Amber Ophthalmics, Inc. announced positive results from the EXPEDE phase 2 trial of NEXAGON (lufepirsen ophthalmic gel) for the treatment of persistent corneal epithelial defect (PCED) secondary to chemical and/or thermal injury (CTI).
Tell me about the study.
The EXPEDE trial was a randomly assigned, double-masked, vehicle-controlled clinical trial of two topically administered concentrations of NEXAGON. The trial assessed the efficacy of NEXAGON on non-infected, PCED from CTI versus the vehicle alone. (via)
A total of 35 patients ranging from 7 to75 years in age were split into three treatment groups: high and low dose (n=12 each) and the vehicle group (n=11). Enrollees had an average baseline corneal defect area of 46.67 mm2 (4 to 176 mm2).
What did they find?
The primary endpoint of the study was corneal epithelial recovery, defined as “corneas exhibiting complete corneal re-epithelialization within the treatment period that is maintained for at least 28 continuous days.” (via.)
Both NEXAGON treatment groups achieved a 66.7% corneal epithelial recovery.
Any adverse reactions?
No adverse reactions were reported, and the dosage concentrations were both reported to be tolerated by patients with a similar safety profile to vehicle alone.
What does this mean for ECPs?
PCED is a rare ophthalmic disease that affects roughly 100,000 patients in the U.S. each year. There are currently no FDA-approved therapies for the treatment of PCED.
Shawn A. Scranton, PharmD, Amber president and CSO, noted that the company has recently received FDA guidance and plans to begin the next phase of trials shortly: “We anticipate the study will begin enrolling subjects with PCED of various etiologies as soon as Q1 2023.”