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On October 3rd, Alnylam Pharmaceuticals (NASDAQ:ALNY) announced positive results of its Phase I study of its experimental drug ALN-PCS for the treatment of hypercholesterolemia. Results of the study showed that a single dose of ALN-PCS, which is administered intravenously, reduced LDL cholesterol an average of 40%. While it was a small and very early study, according to researchers, ALN-PCS -- if successful in large trials -- could potentially replace or complement today's drugs of choice: statins including Lipitor, Crestor, and Zocor.

ALN-PCS is a systemically administered RNAi therapeutic targeting PCSK9, a protein involved in the metabolism of LDL-C, AKA "bad" cholesterol." ALN-PCS inhibits the synthesis of PCSK9, thereby reducing intracellular and extracellular levels of PCSK9, resulting in lowered levels of LDL-C. According to the study, ALN-PCS achieved rapid, dose-dependent, and durable knockdown of PCSK9 protein levels in plasma of up to 84% relative to baseline and placebo. The drug was also shown to be generally safe and well tolerated in the study and there were no serious adverse events.

This could be big news for Alnylam and its partner with ALN-PCS, The Medicines Company (NASDAQ:MDCO), as statins sales are roughly a $19 billion a year business in the U.S. alone. High LDL, along with high blood pressure are two of the main issues that increase ones risk for heart disease, the number 1 killer with over 17 million deaths worldwide each year. According to the Center for Disease Control, 71 million Americans over the age of 20 have high cholesterol levels. High cholesterol and high blood pressure accounts for $1 in every $6 spent on healthcare.

The study's co-author, Kevin Fitzgerald, senior director for research at Alnylam, commented on the next step for the drug: "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 February 2013, Alnylam formed an exclusive global alliance with The Medicines Company for the development and commercialization of the ALN-PCS program. Alnylam will lead the program through the completion of Phase I. The Medicines Company is responsible for leading and funding development from Phase II forward and commercializing the ALN-PCS program if successful.

Alnylam's RNAi Technology: Suppressing Disease-Causing Genes

Researchers are increasingly identifying human genes as the root cause of numerous diseases. The problem is, while certain genes can be identified as causing diseases, traditional medicine had no way to suppress them. In 2006, two Nobel Prize scientists developed RNA interference, or RNAi, technology, which can suppress disease-causing genes in the human body. Alnylam is harvesting this technology and developing a new class of human therapeutic products via RNA-based therapies.

The company has two candidates in phase 2 trials. The first, ALN-TTR, is a drug intended to treat TTR amyloidosis -- a mutation in genes that affects the organs and tissues of the body, including nervous and cardiac tissue. The drug has the potential to switch off the mutated TTR gene as the study has shown ALT-TTR reduced TTR protein levels by up to 94%. Add to that, the safety profile of the drug was very impressive. In October 2012, the company formed an exclusive alliance with Genzyme, part of the French giant healthcare company, Sanofi (NYSE:SNY), to advance the ALN-TTR program in the Japanese and broader Asian market. Alnylam maintained its development and commercialization rights to the drug in North and South America, Europe, and the rest of world.

The other drug in Phase II trials is ALN-RSV01, for the treatment of respiratory syncytial virus (RSV) infections. RSV is a highly contagious lower respiratory tract infection that is the most common in children under one year of age, and accounts for more than 300,000 hospitalizations per year in the U.S. Also, an RSV infection can be found in lung transplant patients, and those infected with RSV are at risk for both acute rejection and a bronchiolitis obliterans syndrome (BOS). Alnylam has formed an alliance with Japan's Kyowa Hakko Kogyo Co. to develop and commercialize ALN-RSV0 in Japan and other Asian regions.

Also, in collaboration with GlaxoSmithKline (NYSE:GSK), the company formed Alnylam Biotherapeutics, designed to focus on the development of RNAi technologies for applications in the $100 billion biologics marketplace, including recombinant proteins and monoclonal antibodies. Alnylam's VaxiRNATM platform applies RNAi technology to improve the manufacturing processes for vaccines including influenza.

Alnylam: A Young But Strong Pipeline

Alnylam has a very solid pipeline in the RNAi. The company has two products in Phase II studies, 3 in Phase I studies, 1 in development, and 4 in the discovery stage. The company expects to have 5 RNAi therapeutic programs in clinical development, including late stages, by 2015.

As noted earlier, the company's strong position on RNAi therapeutics and intellectual property has bolstered Alnylam's ability to form major collaborations and alliances with a number of the larger pharmaceutical companies including platform alliances with Roche, Takeda and Monsanto, and drug development collaborations with Novartis and Iris, along with product alliances with Cubist and Medtronic.

Conclusion

Alnylam is a $4.07 billion market cap company. Its stock has had a banner year, up over 250% YTD, and is just shy of its 52-week high, closing on Friday October 4th at $64.63 per share. At this point, the company is still a development company and therefore has no sales, but generates revenue from research collaborations, which for the previous quarter reported revenues of $8.7 million, including $5.5 million from Takeda, $1.4 million from Monsanto, and $1.8 million from The Medicines Company.

In late September, analysts at Leerink Swann gave Alnylam an outperform rating and raised its price target from $57.00 to $80.00. However, analysts at Zacks reiterated a neutral rating in mid-August and have a $51.00 price target on the stock.

While Alnylam stock has risen dramatically this year, if positive results continue to come from the company's pipeline, I can see the stock maintain its upward trend. However, one must remember it is a long process from Phase II to FDA approval; thus, its promising pipeline still has many challenges ahead. That being said, if any of a number of the drugs in the pipeline receives FDA approval, the company's revenues should soar, as should the stock.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article.

Source: Alnylam Developing The Next Generation Drug Platform In The $19 Billion Cholesterol Market