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

MILAN & SAN MATEO, Calif.--(BUSINESS WIRE)--Dompé, a leading biopharmaceutical company with operations in Italy and the U.S., announced the first patient has been dosed in the US in a Phase 3 study of cenegermin-bkbj ophthalmic solution for the treatment of persistent corneal epithelial defect (PCED). The Phase 3 trial, “Orunea” is a multicenter, randomized, double-masked study comparing cenegermin-bkbj against placebo in achieving complete healing of PCED.

“Expanding research into PCED builds on our established experience in neurotrophic keratitis and reflects our continued effort to explore how rhNGF may address a broader spectrum of corneal diseases." - Marcello Allegretti, Chief Scientific Officer

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“At Dompé, our mission is to ensure that people living with rare and debilitating eye diseases have access to innovative treatments that can meaningfully improve their lives,” said Marcello Allegretti, Chief Scientific Officer at Dompé. “Expanding research into PCED builds on our established experience in neurotrophic keratitis and reflects our continued effort to explore how rhNGF may address a broader spectrum of corneal diseases.”

The cornea is the clear, protective outer layer of the eye. Its surface is made up of specialized cells that constantly renew themselves, to keep the eye healthy and to heal injuries. When this healing process fails and a defect persists for more than two weeks despite standard treatment, it is called PCED. This condition may cause significant pain and blurred vision, and if left untreated, it can lead to serious complications like severe infection, permanent scarring, and partial or complete loss of vision. Affecting fewer than 200,000 people in the U.S., PCED represents a serious, rare disease with a high, unmet clinical need.¹

“Advancing cenegermin-bkbj into a Phase 3 program for PCED reflects the strength of the underlying science and the consistency of the 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,” Allegretti further noted.

“PCED is a highly heterogenous 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,” said Ahmed Enayetallah, Chief Development Officer at Dompé. “This study underscores our long-standing commitment to translating the biology of rhNGF into meaningful therapies for patients with serious ocular diseases.”

The study will enroll approximately 150 people with PCED aged 18 years and above and the primary endpoint will assess the proportion of responders achieving complete epithelial healing of the cornea. Cenegermin-bkbj is an investigational product for PCED, and its safety and efficacy for this indication have not been established or approved by any regulatory authority.

More information about the current clinical trial can be found at www.clinicaltrials.gov (NCT07519902).

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.
Learn more at https://www.dompe.com/us/.

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 Persistent Corneal Epithelial Defect (PCED)
Persistent corneal epithelial defect (PCED) is when the surface of the eye (the cornea) doesn’t heal properly and stays open or damaged for more than two weeks. This can be caused by eye injuries, infections, dryness or chronic inflammation. Conditions like diabetes, nerve damage in the eye or severe allergic eye disease can also make it harder for the cornea to repair itself. Common symptoms of PCED include eye pain, redness, tearing, blurred vision and sensitivity to light. If it is not properly treated or managed, PCED can cause serious infection and permanent scarring of the cornea and loss of eyesight or blindness.

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.

References:

1. Wirostko B, Rafii M, Sullivan DA, Morelli J, Ding J. Novel Therapy to Treat Corneal Epithelial Defects: A Hypothesis with Growth Hormone. Ocul Surf. 2015 Jul;13(3):204-212.e1. doi: 10.1016/j.jtos.2014.12.005. Epub 2015 Mar 28. PMID: 26045234; PMCID: PMC4498999. (Prevalence section pages 2-3)