Perfuse Therapeutics has reported 24-week data from its first-in-human, multi-centre Phase I/IIa trial of the PER-001 intravitreal implant for individuals with glaucoma.
The trial of PER-001 is tailored to evaluate its tolerability, pharmacodynamics, and safety in these patients.
It has reached full enrolment, with 33 subjects receiving PER-001’s single administration alongside existing intraocular pressure (IOP) reducing therapies.
Perfuse noted that the data comprised findings from the Phase I cohort of six subjects and the Phase IIa Dose 1 cohort of 12 subjects, both of which have concluded the trial. The Phase IIa Dose 2 cohort, consisting of 15 patients, is still in progress.
The data showed that PER-001 was well-tolerated, with only a single, mild, and transient drug-related adverse event of vitreous floater reported.
Subjects treated with the implant exhibited a mean increase in optic nerve head blood flow of a minimum of 10% from baseline as early as week 1, a benefit that persisted through week 24.
This improvement was related to a mean enhancement in visual field mean deviation of one decibel (dB) per year and a mean increase in retinal nerve fibre layer thickness of three microns, as measured by optical coherence tomography (OCT) at week 24.
In comparison, the sham control group experienced a decline of 0.63dB per year in visual field mean deviation and a decrease of 1.3 microns in OCT retinal nerve fibre layer thickness at the 24-week mark.
The bio-erodible implant is administered into the eye’s vitreous cavity with a 25-gauge single-use applicator.
It is engineered to offer PER-001’s sustained release, aiming for a “convenient” dosing frequency of every six months.
Perfuse Therapeutics chief medical officer Phil Lai said: “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.”
