Eidos Therapeutics announces positive Phase 2 data for AG10
Eidos Therapeutics announced positive results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin amyloidosis cardiomyopathy. The data were presented in a late-breaking Featured Science oral presentation at the American Heart Association Scientific Sessions. AG10 was well tolerated, demonstrated greater than 90% TTR average stabilization at day 28, and increased serum TTR concentrations, a prognostic indicator of survival in ATTR-CM, in a dose-dependent manner. Subject to discussions with regulatory agencies, these data support the advancement of AG10 into Phase 3 pivotal trials planned to be initiated in the first half of 2019. The study met its primary objective of establishing that AG10 was well tolerated with no safety signals of potential clinical concern related to the administration of AG10 in symptomatic ATTR-CM patients. One serious adverse event of dyspnea deemed unrelated to study drug was observed in one actively-treated subject and SAEs of atrial fibrillation, congestive heart failure, and cellulitis were observed in two placebo-treated subjects. The overall rate of adverse events was 69% in subjects administered 800 mg bid AG10, 63% in subjects administered 400 mg bid AG10, and 88% in subjects administered placebo. As compared to placebo, subjects treated with AG10 demonstrated a statistically significant increase in serum TTR concentrations, a prognostic indicator of survival in ATTR-CM patients, in a dose-dependent manner. Subjects administered 800 mg AG10 twice daily, 400 mg AG10 twice daily, and placebo exhibited mean changes in TTR concentration from baseline of +50%, +36% and -7%, respectively, at day 28. All subjects administered AG10 had serum TTR concentrations within the normal range at day 28, whereas 31% of subjects administered placebo had serum TTR concentrations below the normal range on day 28. AG10 administration resulted in near-complete stabilization of TTR at day 28, across the dosing interval in all actively treated subjects as measured by established ex vivo assays.