Encouraging results have emerged from two phase II clinical trials investigating a novel treatment, PER-001, for glaucoma and diabetic retinopathy, two of the most common causes of blindness globally. Delivered via a sustained-release intravitreal implant every six months, PER-001 improved both vision and retinal structure compared with control groups.

Glaucoma is traditionally managed by lowering intraocular pressure (IOP), yet many patients continue to lose vision despite normal IOP levels.¹ PER-001 targets the underlying ischaemia and neurodegeneration associated with disease progression. In the glaucoma trial, 22.2% of patients receiving a low dose and 37.5% on a high dose experienced significant improvements in visual field, while no improvement was observed in the control group. Additionally, none of the treated patients experienced major vision loss, compared with 12.5% of those in the control group. Improvements in optic nerve blood flow and structure suggest that PER-001 could offer both protection and potential reversal of damage.

In diabetic retinopathy, current treatments tend to focus on halting structural damage, often compromising peripheral vision. PER-001 delivered broad functional improvements, including contrast sensitivity, visual acuity, and peripheral visual field. Treated patients saw up to a 3 dB improvement over controls in contrast sensitivity—equivalent to a three-line gain on a standard eye chart. Structural indicators such as macular ischaemia and vascular leakage also improved in the treatment group, whereas control patients showed worsening in these measures. These results support the therapy’s novel mode of action targeting the endothelin pathway, and point to a potential shift in how diabetic retinopathy is managed.

Phase 2b/3 trials are expected to begin in late 2025.

References:

1. Costagliola C, Agnifili L, Mastropasqua L, di Costanzo A. Low-Tension Glaucoma: An Oxymoron in Ophthalmology. Prev Chronic Dis. 2019;16:180534.

2. Mounirou BAM, Adam ND, Yakoura, et al. Diabetic Retinopathy: An Overview of Treatments. Indian J Endocrinol Metab. 2022;26:111-8

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Disclosure: This article was created by the touchOPHTHALMOLOGY team utilizing AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat.) The content was developed and edited by human editors. No funding was received in the publication of this article.

Citation: PER-100 shows promise in phase II trials for glaucoma and diabetic retinopathy. touchOPHTHALMOLOGY.com. 27 June 2025.