Meet the Game-Changing Drug Designed to Fight America's Leading Cause of Death

By Markets Fool.com


Source: Flickr user Ian McKenzie.

Continue Reading Below

Life expectancies in the United States are now higher than they've ever been, according to the Centers for Disease Control and Prevention. The average American is now living to be 78.8 years old. By comparison, in 1900, the average life expectancy was about 47 years. That's a monumental jump in a fairly short period of time.

But there's more progress to be made -- and there may be an intriguing opportunity to combat one of the leading causes of death.

Per the CDC, some 2.6 million people passed away in 2013, the most recent annual data available. The five leading causes of death accounted for 62% of all deaths in 2013. These included stroke, accidents, and chronic lower respiratory diseases such as COPD and emphysema in the fifth through third spots, respectively. Cancer occupies the second spot, leading to nearly 585,000 deaths. The leading cause of death in the United States? Heart disease, which claimed more than 611,000 lives in 2013.

Source: Centers for Disease Control and Prevention, Facebook.

Continue Reading Below

America's leading cause of death
Heart disease is a fairly broad term describing a cluster of heart or blood vessel related conditions that can be chronic and lead to death. Examples would include coronary artery disease, heart arrhythmias, vascular disease, or heart failure. To be clear, heart failure isn't the same as a heart attack. A heart attack is often caused by coronary artery disease and the blockage or partial blockage of an artery. Heart failure, on the other hand, is a result of the heart being too weak to pump blood adequately throughout the body.

Combined, these diseases and abnormalities lead to nearly a quarter of all deaths in the United States. A major concern, as noted by the CDC, is that a few major risk factors can greatly increase your chance of developing heart disease, such as high blood pressure, high LDL-cholesterol (the bad kind), and smoking. Roughly half of all American possess at least one of these three risk factors, which likely lends to heart disease's high diagnosis rate.

Successes have been few and far between
Researchers have a monumental opportunity to fight various aspects of heart disease in order to improve patient quality of life, and potentially even survival rates. However, heart disease also very expensive to fight, costing an estimated $444 billion in 2010 per the CDC, or roughly $1 out of every $6 spent on healthcare in the United States. Finding a way to fight heart disease could greatly impact heart disease sufferers as well as the entirety of the healthcare system.

Source: Novartis.

Of course, developing drugs designed to fight America's leading cause of death hasn't been easy. A 2012 abstract study published in the European Journal of Heart Failure notes that a sea of acute heart failure therapies have failed to either deliver statistically significant outcomes in late-stage trials, or to win over regulatory committees that are deciding their fate. These failures have included Bayer'scinaciguat, Merck's rolofylline, and most recently,Novartis' breakthrough therapy designated drug serelaxin, which was initially rejected by the Food and Drug Administration and is conducting a phase 3 trial with an expected readout for acute heart failure in 2016.

Despite these failures, it would appear that one game-changing (but currently experimental) heart failure drug is on pace to dramatically improve patient quality of life and survival perhaps beginning as early as this summer.

One game-changing drug taking aim at heart failure
Although Novartis' shareholders and acute heart failure sufferers had to be disappointed by serelaxin needing to run an additional study, Novartis' other prime heart failure drug, LCZ696, looks poised to make a huge impact on heart failure patients with reduced ejection fraction, or HF-REF. In plainer terms, REF simply refers to systolic heart failure.

LCZ696, a twice-daily tablet, is a combination of two therapies: Novartis' Diovan, an angiotensin receptor blocker designed to allow for blood to flow more freely, and newly-developed sacubitril, which promotes cardiovascular homeostasis by protecting neurohormonal systems of the heart via the natriuretic peptide system.


Source: Novartis.

In the company's PARADIGM-HF study, released in August, LCZ696 led to statistically significant improvement in patients in comparison to the control group taking Merck'sVasotec. Specifically, LCZ696 reduced the risk of death by cardiovascular causes by 20%, it reduced the risk of heart failure-related hospitalizations by 21%, and it reduced the risk of all-cause mortality by 16%. Overall, there was a 20% reduction in cardiovascular death or heart failure hospitalization with a p-value (a measure of chance playing a role) of just 0.000002. In other words, without a shadow of a doubt, this was improvement derived from taking LCZ696.

When it comes to safety, LCZ696 has so far also been a winner. Fewer patients discontinued LCZ696 than Vasotec (10.7% vs. 12.3%), with a majority of observed side effects being described in the press release as "manageable."

Understanding these results to be impressive, the FDA in February granted Novarits' LCZ696 a priority review, shortening the potential review process by four months and pushing its PDUFA decision date to this coming August. The Committee for Medicinal Products for Human Use, which is the European equivalent of the FDA's review board, has also granted LCZ696 an accelerated assessment.

One big step for investors, one giant leap for heart failure patients
Just how big could LCZ696 be? Estimates for peak annual sales of the drug have reached as high as $10 billion. While there have been heart failure therapies that have initially worked in the past, such as ACE inhibitors, there have been few therapies that have significantly extended overall long-term survival. LCZ696, though, looks as if it has a chance do that, and it certainly looks on track to be the first heart failure drug for patents with REF. That formula bodes well for investors, even with Novartis having to run an additional study on serelaxin.

I, for one, am excited at the possibility of a revolutionary new heart disease drug coming to market, perhaps by the end of summer, and making an immediate impact on people's lives. Hopefully this is the start of a number of new heart disease therapies hitting the market within the next couple of years.

The article Meet the Game-Changing Drug Designed to Fight America's Leading Cause of Death originally appeared on Fool.com.

Sean Williamshas no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen nameTMFUltraLong, track every pick he makes under the screen nameTrackUltraLong, and check him out on Twitter, where he goes by the handle@TMFUltraLong.The Motley Fool has no position in any of the stocks mentioned. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.