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.
Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) again has two drugs in trials, one intravenous and one subcutaneous. ALN-TTR02 is expected to report top-line phase 2 results mid-year. ALN-TTRsc should report phase 1 results around the same time. Encouraging data would lead to the next phase trials to begin at the end of the year.
Finishing the 5
ALN-AT3 is a pre-clinical hemophilia treatment that should launch a phase 1 trial in late 2013. Alnylam’s seeking a Asian-market partner for the drug.
ALN-AS1 treats acute intermittent porphyria — a rare metabolic disorder that can cause abdominal pain, motor weakness, and cognitive impairments. The drug’s only in the discovery phase but the company’s hoping to move the process into the clinic next year.
ALN-TMP is in the pre-clinical stage and Alnylam hopes to find a partner before moving into phase 1. The drug treats beta thalassemia, which is a genetic blood condition caused by improperly produced hemoglobin that leads to anemia.
Foolish final thoughts
How healthy are Alnylam’s pipes? It’s too soon to say. But the picture will continue to become clearer in the coming year, especially if the company sticks to its 5×15 plans.
For now, I’m adding Alnylam to my watchlist. Want to keep up with the latest news from the company? Add Alnylam to your free, personalized watchlist.
Brandy Betz has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. Brandy is a member of The Motley Fool Blog Network — entries represent the personal opinion of the blogger and are not formally edited.
The article Pipeline Probe: Alnylam originally appeared on Fool.com is written by Brandy Betz.
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