Alnylam Pharmaceuticals Inc. ( ALNY ), a biopharmaceutical company, is focused on the development of novel therapeutics based on RNA (ribo nucleic acid) interference (RNAi). The company is developing several candidates using this technology for the treatment of multiple diseases.
Although most of the candidates at Alnylam are in early or mid-stages of development the company has progressed well with its pipeline in the recent past. ALN-PCSsc, a subcutaneously administered RNAi therapeutic, is an important candidate at Alnylam under the company's ALN-PCS RNAi program. The candidate is being developed for the treatment of hypercholesterolemia.
Earlier this year, Alnylam had entered into a global alliance with The Medicines Co. ( MDCO ) for the development and commercialization of the ALN-PCS RNAi program. The companies announced new data from non-human primate studies which revealed that ALN-PCSsc was successful in reducing PCSK9 by up to 90%. The candidate was also successful in reducing low-density lipoprotein (LDL) cholesterol in the absence of statins by up to 68%.
Alnylam and The Medicines Co. intend to file an investigative new drug application for the candidate in the U.S. towards the end of 2014.
As per the collaboration between the two companies, Alnylam will conduct pre-clinical and phase I studies following which The Medicines Co. will take up funding and development for the rest of the ALN-PCS program. The Medicines Co. will also be responsible for the commercialization of the ALN-PCS program, if successful.
We are encouraged by the pipeline progress at Alnylam. We expect investor focus to remain on the company's pipeline.