Promising Phase 1/2a Trial Results for Glaucoma Treatment PER-001 Presented at AGS Meeting

Promising Results from PER-001 Trials in Glaucoma Treatment



Perfuse Therapeutics, a biopharmaceutical company focused on innovative treatments for ischemic eye diseases, has announced significant findings from the first-in-human Phase 1/2a trial of its investigational drug, PER-001, during the recent American Glaucoma Society (AGS) Meeting in Washington, D.C. The presentation, delivered by Dr. Steven Mansberger, highlighted the safety and efficacy of PER-001 in patients suffering from glaucoma, a leading cause of irreversible blindness globally.

Overview of the Trial



The Phase 1/2a trial is designed to evaluate the safety, tolerability, and pharmacodynamics of PER-001. The trial included 33 enrolled patients who received a single dose of PER-001 alongside their existing intraocular pressure-reducing therapies. This study included two phases: a Phase 1 section with six participants and the first dose of Phase 2 involving 12 participants, both of which have completed their assessments. Meanwhile, the second dose within Phase 2, which involves 15 participants, is still ongoing.

After 24 weeks, results indicated that PER-001 was well-tolerated, with a minimal incidence of side effects—specifically only one mild and transient adverse event reported. Impressively, there were no severe drug-related adverse events or cases of intraocular inflammation noted.

Efficacy Results



Patients who received PER-001 demonstrated a notable improvement in their optic nerve health. This improvement was measured by a mean increase in optic nerve head blood flow of over 10% from the baseline, starting from the first week and continuing through to week 24. Additionally, participants exhibited a mean increase of 3 microns in retinal nerve fiber layer thickness alongside a 1 dB annual improvement in visual field mean deviation — a significant advancement compared to the sham control group, which experienced a decline in both metrics.

Dr. Mansberger remarked on the implications of their findings, stating, "These results are distinctive and noteworthy. There remains an unmet need for neuroprotective therapies for patients with glaucoma, and while this is early data, the observed improvements in the optic nerve head blood flow, as well as the key functional and structural outcomes, are very promising."

Importance of the Trial



The trial's findings are particularly significant given that there are currently no approved therapies aimed specifically at treating glaucoma, potentially allowing for PER-001 to play a transformative role in patient management. The role of endothelin in causing inflammation and nerve cell death makes the development of a targeted treatment urgent and necessary.

Sevgi Gurkan, CEO and Founder of Perfuse Therapeutics, expressed their enthusiasm about the initial results, emphasizing the breakthrough aspects of employing an endothelin antagonist for treating ocular conditions. "Endothelin is a well-established pathway with many approved products for other systemic diseases, but this represents the first clinical application in the ocular domain," she explained.

As the leading cause of irreversible blindness, glaucoma affects millions globally. Most residents with this condition have shown little to no improvement with current therapies. With the promising data presented from the PER-001 trial, there exists hope for a new, effective treatment option that could fundamentally change the lives of those suffering from this disease.

Next Steps



Perfuse Therapeutics and their team continue to monitor patients from the ongoing Phase 2 cohort, with topline data expected to be available by the second quarter of 2025. Should ongoing results continue to highlight the safety and efficacy of PER-001, it may soon pave the way for a revolutionary change in glaucoma treatment.

Headquartered in South San Francisco, California, Perfuse Therapeutics is committed to driving innovative solutions for ocular diseases through its unique sustained-release delivery systems. For further updates, stakeholders are encouraged to follow their progress on official platforms including their LinkedIn page and website.

Topics Health)

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