ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in Rett Syndrome, a Rare Neurodevelopmental Congenital CNS Disorder
“There is no approved treatment for Rett syndrome, which is a rare neurological disease that impacts nearly every aspect of a child’s life, resulting in loss of speech, difficulty breathing, lack of motor control, loss of muscle tone and mobility, seizures, and more,” said
The LAVENDER Phase 3 study is a 12-week, double-blind, randomized, placebo-controlled study evaluating the efficacy and safety of trofinetide in approximately 180 girls and young women 5 to 20 years of age with Rett syndrome. Half of study participants will receive trofinetide and half will receive placebo. Co-primary efficacy endpoints of the study will measure symptom improvement using the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression Scale-Improvement (CGI-I), a clinician assessment.
“For patients living with this debilitating disease, and the families whose dedication to their care inspires us, the LAVENDER study is an important next step in what we hope will result in the first
The LAVENDER study will be followed by LILAC, a nine-month extension study in which all participants, including those on placebo in the LAVENDER study, will be eligible to receive trofinetide. All LILAC participants will be followed to evaluate long-term tolerability, safety, and effectiveness of trofinetide. A second extension study, LILAC-2, will follow in which eligible patients who complete the LILAC trial will continue to receive trofinetide.
“The start of the trofinetide study has been highly anticipated by the Rett community, which currently has no approved treatment for Rett syndrome,” said
In 2018, ACADIA entered into an exclusive North American license agreement with Neuren for the development and commercialization of trofinetide for Rett syndrome and other indications. In an end-of-Phase 2 meeting, the
More information about the LAVENDER study is available at www.rettsyndromestudies.com.
Trofinetide is an investigational drug. It is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by potentially 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 Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Drug Designation in
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 18 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
ACADIA is a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in central nervous system disorders. ACADIA has developed and commercialized a treatment for hallucinations and delusions associated with Parkinson's disease psychosis. In addition, ACADIA has ongoing clinical development efforts in additional areas with significant unmet need, including dementia-related psychosis, schizophrenia-negative symptoms, major depressive disorder, and Rett syndrome. This press release and further information about ACADIA can be found at www.acadia-pharm.com.
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the timing of the commencement of the Phase 3 clinical trial evaluating trofinetide; the likelihood of success of such clinical trial; the prospects for
ACADIA Pharmaceuticals Inc.
Mark Johnson, CFA
ACADIA Pharmaceuticals Inc.