesh and can make the lives of CF patients a priority. The CF patient population deserves access to these new breakthrough drugs as they become available. The mandate of the NHS is to not choose less effective treatments because they are less expensive to treat illness. The mandate is to provide current medical care within budgetary constraints, which can be accomplished by negotiating pricing to provide that treatment. Health care administrators in the developed world have the responsibility ask for additional government funding if needed to provide better care for the population. The voting populace has the right demand the best care available.
CF Trust in UK responding to denial of Orkambi by NICE
As the National Institute for Health and Care Excellence (NICE) opts not to recommend the gene-specific medicine Orkambi to NHS England, while acknowledging the clinical benefits of the drug, the Cystic Fibrosis Trust has put forward a solution that could speed up access and evaluation.
Orkambi has been shown to increase lung function and significantly reduce infection and hospital stays for those carrying two copies of the most common mutation of the cystic fibrosis gene, F508del, and in today’s draft decision NICE finds the treatment to be effective and important for managing cystic fibrosis, but uncertainty around its long-term impact and its high cost means it cannot recommend the drug.
The Trust has proposed a solution whereby Orkambi could be provided to all who need it while further evidence is collected on its long-term clinical impact using the UK Cystic Fibrosis Data Registry.
Ed Owen, Chief Executive of the Cystic Fibrosis Trust said: "It would be ethically unforgiveable if people with cystic fibrosis were treated like pawns in a bigger battle between the NHS and Vertex over price and longer term impact. But there is a solution that requires the NHS in England to work with Vertex to provide access to Orkambi and enable the UK Cystic Fibrosis Data Registry to elucidate its value more clearly over time.”
In a letter to Life Sciences Minister George Freeman MP today, Ed Owen urges intervention to support the Trust's plan, while the Government is currently looking at new arrangements to accelerate access to medicines for those that need them.
Write to your MP
Use this letter to ask your MP to ask the UK Government to support the Trust’s solution, granting access to the drug for those who would benefit, while assessing the longer-term impact of Orkamb
39 employees no institutional owners a device that is not approved and requires implantation by a physician in return for the promise of 90 day readings I'm skeptical of it's theoretical competitive advantages/appeal to patients and have no data as to it's comparative accuracy vs DXCM sensors
Stock price approaching a level where larger diagnostics company would make a take over offer. JNJ Roche Abbott Novartis and Verily/alphabet/google all interested in the glucose testing market. Dexcom, as the CGM technology leader, now with a positive cash flow IMHO is a steal even with 60% premium offer from current prices.- .
Current prices of successful midsized biolech companies are approaching a low enough level where larger pharmaceutical companies with ample cash and market cap looking for drugs in orphan diseases with a diverse pipeline of future drugs, represent profitable acquisition opportunities. Vertex has orphan disease drug pricing with a cash flow already building in CF with a number of additional possible breakthrough drugs advancing in CF, Neurological disease, as well as potential larger market opportunities in oncology,
pain, and influenza. Vertex has little debt, a billion in cash on hand, potential cost savings from reducing it's head count in terms of managers and executives when acquired by a larger drug company with established marketing world wide and the need to keep only the most productive researchers at Vertex. Would not be surprised to wake up one morning soon with a low ball takeover offer from Biogen, SNY, GILD or JNJ all of whom have stated recent interest in taking advantage of current low valuations of potential takeover target companies.. Then it would be interesting to see if Vertex management could fend off a takeover given it's minority ownership and the need for institutional shareholders owning the majority of shares to book some profits this year. Longs in Vertex have to hope that the takeout offer price when it comes at least translates into current analyst target prices averaging 140 to 150 per share.
How can you state 'eventually CMS will cut down reimbursements at this level'? CMS still has not yet approved CGM for use in Type 1 diabetes. However, once it does approve coverage, another one million type one diabetics will be added to the market, essentially doubling the potential user base. The largest private health plans are covering CGM as a pharmacy benefit now and medicare will likely join them in the next year or two. Even without medicare coverage, Dexcom has grown it's revenue 10 fold in the past 5 years and continues that growth going forward, with another doubling of revenue estimated by the end of next year just selling it's G5.platform, and introducing the G6 platform later this year to the Type 1 DM market which so far is less than 20 % penetrated to date. When the Verily/Dexcom partnership debuts it's bandaid size disposable CGM device for use type 2 diabetics, the potential market for Dexcom products will be many times higher, and the stock will seem quite cheap and not 'wildly overvalued'.
BDI rising and latest DSX vessel lease renewal rising with it today. Stock finally moving up short squeeze will propel it higher as long as BDI continues to rise.
With rising BDI, future growth in revenue will materialize with future contract renewals for DSX's expanding fleet. New and used ships being are purchased at severe discounts. DSX turnaround will need sufficient revenue to service it's debt, but stock's price move from lows suggests investors see rise in BDI continuing eventually translating into DSX having significant improvement in revenue and eventual profitability over the next few years
Verity. Thanks for your thoughtful and insightful reply. I realize how very frustrating delays like this are to you and the CF community affected by these decisions. Obviously, the FDA has to be cautious with young children, but the safety of Ivacaftor in the pediatric population is well established and giving the drug a conditional approval requiring clinical phase 4 data to document efficacy over a longer time period in all 23 residual function mutations would give the proof the FDA is demanding and save lives at the same time. Maybe the White House advisers with an interest in 'personalized medicine' could be asked to have the FDA submit this idea to an FDA advisory committee to allow input from experts and the CF community on the idea of an expedited conditional approval for this sub-population of CF patients likely to benefit?
Verity- Are CF advocacy groups not expressing their concern to congressional representatives with FDA oversight over this FDA decision regarding the rejection of the sNDA to treat residual function mutations with Ivacaftor?
The sad part is that these residual function CF patients' health will deteriorate further waiting even though the efficacy and safety in the use of this drug has been proven. The FDA's bureaucracy is now demonstrating that 'fast track' and 'breakthrough' designation status offers little in terms of expediting approval. Ivacaftor was the first drug to get this designation by the FDA. Seems to me the FDA is clearly not making this designation a path for expedited approval, as was intended by the law that by creating this designation, was mandating streamlining the approval process for a drug that makes such a difference in the lives of patients.