Shares of Alnylam Pharmaceuticals (ALNY) are up roughly 2% after the company reported positive results in their phase 2 open label extension study --OLE -- of Patisiran. This phase 2 trial is treating patients with a disease known as TTR-Amyloidosis,  Familial Amyloidotic Polyneuropathy -- FAP -- which is a serious disease that causes mutant and wild type TTR genes to deposit proteins in the body. More specifically excessive protein deposits deposited in the peripheral nerves and heart which can possibly even lead to patient death.

There are about 10,000 people worldwide with FAP. These patients with FAP have a life expectancy of about 5 to 15 years and is a huge unmet medical need. These positive phase 2 results were shown at the 67th Annual Meeting of the American Academy of Neurology -- AAN. The phase 2 OLE study enrolled 27 patients and followed these patients for 12-months to determine safety and tolerability of Patisiran.

The results showed continued safety of Patisiran in these patients with FAP. In addition the study results showed a mean 2.5 decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who reached the 12-month endpoint. This compares to historical data with FAP patients being left untreated who saw a 13 to 18 point increase in mNIS+7 at the 12-month time point. Not only did these patients see a decrease on the Neuropathy Impairment Score, but FAP patients saw an average gene knockdown of 80% throughout the trial for this disease. The TTR gene knockdown observed in this study and other studies have the potential to halt Neuropathy progression in these patients.

Alnylam ran this study primarily as a safety study and had just shown these 12-month results at the conference. Although the company expects to report 18-month data from the same study later this year which should further validate the company's Patisiran drug. The company also has another clinical trial in phase 3 known as the "APOLLO" study which is a global trial enrolling FAP patients which will be the final validating evidence of efficacy and safety in this patient population.

Shares of Alnylam hit a new 52-week high of $132.98 per share today but have since retreated down to roughly around $121 per share. The selling should be a short-term effect as the company continues to prove that RNAi science and its Partisiran drug continue to improve the lives of patients. All eyes now are on the future study results for the FAP program which should further validate Alnylam as the powerhouse in the RNAi space.

Alnylam is considered the powerhouse in the RNAi space because of a few reasons. One reason being that it has been able to develop a delivery vehicle for its RNAi drugs. The company has created the GalNAc-siRNA conjugate delivery platform  in addition to additional iterations of the same delivery technology. This leads to more potent gene knockdown efficacy effect and the ability to deliver siRNA molecules systemeically at lower dosing concentrations. The second reason being that the company has established a large pipeline of other drug compounds that target liver diseases which is a huge space in the biotech industry. We foresee that Alnylam will continue to create shareholder value in the coming years and therefore believe that this biotech is a great long-term investment.