Gene-silencing expert Alnylam ($ALNY) is $7 million richer thanks to some positive mid-stage results for its Genzyme-licensed RNA-interference candidate, and the Cambridge, MA, company is looking to be the first to market with the difficult-to-deliver class of treatments.
Alnylam's ALN-TTR02, now dubbed patisiran, treats transthyretin-mediated amyloidosis (ATTR), a rare genetic defect that can lead to a deadly protein buildup, damaging organs and tissues. The latest milestone stems from the company's partnership with Sanofi's ($SNY) Genzyme, under which the pharma giant will help fund the drug's development in exchange for Asian marketing rights.
Safe and effective delivery has long bedeviled promising RNAi programs, but, in Phase II studies, patisiran has demonstrated a well-tolerated effect on the disease-causing protein TTR, charting knockdown rates of up to 96%. Those data were enough to convince the FDA to put the drug in its fast track program, and the company has kicked off an open-label Phase II extension study and Phase III trial of the novel treatment.
"We believe patisiran holds considerable promise to become a breakthrough therapy for the treatment of ATTR, a progressive and debilitating orphan disease," Alnylam CEO John Maraganore said in a statement. And Genzyme CEO David Meeker said his company believes patisiran is on track to keep racking up positive results on its way to market.
Genzyme paid $22.5 million up front to get in on Alnylam's ATTR program and signed on for undisclosed milestones, also picking up rights to the early-stage ALN-TTRsc, a subcutaneous RNAi treatment for ATTR patients with familial amyloidotic cardiomyopathy.
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