Regulus, Alnylam, and Collaborators Publish In Vivo Efficacy Data for a microRNA Therapeutic in a Disease Model

Regulus Therapeutics LLC, a joint venture between Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. formed to discover, develop, and commercialize microRNA-based therapeutics, announced the publication of new research in the journal Nature on the role of a microRNA, known as miR-21, in heart failure.

The new findings demonstrated that miR-21 is over-expressed in the failing human heart and contributes to heart failure through its regulation of a stress-response signaling pathway associated with changes in heart muscle structure and function.

The study went on to demonstrate that targeting miR-21 with an anti-miR-21, antisense oligonucleotide prevented heart failure in mouse models. Furthermore, administration of anti-miR-21 after established heart failure resulted in a significant treatment benefit in the animal model.

“We view this new study as a landmark event in the advancement of microRNA therapeutics as a new class of innovative medicines. Indeed, we believe that this is the first study to clearly demonstrate therapeutic efficacy for targeting microRNAs in an animal model of human disease,” said Kleanthis G. Xanthopoulos, Ph.D., President and Chief Executive Officer of Regulus Therapeutics. “Moreover, these exciting data highlight microRNA-based therapeutics specifically targeting miR-21 as a promising approach for the treatment of heart failure, extending the scope of disease opportunities for Regulus.”

“This study has revealed a key role for miR-21 in regulating a major stress-response pathway in the failing heart. Administration of anti-miR-21 led to a striking effect in preventing and treating cellular, morphologic, and functional features of heart failure in a well-established animal model,” said Peter Linsley, Ph.D., Chief Scientific Officer of Regulus Therapeutics. “Most importantly, these new in vivo data point to the significant potential for targeting microRNAs, where therapeutic impact can be achieved by interrupting entire pathways of disease, not just single disease targets.