Alnylam scores $2B from Blackstone to propel pipeline prospects

John Maraganore casual
If approved, inclisiran would compete against Amgen's Repatha and Regeneron and Sanofi’s Praluent, which are both antibodies against PCSK9. (Alnylam)

With two approved drugs and a third on the way, Alnylam secured some much-needed capital to keep its RNAi pipeline rolling. Blackstone will provide Alnylam with up to $2 billion in exchange for a 50% cut of the royalties on its Novartis-partnered cholesterol fighter, inclisiran.

Under the deal, Blackstone will buy $100 million in Alnylam stock and pony up $1 billion for 50% of Alnylam’s royalties and commercial milestones for inclisiran. The drugmaker could pick up another $150 million from Blackstone Life Sciences for its cardiometabolic programs vutrisiran and ALN-AGT, as well as a loan worth up to $750 million. The company is developing vutrisiran for ATTR amyloidosis and ALN-AGT for hypertension.

“Alnylam is focused on building a top-tier biopharmaceutical company, advancing RNAi therapeutics as a whole new class of medicines with transformative potential for patients around the world. This exciting new relationship with Blackstone brings us much closer to that goal, securing our bridge towards a self-sustainable financial profile that we believe can now be achieved without any need for Alnylam to access the equity markets in the future,” said Alnylam CEO John Maraganore, Ph.D., in a statement.

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The FDA and the European Medicines Agency are reviewing inclisiran for the treatment of atherosclerotic heart disease and hypercholesterolemia, a genetic disorder that causes very high cholesterol, including in patients who have maxed out the doses of other treatments. The drug landed at Novartis via its $9.7 billion takeover of The Medicines Company, which licensed it from Alnylam.

“If approved, we believe this therapy holds enormous promise as a potential game-changer in hypercholesterolemia management. We are pleased to retain half of the royalties we receive from Novartis, allowing Alnylam to benefit from inclisiran’s anticipated future success,” Maraganore added.

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Inclisiran would compete against Amgen's Repatha and Regeneron and Sanofi’s Praluent, which are both antibodies against PCSK9, a protein that blocks the liver’s ability to clear “bad” LDL cholesterol from the blood. But rather than taking direct aim at the protein, inclisiran works by blocking the expression of specific genes and hindering the production of PCSK9 itself. It also acts longer than its potential rivals. It is given as an injection every six months, compared to once a month or every two weeks.