Agios starts multiple ascending dose Phase I trial of AG-348 to treat PK deficiency

The trial is designed to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of increasing doses of AG-348 for 14 days.

AG-348 is a first-in-class, orally available activator of pyruvate kinase-R (PKR) indicated to treat PK.

The single-center, randomized, double-blind, placebo-controlled Phase I trial will evaluate the safety and tolerability of AG-348 through multiple ascending doses in healthy adult men and women.

Initiation of the trial was based on data from the ongoing single ascending dose (SAD) Phase I trial, in which AG-348 has been determined to be well tolerated to date.

The SAD study began in April 2014 and has completed dosing of more than half of the planned groups and data from both studies are expected to be announced at a medical conference in 2015.

Agios chief executive officer David Schenkein said AG-348 represents a completely new approach to treating patients with PK deficiency, a serious form of inherited hemolytic anemia with no approved therapy that targets the metabolic defect responsible for the underlying disease.

"We are pleased to be moving forward with the MAD study that will allow us to further characterize the product candidate and establish the dose ranges we will study in patients with PK deficiency," Schenkein said.

The preclinical studies have showed that AG-348 activates a broad spectrum of PKR mutant proteins as well as corrects the metabolic defects found in patient-derived blood samples.

Currently, AG-348 is the only drug candidate being assessed for the potential of correcting metabolic defects found in patients with PK deficiency.