Eidos Therapeutics is developing acoramidis, a genetically targeted therapy that stabilizes transthyretin (TTR) proteins to prevent their misfolding and accumulation, which leads to transthyretin amyloidosis (ATTR). This condition is a rare and life-threatening disease that can severely damage the heart and nervous system, with acoramidis aiming to improve patient outcomes and reduce mortality associated with ATTR.
Funding
Funding not disclosed
Founders
Product
Problem
Transthyretin amyloidosis (ATTR) is a rare, underdiagnosed, and life-threatening disease with limited treatment options. ATTR is characterized by the destabilization of transthyretin (TTR) and subsequent accumulation of misfolded TTR protein, which can severely damage the heart and/or nervous system. If left untreated, life expectancy from diagnosis is approximately four years.
Solution
Eidos Therapeutics, an affiliate of BridgeBio, is developing acoramidis, a genetically targeted therapy designed to stabilize TTR proteins and prevent their misfolding and accumulation in patients with ATTR. Acoramidis increases the level of TTR in the serum, which is associated with improved cardiovascular outcomes. By stabilizing the TTR protein, acoramidis aims to improve the morbidity and mortality of patients with ATTR cardiomyopathy (ATTR-CM).
Target Audience
The primary target audience is patients diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM).
Features
- Designed to stabilize the TTR protein, preventing misfolding and accumulation
- Genetically targeted therapy
- Demonstrated a 42% reduction on the composite endpoint of all-cause mortality and recurrent cardiovascular-related hospitalization events
- Increases serum TTR levels, which is associated with improved cardiovascular outcomes