Perfuse Therapeutics, Inc. announced positive 24-week results from its completed phase 1/2a trial of PER-001, a first in class endothelin antagonist intravitreal implant for the treatment of glaucoma. The PER-001 is a bioerodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide sustained release of PER-001, allowing for a dosing frequency of every 6 months.

According to a company press release, 33 participants were enrolled in the trial and were administered a single intravitreal PER-001 administration or sham injection while continuing their existing pre-study IOP-lowering treatments. Twenty-four week results showed increased ocular blood flow and improved both visual function and anatomic structure, each of which are registrable outcomes, as measured by visual field sensitivity and optical coherence tomography retinal nerve fiber layer thickness.

Additionally, the company stated PER-001 was safe and well tolerated over the study period. There were 2 drug-related adverse events of vitreous floaters, both of which were mild, intermittent, and self-resolved. No other drug-related adverse events or serious adverse events were reported, and there were no reports of endophthalmitis, intraocular inflammation, uveitis, or retinal vasculitis. Finally, no changes in IOP therapy occurred, and best-corrected visual acuity was stable during the trial.

"For decades, glaucoma treatments have focused on lowering intraocular pressure (IOP). Yet, many patients still experience vision loss and progress to blindness," said Steven Mansberger, MD, MPH, Chief of Ophthalmology and Director of Glaucoma Services at Legacy Devers Eye Institute, and an investigator in the study. He shared the trial results in an oral presentation at the 2025 Association for Research in Vision and Ophthalmology Meeting. 

"What is particularly remarkable is that treatment with PER-001 led to improvements in ocular blood flow, optic nerve structure, and visual function. These findings suggest a potentially transformative approach for the treatment of glaucoma by targeting the endothelin pathway, a well-established mediator of vascular dysfunction in the disease. Notably, this represents the first time endothelin antagonism has been studied in the human eye, marking the potential start of an exciting new era of treatment for patients with glaucoma."

Perfuse Therapeutics plans to initiate a pivotal-enabling phase 2b trial in the second half of 2025.