Alnylam, with $2 billion from Blackstone, looks to get financially sustainable

The Cambridge biotech is developing treatments for cardiovascular and genetic diseases.

John Maraganore, chief executive of Alnylam Pharmaceuticals, posed for a portrait in his office in Cambridge in 2016.Keith Bedford

The private equity firm Blackstone Group is investing up to $2 billion in Alnylam Pharmaceuticals, a big bet on the Cambridge biotech’s approach to developing drugs for genetic diseases and disorders that need better treatments.

The deal, which was announced Monday, is anchored by Blackstone’s purchase of halfthe royalties owed to Alnylam on global sales of inclisiran, an experimental RNA interference drug for the treatment of high cholesterol.

Inclisiran is a twice-a-year injected RNAi therapeutic that reduced bad cholesterol in a late-stage clinical trial. If approved, the medicine is expected to help reduce a major risk factor for cardiovascular disease, the leading global cause of death, according to Alnylam.

Blackstone’s recently created life sciences unit will also invest up to $150 million in the development of two other Alnylam medicines. Blackstone’s credit arm will provide Alnylam with a loan of up to $750 million. And Blackstone, which is based in New York, will buy $100 million of the biotech’s newly issued stock.

“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," said John Maraganore, Alnylam’s chief executive since 2002, in a statement. “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.”

Nicholas Galakatos, global head of Blackstone Life Sciences, said Alnylam’s RNAi technology "represents one of the most promising and rapidly advancing frontiers in biology and drug development today, and aligns perfectly with our investment strategy.”

In 2018, Alnylam won US approval of the first-ever drug to rely on a Nobel Prize-winning approach to mute disease-causing genes. The drug, marketed as Onpattro, treats hereditary transthyretin-mediated amyloidosis, or hATTR, in adults. That’s an inherited, progressively debilitating rare disease that affects about 50,000 people worldwide, causing nerve degeneration and heart damage. The median survival rate is less than five years after diagnosis.

That approval capped about 20 years of efforts to develop a medicine that mutes genes that aren’t functioning properly. A pair of Americans, including Craig Mello, a professor at the University of Massachusetts Medical School, won the Nobel Prize in 2006 in medicine or physiology for discovering that strands of RNA can silence genes that cause diseases.

Last November, Alnylam won approval of its second RNAi drug, marketed as Givlaari. The medicine treats a rare genetic condition called acute hepatic porphyria, which can cause attacks of severe abdominal pain.

Like many biotech startups that work for years before getting a drug to market, Alnylam has yet to turn a profit. It spends about $1 billion a year, according to Alethia Young, a research analyst at Cantor Fitzgerald. She said the deal with Blackstone “may bridge the company out almost to being profitable.”