Glaucoma is a major cause of blindness worldwide, and although treatments exist to manage symptoms, none stop the disease from progressing. Early clinical studies by Perfuse Therapeutics suggest that its experimental implant, PER-001, could potentially modify the disease itself instead of simply reducing symptoms and slowing vision loss.*

Targeting the Cause

Unlike current treatments that only lower eye pressure, PER-001 is designed to protect and repair the optic nerve. It works by targeting the endothelin pathway, a newly validated part of glaucoma’s disease process. 

Endothelin-1 (ET-1) is a potent vasoconstrictor primarily produced by cells that line the inner walls of blood vessels. Vasoconstrictors cause blood vessels to narrow, reducing blood flow and increasing blood pressure. 

In a 24-week Phase 1 and 2a trial, PER-001 improved several key indicators in the eye: 

• Visual field (over how much area a person can see), 
• Retinal nerve fiber thickness, and 
• Blood flow to the optic nerve head. 

These early results suggest the implant could help slow or even reverse damage caused by glaucoma.

The implant is inserted into the eye and delivers medication for up to six months, reducing the need for frequent treatments.

Blending Science and Real Patient Needs

Dr. Sevgi Gurkan, Perfuse Therapeutics’ founder, explained that the team designed the trial to measure both vision improvements and physical changes in the eye. This strategy helped them understand how well PER-001 works while keeping patients’ experiences in mind. 

For example, they chose clinics already using the necessary diagnostic tools and developed a clear process to train staff. They also focused on including patients with different types of glaucoma, including those with normal eye pressure, which is often harder to treat.

Importantly, the company made sure the trial design considered patients’ and caregivers’ schedules and needs. Visit frequency was reduced where possible, and breaks were built into vision tests to reduce fatigue.

Looking Ahead

Today, over 80 million people worldwide live with glaucoma, and that number is expected to double in 15 years. Even the best current treatments can’t fully prevent vision loss. PER-001 could be the first therapy that changes the course of the disease itself. 

With a larger, adaptive Phase 2b trial for PER-001 set to begin in late 2025, the team at Perfuse Therapeutics says it hopes that this implant could be the first therapy that changes the course of the disease itself and one day becomes the new standard of care.

*Gurkan, S. (2025, April 25). The Future of Glaucoma Therapy: Adaptive Trial Design and Patient-Centric Protocols. Clinical Leader. https://www.clinicalleader.com/doc/the-future-of-glaucoma-therapy-adaptive-trial-design-and-patient-centric-protocols-0001 

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