Company: Alnylam ($ALNY)
Partner: The Medicines Co. ($MDCO)
Technology: RNAi delivery platform
Amount: $205 million
The scoop: Before Sanofi ($SNY) jumped on board with Alnylam to the tune of $700 million early in 2014, The Medicines Co. ($MDCO) took a substantial interest in the RNAi delivery specialist. For $25 million up front and another $180 million in milestones, The Medicines Co. licensed Alnylam's PCSK9 candidate for high cholesterol using the RNAi tech that, when delivered correctly, can knock down a gene that spurs cholesterol synthesis.
Though almost a decade since the technology earned a Nobel Prize in 2006, the RNAi arena has been plagued by delivery issues. The fragile genetic material used to knock down genes is easily denatured in the body before it can reach its target but must also be available to act on the genes once it gets into the cell--a true delivery pickle.
But Alnylam's GalNAc delivery process has gotten its drugs through several important trials and closer to approval. Unlike many delivery platforms for RNAi, Alnylam's doesn't depend on nanoparticles to get into a cell but rather uses a sugar molecule conjugate with subcutaneous administration.
Alnylam handed over development of its early-phase PCSK9 candidate early in 2013 in a deal that seems to have paved the way for a new era of RNAi drugs, which endured a lull in the last few years as biotechs had begun to lose hope in their safe and effective delivery.
For a cholesterol fighter, though, $205 million is considered a relatively small payout. The New York Times' Andrew Pollack noted at the time that this could have been a result of how far the tech was--clinically speaking--behind Sanofi and Regeneron's ($REGN) candidate, as well as Amgen's ($AMGN) in the same area. A year earlier, after efficacy studies for the PCSK9 drug showed solid results, analysts said that the drug could end up being worth billions of dollars a year.
Alnylam posts positive PhI for RNAi cholesterol drug
Alnylam licenses away hot PCSK9 prospect in 'small' $205M deal
Alnylam scores efficacy data with PCSK9 drug