Eidos Therapeutics to present interim analysis of Phase 2 OLE study of AG10
Eidos Therapeutics announced that an interim analysis of its ongoing Phase 2 open-label extension study of AG10 in subjects with symptomatic ATTR cardiomyopathy will be presented in a late-breaking featured science oral presentation at this year's American Heart Association Scientific Sessions. Daniel Judge, M.D., professor in the division of cardiology at the Medical University of South Carolina, will discuss the data in a presentation entitled "Long-term Safety and Efficacy of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy: Interim Analysis of the Ongoing Phase 2 Open-Label Extension Study" at 5:30 PM ET on November 16, 2019 as part of the Early Phase Science oral session. In addition, Eidos will present several posters at AHA. The following summarizes Eidos' presentations at the conference: Long-term Safety and Efficacy of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy: Interim Analysis of the Ongoing Phase 2 Open-Label Extension Study. ATTRibute-CM: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center, Global Phase 3 Study of AG10 in Patients with Transthyretin Amyloid Cardiomyopathy. Differential Transthyretin Binding, Kinetic Stability and Additive Ex Vivo Stabilization by AG10 Compared to Tafamidis. Differential Ex Vivo Stabilization of Transthyretin by AG10 and Tafamidis in Samples from Patients with Moderate or Severely Destabilizing Mutations. AG10 is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin, or TTR, thereby halting at its outset the series of molecular events that give rise to TTR amyloidosis, or ATTR. In a Phase 2 clinical trial in patients with symptomatic ATTR-CM, AG10 was generally well tolerated, demonstrated greater than 90 percent average TTR stabilization at Day 28, and increased serum TTR concentrations, a prognostic indicator of survival in a retrospective study of ATTR-CM patients, in a dose-dependent manner. AG10 was designed to mimic a naturally-occurring variant of the TTR gene that is considered a rescue mutation because co-inheritance has been shown to prevent or ameliorate ATTR in individuals also inheriting a pathogenic, or disease-causing, mutation in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of this rescue mutation. The Phase 3 ATTRibute-CM study of AG10 in patients with ATTR-CM is underway. Part A of the study will assess the change from baseline in 6-minute walk distance at 12 months. Part B of the study will evaluate reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations at 30 months. In addition, Eidos plans to initiate a Phase 3 study of AG10 in ATTR polyneuropathy by the end of 2019.