Biomed reports encouraging NA-921 Rett syndrome data and advances Phase 3 study

Biomed Industries says its investigational Rett syndrome therapy NA-921 posted positive Phase 2/3 results and favorable tolerability at the Orphan Drug Summit in Boston. The company is now moving into a global Phase 3 trial and building named-patient access plans in multiple markets.

Why it matters: - Rett syndrome has few treatment options, and Biomed Industries is trying to position NA-921 as a therapy that can improve symptoms without the safety burden seen with current care options. - If the Phase 3 program confirms the earlier data, NA-921 could expand treatment choices for girls and young women living with a rare, severe neurodevelopmental disorder. - Biomed also wants to widen access through named-patient and compassionate-use programs, which could matter for families in countries where the drug is not yet approved.

What happened: - Biomed Industries CEO Dr. Lloyd L. Tran presented Phase 2/3 clinical results for NA-921, also called Bionetide™, at the Orphan Drug Summit on July 13-14, 2026, in Boston. - The presentation covered a randomized, double-blind, placebo-controlled Phase 2/3 trial in girls and young women with Rett syndrome. - Biomed announced it is advancing a global Phase 3 randomized, double-blind, placebo-controlled trial of NA-921.

The details: - The Phase 2/3 study enrolled 173 girls and women and treated them for 12 weeks. ClinicalTrials.gov lists the study as NCT06849973. - On the Rett Syndrome Behavior Questionnaire, the least squares mean change from baseline to week 12 was -5.5 for NA-921 and -1.6 for placebo, with p = 0.001. - On the Clinical Global Impression–Improvement scale, the week 12 score was 3.60 for NA-921 and 3.83 for placebo, with p = 0.0020 and effect size = 0.42. - Biomed said the data showed a significantly improved safety and tolerability profile for NA-921. - The company said NA-921 had lower rates of diarrhea at 24% versus 82% for DAYBUE and vomiting at 11% versus 29% for DAYBUE. - No patients withdrew from the NA-921 trial because of adverse events. - In the DAYBUE treatment-management guide, severe diarrhea, vomiting and weight loss are listed as important safety concerns. Treatment Management Guide for Healthcare Professionals - The planned Phase 3 study is expected to enroll about 210 patients and use the same clinically meaningful endpoints as earlier studies.

Between the lines: - Biomed is framing NA-921 as both an efficacy story and a tolerability story, which is important in Rett syndrome where long-term treatment burden can drive discontinuation. - The comparison with DAYBUE suggests Biomed wants to differentiate NA-921 not just on symptom improvement but on day-to-day usability for patients and families. - The push into distributors and commercialization partners across Europe, Japan, China, Russia, Turkey, Brazil, Argentina and other markets signals a parallel strategy to prepare for access if development continues to move forward. - Biomed estimates more than 50,000 children worldwide are living with Rett syndrome, which underscores the size of the unmet need the company is targeting.

What's next: - Biomed said the Phase 3 trial will continue to evaluate NA-921’s efficacy and safety in girls and women with Rett syndrome. - The company plans to establish an international network of pharmaceutical distributors and commercialization partners. - Subject to local regulations and regulatory approvals, Biomed intends to make NA-921 available through Named Patient and Compassionate Use programs where permitted. - Dr. Tran said the Phase 2/3 data provide a strong foundation for the ongoing Phase 3 program.

The bottom line: - Biomed is moving NA-921 deeper into development with early data that suggest symptom benefit and fewer side effects, while preparing a broader access strategy if regulators and future trial results support the drug.