Following decades of research, a new standard of care has been introduced that should greatly improve the quality of life and lifespan of hepatitis C patients. Four years ago we'd be talking about an effective cure rate in the neighborhood of 50%. Following the addition of a handful of new compounds with cure rates in the neighborhood of 90% or higher, HCV is finally being eradicated (albeit slowly, as there are 180 million worldwide with the disease).
This victory over HCV certainly leads one to hope that other disease cures could be around the corner. With that in mind, we asked three of our top health care analysts to share what they believed would be the next disease that researchers would cure. Here's what they had to say.
Gilead Sciences (NASDAQ:GILD) is a solid bet to be among the leaders curing the next major disease -- nonalcoholic fatty liver disease.
NASH, or nonalcoholic steatohepatitis, is often billed as the "silent liver disease," since many people with it feel well. But NASH, which is estimated to affect 2-3% of the U.S. population, can eventually lead to liver scarring, liver failure, and death.
More than a dozen biotechs are targeting varying stages of the condition, which is linked to obesity. But Gilead Sciences -- which got into liver disease in a big way with its enormously successful hepatitis C drugs -- is taking a different approach.
Gilead is attempting to leapfrog its competitors with a drug that reverses full-blown liver scarring directly in the most seriously ill patients. By contrast, a drug developed by Intercept Pharmaceuticals' (NASDAQ:ICPT) targets NASH patients who are more moderately ill. Intercept's drug nabbed the FDA's breakthrough therapy designation in late January. It is likely the closest to applying for FDA approval, with Phase III trials expected to begin any day now.
Other biotechs hunting for a cure for NASH include Raptor Pharmaceuticals, Galectin Therapeutics, and Conatus Pharmaceuticals. The potential market for these drugs could rival the success of cholesterol-fighting statins, which generated more than $30 billion a year at their peak.
The company is using their zinc finger therapeutic approach to modify a key gene receptor known as CCR5 that plays a critical role in HIV's ability to infect T-cells in the immune system. If Sangamo is able to effectively shut down HIV's ability to spread by targeting CCR5, then it could revolutionize patient treatment. Currently, HIV is treated with an array of pills that must be taken daily for the rest of a patient's life and that can cause a bevy of side effects.
In February, researchers presented data showing that Sangamo's SB-728-T reduced viral load below the limit of quantification in one out of three patients. If that can be duplicated or improved upon in larger, later stage trials, then Sangamo could end up delivering on its promise of a potential cure to HIV. However, before we get too excited about this possibility, we need to realize that Sangamo's research remains in the early stages, so it will be a while before we know for certain whether or not this approach will work.
I suspect the next disease that researchers may wind up curing is the same disease that's had all but the kitchen sink thrown at it over the past year: Ebola.
Ebola is a particularly scary disease because of its relatively long incubation period. It's possible to develop symptoms of the disease up to 21 days after coming into contact with an infected and ill individual. Ebola also has a notoriously high mortality rate. Of the 26,759 confirmed cases per the World Health Organization, 11,080 people have died. It's a disease that commands a lot of attention and pharmaceutical resources -- and I believe it's a disease that'll have a cure on pharmacy shelves by as early as 2017.
There is certainly no shortage of pharmaceutical and biotech companies racing for the Ebola cure, with somewhere in the neighborhood of a half-dozen to dozen compounds advancing into preclinical and clinical studies. However, the two players that intrigue me the most are GlaxoSmithKline (NYSE:GSK), a veritable vaccine powerhouse already, and Johnson & Johnson (NYSE:JNJ).
The reason I believe GlaxoSmithKline or Johnson & Johnson have the best chances of developing a cure lies in their deep pockets and their ability to rapidly ramp-up commercial production. The problem with smaller drug developers, such as Sarepta Therapeutics, which is relying on RNA-interference, is there aren't concrete plans to scale their operations to meet widespread government and hospital demands.
GlaxoSmithKline was really the first to get the ball rolling, with patients being dosed in phase 1 safety studies in September. In February, Glaxo announced the testing of two Ebola vaccines -- an NIAID/GSK vaccine known as cAd-EBOZ, and the VSV-ZEBOV vaccine -- in a phase 2/3 trial known as PREVAIL in Liberia with an expected completion date of June 2016. Some 27,000 healthy men and women will be enrolled, with Glaxo tracking these patients for 8 to 12 months and comparing the results against a placebo.
J&J, on the other hand, kicked off its phase 1 study in January and received 100 million euros in funding from a private European partnership to speed the development of the vaccine. It may be a bit behind Glaxo in terms of clinical testing, but I believe these two companies hold the edge in the Ebola cure race.