Perfuse Therapeutics announced positive results this week from a pair of phase 2 clinical trials evaluating its investigational drug PER-001 for glaucoma and diabetic retinopathy (DR). The therapy, delivered via a dissolvable intravitreal implant, was associated with improvements in both vision and retinal structure, showing the potential to reverse progressive damage caused by ischemia in the eye.
The trials, each 6 months in duration, tested a slow-release form of PER-001—an endothelin receptor antagonist—administered once every 6 months. A total of 60 patients participated: 33 with glaucoma and 27 with diabetic retinopathy. Results were presented at the 2025 Clinical Trials at the Summit (CTS) meeting in Las Vegas and support the drug’s advancement into phase 2b/3 trials planned for later this year.
In the glaucoma trial, PER-001 was evaluated as an adjunct to existing therapies that lower intraocular pressure (IOP). Although IOP reduction is the current standard of care, many patients with glaucoma continue to lose vision even with normal pressure levels. In this trial, 37.5% of patients receiving the high-dose implant experienced a clinically meaningful ≥7 dB improvement in visual field in predefined regions, compared to 0% in the control group. None of the treated patients lost ≥7 dB of vision, whereas 12.5% of controls did, which is consistent with known disease progression rates, the company said in a press release. The glaucoma data also showed improvement in ocular blood flow and optic nerve structure on optical coherence tomography (OCT) imaging.
PER-001 also showed promise in DR, where current therapies aim primarily to prevent further deterioration. In the phase 2a trial for DR, treated patients had measurable gains in contrast sensitivity and visual acuity under low luminance, as well as improvements in peripheral visual field. Imaging revealed structural changes consistent with reduced macular ischemia, leakage, and microaneurysm burden compared with worsening indicators in the control group.
“These functional gains in peripheral vision, contrast sensitivity, and visual acuity accompanied by structural improvements validate the PER-001 mechanism of action,” said Arshad M. Khanani, MD, who presented the DR data at CTS. “We may finally have a therapy that modifies the course of diabetic retinopathy.”
PER-001 is a first-in-class molecule that targets endothelin, a potent vasoconstrictor implicated in retinal ischemia. The implant uses a 25-gauge injector and is designed for biannual administration. Safety and tolerability were favorable in both trials, with no significant safety concerns reported over 24 weeks.
With the promising early stage results, Perfuse Therapeutics reports it plans to launch pivotal studies in both glaucoma and diabetic retinopathy in the second half of 2025. The company also intends to explore the therapy in other ischemia-driven conditions, including dry age-related macular degeneration. GP