Pipeline Probe: Alnylam Pharmaceuticals, Inc. (ALNY)

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Shares of biotech Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) have soared over 160% in the past year. The company’s a leader in developing drugs and delivery systems centered on RNA interference, or RNAi, It’s an emerging area that could see major gains in the future for the companies at the forefront. And Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)’s at the forefront.

The past year has ushered in new drug alliances with The Medicines Company (NASDAQ:MDCO) and a Sanofi SA (ADR) (NYSE:SNY). But collaborations are only as strong as the drugs behind them.

How healthy are Alnylam’s pipes?

Source: Alnylam.

5 x 15 strategy

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)’s pipeline goals are spelled out in their 5×15 strategy. The company hopes to have five projects pushed into advanced stage trials by 2015. The goal’s ambitious considering most of the projects are in early-stage trials at the moment.

PCSK9 program

Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY)’s ALN-PCS program features two early-stage versions of a PCSK9 inhibitor for the treatment of hypercholesterolemia, or severely high cholesterol. The program includes the intravenous ALN-PCS02 that has finished phase 1 and ALN-PCSsc, a subcutaneous injection currently in pre-clinical development.

PCSK9 drugs won’t replace the off-patent statin class, which are cheap and generally effective. But around 10% of general cholesterol patients have a statin intolerance. Statins also tend not to work in patients with genetic cholesterol issues such as heterozygous familial hypercholesterolemia.

It’s way too early to compare ALN-PCS data to projects from Amgen, Inc. (NASDAQ:AMGN) and Regeneron Pharmaceuticals Inc (NASDAQ:REGN)/ Sanofi SA (ADR) (NYSE:SNY), which are in late-stages. But The Medicines Company (NASDAQ:MDCO) considers ALN-PCS so promising it stepped in earlier this year for a collaboration that paid Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) $25 million upfront and carries the potential for $180 million in development and commercial milestones. The deal involves The Medicines Company (NASDAQ:MDCO) funding development starting in phase 2, and potentially continuing on to market. Alnylam would receive double-digit royalty payments.

TTR-mediated amyloidosis

TTR-mediated amyloidosis is a rare genetic systemic disease that leads to potentially fatal damage to the organs. Rarity means a smaller market but also the potential to price the drug high enough for balance. This is also an area of clear unmet need.

The only treatments for early stages of the disease are Pfizer’s Vyndaquel or a liver transplant. And Vyndaquel currently only has approval in Europe; the Food and Drug Administration issued a complete response letter last summer that requested further efficacy data.

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