Perfuse Therapeutics Reports Positive Phase 1/2a Trial Results for PER-001 in Glaucoma

Perfuse Therapeutics, Inc., a biopharmaceutical firm located in San Francisco, has released promising results from its Phase 1/2a clinical trial of PER-001, an innovative treatment for glaucoma. This trial marks a significant step forward in the quest for a disease-modifying therapy for this leading cause of irreversible blindness worldwide. The findings were presented by Dr. Steven Mansberger during the Association for Research in Vision and Ophthalmology (ARVO) Meeting.

The study demonstrated that a single intravitreal administration of PER-001, when combined with standard intraocular pressure (IOP) reducing therapies, resulted in improved ocular blood flow, visual function, and anatomic structure. These outcomes were measured using visual field (VF) sensitivity and optical coherence tomography (OCT) retinal nerve fiber layer (RNFL) thickness. Importantly, PER-001 was well-tolerated and showed a robust safety profile over the six-month trial period.

Dr. Sevgi Gurkan, CEO and Founder of Perfuse Therapeutics, emphasized the potential of PER-001 as a breakthrough in glaucoma treatment. The company plans to initiate a Phase 2b trial in the latter half of 2025, with aspirations that it could become one of the pivotal studies needed for registration. Unlike traditional glaucoma treatments that solely focus on lowering IOP, PER-001 addresses the endothelin pathway, associated with vascular dysfunction in glaucoma. This novel approach could transform the therapeutic landscape for the disease.

Dr. Mansberger highlighted the significance of the trial results, noting the improvement in ocular blood flow, optic nerve structure, and visual function. This is the first instance of studying endothelin antagonism in the human eye, heralding a potential new era in glaucoma treatment.

The Phase 1/2a study was designed to evaluate the safety and efficacy of the PER-001 intravitreal implant. It included a first-in-human, open-label Phase 1 study that tested two doses on patients with advanced glaucoma. Meanwhile, the Phase 2a component was a randomized, masked study that compared low and high doses of PER-001 against a sham control in patients with mild-to-moderate glaucoma. A total of 33 participants received either PER-001 or a sham injection, while continuing their prior IOP-reducing treatments.

Safety results from the trial were encouraging, with only mild, self-resolving vitreous floaters reported as adverse events. No serious adverse events or drug-related complications were noted, and participants maintained stable best-corrected visual acuity throughout the trial.

The exploratory efficacy results revealed that PER-001 significantly enhanced optic nerve head blood flow from as early as the first week, maintaining this improvement over the 24-week period. Visual function showed a notable improvement in VF mean deviation for both high and low doses of PER-001, while the control group experienced deterioration. Anatomically, PER-001 led to an increase in RNFL thickness, contrasting with the decrease observed in the control group. A strong correlation was identified between structural and functional improvements, underscoring the treatment's potential effectiveness.

PER-001 is an innovative small molecule endothelin receptor antagonist delivered via a bio-erodible implant, allowing for a convenient six-month dosing schedule. This approach targets endothelin, a vasoconstrictor implicated in inflammation and cell death in glaucoma.

Globally, glaucoma remains a primary cause of blindness, and despite existing IOP-lowering treatments, vision loss persists in many patients. Perfuse Therapeutics is committed to developing therapies that modify the disease's course, leveraging its sustained-release drug delivery platform to address validated targets. As the company progresses with PER-001, it aims to offer a groundbreaking treatment option for those with glaucoma.