Acoramidis, an Investigational Medicine Invented at Stanford, Demonstrated Efficacy and Safety in Eidos Therapeutics Phase III Clinical Trial

Transthyretin amyloidosis (ATTR), a medical condition characterized by abnormal misfolding and buildup of a protein called transthyretin (TTR), forms amyloid plaques that can get stuck in the heart and nervous system. Unfortunately, a lack of clinical research and approved therapies, as well as the difficulty in obtaining an accurate diagnosis, hindered progress in treating the disease for many years. But Eidos Therapeutics, a BridgeBio company, recently reported promising results from a phase III clinical trial treating ATTR patients. Eidos' promising ATTR therapeutic, acoramidis, mimics a protective, gain-of-function variant of the TTR protein that glues it together and impairs its ability to travel and affect the body, theoretically preventing further harmful plaque buildup. Acoramidis is based on a technology that Eidos licensed out of Dr. Isabella Graef’s lab at Stanford, who was also supported in these projects by the SPARK program in translational research. Researchers at Stanford also participated in the acoramidis’ phase II clinical trial. In the phase III trials, patients taking acoramidis lowered their risk of death by 25% and lowered their risk of cardiovascular related hospitalization events by 50% relative to placebo over 30 months. “The patients administered acoramidis are not just living longer, they’re living better”, says Ananth Sridhar (B.S. 2010), who worked on acoramidis at the company and now spearheads corporate development for the Eidos team within BridgeBio. It is “very rare to have the opportunity to do something with this kind of impact on patients'', he says. For the acoramidis team at BridgeBio, the next step towards expanding that impact is to file an application with the Food and Drug Administration to seek approval for the drug. They are also now looking into other ATTR patients they can help, with hopes to someday be able to prevent onset of the disease entirely.