Eidos Therapeutics has dosed the first patient in a Phase II clinical trial of AG10 as a treatment for patients with transthyretin amyloidosis (ATTR) cardiomyopathy.

The randomised, placebo-controlled trial will enrol around 45 symptomatic ATTR cardiomyopathy patients.

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The trial will enrol at least 30% of its patients with mutant ATTR cardiomyopathy, while the remaining patients will have wild type ATTR cardiomyopathy.

During the trial, patients will be randomised at 1:1:1 ratio to receive placebo or one of two different doses of AG10 on a background of stable heart failure therapy.

If all doses are well-tolerated, the trial expects to treat the patients for 28 days.

"Our preclinical and Phase I data provide evidence that AG10 could be beneficial for both wild type and mutant ATTR cardiomyopathy patients."

The trial’s primary objective is to investigate the safety and tolerability of AG10. It will also define the pharmacokinetics of AG10 administered twice daily, as well as measure and confirm TTR stabilisation by validated ex vivo assays, including fluorescent probe exclusion and immunoblotting.

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Additionally, the trial intends to provide clinical proof of concept to support potential subsequent pivotal trials.

Topline results from the trial are expected to be reported by the end of this year.

Eidos Therapeutics president and chief medical officer Jonathan Fox said: “The positive results of our Phase I trial of AG10 in healthy volunteers encouraged our rapid advancement into a Phase II trial in patients with ATTR cardiomyopathy.

“Our preclinical and Phase I data provide evidence that AG10 could be beneficial for both wild type and mutant ATTR cardiomyopathy patients, and this Phase II trial aims to provide additional information in these specific populations.”

Eidos plans to launch a second Phase II trial of AG10 in patients with ATTR polyneuropathy later this year.

ATTR is a rare genetic disease and represents a significant unmet need with inadequate standard of care.