Kathie Bishop, PhD, Chief Scientific Officer at Acadia Pharmaceuticals, discusses the positive top-line results from the pivotal phase 3 Lavender trial testing the efficacy of trofinetide to treat Rett syndrome.
Rett syndrome is a rare progressive neurodevelopmental condition that primarily affects girls. These girls appear to have normal psychomotor development during the first 6 to 18 months of life, followed by a developmental “plateau,” and then rapid regression in language and motor skills. Common symptoms include hand-wringing; fits of screaming and inconsolable crying; autistic features; panic-like attacks; bruxism; episodic apnea and/or hyperpnea; gait ataxia and apraxia; tremors; seizures; and slowed head growth. Rett syndrome is most commonly caused by a sporadic mutation in the MECP2 gene on the X chromosome. The majority of cases are not inherited from a parent.
Positive top-line results from the Lavender study were recently announced. As Dr. Bishop explains, the Lavender study (NCT04181723) was a phase 3 study evaluating the safety and efficacy of trofinetide in 187 Rett syndrome patients aged 5 to 20 years. Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression of Improvement (CGI-I). The RSBQ is a caregiver assessment of the core symptoms of Rett syndrome and the CGI-I is a global physician assessment of worsening or improving Rett syndrome. The 12-week study observed the RSBQ scores changed -5.1 in the trofinetide arm vs. -1.7 in the placebo arm (P = .0175). The CGI-I scores were 3.5 vs. 3.8 in the trofinetide arm and the placebo arm, respectively (P = .0030).
A New Drug Admission (NDA) is expected to be submitted to the U.S. Food and Drug Administration later in 2022.
For more information about Rett syndrome and other rare neurological disorders, visit checkrare.com/diseases/neurology