Opko Health (OPK), is expecting to announce top-line results from its Phase III trial for rolapitant, a potentially best-in-class supportive care product for chemotherapy induced nausea and vomiting. Opko's partnership with Tesaro on this product includes an equity position in the company, potential milestone payments up to $121 million, double digit tiered royalties on sales, as well as profits in some other areas of the world if the drug is successfully commercialized.
This global trial program is being conducted at more than 200 sites across 25 countries and includes approximately 2,400 cancer patients. Rolapitant will be evaluated along with the standard of care against a placebo/standard of care combination.
In this Phase III trial, the patients and the company hope to see an improvement in control of nausea and vomiting during the acute (0-24 hours), delayed (24-120 hours) and overall (0-120 hours) time periods post administration of chemotherapy. Additional outcome measures include complete response for other time points, the incidence and severity of nausea, and safety and tolerability. Opko believes this drug will be successful as a longer acting treatment over the course of five days versus comparable drugs which generally last 1-2 days. It also believes the drug will be effective in a more rapid manner once taken compared to the competition.
If this would become the standard of care as the potential indicates, the market opportunity identified by Opko is approximately 1.25 billion. The company expects a high 70% penetration rate because the only drug currently on the market designed as an NK-1 receptor antagonist is Emend from Merck (MRK). U.S. exclusivity is expected through 2028 as well which gives the company a strong asset if eventually approved by the FDA.
Generally, Opko Health makes news because of insider buying from its CEO, Phillip Frost. He now holds a huge stake in the company, approximately 152,000,000 shares. With Dr. Frost continuing to mak
Sentiment: Strong Buy
make purchases, it will be interesting to see how his fortune fluctuates over time. Over the past year, he has seen a nice upswing in his investment as the stock has moved from the mid-$4 range to about $10/share. But, this doesn't deter him from continuing to buy at these levels.
Three weeks ago, I invested all my available funds in OPKO Health Inc. (OPK). I did that when I learned about its new drug, Rayaldy.
I am not a stock analyst, adviser, or investment expert. Instead, I am a physician and an expert on vitamin D. Ten years ago, I established the Vitamin D Council, a non-profit dedicated to educating the public and health professionals about vitamin D. I have written more than 15 peer-reviewed scientific articles on vitamin D. I have been reading, writing, and lecturing about vitamin D for the last 12 years.
Vitamin D (cholecalciferol) is not a vitamin in that it is not contained in any appreciable quantity in the food we eat, including milk. Instead, it is a prohormone made in the skin upon exposure to sunlight -- something that most of us have come to avoid during the last 30 years. Vitamin D is the only known building block for a potent seco-steroid hormone known as calcitriol. Vitamin D is metabolized to calcitriol in two steps via a more active prohormone intermediate known as calcifediol, the circulating form of vitamin D. Like corticosteroids and other steroid hormones, calcitriol directly regulates hundreds if not thousands of human genes. In calcitriol's case, those genes are involved with the repair and maintenance of the human body. It is crucial to realize that steroid hormones have as many mechanisms of action in various diseases as genes they regulate.
Science has discovered much about vitamin D in the last ten years, almost all of it promising. If they know anything about vitamin D, most people think of it as something that may protect against various diseases. Indeed, the evidence is so strong for prevention of disease, four large and very expensive phase 3 trials are currently underway around the world to see if vitamin D prevents common diseases like cancer and heart disease. The largest of these trials is the VITAL study at Harvard where scientists are conducting a randomized controlled trial of vitamin D in 20,000 Americans. Obviously, these large studies would never have been funded unless solid reasons exist indicating these vitamin D trials will be positive. For more on vitamin D, go to Harvard School of Public Health's website on vitamin D.
While everyone seems intent on studying vitamin D's preventative effects, few have noticed that scientists have published dozens of small randomized controlled trials in the last few years, documenting that vitamin D has treatment effects, not just preventative effects. These small "phase 2" trials show over-the-counter (OTC) vitamin D has a treatment effect in several diseases, especially autoimmune diseases like multiple sclerosis. In addition, "phase 2" trials exist, showing vitamin D has efficacy in treating diseases as diverse as major depression, falls in the elderly, atopic dermatitis, congestive heart failure in infants, hypertension in African Americans, and tooth loss in the elderly. Treatment effects are not cures; rather, they are simply clinical or laboratory improvements. Besides the randomized controlled trials, hundreds, if not thousands, of epidemiological studies now exist, suggesting that vitamin D will have treatment effects in an even wider variety of human disease.
OPKO Health's new drug, Rayaldy, is the first and only modified-release formulation of calcifediol, protected by newly issued patents (see, for example, US Patent No. 8,361,488). It is currently in phase 3 trials for the treatment of secondary hyperparathyroidism in vitamin D insufficient patients with chronic kidney disease (CKD). Its phase 2 trials for this indication were very positive. Rayaldy is much more potent than OTC vitamin D and it corrects vitamin D insufficiency more effectively and reliably than does OTC vitamin D. Also, Rayaldy is less prone to degradation in the body than is OTC vitamin D because of gradual delivery of calcifediol from its modified-release formulation.
If the FDA will eventually approve Rayaldy; OPKO Health will certainly study more indications for its new drug than just secondary hyperparathyroidism in CKD. According to the current medical literature, phase 3 trials targeting new indications could confirm treatment effects in a wide variety of human diseases.
Of course, risks exist. I may be wrong about vitamin D; similar enthusiasm for other vitamins in the past did not pan out. Larger randomized controlled trials may fail to find treatment effects with vitamin D. Also, Rayaldy's current phase 3 trials may turn out to be negative. Eventually, other companies may enter the market with different modified-release formulations of vitamin D. OPKO Health may not aggressively seek other indications, settling only for the multibillion-dollar CKD market.
Rayaldy is currently the only product of its type in development by any drug company, as far as I am aware. Currently available prescription vitamin D hormone therapies have significant limitations or side effects, something not seen with Rayaldy. Physicians will soon have a choice: tell their patients to go to the corner drug store to buy OTC vitamin D or prescribe the more potent and reliable and less degradable Rayaldy.
Again, it is important to realize that steroid hormones like calcitriol and corticosteroids have as many mechanisms of action as genes they regulate. The number of genes directly regulated by corticosteroids is estimated to be around 100, almost all having to do with inflammation. However, calcitriol regulates 1,000 human genes. Calcitriol's genetic mechanism of action not only includes anti-inflammatory actions, but extends to mechanisms involving DNA repair, anti-autoimmune actions, mechanisms involving brain development and functioning, profound effects on the immune system, cardiovascular functioning, lung function, liver function, and pancreatic function, to name a few.
In my opinion, sales of Rayaldy-like drugs will eventually easily outstrip the sales of corticosteroids.