Alnylam Pharmaceuticals Inc (ALNY - Analyst Report) recently announced pre-clinical data from a trial evaluating its pipeline candidate ALN-HTT for the treatment of Huntington's disease (HD). ALN-HTT is a novel therapeutic product that uses the potentially revolutionary RNA (ribo nucleic acid) interference (RNAi) technology. The data showed broad central nervous system (CNS) distribution and robust therapeutic silencing of the gene responsible for Huntington's disease. Further, ALN-HTT was well tolerated in these studies.
ALN-HTT is being developed in collaboration with Medtronic, Inc. (MDT - Analyst Report) and the CHDI Foundation. Alnylam expects to file an investigational new drug application (IND) application for the candidate in 2012. Approval of the IND by the US Food & Drug Administration (FDA) will allow the candidate to enter early stage clinical trials.
Besides ALN-HTT, Alnylam is developing ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection and ALN-VSP for the treatment of liver cancer through existing or future partnerships. Alnylam’s core product strategy however is Alnylam 5x15 which aims at developing products for treating genetically defined diseases with unmet medical needs using the ground-breaking RNAi technology. Alnylam expects to have five such RNAi therapeutic programs in advanced clinical development by the end of 2015. Four programs are in pre-clinical/clinical stages of development including ALN-PCS (for hypercholesterolemia), second generation ALN-TTR (ATTR), ALN-HPN (refractory anemia) and ALN-APC (hemophilia). ALN-HTT makes up the fifth candidate.
We currently have an Outperform recommendation on Alnylam. The stock carries a Zacks #2 Rank (“Buy”) in the short term. We like the company’s “5X15” initiative and also the pipeline progress at Alnylam. We believe that 2012 will be an important year for the company due to significant clinical catalysts. Moreover, the company has collaborated with some big pharmaceutical players, such as Roche (RHHBY - Analyst Report), Biogen (BIIB - Analyst Report), Medtronic, Takeda, Kyowa Hakko Kirin, Cubist Pharmaceuticals and Isis Pharmaceuticals (ISIS - Analyst Report), to further develop and utilize its core technology. These partnerships further validate the potential and viability of the RNAi approach.