Alnylam Pharmaceuticals ($ALNY) says it generated promising Phase I trial results for an RNAi compound designed to treat a form of amyloidosis using lipid nanoparticle drug-delivery technology developed by Tekmira Pharmaceuticals.
The company's drug, ALN-TTR01, is designed to treat TTR-meditated amyloidosis by targeting the transthyretin (TTR) gene and silencing it before its mutations are expressed in the liver, and TTR proteins build up in various organs, according to Alnylam. ALN-TTR01 performed well in the trial, generating "statistically significant reductions" in TTR protein levels in patients with TTR-mediated amyloidosis.
Alnylam presented data involving 31 patients, including 8 who received placebo and 23 who received the systemically infused drug treatment in a randomized, placebo-controlled, single-dose escalation study. The drug was well tolerated with no serious adverse events, the Cambridge, MA-based company said. Alnylam presented the data at the International Symposium on Familial Amyloidotic Polyneuropathy, held Nov. 20-22 in Kumamoto, Japan.
RNAi drugs are meant to turn off bad genes that cause disease, but they often need drug delivery technology to work. That's where Tekmira comes in. The Canadian company's nanoparticles are thought to be effective in carrying RNAi drugs through the bloodstream and into cells, and Tekmira has collaborated with Alnylam since at least 2007.
Earlier this year, Tekmira filed suit against Alnylam, alleging patent and license agreement violations over the use of its siRNA delivery tech. Both companies acknowledged the other when separately announcing the amyloidosis news.
- see the Alnylam release
- here's the Tekmira release