Dompé Announces First Patient Enrolled in New Study of Intranasal NGF in Cerebral Palsy

Dompé, a leading biopharmaceutical company with operations in Italy and in the U.S., announced that the first patient has been enrolled at the Agostino Gemelli IRCCS University Hospital in Rome, Italy, in a Phase 2 investigational study of an intranasal form of Nerve Growth Factor (NGF) (Cenegermin-bkbj) for the treatment of spastic cerebral palsy (CP). On top of safety and tolerability, the study will evaluate improvements in daily functioning, quality of life, motor abilities, and neurodevelopmental measures of the Cenegermin-bkbj intranasal formulation. CP is the most common motor disability in childhood, caused by injury or changes during early brain development. The most prevalent form, spastic CP, leads to muscle stiffness and difficulty with movement.²

“Cerebral palsy is a devastating disease, and for patients and families there are still no proven treatments beyond supportive care,” said Marcello Allegretti, Chief Scientific Officer at Dompé. “With our emerging intranasal NGF platform, we hope to deliver a treatment that goes beyond managing symptoms and changes the course of disease, opening the door to a better future for patients and their families.”

Approximately 764,000 people in the U.S. and an estimated 712,000 people in the EU are living with CP^3,4. There are currently no approved therapies that treat the underlying brain damage of CP. Treatment often includes symptom relief and physical, occupational, and speech therapy, which can strengthen muscles, improve coordination, and support daily activities.

“The cause of CP-related disability is directly connected to NGF’s natural role in the body – supporting the growth, survival, and repair of developing nerve cells^5,6,” said Ahmed Enayetallah, Chief Development Officer at Dompé. “With intranasal NGF, Dompé aims to address CP at its source, fundamentally reshape the disease’s trajectory and improve the life-long outlook for patients.”

The Phase 2 investigational study was initiated by Dompé at the Agostino Gemelli IRCCS University Hospital Foundation and will soon be activated at the IRCCS Bambino Gesù Pediatric Hospital. The study will enroll a total of 60 children with spastic CP in 10 different centers across Italy, aged two to six years old with any severity of any level (I–V) on the Gross Motor Function Classification System – Expanded and Revised (GMFCS-E&R).

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 950 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 Cerebral Palsy (CP)

CP refers to a group of permanent disorders in the development of movement and posture, which causes activity limitations, and is attributed to non-progressive disturbances that occurred in the developing fetal or infant brain. CP has multiple subtypes which are characterized by a range of symptoms, including but not limited to: hypertonicity, hyperreflexia, involuntary uncontrolled, repetitive movements, altered muscle tone, abnormal posture. The severity of CP presentation varies depending on the movement disorder, area affected, and extent of damage. Various factors can disrupt normal brain development and lead to CP, including infections, encephalopathy, trauma, or genetic contributions. Disruption at critical developmental stages of brain plasticity, such as in utero, during childbirth, or during infancy can lead to CP.

References:
^[1] Rosenbaum P, et al. Dev Med Child Neurol Suppl. 2007;109:8-14. https://pubmed.ncbi.nlm.nih.gov/17371509/
^[2]Salomon, I. Brain and Behavior. 2025;14(10)https://onlinelibrary.wiley.com/doi/10.1002/brb3.70065
^[3] Cerebral Palsy Guidance. Cerebral Palsy Facts and Statistics. Available at: https://cerebralpalsyguidance.com/cerebral-palsy/research/facts-and-statistics
^[4] McIntyre S; Global CP Prevalence Group*. Global prevalence of cerebral palsy: A systematic analysis. Dev Med Child Neurol. 2022 Dec;64(12):1494-1506. doi: 10.1111/dmcn.15346. Epub 2022 Aug 11. PMID: 35952356; PMCID: PMC9804547 https://pubmed.ncbi.nlm.nih.gov/35952356/
^[5] Chen X, et al. Adv Clin Exp Med. 2025;34(6):911-923. Available here.
^[6] Di Sarno L, et al. Pharmaceuticals. 2025; 18(6):929. Available here.

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