Key Takeaways

  • Dompé has enrolled the first patient in the global phase 3 Galassia-NAION-301 trial evaluating investigational intranasal cenegermin-bkbj for non-arteritic anterior ischemic optic neuropathy (NAION)
  • The placebo-controlled study will enroll approximately 272 participants at more than 130 sites worldwide and assess visual outcomes using BCVA, visual field testing, and OCT
  • Dompé's investigational NGF therapy is being studied as a noninvasive approach to support optic nerve cell survival and repair

Dompé announced the first patient has been enrolled in a phase 3 study of cenegermin-bkbj for the treatment of non-arteritic anterior ischemic neuropathy (NAION). Involving more than 130 centers globally, “Galassia-NAION-301” is a multicenter, double-blind study comparing cenegermin-bkbj against placebo in improving vision loss caused by NAION.

"Our NGF R&D program continues to accelerate with our third clinical study of 2026,” said Marcello Allegretti, Chief Scientific Officer at Dompé. “Dosing the first patient in our NAION trial marks a critical step in our mission to unlock the full potential of NGF. By advancing our proprietary intranasal NGF in clinical investigation, we are taking a bold, noninvasive approach that bypasses the blood-brain barrier and has the potential to redefine the standard of care.”

The study will enroll approximately 272 people globally with NAION aged 50 to 80 years. The study endpoints will assess functional and structural changes in the affected eye, as measured by best corrected visual acuity (BCVA), visual field testing, and optical coherence tomography (OCT). 

NAION refers to a loss of blood flow to the optic nerve which causes rapid vision loss that usually occurs spontaneously. There are approximately 6,000 new cases per year in the US and 10,000-46,000 new cases in the EU per year. According to Dompé, 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. As many as three out of four patients experience substantial visual impairment, and approximately one in 10 patients experience profound loss of vision and legal blindness.

“With no FDA-approved treatments currently available for NAION, there is an urgent need for novel therapies,” said Ahmed Enayetallah, Chief Development Officer at Dompé. “Dompé’s 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.”