Eidos Therapeutics to Host Conference Call and Webcast to Discuss ATTR-CM Phase 3 Trial (ATTRibute-CM) Design
Conference call and webcast
To participate in the conference call, dial +1-844-293-0174 (U.S. toll free) or 1-916-582-3546 (international), conference ID 7365928. The webcast will be available live and for replay on the company’s website at ir.eidostx.com.
About transthyretin amyloidosis (ATTR)
ATTR represents a significant unmet need of a comparatively large patient population in the context of rare genetic diseases with an inadequate current standard of care. There are three distinct diseases that comprise the ATTR family: wild-type ATTR cardiomyopathy (ATTRwt-CM), mutant ATTR cardiomyopathy (ATTRm-CM), and ATTR polyneuropathy (ATTR-PN). The worldwide prevalence of each disease is approximately 400,000 patients, 40,000 patients and 10,000 patients, respectively.
All three forms of ATTR are progressive and fatal. For patients with ATTRwt-CM and ATTRm-CM, symptoms usually manifest later in life (age 50+), with median survival of three to five years from diagnosis. ATTR-PN either presents in a patient's early 30s or later (age 50+), and results in a median life expectancy of five to ten years from diagnosis. Progression of all forms of ATTR causes significant morbidity, impacts productivity and quality of life, and creates a significant economic burden due to the costs associated with progressively greater patient needs for supportive care.
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 amyloidosis, or ATTR. In a Phase 2 clinical trial in subjects with symptomatic ATTR-CM, AG10 was well tolerated and increased serum TTR concentrations, a biomarker associated with survival in an observational study of ATTRwt-CM patients, in a dose-dependent manner. AG10 is currently being studied in an open-label extension of a Phase 2 clinical trial in patients with ATTR cardiomyopathy.
AG10 was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. To our knowledge, AG10 is the only TTR stabilizer in development that has been observed to mimic the structure of this rescue mutation.
Source: Eidos Therapeutics, Inc.