RNAi pioneer Alnylam announced that Genzyme will be paying it $22.5 M in upfront fees to license its phase I drug ALN-TTR02 in Asia. The drug performed very well in studies completed in July 2012 for the treatment of transthyretin-mediated amyloidosis. The disease effects primarily adults in the 30-40 yrs range and can damage their nerves and/or heart.
Genzyme agreed to the upfront and a royalty that could run to the mid-teens for the TTR program. It is an orphan disease that will fit well with the Genzyme portfolio. It is somewhat unusual for a company to pay this much upfront for a phase I program, but the results we very good in a phase 1 trial (knocking down the disease causing TTR protein by 94% when 50% is considered therapeutically relevant) and it is an orphan disease. A defined patient population in a growing part of the world, a very good clinical result and a prospect for quicker/cheaper clinical trials and commercialization means you cut the deal.
Posted by Bruce Lehr October 22nd 2012.