Alnylam Going After World's Most Expensive Drug in New Trials

  • CEO says $440,000 price on existing drug gives room to compete
  • Alnylam says experimental drug will be in late trials in 2017

Alnylam Pharmaceuticals Inc. is taking on the world’s most expensive drug, betting that its experimental treatment for a rare disease can compete in a narrow market dominated by one pricey therapy.

In 2017, the biotechnology company plans to start final-stage trials of ALN-CC5 in patients with a rare and often deadly blood disease known as PNH. It’s a space dominated by Alexion Pharmaceuticals Inc., which charges about $440,000 a year for its drug Soliris, the only approved treatment for the disorder.

John Maraganore

Photographer: Ruby Arguilla Tull/Bloomberg

“Alexion’s drug is the world’s most expensive,” Alnylam’s Chief Executive Officer John Maraganore said in an interview. “So we can think about that space with a pretty high ceiling.”

The company hopes it’s giving Alexion "indigestion" with the candidate treatment, Maraganore said at his office in Cambridge, Massachusetts. If the trials prove ALN-CC5 can treat PNH, or paroxysmal nocturnal hemoglobinuria, a potentially fatal condition that causes clotting and anemia, being competitive on pricing shouldn’t be too difficult, he said. 

Maraganore laid out his strategy ahead of Alnylam’s investor conference that takes place Thursday, when the company will highlight trials of a number of experimental drugs for rare diseases. The drugmaker is looking to challenge companies that make treatments for the blood disorder hemophilia with two late-stage trials next year, and expects to file for its first drug to be approved in 2017 for a rare, inherited disease caused by deposits of an abnormal version of the protein transthyretin. Alnylam aims to have a total of five or more late-stage trials running by the end of 2017.

Ritu Baral, an analyst at Cowen & Co., said that a head-to-head comparison study may be needed for Alnylam’s candidate to prove that it’s as good as Alexion’s drug.

“Is it a drug in PNH in a world where Soliris doesn’t exist? Yes,” Baral said in a phone interview before the conference. “Is it a PNH drug in a world where Soliris does exist? Unclear.”

“There is a minority of investors that think this could absolutely go toe-to-toe with Alexion,” she said.

Alnylam isn’t planning a head-to-head trial, Maraganore said, nor does he see it as necessary for approval. If Alnylam’s drug for PNH is approved, the company may take a different approach to pricing than Alexion, which sells Soliris at similar prices in different countries around the world.

“Global parity is an important aspiration for any new medicine,” Maraganore said. “But you also have local market situations that you need to be responsive to.”

Competition among expensive treatments can send prices plunging, as was shown last year when AbbVie Inc. came out with a hepatitis C drug that challenged Gilead Sciences Inc.’s dominant therapy, which originally listed at $1,000 per pill. Payers quickly negotiated exclusive deals with both companies for significant discounts.

“We have an opportunity for introducing competition in the space, which is always good for patients, it’s always good for payers,” Maraganore said.

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