Acadia Pharmaceuticals announced that its experimental drug being developed for a rare neurological condition met both goals of a late-stage clinical trial, edging the San Diego-based biotech closer to an FDA approval.
The company shared positive top-line results from the pivotal, phase 3 Lavender study evaluating the efficacy and safety of experimental treatment trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome. Rett syndrome is a rare, debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life. Acadia's drug 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. Trofinetide has been granted FDA Fast Track Status and Orphan Drug Designation for Rett syndrome in addition to being given Rare Pediatric Disease designation by the FDA.
The 12-week study demonstrated a statistically significant improvement over placebo for both co-primary endpoints. Improvements were seen in the caregiver assessment (RSBQ) of the core symptoms of Rett syndrome as well as the global physician assessment (CGI-I) of worsening or improving of Rett syndrome. Patients reported improvements in core symptoms, like being able to respond to a choice when asked by parents, or experiencing more freedom from repetitive hand movements.
Additionally, trofinetide demonstrated a statistically significant separation over placebo on the key secondary endpoint, the Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist–Social composite score.
Acadia is preparing for a pre-NDA meeting with the FDA in the first quarter of 2022 and plans to submit a New Drug Application around mid-year 2022.
