Perfuse Therapeutics Announces Positive Results from Phase 2 Clinical Trials in Glaucoma and Diabetic Retinopathy Patients

• PER-001 intravitreal implant shows potential to be the first disease-modifying treatment for both glaucoma and diabetic retinopathy
   
• Progressive vision loss reversed in glaucoma patients as optic nerve blood flow and structure improved
   
• Diabetic retinopathy patients experience improvements in vision, retinal ischemia and structure
   
• PER-001 demonstrated a favorable safety and tolerability profile in diabetic retinopathy and glaucoma

SAN FRANCISCO, June 24, 2025 /PRNewswire/ -- Perfuse Therapeutics, Inc., a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases, today announced positive results from two Phase 2 clinical trials of PER-001, a novel therapeutic designed to address two leading causes of global blindness: glaucoma and diabetic retinopathy. Each of the six month, randomized, controlled trials evaluated PER-001, a first-in-class endothelin antagonist delivered in a slow-release, dissolvable implant that is injected into the back of the eye (intravitreal) every six months. Significant improvement in vision was demonstrated compared to control in each trial. 

With a therapy that targets the underlying cause of both diseases, early results of PER-001 show promise for transforming the trajectory of human blindness and offer evidence of PER-001's ability to reverse the course of progressive vision loss. The goal of this approach is to improve retinal blood flow and prevent the death of retinal cells that occur in ocular diseases. Ph2b/3 trials are planned to begin in the second half of 2025. 

"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 Phil Lai, MD, chief medical officer of Perfuse Therapeutics. "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."

New Hope for Glaucoma Patients: Restoring Vision by Targeting the Root Cause 

Glaucoma is the leading cause of global blindness worldwide. An estimated 4.2 million people in the U.S. have glaucoma, but only about half are aware of their condition. Currently there are no glaucoma therapies approved by the U.S. Food and Drug Administration; treatments primarily consist of pharmacological, laser or surgical solutions to reduce intraocular pressure (IOP), which is normal in around half of glaucoma patients. Unfortunately, even with reduction of IOP, many patients progress to blindness.

Key findings from the Phase 2a glaucoma trial showed that after single intravitreal administration of PER-001 released for six months, added to existing standard-of-care IOP-reducing therapies:

• 22.2% of low-dose and 37.5% of high-dose patients experienced ≥7 decibel (dB) improvement in a pre-defined retina region of minimal 5 test points compared to 0% in control in six months. This improvement is 8x to 14x better than natural history of disease (2.7%) with currently available treatments.
• No PER-001 treated patient experienced ≥7 dB loss compared to 12.5% in control in six months. Control rate of ≥7 dB loss observed in this study is consistent with the natural history of disease (13%) with currently available treatments.
• Early results of the PER-001 glaucoma clinical trial were reported at the Association of Research in Vision and Ophthalmology (ARVO) annual meeting in May 2025. This initial dataset demonstrated improvement in ocular blood flow, optic nerve structure (OCT RNFL) and function (visual field rate of change) outcomes with PER-001 in glaucoma patients.

"Many people continue to have worsening glaucoma despite what is considered to be safe IOP. A holy grail in the treatment of glaucoma is the ability to reduce optic nerve damage by means other than lowering IOP," said Joel Schuman, MD, professor and K.L. Roper Endowed Chair, Department of Ophthalmology at Wills Eye Hospital and president of Collaborative Community on Ophthalmic Innovation (CCOI). "For the first time, Perfuse's Phase 2a 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. The 7 dB scale of improvement in the Garway-Heath visual field sectors at a minimum of 5 testable points, is generally considered clinically significant and has been employed in the past by the U.S. Food and Drug Administration (FDA) as one criterion in considering approval of a new agent for glaucoma. In addition to enhancement of visual function, patients receiving PER-001 demonstrated improvements in ocular blood flow and optic nerve structure. Importantly, there is currently no FDA-approved drug for the treatment of glaucoma. The current study results are most encouraging, demonstrating the potential for a radically new, transformational treatment approach for this disease."

Improving Vision and Ischemia in Diabetic Retinopathy

An estimated 9.6 million people in the U.S. are living with diabetic retinopathy, including more than 1.8 million with vision-threatening complications. Globally, diabetic retinopathy remains the leading cause of blindness among working age adults.

Current treatment typically revolves around stabilization or improvement of a structural outcome, the Diabetic Retinopathy Severity Scale (DRSS), with no long-term benefit in vision. In many cases, peripheral vision is destroyed by laser treatment with hopes of maintaining central vision. Unfortunately, many patients continue to experience vision loss, even with available therapies. 

Key findings from the diabetic retinopathy Phase 2a trial showed a single intravitreal administration of PER-001 released for six months led to:

• Improved function (contrast sensitivity, visual acuity and visual field). There was a mean of +0.9 dB improvement in low luminance contrast sensitivity in the high-dose group and +0.65 dB in the low-dose group across multiple frequencies measured at week 20. In contrast, a mean of -2.1 dB worsening occurred in the control group over the same period. This corresponds to a difference of 2.75 dB to 3 dB magnitude for low- and high-dose groups compared to control; 3 dB is equivalent to ETDRS 15 letters (three lines). The low luminance, low contrast visual acuity was better by a mean difference of 5.5 and 5.1 letters from baseline in low- and high-dose groups compared to control measured at week 20. Patients with reduced peripheral vision showed an improvement of +1.8 dB per year with PER-001 treatment compared to +0.01 dB per year in the control group at six months.
   
• Improved structure (macular ischemia, macular leakage and macular microaneurysms by ultrawide field fluorescein angiography): PER-001 treatment improved macular ischemia index, macular leakage and macular microaneurysms across both doses compared to worsening in control at six months. DRSS remained largely stable across all groups.

Details of this phase are published in "PER-001, A Novel, Long Acting Endothelin Antagonist IVT Implant for the Treatment of Retinal Ischemia in Glaucoma, Diabetic Retinopathy and Age Related Macular Degeneration," which was presented at Clinical Trials at the Summit (CTS) meeting on June 21.

"The results from the Ph2a 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 Arshad Khanani, MD, Clinical Professor of Ophthalmology at University of Nevada and Director of Clinical Research at Sierra Eye Associates, who presented the results at the CTS meeting. "These functional gains in peripheral vision, contrast sensitivity and visual acuity accompanied by structural improvements in retinal ischemia, leakage, and microaneurysm validate the PER-001 mechanism of action. By targeting the endothelin pathway, PER-001 introduces a novel, every 6 months therapy, that can address the underlying neurovascular disease and offer vision benefits with the potential to be disease modifying for diabetic retinopathy and other ischemic retinal conditions."

A total of 60 patients were treated in both trials; 33 participants were enrolled in the glaucoma Ph1/2a and 27 participants in the diabetic retinopathy Ph2a study. The 24-week data demonstrated that PER-001 was safe and well-tolerated in both trials.

With these encouraging results, Perfuse Therapeutics plans to initiate pivotal trials across both indications with a larger population of patients over a longer period, starting in the second half of 2025. 

About PER-001
PER-001 is a novel (new chemical entity), first-in-class small molecule endothelin receptor antagonist. Endothelin is the most potent vasoconstrictor in the human body and is upregulated in glaucoma, diabetic retinopathy, age related macular degeneration and retinal vein occlusion. Endothelin is involved in inflammation and cell death through its receptors expressed in retinal vascular and neuroretinal cells. The PER-001 intravitreal implant is a bio-erodible implant administered into the vitreous cavity of the eye using a single-use, 25-gauge applicator and designed to provide a sustained release of PER-001, allowing for a convenient dosing every six months.

About Perfuse Therapeutics
Perfuse Therapeutics is a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases. Enabled by its proprietary sustained-release drug delivery platform, the company is developing a robust pipeline of novel assets against validated targets. Headquartered in South San Francisco, Calif., and with R&D facilities in Durham, N.C., Perfuse Therapeutics has established a strong team committed to advancing disease-modifying therapies to patients with ocular disease around the world. For more information, visit www.perfusetherapeutics.com and LinkedIn.

Investor Relations and Media Contact info@perfusetherapeutics.com.

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