AG10 is designed to stabilise TTR and in turn prevent the molecular events leading to amyloidosis. Credit: Ed Uthman.

Eidos Therapeutics has commenced a pivotal Phase III clinical trial of its investigational small molecule AG10 for the treatment of transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).

ATTR is caused by tetrameric transthyretin (TTR) destabilisation due to inherited mutations or aging. AG10 is designed to stabilise TTR and prevent the molecular events leading to amyloidosis (ATTR).

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The global Phase III trial is dubbed ATTRibute-CM and will compare 800mg AG10 with placebo in a total of 510 patients suffering from symptomatic ATTR-CM associated with either wild-type or mutant TTR.

ATTRibute-CM will include two potentially registrational primary endpoints.

“The trial is designed to evaluate preservation of function and quality of life on an accelerated timeframe.”

Part A will measure the benefit of a change from baseline in six-minute walk distance (6MWD) at 12 months, while Part B will evaluate the decrease in all-cause mortality and the frequency of cardiovascular-related hospitalisations at 30 months.

The trial will also involve secondary endpoints such as quality of life assessment, safety parameters, serum TTR levels, and TTR stabilisation.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Eidos Therapeutics president and chief medical officer Jonathan Fox said: “ATTRibute-CM aims to generate registrational evidence that AG10 provides meaningful benefit to ATTR-CM patients.

“The trial is designed to evaluate preservation of function and quality of life on an accelerated timeframe in addition to evaluating longer-term benefit on mortality and cardiovascular-related hospitalisations.

Positive data from the trial is expected to support the approval of AG10 in ATTR-CM treatment.

A previous Phase II trial involving symptomatic ATTR-CM patients demonstrated that AG10 was generally well-tolerated and significantly increased serum transthyretin in a dose-dependent manner.

Participants who received AG10 experienced normal serum transthyretin levels within 28 days of treatment.