Perfuse Therapeutics, Inc., a US biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases, has 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.
A media release from the company said each of the six-month, randomised, 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.
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.
Significant improvement in vision was demonstrated compared with control in each trial, the release said.
It said the therapy targeted the underlying cause of both diseases, and early results of PER-001 had shown 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 Dr 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.”
Glaucoma is the leading cause of global blindness worldwide, but only about half are aware of their condition.
Currently there are no glaucoma therapies approved by the US 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.
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