On 12/06/2021, US time, Acadia Pharmaceuticals (Nasdaq: ACAD) announced positive top-line results from its Phase III clinical trial of its drug candidate Trofinetide. The phase III study, called Lavender, will be used primarily to evaluate the safety and effectiveness of trofinetide in treating Rett Syndrome (RS). A total of 189 subjects were enrolled, all females aged 5 to 20 years RS.

Lavender was a 12-week, double-blind, randomized, placebo-controlled study with composite primary endpoints of the RS Behavioral Questionnaire (RSBQ) and the Clinical Outcome Global Rating Scale (CGI-I), assessed by nurses and physicians, respectively; The secondary endpoint is the Communication and Symbolic Behavior Development Scale for Infants and Toddlers (CSBS-DP-IT-Social), designed primarily to assess the development of social, verbal, and symbolic behaviors in infants and toddlers aged 6 to 24 years used is months old and can be used Autism Early Detection for symptoms of developmental delay and other developmental delays as assessed by the nursing staff.

The results showed that trofinetide had significant improvements in both primary endpoints compared to placebo. Changes from baseline in RSBQ for placebo and trofinetide at week 12 were -1.7 vs -5.1 (p=0.0175); CGI-I scores were 3.8 vs. 3.5 (p=0.0030). Meanwhile, the change from baseline in CSBS-DP-IT-Social was -1.1 and -0.1 for placebo and trofinetide, respectively.

Both Lavender’s primary and primary secondary endpoints demonstrated the potential of trofinetide for the treatment of RS, however, it should be noted that the rate of study discontinuations related to emerging adverse events (TEAEs) was higher in the trofinetide arm was than placebo , The two were 2.1% and 17.2%, respectively. Among them, the most common adverse events were:

① Diarrhea – trofinetide was 80.6% (of which 97.3% was mild to moderate) and placebo 19.1%;

② Vomiting – Trofinetide was 26.9% (of which 96% was mild…