From fda trail site mod-4023 which is the HGH drug:
Estimated Enrollment: 189
Study Start Date: June 2013
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
knows the ckd/vitamin D market. After all the drug his company pioneered and brought to market is the leading ckd/vitamin D replacement therapy, a drug called Hectorol. Would he waste his time developing a bio equivalent to Hectorol, or a bio better. Hectorol can and does impact phosphorous and blood calcium levels. His new drug eliminates that issue from the equation.
An excerpt from his Opko Bio follows:
Dr. Bishop was responsible for the successful preparation and execution of three NDAs that ultimately led to U.S. marketing approvals for a novel vitamin D drug, named doxercalciferol, for SHPT in dialysis patients (Hectorol® 2.5 mcg Capsules and Hectorol® Injection) and pre-dialysis patients (Hectorol® 0.5 mcg Capsules). Hectorol® is currently the leading vitamin D hormone replacement therapy in the U.S. Bone Care was acquired for $720 million by Genzyme Corporation in July 2005.
He not only thinks he got it right this time by eliminating the serious side effects, but that he can dominate this niche.
Prior to joining Cytochroma, he was Senior Medical Director in the Global Pharmaceutical Research and Development Group at Abbott Laboratories (North Chicago, IL). While at Abbott, Dr. Melnick managed clinical trials, regulatory submissions, marketing approvals and post-launch support for Zemplar® (paricalcitol) Capsules and Injection, currently one of the world's leading vitamin D hormone therapies for SHPT in CKD. Prior to Abbott, Dr. Melnick served as Assistant Professor of Pediatrics at Northwestern University, where he conducted research in metabolism and cardio-renal disease. Dr. Melnick completed his medical degree, internship, and residency in internal medicine and pediatrics at the Creighton University Medical Center, and completed a post-doctoral fellowship in pediatric and adult nephrology at the University of Texas Southwestern Medical Center.
Dr. Bishop held various senior management positions at Bone Care International, Inc. (NASDAQ: BCII), a public specialty pharmaceutical company focused on developing and commercializing vitamin D hormone therapies. Dr. Bishop’s positions with Bone Care included President, CEO, Director, Executive Vice President of Research and Development, and Chief Scientific Officer. He was the first full-time employee in Bone Care and he ran the Company from its inception until after the commercial launches of its first two products. Under Dr. Bishop's direction, Bone Care advanced a portfolio of proprietary vitamin D hormones and analogs to varying stages of development for SHPT, metabolic bone disease, psoriasis and cancers of the prostate, breast and colon. Dr. Bishop was responsible for the successful preparation and prosecution of three NDAs that ultimately led to U.S. marketing approvals for a novel vitamin D drug, named doxercalciferol, for SHPT in dialysis patients (Hectorol® 2.5 mcg Capsules and Hectorol® Injection) and pre-dialysis patients (Hectorol® 0.5 mcg Capsules). Hectorol® is currently the leading vitamin D hormone replacement therapy in the U.S. Bone Care was acquired for $720 million by Genzyme Corporation in July 2005. Prior to joining Bone Care, Dr. Bishop held various management positions in the Health Care Division of the Procter & Gamble Company. Dr. Bishop completed a four-year National Institutes of Health Postdoctoral Fellowship in vitamin D Biochemistry at the University of Wisconsin-Madison and received his PhD degree in Nutritional Biochemistry from Virginia Polytechnic Institute and State University, after earning an undergraduate degree in Chemistry from the University of Virginia.
I have found relying on a companies PR and CC's can create investing mistakes. It is not that they do not present factual data, but it is the data they have at that time and usually overly optimistic. Your number 2, are you talking patent protection? As far as insurance, that will not be an issue, IMHO. These guys have bought hundreds of drugs to market, they got that covered. My concern is not the label. My concern is market share, and total revenue. I have held that peak sales will be way lower, in the 300m-800m range for a long time. Looking at the sales of the calcitriol type drugs, umm, makes me skeptical. That said, the Rayaldee profile COULD bring in a slice of the untreated and otc population. Rayaldee will gain serious traction from the calcitriol group but sales in that area have declined in the past couple years. Those in charge invented the calcitriol drugs. Sold to Amgen? I think it was Amgen? They know what they are doing.
Just from owning Opko I found it curious that very few patients reach esrd, the majority of the stage 3-4 patients and there are 25m of them, end up dying from heart issues. There are D receptors in every organ, makes me wonder if the shpt problem, and resultant higher blood calcium issues that can occur, are grossly UNDERSTATED. If blood calcium is high for an extended period it is known that it settles in the organ tissues.
At this point I would like to talk to the Dr. Sort of as if he threw a bunch of stuff out there, and if you had no research background, or knowledge about setting up a phase study, you would go OH wow, he knows stuff. His stuff is dribble. My thinking now is he has never treated one patient, or has never designed a research study, or both.
I have trouble believing any Dr. could possibly have written this dribble. For one, survival is not an end point in stage 3-4, why would ANY DR. make that statement ??? In fact most CKD patients never die from ckd, but usually CVD, second if you have stage 3-4 you indeed have an shpt issue, and a D issue. All literature over the past decade, regardless of origin points to vitamin d, hence the calcitriol type drugs. As far as the end points, umm identical to the calcitriol type drugs, so to say the study lacks valid end points is nonsense. Get a new Dr.
We all should welcome that stuff. Not sure who that Dr. is but great stuff to go look up to find out how false or true it is. Claim there is nothing in the literature to warrant the DRUG is hilarious. ONE thing people might not know is that the inventors of Rayaldee are ALSO the inventors of the GENERICS they intend to compete against. THEY KNOW these other drugs inside and out, and sold their companies to big pharma. They then formed cytochroma to develop a better drug. THE LITERATURE is REPLETE with D, and kidney function.
Again: nothing in literature, and no work done by non pharma? REALLY ?
MANAGING THE SPECTRUM OF COMORBIDITIES IN CKDPRESENTED BY STEVEN FISHBANE, MD
Let's think about why that might be important. Vitamin D deficiency exists for years when patients have chronic kidney disease. If you take any of your patients with a 1.5-mg/dL creatinine or a 2-mg/dL creatinine and measure a 25-hydroxy-vitamin D level, you're going to find that the levels are deficient. If you're deficient of vitamin D for years and it increases renin and angiotensin II levels, what is the effect of that on increasing the progression of kidney disease? What is the effect of that on proteinuria, and what is the effect of that in terms of left ventricular hypertrophy and cardiovascular outcomes? We don't know yet, and it's an area of very active investigation. I'd guess that over the next decade, this is going to be an area of medicine that will get more attention.
(Enlarge Slide)(Enlarge Slide)
Calcitriol vitamin D levels decline progressively in chronic kidney disease. It drives another problem that is potentially important in many of these patients, which is hyperparathyroidism and progressive bone disease. This corresponds with increasing levels of left ventricular hypertrophy.
You might need a different Dr. He also states he cannot find a thing in medical literature that would warrant his type drug??? REALLY from the national kidney foundation, key data from JUST one study follows:
development of more than 5,800 men and women without protein in their urine, 3.8 percent developed
albuminuria during a five-year follow-up period. Those patients deficient in a form of vitamin D known as serum 25-hydroxyvitamin D, were found to be 84 percent more likely to have albuminuria. Deficiency was defined as having less than 15 nanograms of vitamin D per milliliter of blood.
Adjusted for age and other lifestyle factors, individuals with vitamin D deficiency were found to have a 70 percent increase in albuminuria.
“There is mounting evidence of the benefits of correcting vitamin D levels to prevent or delay the development of albuminuria in the general population,” said the study’s lead researcher, Matthew Damasiewicz, MD, of Monash Medical Centre in Melbourne , Australia. “It is also likely that patients with chronic conditions such as CKD may need higher vitamin D levels than the general healthy population.”
Urine Protein and Vitamin D Facts from the National Kidney Foundation
Albumin is a type of protein. When found in the urine it is known as albuminuria. The presence of albuminuria indicates the kidneys have some damage that prevents them from retaining protein in the body.
Two positive tests for albuminuria over several weeks are an early indication of kidney disease.
For patients with diabetes and/or established chronic kidney disease, albuminuria is associated with more rapid progression of their condition and a greater chance that kidney failure will develop.
Vitamin D is important for maintaining healthy bones. Muscle weakness may also occur if vitamin D levels are insufficient.
At what UNIVERSITY did you take your experimental research courses?? Thought so. You are clueless. Opko does not need get the 25M patients in their target market, but only 4-5% in the US, 4-5% globally to have a great drug. That is an achievable number.
Look up those Drugs the Dr. is thrilled about, study them, get to what they are. Both are generally used in end stage renal care, cancer, anemia patients usually in conjunction with dialysis. NOTE, too, there is much in the post to think on. In stage 3-4 showing prolonged survival as a primary end point DOES not FIT. Now if it were stage 5 which those two drugs apply, and time to death was a variable then it would be measured. Controlling SHPT by a new mechanism, regulating blood D levels, IS INDEED a valid primary end point. Prolonging life in stage 3-4 with patients who might live 30-40 more years has no place in the opko study.
There are 25 million Americans with varying degrees of stage 3-4 kidney disease, under 1 million on dialysis. Those 25 million 3-4 ckd patients have varying degrees of shpt, and related issues. Opko is trying to show that Rayaldee is a VIABLE alternate therapy in THAT population vis a vis " non-calciferol forms of vitamin D. " Of the 25 million ckd patients in stage 3-4 Rayaldee might be the preferred treatment for say 1 million, a small percent of the 25 million, 1m x 3,000 =
Since the Yellen call, most large cap pharma has recovered and gone up, ie., gild new highs every week. Nearly all the small cap bios got knocked down, and have yet to gain traction. There are bound to be a few exceptions in the smaller cap area, a phase 3, new drug approval, but by and large of the 25-30 I keep watch nearly all are down or have flat lined for about 3-6 months. Opko has actually held up better than most in the most recent 3 mo, frame, opko down 1%, CGIX down 23%, nwbo down 8%m vrml down 20%, agen down 30%,irwd down 10%, arry down 20% and the list is long and huge.
So Opko is holding up well in a very weak small cap bio sector. It is up slightly y/y and ytd. This will get better as news materializes.
Ron is always talking about future events that will transform Array. There are no approved drugs yet, no WAY to speed up phase studies. The one bright light might be Binimetinib, that phase 3 is about DONE or will be in under 6 months. Not sure of the potential revenue, but if it enough to fund other phase studies that could be huge for a micro cap like arry. So hoping this happens sooner than later, and that the phase 3 is great.
The RSU's vest upon achievement of certain milestones relating to a program licensed by the Company to Novartis International Pharmaceutical Ltd., with one half vesting upon achievement of the first milestone relating to the return of commercialization rights for binimetinib to the Company that have been licensed to Novartis International Pharmaceutical Ltd. and one half vesting upon achievement of the second
milestone on or prior to the fourth anniversary of the grant date, in each case subject to continued service with the Company as of each vesting date.
Sort of BOGUS way to get FREE shares but you can likely bank on the fact they are getting the drug back since the insiders saw it as a money grab situation. Not sure what the second milestone is for vesting but hope it is FDA approval, or a sales number. From what I could find it appears arry gets the drug back around or before March 2015. So anywhere from 1-7 months.
Reread the conference call. They R doing the comparative, they are in touch with insurors. The phase 3 data was stellar. Bottom line is calcitriol lowers SHPT fast, but with side effects. Rayaldee takes a few weeks to lower it, but gets it right, with no side effects. Until I see data other wise I am convinced this becomes the go to treatment. I too want to see data from the calcitriol study vs Rayaldee. You might want to wait on that data, but based on what I have read about Calcitriol I am convinced NOW that Rayaldee proves superior. I think that data will be out early 2015, but after they file the NDA, it has no impact on the NDA. It will have a MAJOR impact on labeling, ie., what claims OPKO can make.
The shorts will be gone by 2016, Rayaldee will be WAY bigger than they think. Just my take. Longs and shorts do research, there are big bets on both sides, I am confident in my position. I think they do not fully get Rayaldee and all they initially saw was some vitamin, that was my take then too. I thought garbage drug, vitamin D #$%$.
Not contradicting myself. Calcitriol does not address Vitam D insufficiency. It is the current treatment standard for SHPT, it ignores D levels. Like I said, if you start out vitamin D insufficient and take calcitriol, you stay vitamin D insufficient. Your SHPT level will drop, but it HAS NO impact on D. So I guess from my perspective since calcitriol does not raise D levels, I would say it is standard of care for SHPT/CKD patients and leave the D insufficiency part out.
Read the CC, the calcitriol vs rayaldee study will be finished before drug approval. The results will show that both lower SHPT, they will also show increased blood calcium for the calcitriol group and not the rayaldee group.
The FDA gave Opko the Clearance to design this study without including calcitriol under an SPA. The DRUG will get approved based on it's OWN merits without showing superiority to calcitriol. It is like this, it corrects Vitamin D insufficiency in 96% of all CKD patients and if it had no impact on SHPT, the drug gets approved for that alone. The SHPT is a bonus, They are doing the head to head anyway, but it is not necessary to submit the NDA.
It is the only drug to correct vitamin D insufficiency in ckd patients. So find a drug that does that, and they would have had to include it in the initial phase 3, but there isn't one. The bonus is it ALSO lowers SHPT as well as Calcitriol without the side effects.
Print the earnings conference call, can be found at seeking alpha. Then take out the Rayaldee sections including those in the question and answer section. Give those to the Drs. Opko believes Rayaldee will be prescribed to those who get no D, those that get OTC D, and those that get Calcitriol. It should become standard of care, and WAY bigger than most think. I am convinced it replaces Calcitriol.