The biopharmaceutical sector is built to breed innovation from the ground up. If you think about it, drug patents are only scheduled to last for a grand total of 20 years, and that is sometimes from the point at which a company begins preclinical trials. In other words, around half of a drug's protected period can be spent running pre-clinical and clinical tests on efficacy and safety. This means the bread-and-butter period of success for even the biggest blockbusters tends to only be about one decade.
Source: Missy Schmidt, Flickr.
To add, the march toward creating better drugs never stops. Just because a new hepatitis-C drug hit the market two years ago doesn't mean the other four companies making hep-C drugs are suddenly going to go home with their hands in their pockets pouting about what could have been. Biopharmaceutical companies need to always be on their toes and trying to outdo their last drug if they hope to maintain success. Like technology, these drugs become bull's-eyes as soon as they're approved by the Food and Drug Administration for the next group of biopharma companies to unseat.
That's why today I'm looking at seven drugs that I feel have the power to remain a disruptive force in their treatment field throughout the next 10 years. Some are already approved by the FDA; others are not. Obviously, there could be a lot of variables here as it's difficult to see 10 years out and understand what discoveries could be made, as well as what monkey wrench clinical trials or the FDA could throw out to the handful of drugs still in clinical stages mentioned below. What I do believe, though, is that each drug below has the tools to become a dominant force over the next decade and you should, at minimum, be watching their progress.
Let's begin with three recently approved drugs that have incredible potential.
Three game-changing FDA-approved drugs
1. Tecfidera -- Biogen Idec (BIIB +0.89%)
Approved in March, Tecfidera is the latest and greatest oral treatment for relapse-remitting multiple sclerosis patients. It certainly wasn't the first oral MS treatment to make it to market with Novartis' Gilenya and Sanofi's Aubagio clearing the hurdle well before Tecfidera. However, there are differences between these treatments.
Aubagio comes with a black-box warning on its packaging that it can cause severe liver problems, which can lead to death. In addition, Aubagio comes with side effects including kidney and breathing problems, as well as high blood pressure. Novartis' Gilenya has no black-box warning, but it nonetheless comes with the side effect of lowering heart rate, which can, in rare cases, lead to severe cardiovascular problems.
Gilenya, a once-a-day pill, does have a dosing advantage over the twice-a-day treatment Tecfidera. However, with a strong safety profile and efficacy -- 49% reduction in MS relapse rates and 71%-99% reduction in new or expanding lesions in trials -- I'd say Tecfidera could live up to expectations and be a $4 billion-plus peak sale drug.
2. Eliquis -- Bristol-Myers Squibb (BMY 0.63%) and Pfizer (PFE +0.21%)
Warfarin has been the mainstay blood-thinning agent for more than a decade, but its reign appears to be coming to an end. Boehringer can certainly relish in the fact that Pradaxa had strong clinical trial results and was shown to be superior to warfarin at a 150 mg dose taken twice a day. However, Eliquis in a head-to-head, double-blind study against warfarin exhibited fewer strokes or system embolisms, less major bleeding, fewer hemorrhagic strokes, and fewer deaths during trial than those on warfarin. Based on these results, Eliquis has the potential to become a strong competitor in this space.
At the moment, Eliquis is only approved to reduce the risk of stroke and blood clots in patients with atrial fibrillation. Just imagine what sales would be like if Eliquis gets expanded indications of use into blood clot prevention following surgery? I suspect Eliquis could top $5 billion in global sales by 2020.
3. Kadcyla -- ImmunoGen (IMGN +0.00%) and Roche
With better technology comes the potential for more efficient ways to target cancer care. Thus we have Kadcyla, a drug approved by the FDA in February to treat HER2-positive metastatic breast cancer (one of the more aggressive types of breast cancer).
What makes Kadcyla so unique is that it combines Roche's already-proven cancer drug Herceptin with ImmunoGen's revolutionary targeted-antibody payload technology to deliver improved results. In plainer English, ImmunoGen has found a way to "strap" chemotherapy toxins (i.e., Herceptin) onto antibodies, which will release that toxin once it comes into contact with a specific protein signature found in the targeted cancer cells. The end result is less healthy cell death and more targeted treatment as the late-stage trials showed where Kadcyla improved median overall survival to 30.9 months from 25.1 months for the placebo.
This is just the beginning for Kadcyla, and it very well could find its indications expanding as ImmunoGen and Roche are currently testing Kadcyla as an early-stage HER2-positive breast cancer therapy and in the very tough to treat indication of gastric cancer. On just its current approval alone, Kadcyla likely has a $2 billion drug on its hands. That figure will only go higher in my eyes if Kadcyla expands its indications in the next couple of years.
Source: e-Magine Art, Flickr.
And four pipeline therapies that have worlds of potential
4. Sofosbuvir -- Gilead Sciences (GILD +2.08%)
You could very easily make an argument to include AbbVie's direct-acting antiviral combo drug here and I probably wouldn't put up too much of a stink, but Gilead's sofosbuvir has simply been so dominant in hepatitis C trials that I believe its success is a near-given.
Sofosbuvir is part of a new line of oral therapies for hepatitis C patients that are completely independent of interferon. If you recall, Vertex Pharmaceuticals' Incivek (which is a pill) greatly improved sustained virologic response (i.e., no detectable levels of the disease) in hep C patients after 24 weeks, but it needs to be taken in combination with IV interferon, which has the side effect of making people feel like they have the flu for up to 48 weeks. Sofosbuvir is changing that. Sofosbuvir doesn't need to be taken with interferon for the treatment of the most common hepatitis C genotype and it demonstrated statistically significant improvement in SVR in just 12 weeks across the most common and lesser common genotypes. If sofosbuvir wins FDA approval, Gilead should have an easy blockbuster on its hands.
5. Ibrutinib -- Pharmacyclics (NASDAQ: PCYC) and Johnson & Johnson
Few experimental therapies have wowed Wall Street like ibrutinib has over the past two years.
Ibrutinib is currently being studied as a B-cell malignancy therapy to treat mantle cell lymphoma (MCL), small lymphocytic lymphoma (SLL), and chronic lymphocytic leukemia (CLL). As it relates to MCL and CLL, ibrutinib returned the most impressive overall response rates ever recorded in a clinical trial. In its MCL trial, ibrutinib exhibited a 68% overall response rate and a median response duration of 17.5 months. In CLL, which is the huge application since it's by far the most common in adults, the overall response rate of the combined doses was a whopping 71% with an estimated 75% of patients still in the progression-free stage after 26 months!
These are knockout numbers that have allowed ibrutinib under the breakthrough therapy designation to file for a new drug application. Barring something truly out of left field from the FDA, an ibrutinib approval could quickly vault it to the No. 1 treatment for CLL and MCL.
6. Eteplirsen -- Sarepta Therapeutics (SRPT +5.60%)
Unlike the previous two experimental therapies, we're stepping into a riskier proposition with Sarepta's eteplirsen -- which is designed to treat Duchenne muscular dystrophy -- but also a world of potential.
Eteplirsen's claim to fame came about a year ago when a mid-stage trial of 12 patients demonstrated not only a lessening in disease progression, but an improvement in six-minute walking distance, abbreviated as 6MWD. The drug, which spurs dystrophin production, essentially managed to reverse the effects of the disease, albeit in a small subset of patients. The danger, of course, is that the FDA may not see the connection between dystrophin production and the end result of an improvement 6MWD test. Also, GlaxoSmithKline and Prosensa's similar exon-skipping drug, drisapersen, failed miserably in a late-stage study. In other words, the FDA has all the more reason to take the cautious approach with eteplirsen moving forward despite its groundbreaking mid-stage study.
Should eteplirsen clear all its hurdles -- and nothing would point contrary to that fact -- it could become the priciest drug in the world and would have a clear path to at least 13% of the DMD market.
7. LX4211 -- Lexicon Pharmaceuticals (LXRX 3.03%)
OK, so I'm really going early stage here with a proof-of-concept study that isn't but a few days old, but Lexicon's LX4211 appears impressive thus far.
What's unique about LX4211 is that it inhibits both SGLT1 and SGLT2 in the body to help control glycemic balance in patients with type 2 diabetes. If you recall, I believe that SGLT2 inhibitors could become the dominant diabetes drug class; these inhibitors have been shown to induce weight loss and lower A1C levels in patients. LX4211 is similar to J&J's Invokana, save for the fact that it also targets SGLT1 inhibitors. It'll probably be a little while before we see if there's a statistically meaningful difference between Invokana and LX4211, but if its early-stage data holds consistent -- increased GLP-1, which is a hormone that controls glucose levels and appetite, and decreased post-prandial glucose levels -- this could become a monster of a glycemic balance drug with about 90% of the 25.3 million people in the U.S. having type 2 diabetes.
Editor's note: The comparison between Tecfidera and other oral MS drugs was not accurate in a previous version of this article and has been updated. Also, a section on Eliquis and Pradaxa has been edited for additional clarity. The Fool regrets the errors.
