Dompé recently announced that the first patient has been enrolled in a phase 3 study of intranasal cenegermin-bkbj (OXERVATE) for the treatment of non-arteritic anterior ischemic neuropathy (NAION).
Give me some background on cenegermin.
Dompé’s foray into ophthalmic clinical research began with developing a treatment for neurotrophic keratitis (NK) based on nerve growth factor (NGF) research.
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 and first recombinant human nerve NGF (rhNGF)-based therapy for NK.
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 NAION?
NAION is the most prevalent acute optic neuropathy affecting middle-aged and older individuals.
- Its cause: a lack of sufficient blood flow to the optic nerve, which can lead to damage and vision loss.
- Its prevalence: There are approximately 6,000 new cases per year in the U.S. and 10,000-46,000 new cases in the European Union per year.
Moreover: The number of cases is growing because of the aging population and rising prevalence of risk factors such as diabetes, hypertension, and other obesity-related conditions like sleep apnea.
And to make matters worse, there are currently no FDA-approved treatments currently available for NAION.
… go on …
Consequently: As Dompé Chief Development Officer Ahmed Enayetallah, explained:
- “[The] intranasal NGF therapy represents a promising approach, leveraging NGF’s ability to support the survival and repair of optic nerve cells, with the potential to reverse and/or prevent vision loss associated with NAION if diagnosed and treated quickly.”
Got it. Now, wasn’t Dompé recently in the news?
Indeed. In fact, just last month the company reported that the first U.S. patient had been dosed in the phase 3 ORUNEA study of OXERVATE for the treatment of persistent corneal epithelial defect (PCED).
The relevancy to this intranasal therapy: In October 2025 the company was selected for the Commissioner’s National Priority Voucher (CNPV) pilot program to accelerate the development and review of drugs that address critical U.S. national health priorities.
- Why: To accelerate a potential Biologics License Application (BLA) for the therapy.
Sounds like it’s been a busy year … now move on to this new study.
Involving more than 130 centers globally, the phase 3 trial Galassia-NAION-301 (NCT07453888) is a 24-week, multicenter, double-blind study comparing OXERVATE against placebo in improving vision loss caused by NAION.
Regarding enrollment: Dompé plans to enroll approximately 272 people globally with NAION aged 50 to 80 years.
The study endpoints will assess safety and functional and structural changes in the affected eye, such as the change from baseline in:
- Best-corrected visual acuity (BCVA) measured using the Early Treatment in Diabetic Retinopathy (ETDRS) chart
- Visual field (VF) mean sensitivity in decibels and in a prespecified region consisting of at least five separate loci on VF testing
- Ganglion cell layer-inner plexiform layer (GCL-IPL) thickness on optical coherence tomography (OCT)
Last question: When can we expect an update?
Per ClinicalTrials, the estimated primary completion date is September 2027; however, with all of the ongoing clinical trials, we’ll likely hear from Dompé again soon.
As usual, stay tuned for more updates!