According to the company, the primary endpoint for the NEXPEDE-1 study of lufepirsen ophthalmic gel will be complete corneal healing as determined by corneal fluorescein staining.
Amber Ophthalmics Inc. has enrolled the first patient in the AMB-01-006 (NEXPEDE-1) study. The NEXPEDE-1 study is a randomly assigned, double-masked, vehicle-controlled Phase 2/3 clinical trial (NCT05966493) designed to evaluate two concentrations of lufepirsen ophthalmic gel (Nexagon) for the treatment of persistent corneal epithelial defects (PCED).
According to a company news release, the study evaluates the safety and efficacy of lufepirsen ophthalmic gel in a dosing regimen that utilizes as few as five topical in-office administrations for subjects with confirmed non-infectious PCED. Subjects who do not re-epithelialize within the first 4 weeks of treatment will continue to receive weekly administrations until subjects either achieve re-epithelialization or complete 8 weeks of therapy.
Amber Ophthalmics noted in its news release the primary endpoint for the NEXPEDE-1 study will be complete corneal healing as determined by corneal fluorescein staining.
"We are excited to have enrolled the first subject into the NEXPEDE-1 study and are fortunate to have a strong contingency of clinical sites dedicated to the treatment of PCED," Shawn A. Scranton, PharmD, president and CSO of Amber, said in the news release. "We look to expeditiously complete this study, as it will further guide the development of this first-in-class PCED therapy that we believe can address a variety of the underlying PCED etiologies."
According to the company, lufepirsen ophthalmic gel is an unmodified antisense oligonucleotide that inhibits connexin 43 protein translation, a cell membrane hemichannel forming protein associated with ocular inflammation and progression of PCED disease pathology. As a result of the gel’s mechanism of action, the medication may be administered topically in the office by the physician at a frequency far less than the current treatment paradigm consisting of multiple daily patient-administered drops.
The company also noted in its news release PCED is a rare and progressive disease impacting about 100,000 patients in the United States each year that arises from the failure of corneal re-epithelialization within 10 to 14 days of a corneal insult, even with the use of standard supportive treatment.
Moreover, the company noted in its release that despite treatment, and regardless of underlying etiology if progression is not halted, PCED can result in corneal scarring, vascularization and/or severe vision loss. There are currently no FDA-approved therapies for the treatment of PCED, with many patients progressing to corneal transplant.