Alnylam Pharmaceuticals Inc. (ALNY) is a biopharmaceutical company developing novel therapeutics based on a biological pathway known as RNA (ribo nucleic acid) interference (RNAi). Alnylam is developing several candidates using this technology for the treatment of multiple diseases.
ALN-PCS, a systemically administered RNAi therapeutic, is one of the important candidates at Alnylam being developed for the treatment of hypercholesterolemia. Positive results from a phase I study on ALN-PCS were published in The Lancet. The study evaluated a single dose of the candidate without concomitant lipid-lowering agents such as statins.
Results from the study revealed that a rapid, dose-dependent reduction in plasma PCSK9 of up to 84% from baseline was observed in patients in the ALN-PCS arm compared to placebo. Moreover, serum levels of low-density lipoprotein cholesterol (LDL-C) were reduced by up to 57% from baseline in these patients compared to placebo. Results also revealed that the candidate was generally safe and well tolerated in this phase I study.
We remind investors that Alnylam had formed a global alliance with The Medicines Co. (MDCO) for the development and commercialization of the ALN-PCS RNAi program earlier this year. As per the terms of the agreement Alnylam is eligible to receive up to $180 million on the achievement of development and commercial milestones apart from an upfront cash payment of $25 million. The company could also receive scaled double-digit royalties from The Medicines Company on global sales of ALN-PCS products commercialized under this agreement.
Alnylam, along with The Medicines Company, is currently advancing the development of another RNAi therapeutic candidate targeting PCSK9, ALN-PCSsc. In preclinical studies, administration of ALN-PCSsc resulted in more than 80% reduction in plasma PCSK9 with more than 50% reduction in LDL-C. Alnylam intends to nominate a development candidate for ALN-PCSsc by year end.
We are encouraged by the pipeline progress at Alnylam. However, most of the candidates at the company’s pipeline are in the early or middle stages of development. We expect investor focus to remain on Alnylam’s pipeline going forward.
Alnylam presently carries a Zacks Rank #2 (Buy). Stocks such as Endo Health Solutions Inc. (ENDP) and Actelion Ltd. (ALIOF) currently look better positioned with a Zacks Rank #1 (Strong Buy).