EyePoint reports midstage trial fail for Duravyu, raising serious questions about the prospect

Targeting a major pharmaceutical market in retinal diseases, EyePoint Pharmaceuticals has revealed that its lead drug candidate failed to meet its endpoint in a phase 2 trial.

In the PAVIA study in patients with non-proliferative diabetic retinopathy (NPDR), EyePoint's Duravyu failed to sufficiently improve patient scores on the Diabetic Retinopathy Severity Scale (DRSS). With this, the drug fell short of the trial's pre-specified primary endpoint, EyePoint said in a release.

Trial investigators were looking for patients to experience at least a two-point improvement on the DRSS scale nine months after a single injection with Duravyu, also known as EYP-1901. 

Only 5% of patients in the 3-mg treatment arm—and none in the 2-mg arm—achieved the two-step DRSS improvement or greater, EyePoint said. That's compared with 5% in the control arm.

The company noted that at least 80% of patients in each treatment arm "demonstrated stable or improved disease" after nine months. Seventy percent of patients in the control group experienced the same.

While the company touted the prospect's "biologic effect" and "favorable safety and tolerability profile," investors sold the company's stock off by about 35% Monday morning.

EyePoint said it will update on its plans for Duravyu in NPDR after reviewing 12-month data.

In a research note, Mizuho analyst Graig Suvannavejh, Ph.D., said the negative trial result "is a significant disappointment" for the company. Based on the result, the analyst said he expects "questions to rise to the surface regarding Duravyu's potential utility" in diabetic macular edema and wet age-related macular degeneration, two diseases the company is targeting with its treatment prospect.

The NPDR market for the drug could be worth about $250 million, Suvannavejh wrote, while the wet AMD field presents a much larger opportunity worth $1.5 billion.

Duravyu is an intravitreal, sustained-release insert that delivers vorolanib, a tyrosine kinase inhibitor that EyePoint licensed from Equinox Science in February 2020. 

In China, EyePoint partner Betta Pharmaceuticals has won regulatory approval for vorolanib tablets to treat certain patients with renal cell carcinoma when used in combination with everolimus.

In addition, regulators in the country have approved a separate EyePoint implant containing the corticosteroid fluocinolone acetonide. EyePoint has since sold the China-approved Yutiq product to Alimera Sciences in a deal worth $82.5 million plus royalties.