Date: 2016-06-09
Type of information: Initiation of the trial
phase: 1
Announcement: initiation of the trial
Company: Alnylam Pharmaceuticals (USA - MA)
Product: ALN-TTRsc02
Action
mechanism: RNAi. ALN-TTRsc02 is a subcutaneous RNAi therapeutic targeting the transthyretin (TTR) gene. ALN-TTRsc02 utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugate delivery platform, which enables high potency and durability with a very wide therapeutic index.
Disease: transthyretin-mediated amyloidosis (ATTR amyloidosis)
Therapeutic area: Rare diseases - Genetic diseases
Country: UK
Trial
details: The Phase 1 trial of ALN-TTRsc02 is a randomized, placebo-controlled, single ascending-dose study designed to enroll up to a total of 100 normal healthy volunteers (NHVs). The primary objective of the study is to evaluate safety and tolerability of a single subcutaneous dose of ALN-TTRsc02. Secondary objectives include evaluation of pharmacokinetics and clinical activity for ALN-TTRsc02, as measured by knockdown of serum TTR levels in NHVs, and identification of the appropriate dose and regimen for a pivotal study. (NCT02797847)
Latest
news: * On June 9, 2016, Alnylam Pharmaceuticals announced that it has initiated a Phase 1 clinical trial with ALN-TTRsc02, a subcutaneously administered investigational RNAi therapeutic for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis). The Phase 1 trial will be conducted in normal healthy volunteers. Initiation of this trial is based on encouraging pre-clinical results, including data presented last year at the Oligonucleotide Therapeutics Society (OTS) meeting held October 11 - 14, 2015. The Company has guided that it expects to report initial clinical data from this study in late 2016, and if positive, plans to initiate a Phase 3 study in 2017.
In pre-clinical studies, including those in non-human primates (NHPs), ALN-TTRsc02 achieved potent and highly durable knockdown of serum TTR of up to 99% with multi-month durability achieved after just a single dose at 1 mg/kg, supportive of a potentially once quarterly dose regimen. In 13-week toxicology studies, ALN-TTRsc02, when given monthly for four doses, was generally well-tolerated with no significant adverse events at doses as high as 120 mg/kg in rats or 300 mg/kg in NHPs. There were no effects on platelet counts observed in either study.
