Dompé Initiates Phase 2 Trial of Intranasal NGF for Spastic Cerebral Palsy in Italy
核心洞察
Dompé (搜索) has enrolled the first patient in a Phase 2 clinical trial evaluating intranasal nerve growth factor (NGF) for treating spastic cerebral palsy (搜索) at Agostino Gemelli University Hospital in Rome.
The study will enroll 60 children aged 2-6 years with spastic cerebral palsy (搜索) across 10 Italian centers, marking the first potential disease-modifying therapy for the underlying brain damage in CP.
Approximately 764,000 people in the U.S. and 712,000 in the EU live with cerebral palsy (搜索), with no current approved therapies addressing the root cause of brain damage.
Dompé (搜索), an Italian biopharmaceutical company, has announced the enrollment of the first patient in a Phase 2 clinical trial investigating intranasal nerve growth factor (NGF) for the treatment of spastic cerebral palsy (搜索). The milestone was achieved at the Agostino Gemelli IRCCS University Hospital (搜索) in Rome, marking a significant step toward developing the first disease-modifying therapy for this devastating childhood condition.
Addressing an Unmet Medical Need
Cerebral palsy (搜索) represents the most common motor disability in childhood, affecting approximately 764,000 people in the United States and an estimated 712,000 people in the European Union. In Italy alone, approximately 740 children with cerebral palsy are born each year. The condition refers to a group of permanent disorders in the development of movement and posture, attributed to non-progressive disturbances that occurred in the developing fetal or infant brain.
"Cerebral palsy (搜索) 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 (搜索). "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."
Currently, no approved therapies exist to treat the underlying brain damage of cerebral palsy (搜索). Available treatments primarily focus on symptom relief and include physical, occupational, and speech therapy to strengthen muscles, improve coordination, and support daily activities.
Novel Therapeutic Approach
The Phase 2 investigational study will evaluate Cenegermin-bkbj, an intranasal formulation of NGF, in children with spastic cerebral palsy (搜索). Beyond safety and tolerability, the trial will assess improvements in daily functioning, quality of life, motor abilities, and neurodevelopmental measures.
Nerve growth factor is a naturally-occurring protein that supports damaged nerve cells and improves nervous system signaling. The therapeutic rationale is based on NGF's natural role in supporting the growth, survival, and repair of developing nerve cells.
"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," explained Ahmed Enayetallah, Chief Development Officer at Dompé (搜索). "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."
Study Design and Patient Population
The Phase 2 trial will enroll a total of 60 children with spastic cerebral palsy (搜索) across 10 different centers throughout Italy. Eligible participants are aged two to six years old with any severity level (I–V) on the Gross Motor Function Classification System – Expanded and Revised (GMFCS-E&R).
The study was initiated at the Agostino Gemelli IRCCS University Hospital (搜索) Foundation and will soon be activated at the IRCCS Bambino Gesù Pediatric Hospital (搜索). The multi-center approach will provide a comprehensive evaluation of the therapy's potential across diverse patient populations.
Building on Nobel Prize-Winning Science
Dompé (搜索)'s intranasal NGF platform builds on the Nobel Prize-winning discovery of Italian neurobiologist Professor Rita Levi-Montalcini and American biochemist Dr. Stanley Cohen, who earned the Nobel Prize in Physiology or Medicine in 1986 for their groundbreaking research on nerve growth factor.
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 (搜索). Dompé (搜索) has positioned itself as the first company to unlock the therapeutic potential of NGF, having previously developed the first FDA-approved NGF treatment.
The company's clinical pipeline reflects its commitment to redefining treatments across ophthalmic, neurological, and pain-related conditions, with NGF serving as the cornerstone of its therapeutic approach to address previously untreatable neurological conditions.