PER-001 Shows Promise in Eye Disease Trials

Perfuse Therapeutics, Inc. has reported promising topline data from two Phase II clinical trials evaluating its investigational therapy, PER-001. This first-in-class endothelin antagonist is delivered via a dissolvable intravitreal implant designed for slow release over six months. The therapy demonstrated significant improvements in vision in patients with glaucoma and diabetic retinopathy. A Phase IIb/III clinical program is set to launch in the second half of 2025.

Advancing a Disease-Modifying Approach

Dr. Phil Lai, Chief Medical Officer of Perfuse Therapeutics, highlighted the company’s strategy to address ischemia-driven ocular diseases. He stated that the aim is to introduce the first disease-modifying treatment for conditions such as glaucoma and diabetic retinopathy, with plans to expand into dry age-related macular degeneration. According to Dr. Lai, PER-001 has shown potential not only to prevent blindness but also to improve visual function.

Glaucoma Trial Highlights

In the Phase 2a glaucoma trial, a single intravitreal injection of PER-001 provided six months of sustained release, added to standard-of-care IOP-lowering treatments. Results indicated that 22.2 percent of low-dose patients and 37.5 percent of high-dose patients experienced a seven-decibel or greater improvement in visual field sensitivity across a defined retinal region, compared to zero percent in the control group. These outcomes are eight to fourteen times better than the typical progression rate seen with existing treatments.

No patients in the PER-001 treatment groups experienced a loss of seven decibels or more, while 12.5 percent of the control group did. These results are consistent with the natural disease progression rate of approximately 13 percent. Early data from the trial, presented at ARVO 2025, also showed improvements in ocular blood flow, retinal nerve fiber layer thickness, and visual field trends.

Diabetic Retinopathy Trial Outcomes

In the diabetic retinopathy Phase 2a trial, a single dose of PER-001 led to measurable improvements in both functional and structural outcomes over six months. High-dose participants experienced a mean contrast sensitivity gain of 0.9 decibels, while low-dose participants gained 0.65 decibels. In contrast, the control group saw a mean reduction of 2.1 decibels. This difference corresponds to a visual improvement equivalent to 15 ETDRS letters.

Visual acuity under low-luminance, low-contrast conditions improved by an average of 5.5 letters in the low-dose group and 5.1 letters in the high-dose group compared to baseline. Patients with impaired peripheral vision demonstrated an improvement rate of 1.8 decibels per year, versus only 0.01 decibels in the control group. Additionally, the treatment improved structural parameters such as macular ischemia index, leakage, and microaneurysms, while diabetic retinopathy severity scores remained stable across all groups.

Trial Design and Safety Profile

A total of 60 patients were enrolled across both studies, with 33 in the glaucoma trial and 27 in the diabetic retinopathy study. The 24-week data showed that PER-001 was well tolerated and demonstrated a favorable safety profile. Findings were presented at the Clinical Trials at the Summit (CTS) meeting on June 21. Perfuse Therapeutics intends to initiate pivotal Phase IIb/III trials for both indications in the second half of 2025.