Dear GP2C Community,
Here is a significant announcement about pharma test results that is promising. I’ve highlighted specific sections of significant for your review.
Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome
– Trofinetide met co-primary efficacy endpoints demonstrating statistically significant improvement over placebo in the Rett Syndrome Behaviour Questionnaire (RSBQ) (p=0.0175) and the Clinical Global Impression of Improvement (CGI-I) (p=0.0030)
– Trofinetide met key secondary endpoint demonstrating statistically significant improvement over placebo in CSBS-DP-IT–Social (p=0.0064), caregiver scale of ability to communicate
– Pre-New Drug Application meeting with the U.S. FDA planned for the first quarter 2022
– Conference call and webcast to be held today at 4:30 p.m. Eastern Time
SAN DIEGO–(BUSINESS WIRE)–Dec. 6, 2021– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced positive top-line results from the pivotal, Phase 3 Lavender™ study evaluating the efficacy and safety of trofinetide in 187 girls and young women aged 5-20 years with Rett syndrome. The 12-week placebo-controlled study demonstrated a 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 caregiver assessment of the core symptoms of Rett syndrome and the CGI-I is a global physician assessment of worsening or improving of Rett syndrome.
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 (CSBS-DP-IT–Social) change from baseline to week 12 was -0.1 vs. -1.1 (p=0.0064; effect size=0.43).
“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,” said Jeffrey L. Neul, M.D., Ph.D., 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. “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.”
Study treatment discontinuation rates related to treatment emergent adverse events (TEAEs) were 17.2% in the trofinetide group as compared to 2.1% in the placebo group. The most common adverse events were diarrhea (80.6% with trofinetide vs. 19.1% with placebo), of which 97.3% in the trofinetide arm were characterized as mild-to-moderate, and vomiting (26.9% with trofinetide vs. 9.6% with placebo), of which 96% in the trofinetide arm were characterized as mild-to-moderate. 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.