Alnylam Pharmaceuticals has completed enrolment in a Phase 1 trial and commenced a Phase 2 trial of ALN-TTR02, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).

The Phase 2 trial, an open-label, multicentre, multi-dose, dose-escalation trial, will evaluate the clinical activity, safety and tolerability of multiple once-monthly doses of ALN-TTR02 in ATTR patients.

Alnylam senior vice president and chief medical officer, Akshay Vaishnaw, said that with dosing completed in the Phase 1 study, the company is on track to report results early in the third quarter of 2012.

"ALN-TTR02 uses the company’s second-generation lipid nanoparticle (LNP) delivery technology."

"Our Phase 2 study is now underway and will evaluate a once-monthly multi-dose regimen in ATTR patients," Vaishnaw added.

"We look forward to continuing to share clinical data from our ALN-TTR02 programme, and, assuming positive results in the current Phase 2 study, we plan to advance to a pivotal trial in 2013."

The subjects will be enrolled into cohorts of increasing doses and will receive the drug once every four weeks for two cycles in the Phase 2 trial.

The clinical activity of ALN-TTR02 will be evaluated based on measurement of serum levels of TTR, the disease-causing protein in patients with ATTR.

ALN-TTR02 uses the company’s second-generation lipid nanoparticle (LNP) delivery technology, using the MC3 lipid.

Assuming positive results from the Phase 2 study, Alnylam expects to start a pivotal trial for ALN-TTR02 in 2013.

The company also plans to advance ALN-TTRsc, which uses a GalNAc-conjugate delivery approach and subcutaneous dose administration.