Dompé initiates phase 3 Orunea trial of cenegermin-bkbj in PCED

Dompé has dosed the first US patient in a phase 3 study of cenegermin-bkbj ophthalmic solution for the treatment of persistent corneal epithelial defect (PCED).¹ The multicenter, randomized, double-masked trial—named Orunea—will compare cenegermin-bkbj against placebo in approximately 150 adult patients, with a primary endpoint of complete epithelial healing of the cornea.¹

PCED affects fewer than 200,000 people in the US and represents a condition with a high unmet clinical need, as current treatment options remain limited.¹ Dompé's entry into PCED builds on its established rhNGF development program—cenegermin-bkbj received FDA approval in 2018 under the trade name Oxervate for the treatment of neurotrophic keratitis, becoming the first topical biologic approved in ophthalmology.² “Expanding research into PCED builds on our established experience in neurotrophic keratitis and reflects our continued effort to explore how rhNGF may address a broader spectrum of corneal diseases,” said Marcello Allegretti, chief scientific officer at Dompé.¹

Orunea trial design and endpoints

The Orunea trial (NCT07519902) is a multicenter, randomized, double-masked, placebo-controlled study enrolling approximately 150 adults aged 18 years and older with PCED.¹ The primary endpoint will assess the proportion of patients achieving complete epithelial healing of the cornea. Cenegermin-bkbj is a recombinant human nerve growth factor (rhNGF)—a neurotrophin essential for the growth, maintenance, and survival of neurons—formulated as a topical ophthalmic solution.¹

The rhNGF mechanism targets the biological drivers of corneal epithelial repair, including corneal innervation and epithelial-stromal interaction pathways established in cenegermin's NK clinical program.² In the pivotal trials supporting the NK approval, 69.6% of patients treated with cenegermin achieved complete corneal healing within 8 weeks, compared with 29.4% of vehicle-treated patients.² PCED represents a broader heterogeneous corneal healing failure phenotype with overlapping but distinct pathophysiology, and no agent has been approved specifically for this indication.³

Safety profile and unmet need

No safety or efficacy data for cenegermin-bkbj in PCED have been established, as Orunea is the first phase 3 evaluation of this agent in the indication.¹ The known safety profile of cenegermin-bkbj from the NK program includes eye pain as the most common adverse reaction, occurring in approximately 16% of patients, with other reactions including corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation, photophobia, tearing, and headache occurring in 1% to 10% of patients.¹

PCED arises from a range of etiologies—including limbal stem cell deficiency, neurotrophic keratopathy, dry eye disease, and systemic conditions such as diabetes—and carries significant morbidity risk, including corneal scarring, infection, and vision loss if inadequately managed.³ Current standard-of-care approaches, including lubricants, therapeutic contact lenses, autologous serum eye drops, and amniotic membrane transplantation, address supportive healing but do not directly target the biological mechanisms of re-epithelialization.³ The Orunea trial is designed to evaluate whether rhNGF can provide clinically meaningful benefit in a population where no approved pharmacologic option currently exists.

"PCED is a highly heterogenous condition, and many of the underlying corneal diseases associated with it have limited treatment options. This underscores an urgent need for novel therapies that directly stimulate epithelial cells involved in the corneal healing and defect closure," said Ahmed Enayetallah, chief development officer at Dompé.

Dompé's rhNGF pipeline extends beyond NK and PCED, with cenegermin-bkbj under evaluation in Sjögren's dry eye disease and other anterior segment conditions.¹ Orunea marks the company's first phase 3 program in a PCED-specific indication and, if successful, would represent the first approved pharmacologic agent for PCED in the US.

References:

1. Dompé. Dompé doses first patient in phase 3 study of cenegermin-bkbj in PCED. Published June 10, 2026. Accessed June 10, 2026. https://www.biospace.com/press-releases/dompe-doses-first-patient-in-phase-3-study-of-cenegermin-bkbj-in-pced

2. Pflugfelder SC, Massaro-Giordano M, Perez VL, et al. Topical recombinant human nerve growth factor (cenegermin) for neurotrophic keratopathy: a multicenter randomized vehicle-controlled pivotal trial. Ophthalmology. 2020;127(1):14-26. doi:10.1016/j.ophtha.2019.08.020

3. Thia ZZ, Ho YT, Shih KC, Tong L. New developments in the management of persistent corneal epithelial defects. Surv Ophthalmol. 2023;68(6):1093-1114. doi:10.1016/j.survophthal.2023.06.001