(By Balaseshan) Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) said the results of phase 1 clinical trial of ALN-TTR02 showed robust knockdown of serum transthyretin (TTR) protein levels in TTR-mediated amyloidosis (ATTR) patients.
The data were presented July 16 in a seminar by Alnylam scientists at Boston University School of Medicine. The potential clinical activity of ALN-TTR02 would be evaluated based on measurement of serum levels of TTR, the disease-causing protein in patients with ATTR.
Alnylam said suppression of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, durable, and specific after just a single dose.
ALN-TTR02 was found to be generally safe and well tolerated in this Phase I study, consistent with Alnylam's broader clinical experience with LNP-formulated siRNA which now includes 102 patients or subjects, 334 total doses administered, and a length of treatment exceeding two years. There were no serious adverse events or discontinuations in the study related to ALN-TTR02.
Alnylam believes that these robust and durable knockdown data support a once-a-month or possibly once-every-other month dosing regimen, and plans to examine this further in the ongoing Phase 2 study.
The company recently reported that it has initiated a Phase 2 study of ALN-TTR02 in patients with ATTR and has guided that its goal is to start a pivotal trial in 2013.
Alnylam is advancing ALN-TTR02 in a Phase 2 study which is designed as an open-label, multi-center, multi-dose, dose-escalation trial expected to enroll approximately 20 ATTR patients. Patients would be enrolled into cohorts of increasing doses and will receive drug once every four weeks for two cycles.
The primary objectives of the phase 2 study are to evaluate the safety and tolerability of multiple doses of ALN-TTR02 and to measure clinical activity based on serial measurement of circulating serum TTR levels. Assuming positive results from the Phase 2 study, Alnylam expects to start a pivotal trial for ALN-TTR02 in 2013.
Alnylam also intends to advance ALN-TTRsc, which utilizes a GalNAc-conjugate delivery approach and subcutaneous dose administration. Alnylam's goal is to advance ALN-TTRsc to an investigational new drug (IND) filing in the second half of 2012 with data in the first half of 2013.
ALNY closed Friday's regular session up 0.72% at $12.52. The stock has been trading between $5.88 and $13.75 for the past 52 weeks.