Twenty-four-week data from the Ph1/2a trial in glaucoma patients reveals PER-001 to be well-tolerated. Proof of concept is established with both strong functional and structural outcomes
Glaucoma patients treated with PER-001 on top of IOP reducing therapies experienced a mean improvement in visual field mean deviation of 1 decibel (dB) per year and a mean increase in retinal nerve fiber layer thickness of 3 microns at 24 weeks
SAN FRANCISCO, March 3, 2025 /PRNewswire/ -- Perfuse Therapeutics, a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases announced today the presentation of 24-week data from its first-in-human Phase 1/2a trial of PER-001 intravitreal implant in patients with glaucoma. The data was presented by Steven Mansberger, MD, MPH, Chief of Ophthalmology, Director of Glaucoma Services at Legacy Devers Eye Institute, March 1, 2025, at the American Glaucoma Society Meeting in Washington DC.
The trial is fully enrolled with glaucoma patients (n=33) receiving a single administration of PER-001 on top of IOP reducing therapies. Data presented included results from the Ph1 (n=6) and Ph2a Dose 1 (n=12) cohorts, both of which completed the trial. The Ph2a Dose 2 (n=15) cohort is ongoing.
The 24-week data demonstrated that PER-001 was well-tolerated, with only one drug-related adverse event of vitreous floater (mild and transient), no drug-related serious adverse events and no reports of intraocular inflammation. Patients treated with PER-001 showed a mean increase in optic nerve head blood flow of at least 10% from baseline starting at Week 1, which was sustained out to Week 24 and was associated with a mean improvement in visual field mean deviation of 1 dB/yr and a mean increase in retinal nerve fiber layer thickness of 3 microns, as measured by optical coherence tomography (OCT) at Week 24. In contrast, the sham control group revealed a 0.63 dB/yr decline in visual field mean deviation and a decrease of 1.3 microns in OCT RNFL at Week 24.
“These results are distinctive and noteworthy,” stated Steven Mansberger, MD, MPH. “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 key functional and structural outcomes are very promising.”
“We are excited to share first-in-human data from the ongoing PER-001 trials,” said Sevgi Gurkan, MD, CEO and Founder. “Endothelin is a well-established, clinically validated pathway with many approved products in systemic diseases. The Ph1/2a trial marks the first ever clinical application of an endothelin antagonist for local treatment of back of the eye diseases, and we are encouraged by the safety profile and efficacy data to date.”
“Glaucoma is the leading cause of irreversible blindness worldwide, and there are no currently approved therapies. This trial provides the first clinical validation of the key role of endothelin pathway in glaucoma. The results of this trial are highly encouraging and the data supports the potential of PER-001 intravitreal implant as a disease-modifying therapy that could significantly improve the vision and lives of glaucoma patients,” said Phil Lai, MD, Chief Medical Officer.
About PER-001 Intravitreal Implant, a Long Acting Endothelin Receptor Antagonist
PER-001 is a novel (NCE), first-in-class small molecule endothelin receptor antagonist. Endothelin is the most potent vasoconstrictor in the human body and is upregulated in glaucoma. 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 every 6 months dosing frequency.
About the Ph1/2a Trial
The Phase 1/2a trial is a multi-center two-part trial designed to assess the safety, tolerability and pharmacodynamics of PER-001 in glaucoma patients. The Phase 1 part of the trial was a first-in-human, open-label, single ascending dose (SAD) study that evaluated the safety and tolerability of two doses of PER-001 intravitreal implant in patients with advanced glaucoma. The Phase 2a part of the trial was a patient and reading center masked, randomized, sham-controlled study that evaluated the safety, tolerability, and pharmacodynamics of two doses of PER-001 in patients with progressing, mild to moderate glaucoma.
Patients in both phases received a single intravitreal administration of PER-001 while maintaining their pre-study IOP-lowering therapies. Two doses of PER-001 intravitreal implant were tested sequentially in cohorts of 3 patients each during the Ph1 SAD portion and the same two doses were subsequently tested in a larger cohort of patients during the Ph2a portion of the trial. All patients were followed for 24 weeks from their first and single dose. The Ph1 cohorts and Ph2a Dose 1 cohort have completed the trial. Ph2a Dose 2 cohort is ongoing and the topline data will be available in second quarter 2025.
About Perfuse Therapeutics, Inc.
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, California and with R&D facilities in Durham, North Carolina, Perfuse Therapeutics has established a strong team committed to advancing disease modifying therapies to patients with ocular disease around the world. For more information, please see www.perfusetherapeutics.com and on LinkedIn.
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