Serious adverse events were observed in 3.2% of study participants in both the trofinetide and placebo groups. Patients completing the Lavender study had the opportunity to continue to receive trofinetide in the open-label Lilac and Lilac-2 extension studies. More than 95% of participants who completed the Lavender study elected to roll-over to the Lilac open-label extension study.
The results from this study will be submitted for presentation at upcoming medical meetings.
“The consistent efficacy across primary and key secondary endpoints in the Lavender study demonstrates the potential of trofinetide to treat Rett syndrome,” said Kathie Bishop, Ph.D., Acadia’s Senior Vice President, Chief Scientific Officer and Head of Rare Disease. “We want to thank the patients, their caregivers, study site personnel, physicians and everyone who participated in the Lavender study for their contribution to making this milestone a reality. We look forward to continuing this important work and potentially delivering an FDA-approved treatment for this rare and devastating disease.”
Acadia is preparing for a pre-NDA meeting with the U.S. Food and Drug Administration (FDA) in the first quarter of 2022 and plans to submit a New Drug Application (NDA) around mid-year 2022. Trofinetide has been granted Fast Track Status and Orphan Drug Designation for Rett syndrome. Trofinetide has also been granted Rare Pediatric Disease (RPD) designation by the FDA. An NDA with Orphan Drug Designation is eligible for priority review. With an RPD NDA we would expect to be awarded a Priority Review Voucher if approved, subject to final determination by the FDA.
In 2018, Acadia entered into an exclusive license agreement with Neuren Pharmaceuticals Limited (ASX: NEU) for the development and commercialization of trofinetide for Rett syndrome and other indications in North America.
Conference Call and Webcast Information
Acadia will discuss top-line results from its Lavender study of trofinetide for the treatment of Rett syndrome via conference call and webcast today at 4:30 p.m. Eastern Time. The conference call can be accessed by dialing 855-638-4820 for participants in the U.S. or Canada and 443-877-4067 for international callers (reference passcode 7989366). A telephone replay of the conference call may be accessed through December 20, 2021 by dialing 855-859-2056 for callers in the U.S. or Canada and 404-537-3406 for international callers (reference passcode 7989366). The conference call will also be webcast live on Acadia’s website, www.acadia-pharm.com, in the investors section and archived until January 3, 2022.
About Lavender™
The Lavender study was a Phase 3, 12-week, double-blind, randomized, placebo-controlled study of trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome, designed to evaluate its efficacy and safety. The co-primary endpoints of Lavender included both a caregiver (Rett Syndrome Behaviour Questionnaire [RSBQ]) and physician (Clinical Global Impression–Improvement [CGI-I]) assessment. The key secondary endpoint was also a caregiver assessment designed to evaluate communication skills, the Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist – Social Composite Score (CSBS-DP- IT–Social).
About 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. Rett syndrome is 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.
