Alnylam Pharmaceuticals, one of the leaders in developing drugs through RNA interference, has achieved another big first for the emerging field. The Cambridge, MA-based drug developer (NASDAQ: [[ticker:ALNY]]) is announcing today that it has filed an application with the FDA to start the first clinical trial of an RNAi drug that can circulate throughout the body.
The drug candidate is built through technology Alnylam licensed from Vancouver, BC-based Tekmira (TSX: [[ticker:TKM]]). The treatment is designed to deliver two gene-silencing molecules developed by Alnylam via a lipid-nanoparticle capsule from Tekmira. That package (essentially a grease ball) is supposed to protect the drug from breaking down in the bloodstream, making it possible to distribute the gene-silencing molecule throughout the body, and particularly where it needs to be inside liver cancer cells.
This is an important step for the field of RNA interference, says Alnylam CEO John Maraganore. Such drugs have attracted intense interest among pharmaceutical and biotech companies for their potential as a new class of treatments that can shut down disease at its root genetic cause. But one of the biggest challenges is that these gene-silencing drug candidates are broken down and excreted by the kidneys within minutes if given in a straight injection. So researchers have spent years trying to work around this problem, through local delivery for eye diseases, or inhalable forms made for the lungs. By developing a protective lipid-capsule that’s stable, Alnylam hopes this new candidate will blaze a trail for delivering these drugs throughout the body, the same way most effective pills and biotech drugs are delivered.
“This opens the door to a brand new approach to delivering RNAi therapeutics,” Maraganore says. “Systemic delivery is the key enabling event to make this field much more like monoclonal antibodies or small-molecules, in terms of the breadth of diseases we can go after.”
The new candidate, called ALN-VSP, has been designed to treat primary liver tumors and other malignancies that have spread to that organ, Alnylam says. The drug is designed to shut down
