Dompé Launches Phase 3 Trial for NAION Treatment
Biopharmaceutical firm Dompé has enrolled the first patient in a Phase 3 clinical trial for cenegermin-bkbj, an investigational treatment targeting non-arteritic anterior ischemic optic neuropathy (NAION). The multicenter study, dubbed Galassia-NAION-301, evaluates the efficacy and safety of intranasal nerve growth factor (NGF) in a patient population that currently lacks any FDA-approved therapies.
A Sudden Threat to Vision
NAION occurs when blood flow to the optic nerve is spontaneously interrupted, leading to rapid vision loss. The condition strikes roughly 6,000 new patients each year in the United States, with an additional 10,000 to 46,000 cases annually across the European Union. Hypertension, diabetes, and obesity-related issues like sleep apnea serve as primary risk factors.
The consequences are often severe. Data shows up to three patients in four endure substantial visual impairment, while roughly one patient in ten faces profound vision loss or legal blindness. With no existing FDA-approved treatment to address the underlying optic nerve damage, the medical community is now turning toward neurotrophic support as a potential lifeline.
Testing Intranasal Nerve Growth Factor
The Galassia-NAION-301 trial spans more than 130 global sites and aims to enroll approximately 272 participants aged 50 to 80. This double-blind investigation pits intranasal cenegermin-bkbj against a placebo to determine if the drug can offer meaningful clinical improvements.
Marcello Allegretti, Chief Scientific Officer at Dompé, notes that the intranasal delivery method is specifically engineered to bypass the blood-brain barrier. The trial measures success through three primary lenses: Best Corrected Visual Acuity (BCVA) for central vision, visual field tests for peripheral loss, and Optical Coherence Tomography (OCT) to track structural changes in the optic nerve.
Accelerating Regulatory Pathways
Dompé’s push into NAION follows its prior success with nerve growth factor. In October 2025, the FDA granted the company a Commissioner’s National Priority Review Voucher (CNPV) to expedite the regulatory process for this urgent medical need. While cenegermin-bkbj is already approved for neurotrophic keratitis under the name Oxervate, its application for NAION remains unproven. The trial is registered on ClinicalTrials.gov (NCT07453888), with further details hosted at galassia-naion301study.com.
Leveraging Nobel-Winning Science
The study hinges on the properties of NGF, a protein identified by Nobel laureates Rita Levi-Montalcini and Stanley Cohen. Because NGF is vital for the survival and maintenance of neurons, researchers hope to harness its power to protect the optic nerve.
“The goal is to leverage NGF’s capacity to promote the survival and repair of optic nerve cells, which could potentially prevent or reverse vision loss if administered rapidly after the onset of the condition,” said Ahmed Enayetallah, Head of Development at Dompé. As the trial moves forward, clinicians are watching closely to see if this neurotrophic approach can establish a new standard of care for patients who currently have no approved medical options.
Keep reading