Alnylam Pharmaceuticals’ (ALNY) New Cholesterol Treatment Could Be A Next Generation Blockbuster

Earlier this month, Alnylam Pharmaceuticals (ALNY) impressed investors with positive results from its phase 1 study of its experimental high cholesterol drug, ALN-PCS. The results showed that ALN-PCS reduced LDL ("bad") cholesterol by an average of 40%. Although this is an early study, it shows potential of becoming a new, more effective treatment in a market crowded with older statin based treatments like Lipitor, Crestor, and Zocor.

Shares of Alnylam have risen 55% over the past three months due to the positive buzz regarding ALN-PCS. Daily Chart
ALN-PCS, which is an intravenous treatment, directly targets a protein which aids in the production of bad cholesterol. It is part of a new class of drugs which interfere with RNA sequencing, known as RNAi technology, which can directly suppress disease-causing genes in the body. There were no serious adverse events from the phase 1 trial and the drug was found to have a mild side effect profile. Alnylamâ s success could also boost sales for its partner on the drug, The Medicines Company MDCO, a $2 billion company which currently produces three intravenously injected drugs for critical care patients. The Medicines Company will fund trials from phase 2 forward and commercialize the treatment if successful. The market for statin-based cholesterol treatments, which have mostly gone generic over the past few years, represents a $19 billion per year business in the United States. Alnylam will now move ALN-PCS onto a larger study. Kevin Fitzgerald, the senior director for research at the company, stated, "The next step will be larger, multi-dose studies to address the long-term safety and tolerability of ALN-PCS in various patient populations, including those on statins and those who are statin-intolerant." In addition to ALN-PCS, the company is also creating a RNAi treatment for amyloidosis, a mutation of the genes which affects multiple tissues and organs in the body. It is also producing another treatment for respiratory syncytial virus (RSV) -- a rare respiratory disease which affects children under a year old. Both drugs are currently in phase 2 trials. The company also has several major collaborations with large companies. A key one is with pharma giant GlaxoSmithKline GSK to develop new RNAi technologies for next-generation biologics treatments. Other collaborations include Takeda Pharmaceutical, Monsanto MON, Roche, Medtronic MDT, and The Medicines Company, among others. Its revenue of $8.7 billion last quarter was completely generated by these collaborations. Other News About ALNY Alnylam Developing The Next Generation Drug Platform Will new RNAi treatments be the next generation of high cholesterol treatments? Alnylam Adds Hepatic Porphyria Drug to Official Pipeline Alnylamâ s pipeline gets a little bit stronger. Other Stocks in the News The Second-Screen Phenomenon Is Bigger Than TwitterVs. Facebook Is the second screen phenomenon hype, attention deficit disorder, or really the future of advertising? Starbucks Corporation Is Today's Momo Momentum Stock Will Starbucks keep soaring higher? Copyright 2013 by, Inc. InvestorGuide has no control over the sites we link to, is not affiliated with these sites, and cannot take responsibility for their quality or suitability. The news, analysis, commentary and profile information is not meant to be comprehensive, and the data provided is not guaranteed to be accurate. WebFinance Inc., the publisher of this newsletter, is not a registered investment advisor or a broker/dealer. This is not a stock recommendation newsletter but rather a source for investment ideas, and we encourage you to fully research any company before considering investing. The opinions expressed herein are those of the author and do not necessarily represent the views of nor are they endorsed by WebFinance Inc. No employee of WebFinance has owned or currently owns any shares in the company described above. The above is neither an offer nor solicitation to buy or sell any securities. The trading of securities may not be suitable for all potential readers of this newsletter, and the purchase of stocks mentioned in this newsletter may result in the loss of some or all of any investment made. We recommend that you consult a stockbroker or financial advisor before buying or selling securities or making investment decisions. We are not responsible for claims made by advertisers and sponsors. Anyone who makes decisions based on what they read here does so at their own risk and cannot hold WebFinance Inc. (DBA, Inc.) or its employees responsible.

Published on Oct 17, 2013
By Leo Sun
Leo Sun
Leo Sun is a freelance finance writer and position trader. He focuses on a combination of value and momentum investing, with a strong interest in the trading philosophies of Warren Buffett and Peter Lynch. Leo also has experience writing articles to help small business owners acquire loans and manage their finances. He regularly contributes to the Stock of the Day analysis.

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