Acadia Pharmaceuticals and Neuren Pharmaceuticals announced that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications.
Trofinetide is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical for both normal development and for response to injury and disease. Trofinetide has been granted U.S. FDA Fast Track Status and Orphan Drug Designation in the U.S. and Europe for both Rett syndrome and Fragile X syndrome.
Acadia plans to initiate a Phase 3 randomized, double-blind placebo-controlled study evaluating trofinetide in the second half of 2019 following completion of additional manufacturing activities. This study will evaluate trofinetide and placebo in approximately 180 girls with Rett syndrome and will measure the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression of Improvement (CGI-I), a physician assessment, as co-primary efficacy endpoints.
“A potential treatment for Rett syndrome is a perfect fit with Acadia’s mission to develop novel therapies to improve the lives of patients with central nervous system disorders,” said Serge Stankovic, MD, Executive Vice President, Head of Research and Development at Acadia. “Today there are no approved treatments for the girls and women suffering from Rett syndrome. We look forward to initiating a Phase 3 study in the second half of 2019 to further explore the potential benefits of trofinetide for patients and their caregivers.”
Neuren conducted a Phase 2 double-blind placebo-controlled dose ranging study in girls aged 5 to 15 years with Rett syndrome, in which statistically significant and clinically meaningful improvement was demonstrated on the RSBQ and the CGI-I. This followed positive trends observed in an earlier Phase 2 trial in adolescents and adults aged 16 to 45 years with Rett syndrome3. In addition, Neuren has completed an exploratory study in Fragile X syndrome.
Dr. Daniel Glaze, Medical Director at the Blue Bird Circle Rett Center, Texas Children’s Hospital commented, “The trofinetide Phase 2 results in Rett syndrome are very promising in terms of both safety and clinical benefit. For many families, these improvements would provide meaningful improvement in their child’s quality of life.”
About Rett Syndrome
Rett syndrome is a debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Rett syndrome has been most often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Rett syndrome is caused by mutations on the X chromosome on a gene called MeCP2. There are more than 200 different mutations found on the MeCP2 gene that interfere with its ability to generate a normal gene product. Rett syndrome occurs worldwide in approximately one of every 10,000 to 15,000 female births causing problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. Typically, between six to eighteen months of age, patients experience a period of rapid decline with loss of purposeful hand use and spoken communication and inability to independently conduct activities of daily living. Symptoms also include seizures, disorganized breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis) and sleep disturbances. Currently, there are no approved medicines approved for the treatment of Rett syndrome.