Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The study is aimed at evaluating the safety and tolerability of ALN-TTR02 in healthy volunteers. In addition, the study will evaluate the clinical activity of ALN-TTR02 based on measurements of serum levels of TTR, the disease-causing protein in patients with ATTR. ALN-TTR02 utilizes the company's proprietary second-generation lipid nanoparticle (LNP) technology using the "MC3" lipid. Alnylam expects to present data from this study in the third quarter of 2012.
“Advancement of ALN-TTR02 into the clinic is an important milestone in our 'Alnylam 5x15' product strategy. Indeed, ALN-TTR02 is our lead program in this effort and we expect to initiate a Phase II trial later this year, followed by a pivotal trial starting in 2013”
"Advancement of ALN-TTR02 into the clinic is an important milestone in our 'Alnylam 5x15' product strategy. Indeed, ALN-TTR02 is our lead program in this effort and we expect to initiate a Phase II trial later this year, followed by a pivotal trial starting in 2013," said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. "ALN-TTR02 utilizes our proprietary second-generation LNP technology which has shown significant potency improvements in both pre-clinical and clinical studies. We very much look forward to our expected reporting of TTR knockdown data from the Phase I study in the third quarter of this year."
"RNAi therapeutics represent a novel and exciting approach for the treatment of ATTR, as this new drug modality has the potential to make a meaningful impact in the treatment of this devastating disease. I am very encouraged by the recent results from Alnylam's Phase I study with ALN-TTR01 which showed knockdown in serum levels of TTR, the pathogenic protein in this disease. These results are important because we believe TTR suppression has the potential of halting or even reversing disease pathology in ATTR patients," said Teresa Coelho, M.D., Director, Unidade Clinica de Paramiloidose. "I support the continued advancement of this innovative medicine, and look forward to results from this new study, as well as additional clinical studies."
The Phase I trial of ALN-TTR02 is being conducted in the U.K. as a randomized, single-blind, placebo-controlled, single-ascending dose study, enrolling approximately 32 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of a single dose of ALN-TTR02, with subjects being enrolled into five sequential cohorts of increasing doses ranging from 0.01 to 0.50 mg/kg. Secondary objectives include serial measurement of circulating TTR serum levels through at least day 56 following a single dose. Additional secondary objectives include plasma and urine pharmacokinetics of ALN-TTR02.
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