This New Cholesterol Treatment Has Blockbuster Potential

Here's a bit of news that shouldn't surprise anyone -- America has a problem with high cholesterol. According to the Centers for Disease Control and Prevention, a third of Americans currently have high levels of LDL (bad) cholesterol, but less than half of them seek medical treatment. People with higher LDL cholesterol levels, defined as over 200 mg/dL, are at twice the risk of heart disease compared to those with healthy levels.

This epidemic created a huge demand for LDL cholesterol-lowering drugs over the past decade, with the treatment of high LDL cholesterol rising from 28.4% between 1999 to 2002 to 48.1% between 2005 and 2008.

Today, demand for high LDL treatments remains elevated as poor diets and sedentary lifestyles continue to take their toll on the American population. Although the era of blockbuster high LDL treatments like Pfizer's Lipitor has passed, more sophisticated treatments are starting to appear.

Let's take a look at current and future high LDL treatments to better understand what this evolving market means for investors.

The glory days of statin
Over the past decade, three companies -- Pfizer, AstraZeneca , and Merck -- rode high on billions of dollars in annual revenue generated by their top-selling LDL treatments. All three of their respective treatments -- Lipitor, Crestor, and Zocor -- were statin-based, meaning that they inhibit the production of cholesterol in the liver by targeting a specific enzyme known as HMG-CoA.

However, patent expirations for Lipitor in 2011 and Zocor in 2006 took a bite out of Pfizer and Merck's top line. Even though Crestor's patent hasn't expired yet, the widespread availability of statin-based cholesterol drugs has slowed its growth.



Peak Sales (Year)

2012 sales



$13.7 billion (2006)

$3.9 billion



$7.0 billion (2011)

$6.3 billion



$5.4 billion (2002)

$244 million

Sources: Forbes, company 2012 annual reports.

Since generic statin cholesterol treatments are now widely available from companies like Teva Pharmaceutical and Dr. Reddy's Laboratories, it is unlikely that a new statin treatment for high cholesterol will become the next blockbuster. In addition, statin can cause muscle and liver damage, digestive problems, increased blood sugar and diabetes, and neurological side effects -- causing the drawbacks to outweigh the benefits.

Therefore, investors should look to new kinds of high LDL treatments for a clearer glimpse into the future.

Could this new treatment be safer and more effective?
One new approach to treating high cholesterol is to silence certain genes. A top contender in this space is Alnylam Pharmaceuticals .

Alnylam is focused on creating a new class of drugs, which interfere with RNA expression, known as RNAi treatments, which can directly silence disease-causing genes in the body. Alnylam's experimental high-cholesterol drug, ALN-PCS, reduced patients' LDL levels by an average of 40% and up to 57% on a higher dose during a phase 1 trial. It also reduced LDL cholesterol levels by 68% during a test on primates. By comparison, Pfizer originally claimed that Lipitor reduced high cholesterol in 39% to 60% of patients.

If ALN-PCS can make it through mid-stage and late-stage trials, it could become a revolutionary next-generation treatment for high cholesterol, since its side effect profile compares favorably to statins, with muscle pain and memory loss being the most common problems reported. In addition, no serious adverse events occurred.

Alnylam is currently partnered with critical care treatment company The Medicines Company , which will fund clinical trials of ALN-PCS from phase 2 onwards and commercialize the treatment if successfully approved.

ALNY Chart
ALNY Chart

Source: YCharts.

In addition to ALN-PCS, Alnylam has two other promising products in its pipeline -- a treatment for amyloidosis, a mutation which can affect multiple tissues and organs in the body, and another one for respiratory syncytial virus, a rare respiratory disease that affects children under a year old. Both drugs have reached phase 2 trials.

However, the biggest indicator of Alnylam's huge growth potential is its list of collaborators, which include major companies like GlaxoSmithKline, AstraZeneca, Sanofi, Takeda Pharmaceutical, and Cubist Pharmaceuticals, as well as the U.S. government. Last quarter, Alnylam's revenue of $8.7 billion was completely funded by these collaborations.

The Foolish takeaway
We are at the dawn of a new generation of high-cholesterol treatments, which could be both safer and more effective for patients.

Whereas older blockbuster treatments are fading away into generic obscurity, newer treatments like ALN-PCS could open the door to a new age of blockbuster high LDL drugs and wipe away the previous generation of statin-based treatments.

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Leo Sun has no position in any stocks mentioned. The Motley Fool recommends Alnylam Pharmaceuticals. 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.

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