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Alnylam inks broad R&D deal with Regeneron

  • Alnylam Pharmaceuticals on Monday announced a major drug development collaboration with Regeneron, taking its expertise in RNA interference to a new partner as it winds down research under an existing alliance with Sanofi.
  • For Regeneron, the deal with Alnylam allows the New York biotech to go in directions ill-suited to the antibody technology for which it’s known. “This collaboration enables us to reach targets inside the cell complementing our expertise in antibodies, which are ideal for extracellular targets and those on the cell surface,” said George Yancopoulos, Regeneron’s chief scientific officer, in an April 8 statement.
  • Regeneron will pay Alnylam $800 million upfront, evenly split between cash and an equity investment. The two companies aim to develop therapies for central nervous system and eye diseases over a five-year discovery period. Based on clinical progress, Alnylam could receive a further $200 million in milestone payments as well as $30 million per year for program advancement.

Settling in as a commercial-stage biotech, Alnylam is shaking up its research partnerships, significantly expanding its work with Regeneron well beyond an earlier deal to develop a treatment for non-alcoholic steatohepatitis (NASH).

News of the deal was well received by Wall Street analysts, who indicated Monday the deal makes strategic sense for both companies. For Alnylam, the financial infusion will extend its cash runway and provides further validation of an RNAi research platform that’s at the heart of the biotech’s drug development.

Regeneron, meanwhile, has a genetics center capable of generating numerous potential targets, not all of which can be addressed by the biotech’s antibody approach, noted SVB Leerink analyst Geoffrey Porges. Partnering with Alnylam enables the companies to explore targets within the cell using Alnylam’s RNAi technology.

While the bulk of the deal will focus on preclinical discovery and development, an early priority will be on two clinical-stage assets. The companies plan to test a combination of pozelimab, Regeneron’s C5 antibody in Phase 1 testing and cemdisiran, Alnylam’s RNAi anti-C5 therapy, now in Phase 2.

Details were more sparse for plans beyond the initial first steps in the collaboration. But Alnylam CEO John Maragnore, speaking on a Monday call with analysts, hinted at the potential of an antibody and RNAi combination.

“We’re not ready to disclose some of the thinking, but we do believe that the concept in some settings of knocking down the target’s production and then obviously sopping up what might be left in the circulation can yield some very creative and innovative approaches to new medicines,” Maraganore said.

“You should not be surprised to see other examples like that emerge from the power of RNAi combined with antibody development,” he added.

Another Alnylam executive likened the partnership to a “big sandbox” that will speed up its pipeline progress. The company has previously expected one or two Investigational New Drug application each year beginning in 2020. Now, they expect between two and four INDs per year starting in 2020.

Per the deal, the two biotechs will alternate on leading development for each program, with the lead company keeping global development and commercial responsibility, with the other having the option to get an equal share of potential future profits.

Monday also marked a close to the research and option phase of Alynlam’s deal with Sanofi, as the two agreed to exit a 2014 rare disease collaboration.

The alliance will continue unchanged, however, for work on Alnylam’s Onpattro (patisiran), vutrisiran and fitusiran. Alnylam will also advance an undisclosed, preclinical therapy through IND-enabling studies, after which Sanofi will take over development.

Regeneron’s and Alnylam’s shares opened relatively even Monday, before falling modestly in morning trading. By noon, Regeneron’s stock was down more than 2% and Alnylam was down 1%.