Could better blood flow be the key to treating glaucoma and diabetic retinopathy? New findings suggest it just might.
Sometimes in ophthalmology, the stars align…and sometimes it’s the blood flow—especially in retinal ischemia.
Perfuse Therapeutics (California, USA) has unveiled encouraging Phase IIa trial results for PER-001, a novel endothelin antagonist delivered via a slow-release intravitreal implant. The therapy showed significant improvements in both glaucoma and diabetic retinopathy (DR) patients, with up to 37.5% of glaucoma participants gaining more than seven decibels (dB) in visual field performance, and DR patients showing contrast sensitivity improvements of nearly one dB.
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Perfuse Therapeutics announces the Phase IIa results on its LinkedIn page.
PER-001 is designed to target a key underlying pathology shared by both diseases: retinal ischemia. By improving blood flow to oxygen-deprived retinal tissue, the therapy aims to restore perfusion, reduce cellular damage and potentially reverse vision loss.
The twin six-month, randomized, controlled studies evaluated PER-001’s efficacy as a potential first disease-modifying treatment for these leading causes of blindness. Results suggest that improving retinal perfusion may help halt, or even reverse, progressive visual decline.
“Our goal is to pioneer the first disease-modifying treatment for ocular diseases that share a common underlying pathology of ischemia—starting with glaucoma and diabetic retinopathy and rapidly expanding into dry age-related macular degeneration,” said Dr. Phil Lai, chief medical officer of Perfuse Therapeutics, in a news release.
“Our studies demonstrated that PER-001 has the potential to transform care for leading causes of blindness, delivering sustained benefits that not only prevent blindness but also improve vision.”
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Glaucoma trial results
In the glaucoma trial, PER-001 was used as an adjunct to standard intraocular pressure (IOP)-lowering therapies. Here’s what stood out:
- 37.5% of high-dose and 22.2% of low-dose patients experienced ≥7 dB improvement in visual field performance, compared to 0% in the control group
- No PER-001-treated patients had ≥7 dB loss, while 12.5% of control patients did
- Improvements in ocular blood flow and optic nerve structure were also observed
Dr. Joel Schuman, professor at Wills Eye Hospital and president of Collaborative Community on Ophthalmic Innovation, noted the significance of these results, saying, “[f]or the first time, Perfuse’s Phase IIa glaucoma trial demonstrates neuroenhancement, improvement in the function of existing optic nerve tissue, in humans with glaucoma. Patients treated with PER-001 in this trial remarkably showed improvement in visual field performance, while those in the control group continued to deteriorate.”
Diabetic retinopathy results
Results from the DR trial, presented at Clinical Trials at the Summit (CTS), revealed meaningful improvements in functional vision after just a single administration.
The diabetic retinopathy trial showed that a single administration of PER-001 led to:
- Contrast sensitivity improved by +0.9 dB (high dose) and +0.65 dB (low dose), compared to -2.1 dB decline in controls
- Low luminance, low contrast acuity improved by ~5 letters over control in both treatment arms
- Improvements in peripheral were seen as well as reductions in macular ischemia, leakage and microaneurysms
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“The results from the Phase IIa study of PER-001 represent a major advancement in the treatment of diabetic retinopathy. For the first time, we’re seeing a therapy for diabetic retinopathy that shows improvement in visual function,” said Dr. Arshad Khanani, clinical professor of Ophthalmology at University of Nevada, who presented the results at the CTS meeting.
With these Phase IIa findings in hand, Perfuse Therapeutics plans to launch pivotal trials for both glaucoma and DR in the second half of 2025. If future results hold steady, PER-001 could mark a shift in how clinicians treat ischemia-driven vision loss. Perhaps it’s not about chasing pressure or patching damage…it’s about getting the blood flowing.
Editor’s Note: This content is intended exclusively for healthcare professionals. It is not intended for the general public. Products or therapies discussed may not be registered or approved in all jurisdictions, including Singapore.