Orphan Drug Designation Granted to Transthyretin Amyloidosis Treatment, AG10

The US Food and Drug Administration (FDA) has granted an orphan drug designation to Eidos Therapeutics, Inc.’s AG10, an investigational, orally-administered small molecule engineered to potently stabilize transthyretin (TTR) amyloidosis (ATTR).

“Orphan drug designation recognizes AG10’s potential to become an effective treatment for ATTR,” said Eidos’ chief medical officer, Jonathan Fox, to Rare Disease Report^®. “We’ve seen that increasing levels of transthyretin stabilization lead to improved clinical outcome in this disease, so believe that the near-complete stabilization we observed in our phase 1 study will translate to meaningful long-term benefit to ATTR patients.”

Data from its phase 1 clinical trial, which were presented this past September at the 22^nd Annual Scientific Meeting of the Heart Failure Society of America, indicated that AG10 was well-tolerated at doses resulting in target therapeutic blood levels.

Patients administered AG10 at its highest dose achieved 100% transthyretin (TTR) stabilization at peak concentration and over 95% TTR stabilization on average at steady state.

The first patient was also dosed in its phase 2 clinical trial this past May.

Approximately 45 symptomatic ATTR cardiomyopathy patients are anticipated to enroll in the randomized, double-blind, placebo-controlled trial. A minimum of 30% of patients with mutant ATTR cardiomyopathy will be included in the trial while the remainder will have wild type ATTR cardiomyopathy. Patients will be randomized in a 1:1:1 fashion to receive either placebo or 1 of 2 different doses of AG10 on a background of stable heart failure therapy.

The primary endpoint of the study is safety and tolerability of AG10; pharmacokinetics of the drug administered twice-daily will also be characterized.

Treatment will continue for 4 weeks if all doses are well-tolerated by participants.

Trial results are anticipated by the end of 2018.