NA-921 shows potential for Rett, with fewer side effects than Daybue
NA-921 less likely to cause gastrointestinal side effects, Phase 2/3 trial shows
Treatment with NA-921 was found to improve measures of abnormal behavior and overall health in girls and young women with Rett syndrome in a Phase 2/3 clinical trial.
Data also suggested the oral therapy is less likely to cause digestive side effects than Daybue (trofinetide), which is marketed by Acadia Pharmaceuticals and currently the only approved treatment for Rett.
The results “highlight NA-921’s potential as a breakthrough treatment with fewer side effects and improved patient retention rates,” stated Biomed Industries, the company that is developing NA-921, in a company press release.
Phase 3 trial currently recruiting girls, young women in US, Australia
Biomed is currently recruiting girls and young women with Rett to participate in a Phase 3 trial of NA-921 (NCT06840496), which will explore NA-921’s efficacy versus a placebo. That trial will take place at multiple locations in the U.S. and Australia.
Rett syndrome is a genetic disorder marked by abnormalities in development, behavior, and communication. NA-921, also known as bionetide, is able to cross into the brain and promote the growth and survival of nerve cells, according to Biomed’s website.
Biomed’s Phase 2/3 clinical trial (NCT06849973) tested NA-921 against a placebo in more than 180 girls and young women, ages 5 to 20, with Rett syndrome. Patients were given NA-921 or a placebo twice daily, with dosage based on weight, for 12 weeks.
The study’s main goals were to assess the impact of treatment on the Rett Syndrome Behavior Questionnaire (RSBQ) — a standardized measure of Rett behavioral abnormalities that’s completed by caregivers — and on the Clinical Global Impression-Improvement (CGI-I) score, which is a clinician-associated measure of overall health. Of note, these two measures will also be primary endpoints in the upcoming Phase 3 trial, which will further assess impact on communication.
Results showed average RSBQ scores improved by 5.5 points for patients given NA-921. By contrast, for patients given the placebo, scores improved by less than two points on average.
For the CGI-I, where lower scores reflect better outcomes, the average score at week 12 was 3.6 points for patients given NA-921 and 3.83 points for those given the placebo.
Fewer side effects reported than for Daybue
One of the main drawbacks of Daybue is that it commonly causes digestive side effects like diarrhea and vomiting. Exploratory analyses were conducted to compare rates of these side effects in patients given NA-921 during this study against rates that were seen in clinical trial data of Daybue. Results showed fewer patients given NA-921 experienced diarrhea (14% vs. 82%) or vomiting (8% vs. 29%).
Also, more than 1 in 3 patients given Daybue in a clinical trial discontinued treatment due to safety issues. By contrast, no one in the NA-921 trial discontinued due to side effects.
“Our latest clinical data reinforces NA-921’s potential to provide a more effective and well-tolerated treatment option,” said Lloyd L. Tran, PhD, chairman and CEO of Biomed. “Unlike existing therapies, which are often associated with severe gastrointestinal side effects and high discontinuation rates, NA-921 has demonstrated a remarkably low incidence of adverse events. With a favorable safety profile, NA-921 represents a promising treatment that could enhance patient adherence and long-term outcomes.”
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