Shares of Alnylam Pharmaceuticals (ALNY) spiked as high as 18% yesterday after the company announced results from a phase 2 open-label study in patients with TTR-Amyloidosis in FAP. For starters, open-label study means that Physicians and patients know that they are getting the Patisiran drug -- Alnylam's RNAi drug -- and no placebo counterpart. The company is currently conducting a phase 3 trial for Patisiran in FAP known as the "APOLLO" trial. The additional phase 2  was to run a study where investigators can see how the drug performs over a period of time instead of having a closed analysis. TTR-Amyloidosis is a disease characterized by excessive folding of the protein thereby depositing into pockets of the peripheral nerves and heart.

Alnylam is using Patisiran to knock down the TTR-genes that are produced in the liver itself. The RNAi drug itself, using Tekmira Pharmaceuticals (TKMR) LNP -- Lipid Nanoparticle -- delivery technology, is intended to knock down the genes of the diseases. The knocking down of theses genes is intended to improve the patient's life expectancy and/or prevent further damage in their body.  In these phase 2 results reported the other day, the Patisiran drug was able to achieve a knockdown of 90% of the TTR-Amyloidosis FAP disease over a 9 month period. Life expectancy for patients with TTR-Amyloidsosis in FAP is around 5 to 15 years from the moment the disease takes effect. There are about 10,000 patients worldwide with TTR-Amyloidosis FAP disease, therefore Alnylam should be able to give the Patisiran drug to this orphan disease. Orphan diseases are diseases that are an unmet medical need that affect less than 200,000 patients worldwide. This gives Alnylam special perks if this drug makes it past the final phase 3 clinical trial like longer exclusivity in sales, and quicker review from the FDA.

In addition, the efficacy of Patisiran was measured using a score technique known as the Modified Neuropathy Impairment Score -- (mNIS+7). The basic thing to know about this is that patients with TTR-Amyloidosis end up with 10 points or higher on the score taking a placebo drug. On the other hand taking Patisiran the patients actually decreased by about 1 point. Generally this is good because patients taking Alnylam's drug were able to avoid going up in score like placebo patients have done in the past. This shows the true efficacy of RNAi science, and the promise of RNAi for the future.

Alnylam is currently the leader in RNAi at the moment but that doesn't mean it won't change later down the line. Despite that this company continues to see excellent clinical trial results and already has an RNAi drug in a phase 3 clinical trial, there are no approved RNAi drugs on the market; so Alnylam has the opportunity to become the first RNAi biotechnology company to have an approved RNAi drug. We believe the company to be a great long term buy as the company advocates its 5x15 program. This means that Alnylam expects to have 5 mid stage drugs by 2015 in the RNAi space. Sanofi (SNY) is already excited about Alnylam's prospects as it has already acquired about 18% of the company. We believe that Alnylam is positioned well in the RNAi space and will continue to post positive results for other drug compounds in the clinic.