Blackstone and Alnylam close $150 million R&D funding to advance RNAi therapeutics for cardiovascular disease

Blackstone, a private investment platform that invests across the life-cycle of companies and products within the key life science sectors, and Alnylam Pharmaceuticals, one the leading RNAi therapeutics companies, today announced the closing of $150 million R&D funding to advance RNAi therapeutics for cardiovascular disease. The funding is part of the R&D funding component of the companies’ previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.

As part of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylam’s cardiometabolic disease programs vutrisiran and ALN-AGT. The investment includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 and Phase 3 development of ALN-AGT, in development for the treatment of hypertension.

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.

By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

In April 2020, Blackstone and Alnylam entered into a broad $2 billion strategic financing collaboration anchored by Blackstone’s purchase of 50 percent of royalties owed to Alnylam on global sales of inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, currently under review by the U.S. Food and Drug Administration and other regulatory authorities. The strategic financing collaboration is expected to enable Alnylam’s achievement of a self-sustainable financial profile without need for future equity financing, accelerating the commercial potential of Alnylam’s rapidly advancing product portfolio.

Founded in 2002, Alnylam translates RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases.