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
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Published on Oct 17, 2013
By Leo Sun