Acadia Pharmaceuticals achieve positive Phase III results in Rett Syndrome trial

pharmafile | December 7, 2021 | News story | Manufacturing and Production  

Acadia Pharmaceuticals have announced positive top-line results from the pivotal, Phase III Lavender study, which evaluated the efficacy and safety of trofinetide in 187 girls and young women aged five to 20 years with Rett syndrome. The 12-week placebo-controlled study demonstrated statistically significant improvement over placebo, for both co-primary endpoints.

On the Rett Syndrome Behaviour Questionnaire (RSBQ), change from baseline to week 12 was -5.1 vs -1.7 (p=0.0175; effect size=0.37). The Clinical Global Impression-Improvement (CGI-I) score at week 12 was 3.5 vs 3.8 (p=0.0030; effect size=0.47). The RSBQ is a caregier assessment of the main symptoms associated with Rett syndrome, and the CGI-I is a global physician assessment of the worsening or improving of the syndrome.

In the study, trofinetide also demonstrated a statistically significant separation over placebo on the key secondary endpoint, the Communication and Symbolic Behaviour Scales Developmental Profile™ Infant-Toddler Checklist-Social composite score (CSBS-DP-IT-Social) change from baseline to week 12 was -0.1 vs -1.1 (p=0.0064; effect size=0.43).

Rett syndrome is a rare genetic disorder, affecting brain development and causing severe muscle movement disability. It affects approximately 1 in 12,000 girls born every year, and is only rarely seen in boys.

Jeffrey L Neul, MD, PhD, Annette Schaffer Eskind Chair and Director, Vanderbilt Kennedy Center; Professor of Pediatrics, Division of Neurology, Pharmacology, and Special Education, Vanderbilt University Medical Center and Lavender study investigator, commented: “These are encouraging results for patients and families affected by Rett syndrome. Patients reported improvements in core symptoms, like being able to respond to a choice when asked by their parents, or experiencing more freedom from the repetitive hand movements that create obstacles in other areas of their lives.

“The positive Lavender study results support a potential treatment for Rett syndrome and represent an important step forward in addressing this rare and serious neurological disease.”

Lina Adams

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