Dompé Doses First Patient in Phase 3 Study of Cenegermin-bkbj in NAION

Non-arteritic anterior ischemic optic neuropathy (NAION) is a loss of blood flow to the optic nerve which causes spontaneous and rapid vision loss, for which there are currently no FDA-approved treatments. Given the substantial unmet need for effective treatments in NAION, in October 2025, Dompé was awarded a Commissioner’s National Priority Review Voucher (CNPV) by the US FDA to accelerate a potential Biologics License Application for intranasal nerve growth factor (NGF) in NAION.

Dompé, a biopharmaceutical company with operations in Italy and the US, 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, the Phase 3 trial, “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, non-invasive approach that bypasses the blood-brain barrier and has the potential to redefine the standard of care.”

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. 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.”

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). Cenegermin-bkbj is an investigational product for NAION, and its safety and efficacy for this indication have not been established or approved by any regulatory authority.

About Dompé
Dompé farmaceutici S.p.A. is a privately held, global biopharmaceutical company on a mission to bring the full potential of nerve growth factor (NGF) to patients. As the first company to unlock the therapeutic potential of NGF, Dompé developed the first FDA-approved NGF treatment. Today, our clinical pipeline reflects our commitment to redefining treatments across ophthalmic, neurological and pain-related conditions. Building on 130 years of independence, we are embracing the challenge to transform our science and ourselves by delivering first-in-class, disease-modifying therapies through NGF and other breakthrough molecules to help people improve their sight, support neurological recovery and manage pain effectively. Today, Dompé employs more than 1,100 employees worldwide and maintains a US commercial operations hub in the San Francisco Bay Area.

More information about the current Galassia-NAION-301 clinical trial can be found at www.clinicaltrials.gov (NCT07453888) or at: https://galassia-naion301study.com/.

About Nerve Growth Factor (NGF)
Nerve Growth Factor (NGF) is a neurotrophin, a member of a family of proteins essential for the growth, maintenance and survival of neurons, with broad therapeutic potential. NGF was discovered by the Italian neurobiologist Professor Rita Levi-Montalcini and American biochemist Dr. Stanley Cohen, earning them the Nobel Prize in Physiology or Medicine in 1986. In their research, Drs. Levi-Montalcini and Cohen uncovered that NGF plays a crucial role in the proliferation, differentiation, and survival of sympathetic and sensory neurons. Dompé is the first company to unlock the therapeutic potential of NGF, building upon a Nobel Prize-winning discovery to deliver a breakthrough treatment for a rare neurotrophic eye disease.

About Non-Arteritic Anterior Ischemic Neuropathy (NAION)
Optic neuropathies are disorders caused by damage to the optic nerve, disrupting visual signals from the retina to the brain due to injury, inflammation, poor blood flow, or genetic and degenerative factors. The result is progressive or rapid loss of vision, often affecting central or peripheral sight, color discrimination, and in severe cases, leading to permanent blindness. Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common cause of acute optic nerve injury and optic neuropathy in people over 50. It affects men and women equally, usually starting around age 66. While the exact cause isn’t fully understood, common risk factors include diabetes, high blood pressure, high cholesterol, and smoking. Despite its frequency, and risk of severe permanent vision loss, there is no proven treatment to improve visual outcomes.

About the Commissioner's National Priority Voucher (CNPV) Pilot Program
The U.S. Food and Drug Administration (FDA) launched the Commissioner’s National Priority Voucher (CNPV) pilot program to accelerate the development and review of prescription drugs and biological products that address critical U.S. national health priorities. Announced in June 2025, the program offers companies the opportunity to reduce standard application review times from 10–12 months to just 1–2 months through a collaborative, multidisciplinary review process. Selected companies receive a voucher that provides enhanced communication with the FDA and a rolling review to facilitate a faster timeline, while maintaining the agency’s rigorous standards for safety and efficacy. The program focuses on transformative therapies that tackle major public health challenges and large unmet medical needs.

About Oxervate
OXERVATE^® (cenegermin-bkbj) ophthalmic solution 0.002% is indicated for the treatment of neurotrophic keratitis. Important Safety Information: Contact lenses, either therapeutic or corrective, should be removed before applying OXERVATE. Contact lenses may be reinserted 15 minutes after OXERVATE administration. Eye Discomfort, such as eye pain, that can be mild to moderate can occur with OXERVATE. Patients should contact their health care provider if a more serious eye reaction occurs. The most common adverse reaction with OXERVATE (~16%) was eye pain. Other adverse reactions with OXERVATE (1% to 10%) included corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation, photophobia, tearing, and headache.

View source version on businesswire.com: https://www.businesswire.com/news/home/20260713100379/en/