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Last week was another great one for clinical trial results, although you wouldn't know it looking at biotech and pharmaceutical stock prices. Amgen (NASDAQ: AMGN ) released robust data from an enormous late-stage program. Isis Pharmaceuticals (NASDAQ: ISIS ) furthered the case for RNA antisense therapies. The biggest win of the week belongs to Novartis (NYSE: NVS ) , the pharmaceutical giant that shocked the industry, and perhaps itself, with a surprise victory just days after a major setback.
Look out Zetia
Amgen continued marching ahead in the race to develop next-generation cholesterol-lowering drugs for the statin intolerant. Last weekend, the company released some detailed data from five Phase 3 studies involving its PCSK9 inhibitor, evolocumab.
The amount of data released could fill volumes, but one trial in particular illustrates evolocumab's potential. The Gauss-2 study compared Amgen's candidate to Merck's (NYSE: MRK ) blockbuster Zetia. Last year, Merck recorded $2.66 billion in Zetia sales. It's a popular alternative to statins that reduces the amount of cholesterol absorbed by the small intestine. During the study, evolocumab patients showed a 38 percentage point reduction from baseline in LDL cholesterol compared to Zetia at 12 weeks. The observed 38 percentage point superiority over Zetia was the same for patients receiving both monthly and biweekly evolocumab injections. Nobody likes needles, but if the data continue to be strong, I could see people trading in their daily pills for the more effective monthly injections.
Isis and its proprietary development platform was vindicated last January when the FDA approved its first RNA antisense drug, Kynamro. Unfortunately, patients and physicians don't seem interested.
Developed in partnership with Sanofi's (NYSE: SNY ) Genzyme, Isis is entitled to between 30% and 50% of Kynamro's profits. The cholesterol-lowering therapy reportedly isn't setting any records, however, with Sanofi expanding its sales force to juice sales.
Last week, one of Isis' 31 candidates took a big step toward commercialization. Its leading unpartnered compound, ISIS-APOCIII, inhibits production of apolipoprotein C3. Patients with too much apo-C3 typically have dangerously elevated levels of triglycerides in their bloodstream. They're at high risk for cardiovascular disease and pancreatitis. With a lack of highly effective therapies, this compound could also reduce elevated levels of red ink on the company's income statements.
In a recent phase 2 trial, patients receiving 200 mg or 300 mg doses ISIS-APOCIII achieved triglyceride reductions of 51% and 64%, respectively. Average levels of high-density lipoprotein -- the good kind -- increased up to 51% and 52%, respectively.
A big concern regarding RNA antisense drugs is potential liver damage. Kynamro comes with a boxed warning citing a risk of hepatic toxicity. There were "no elevations of liver enzymes greater than three times the upper limit of normal" among the 20 patients dosed with ISIS-APOCIII. This is a step in the right direction, but waiting for more safety data from a larger trial is probably a good idea.
The cardiology segment at Novartis has been through some ups and downs lately. Serelaxin was one of the first drugs to receive Breakthrough Therapy status from the FDA last year. Late last month, however, the agency's independent advisory committee voted unanimously against it while citing issues with Novartis' pivotal trial design and data.
Just a few days later, Novartis announced what was easily last week's biggest clinical win. An independent data monitoring committee unanimously voted in favor of ending the Paradigm-HF trial early. The 8,436 patient trial pitted the company's LCZ696 against a heart failure treatment standard, the ACE inhibitor enalapril. A statistically significant number of patients with chronic heart failure in the LCZ696 arm lived longer without being hospitalized.
The potential market for an improvement over ACE inhibitors in the treatment of chronic heart failure is hard to overstate. The American Heart Association estimates more than 5 million people in the U.S. now have heart failure. This figure, unfortunately, should continue growing along with the aging population and the increasing population of heart attack survivors. If the existing data holds up to regulator scrutiny, LCZ696 could add a great deal to Novartis' topline in the near term.
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