Alnylam ($ALNY) chugged along this week in its efforts to advance its line of RNAi drugs to the market, notching positive results for two of its candidates, one in Phase II trials and the other preclinical.
The Cambridge, MA-based company known for its RNAi gene-silencing drugs in development, collected positive data for its Phase II treatment ALN-TTR02 for TTR-mediated amyloidosis (ATTR) and for its preclinical ALN-AT3 to treat hemophilia and other rare bleeding disorders.
In 25 patients dosed to date, ALN-TTR02 "knocked down" the offending protein, TTR, which causes ATTR, by 93%, according to the company. And ALN-AT3 reduced plasma AT levels by 90% in animal trials, which led to a normalization of hemophilia-causing thrombins.
RNA interference, a discovery that garnered the 2006 Nobel Prize for Physiology or Medicine, uses the natural process of gene silencing to shut down the ones that lead to certain diseases. The potential for the class of drugs has in the past been dampened by the difficulty with which the small strands of RNA--called small interfering RNA, or siRNA--are delivered.
"The historical challenge is getting siRNA into the cells' genetic machinery," Alnylam President and COO Barry Greene told FierceDrugDelivery. He said that ALN-TTR02 is expected to reach Phase III by the end of this year, and that the company hopes to begin clinical trials of ALN-AT3 by 2013.
Using a combination of intravenous and subcutaneous delivery, Alnylam expects to have "5 RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015," according to the company. And Greene says it is ahead of pace.
"We're exceeding multiple people's expectations of where we'd be right now," Greene said. "All these programs have fairly expeditious paths and potential commercial value."
- here's the ALN-TTR02 release
- and here's the ALN-AT3 release