Phase 1 safety and tolerability assessment of ANX776 in DARC (Detection of Apoptosing Retinal Cells) Technology

Abstract

Purpose : We have previously demonstrated experimentally the use of a new fluorescent –labelled annexin 5 (ANX776) to detect apoptosing cells in the retina (DARC - Detection of Apoptosing Retinal Cells), and have investigated ANX776 in different experimental models of retinal neurodegeneratiom. Here we report the safety and tolerability of intravenous ANX776 in a Phase 1 study.

Methods : 8 healthy and 8 glaucoma subjects were treated in four ascending-dose cohorts (n=4 per cohort) in which participants were randomly assigned to receive a single intravenous injection of ANX776 (0.1, 0.2, 0.4 or 0.5 mg).

Results : ANX776 was found to demonstrate favourable safety, tolerability, and pharmacokinetic profiles at all doses tested. No serious adverse events (defined as Grade 3 or above by the CTCAE - common terminology criteria for adverse events), discontinuations as a result of adverse events, or dose-limiting toxicities were reported. Pharmacokinetics confirmed that ANX776 had a short half-life inversely proportional to the dose administered, ranging from 10-36 minutes.

Conclusions : This study demonstrates that intravenous ANX776 is safe to use in humans. These findings support continued development of ANX776, including further characterization of its efficacy, safety, tolerability and pharmacodynamic effects in patients with neurodegenerative conditions

This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.