Under the terms of the RNAi agreement, Genzymewill make an upfront cash payment of $22.5 million to Alnylam. The agreementalso includes development milestone payments and tiered royalties expected toyield an effective rate in the mid-teens to mid-twenties on Genzyme's sales ofALN-TTR products in their territory. In addition, each party will beresponsible for the development and commercialization activities in theirrespective territories.
"Given our confidence in ALN-TTR's potential, weview such a royalty-weighted deal with an industry leader in the Asian marketas a highly valuable and attractive proposition from Alnylam," noted LaurenceReid, senior vice president and chief business officer at Alnylam, in aconference call with investors and media about that deal. "Overall, we are veryexcited to form this alliance with Genzyme, we believe that as a result of thisnew alliance, ALN-TTR will get to patients in Japan, and the other Asia-Pacificcountries sooner and our drug will reach that market much faster."
"OurALN-TTR program holds promise as a breakthrough therapy for the treatment ofATTR, a debilitating orphan disease," said Dr. John Maraganore, CEO of Alnylam,in the news release about the deal, which further explained that ATTR is a rare,hereditary disease that damages the nervous system and heart, resulting in alife expectancy of five to 15 years.
"As the lead program in our 'Alnylam 5x15' productstrategy, we also view this program as a key part of building Alnylam for thefuture," Maraganore added. "In this important collaboration, Genzyme willadvance our ALN-TTR program with their proven capabilities in the Japanese andbroader Asian market, while we maintain our plans to develop and commercializethis potential breakthrough medicine in the U.S., Europe, and rest of world. Inaddition, a key part of the value proposition in this alliance for Alnylam isthe potential for significant royalty payments on sales of products."
In a conference call to investors and media, healso expressed eagerness to work with Genzyme, citing that company as being "clearlythe industry innovator and leader in bringing orphan drugs to patients thatneed them."
ATTR is an endemic disease in Japan, with asignificant number of patients carrying the V30M TTR mutation that leads toonset of a severe form of ATTR known as familial amyloidotic polyneuropathy(FAP). Alnylam and Genzyme plan to maximize the value of ALN-TTR worldwide bydeveloping the RNAi program in FAP and other ATTR indications, such as familialamyloidotic cardiomyopathy and senile systemic amyloidosis.
Alnylam's ALN-TTR program currently includesALN-TTR02, which is in a Phase II clinical trial, and ALN-TTRsc, asubcutaneously administered RNAi therapeutic in late-stage preclinicaldevelopment.
Saying that his company is encouraged by Alnylam'sprogress with the ALN-TTR program, Dr. David Meeker, president and CEO ofGenzyme, noted, "The results to date demonstrate impressive clinical activityand support advancement of this promising therapeutic into pivotal studies andtoward the market." Further, he cast the alliance as an important part ofGenzyme's efforts to build its pipeline not just through internal research anddevelopment but also external collaborations.