Dompé recently announced that the first patient has been dosed in the United States in the phase 3 ORUNEA study of cenegermin-bkbj ophthalmic solution for the treatment of persistent corneal epithelial defect (PCED).
Let’s begin with Dompé.
Operating its international business out of Italy—with U.S. headquarters in San Mateo, California—the privately-held Dompé is active in all areas of the pharmaceutical value chain.
From the research and development phase to the production and commercialization phases, the biopharma giant boasts a pipeline targeting three therapeutic areas: the eye, the brain, and pain.
And zeroing in on the eye …
Dompé’s ophthalmic involvement began with developing a treatment for neuropathic keratitis (NK) based on nerve growth factor (NGF) research and a biotechnological process.
- To note: That drug has since been approved by the FDA (in 2018) and marketed as OXERVATE (cenegermin-bkbj) ophthalmic solution, 0.002% (20 mcg/mL).
- Its claim to fame: This prescription eye drop is the:
- First topical biologic FDA-approved treatment for NK
- First recombinant human nerve NGF (rhNGF)-based therapy for NK.
- Its claim to fame: This prescription eye drop is the:
Back up: Refresh me on NGF.
NGF is essentially an endogenous protein that plays a role in neuron differentiation and maintenance.
- During its discovery, neurobiologists determined that NGF is also critically involved in the proliferation, differentiation, and survival of sympathetic and sensory nerves—as well as pain perception.
And in recent research?
NGF’s therapeutic properties have been found to benefit human cutaneous and corneal ulcers, pressure ulcers, glaucoma, maculopathy, diabetic retinopathy (DR), retinitis pigmentosa (RP), and other ophthalmic diseases.
- As for Dompé: The company acquired the rights to produce and market NGF-based treatments in 2010.
Why test its efficacy in PCED?
As Dompé Chief Development Officer Ahmed Enayetallah noted:
- “PCED is a highly heterogeneous 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.”
Let’s move on to the phase 3 study.
ORUNEA (NCT07519902) is a multicenter, randomized, double-masked study comparing cenegermin-bkbj against placebo in achieving complete healing of PCED.
The study will enroll approximately 150 people with PCED aged 18+ years and comprise three periods:
- An 8-week initial treatment period (Day 1 to Week 8)
- An 8-week extension treatment period (Weeks 9 to 16)
- A 24-week follow-up period (Weeks 17 to 40)
Participants with PCED in the study eye are included if the PCED is ≥1.0 mm in greatest diameter and of at least 14 days duration and refractory to one or more conventional nonsurgical treatments, showing no clinical resolution.
What are the main outcome measures?
The primary endpoint will assess the proportion of responders achieving complete epithelial healing of the cornea after 4 weeks of treatment and maintained at 8 weeks.
The secondary outcome measures include:
- Percentage change from baseline in maximum diameter of PCED at Weeks 4 and 8
- Proportion of responders achieving complete epithelial healing of the cornea at Week 8 and maintained at Week 10
- Linear change from baseline in maximum diameter of the PCED at week 4
- Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
- Number of participants discontinuing the study due to intolerability
Any comments from the company on this news?
Per Marcello Allegretti, Dompé’s chief scientific officer: “Advancing cenegermin-bkbj into a phase 3 program for PCED reflects the strength of the underlying science and the consistency of the recombinant human NGF (rhNGF) mechanism across corneal pathologies characterized by impaired healing.”
“This study is designed to rigorously evaluate whether targeting the biological drivers of epithelial repair can offer a clinically meaningful benefit for patients with limited therapeutic options,” he further noted.
Any other news from Dompé?
Indeed. In October 2025 the FDA selected Dompé’s investigational intranasal formulation of cenegermin for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION) for the Commissioner’s National Priority Voucher (CNPV) pilot program.
Why this matters: The program aids in expediting the development and review of prescription drugs and biological products that have significant potential to address critical U.S. national priorities.
And most recently: A phase 3 clinical trial (NCT07453888) evaluating the intranasal spray for NAION is currently marked as not yet recruiting, so we’ll likely hear more news from the company soon.