Alnylam Pharmaceuticals, Inc. (NasdaqGS:ALNY - News) recently announced that it has received a notice of allowance from the US patent office for a new patent which covers the company’s proprietary second generation lipid nanoparticle (:LNP) platform, including the “MC3” lipid.
Alnylam, which currently has no product on the market, develops novel therapeutics based on a biological breakthrough technology known as RNA (Ribo Nucleic Acid) interference (RNAi). The company wants to focus its resources on its core product strategy, Alnylam 5x15, which is aimed at developing five RNAi therapeutic products for the treatment of genetically defined diseases addressing major unmet medical needs. The LNP platform is being used to develop many programs under the Alnylam 5x15 strategy. These include ALN-TTR02, an RNAi therapeutic that employs a second-generation LNP formulation to treat transthyretin (:TTR) mediated amyloidosis or ATTR and ALN-PCS, another RNAi therapeutic for the treatment of hypercholesterolemia.
Under the ATTR program, Alnylam is also developing ALN-TTR01, a systemically delivered RNAi therapeutic that employs a first-generation LNP formulation to treat ATTR. The other programs which form part of the Alnylam 5x15 strategy include ALN-HPN (refractory anemia), ALN-APC (hemophilia) and ALN-TMP (hemoglobinopathies). Among these, Alnylam aims to accelerate development of ALN-TTR and ALN-APC which it believes holds the most promise.
We currently have a Neutral recommendation on Alnylam. The stock carries a Zacks #2 Rank (“Buy”) in the short term.
We like the company’s “5X15” initiative and the pipeline progress. Moreover, the company has collaborated with some of the big pharmaceutical players to further develop and utilize its core technology. These partnerships validate the potential and viability of the RNAi approach. The year 2012 is important for the company with many a program expected to report data. We therefore prefer to wait and see how the year shapes up for Alnylam and whether the data presentations increase visibility on the approval timeline.
More From Zacks.com