Alexion buys RNAi therapies in Dicerna deal

ludwig hantson
Alexion CEO Lud­wig Hantson. (Alexion)

Alexion has teamed up with Dicerna to branch out into RNAi therapies for complement-mediated diseases. The deal gives Alexion the rights to two preclinical assets, plus an option on two more, in return for $22 million upfront.

Boston-based Alexion started out focused on diseases mediated by the complement component of the immune system and, under the leadership ex-Baxalta CEO Lud­wig Hantson, is looking to use that traditional strength to secure its post-Soliris future. The Dicerna agreement furthers that effort by giving Alexion new ways to manipulate familiar biology. 

Working with Dicerna, Alexion plans to advance RNAi therapies that block production of complement pathway factors. In doing so, Alexion thinks it can stop the uncontrolled complement activation that underpins many diseases, including its key indication: paroxysmal nocturnal hemoglobinuria.

Alexion is a long way from showing it can make the idea work in practice, though. The agreement with Dicerna gives Alexion the chance to collaborate on the discovery and development of two RNAi molecules that hit complement pathway targets. Dicerna will lead the preclinical programs, before handing over to Alexion once the drugs get to human testing.

The early-stage nature of the work is reflected in the financials. Alexion is paying $22 million to enter into the deal, committing to milestones of up to $105 million per target and investing $15 million in Dicerna. In return, Alexion gets two discovery-stage programs and an option on two more assets.

Striking the deal continues the pipeline rebuild that has taken place since Alexion jettisoned drugs and alliances last year. Hantson tossed out pacts with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma—and put two internal programs on hold—when he took over last year. Since then, the CEO has stamped his mark on Alexion’s pipeline with a series of deals.

Alexion spent $40 million on a license to Halozyme’s drug delivery technology, paid $855 million to buy Wilson Therapeutics and struck a small deal to gain an option on Complement Pharma. Hantson and his team then returned to the deal table to bid $400 million upfront to buy Syntimmune.

Collectively, the agreements strengthen Alexion’s pipeline from end to end, starting with the discovery-stage Dicerna assets and running right up to the phase 3 drug in development at Wilson. The question is whether the deals will be more successful than Alexion’s earlier efforts to lessen its reliance on Soliris, which hit a nadir with the $8.4 billion takeover of Synageva.