Drug Development

Step 3: Sanofi commits up to $100M to opt-in on Alnylam’s PhIII RNAi hemophilia trial

John Maraganore, Alnylam

John Maraganore, Alnylam

Alnylam is stepping up to the starting line for a Phase III study of its hemophilia drug fitusiran with Sanofi on board as a partner. The pharma company lined up the opt-in rights under their earlier deal to buy a minority stake in the biotech for $700 million. And Sanofi is now committed to providing $75 million in development and regulatory milestones following a $25 million upfront due when Phase III starts in early 2017.

Cambridge, MA-based Alnylam $ALNY and its collaborator Sanofi $SFY will now partner up on development of the RNAi program with the prospect of a marketing alliance in the US, Canada and Western Europe, while Sanofi Genzyme in Boston will have rights to develop the drug for hemophilia and rare bleeding disorders in the rest of the world.

Execs from both sides heralded the move as another big step forward for an alliance announced with great fanfare in early 2014, which greatly expanded an existing relationship. For Sanofi, the Alnylam deal reflected their enthusiasm for a similar relationship they have with Regeneron, which has been their most productive pact.

Sanofi Genzyme, though, is leaving another program on the sideline at Alnylam. There will be no opt-in for ALN-AS1, another RNAi drug aimed at acute hepatic porphyrias. Sanofi bowed out after taking a look at the data from the first two stages of a Phase I study. A third stage is coming up for review as Alnylam plans to proceed on its own.

The opt-in on hemophilia comes as a boon to Alnylam, which had to scrap the number two drug in its pipeline — revusiran — after tracking a disproportionate number of deaths on the treatment arm of a late-stage program. On a brighter note, Alnylam partner The Medicines Company enjoyed a clinical success with their PCSK9 drug.

Alnylam CEO John Maraganore had this to say:

“This decision allows us to broaden the global reach and accelerate the commercial development of fitusiran, a potentially transformative approach to the treatment of hemophilia and rare bleeding disorders, with a well-established partner in Sanofi Genzyme. We look forward to continued collaboration with Sanofi Genzyme to advance fitusiran as Alnylam transitions toward the commercial stage with our late-stage pipeline.”


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