Alnylam Pharmaceuticals, Inc. (ALNY) announced several study results on its pipeline candidates which were presented at the 10th annual meeting of the Oligonucleotide Therapeutics Society.
Alnylam announced additional results from a phase I study on ALN-TTRsc, a subcutaneously administrated ribo nucleic acid interference (RNAi) therapeutic being developed for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR) in patients suffering from TTR cardiac amyloidosis. Results from the study showed that rapid, dose-dependent, stable and durable knockdown of serum TTR of up to 96.2% was observed in patients treated with ALN-TTRsc.
Alnylam intends to advance ALN-TTRsc in phase III development which is expected by the end of this year. The candidate is currently being developed in a phase II study in ATTR patients suffering from TTR cardiac amyloidosis. Initial data from the study is expected next month.
Preclinical data on ALN-GO1 being developed for the treatment of primary hyperoxaluria type 1 was also announced. Data revealed efficacy in rodent disease models treated with the candidate. The company plans to file an investigational new drug application for the candidate in 2016.
Alnylam also announced pre-clinical results on ESC-GalNAc-siRNA conjugates which revealed that the candidate achieved comparable knockdown activity in mouse liver when delivered through a Microsprayer device as against a subcutaneous injection.
Alnylam has progressed well with its pipeline so far. Earlier in the week, Alnylam announced encouraging results from an ongoing phase II open-label extension study (n=27) on one of its most important pipeline candidates, patisiran. Patisiran is a subcutaneously administrated RNAi therapeutic being developed for the treatment of ATTR in patients suffering from familial amyloidotic polyneuropathy (read more: Alnylam Jumps on Positive Patisiran Data from Phase II Study).
Alnylam currently has no marketed products. Moreover, most of the candidates at its pipeline are in early or mid-stages of development. Successful development of its pipeline is crucial for the company.
Alnylam carries a Zacks Rank #2 (Buy). Some other stocks in the healthcare sector that are worth considering include Medivation, Inc. (MDVN), Emergent BioSolutions, Inc. (EBS) and Ligand Pharmaceuticals Incorporated (LGND). All three stocks hold a Zacks Rank #1 (Strong Buy).
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