Biomed Industries Presents Promising Phase 2/3 Clinical Results of NA-921 for Rett Syndrome at Orphan Drug Summit 2026 Advancing Named Patient Access Initiative

SAN JOSE, CA, UNITED STATES, July 16, 2026 /EINPresswire.com/ -- Biomed Industries, Inc., a clinical-stage biopharmaceutical company, announced that its Chief Executive Officer, Dr. Lloyd L. Tran, presented encouraging clinical findings from the company's investigational therapy NA-921 (Bionetide™) during the Orphan Drug Summit, held July 13-14, 2026, in Boston, Massachusetts.

The presentation, titled "Reimagining Rett Syndrome Treatment – Phase 2/3 Clinical Results of NA-921 Demonstrating Efficacy and Favorable Tolerability," highlighted positive efficacy and safety results from the company's randomized, double-blind, placebo-controlled Phase 2/3 clinical trial evaluating NA-921 in girls and young women with Rett syndrome.

Rett syndrome is a rare, severe neurodevelopmental disorder caused primarily by mutations in the MECP2 gene. Affecting approximately one in every 10,000 live female births, the disease results in progressive loss of motor skills, communication abilities, purposeful hand movements, and cognitive function, while many patients also experience seizures, breathing abnormalities, scoliosis, and cardiac complications. Despite its devastating impact on patients and families, treatment options remain extremely limited.

During his presentation, Dr. Tran reviewed the scientific rationale behind NA-921, an orally administered investigational therapy designed to improve neurological function while offering a favorable safety profile.

POSITIVE PHASE 2/3 CLINICAL RESULTS
The Phase 2/3 study enrolled 173 girls and women with Rett syndrome, randomized to receive either NA-921 or placebo over a 12-week treatment period. (ClinicalTrials.gov ID NCT06849973)

The topline results from the study demonstrate compelling proof of safety and efficacy for NA-921, an orally administered small molecule. Biomed has conducted a comparative analysis of adverse reactions observed in the clinical trials, showing a significantly improved safety and tolerability profile for NA-921.

Rett Syndrome Behavior Questionnaire (RSBQ): The least squares mean (LSM) change from baseline to week 12 was -5.5 for NA-921 versus -1.6 for placebo (p = 0.001; n = 86 for NA-921, n = 87 for placebo).
Clinical Global Impression–Improvement (CGI-I): At week 12, the score was 3.60 for NA-921 versus 3.83 for placebo (p = 0.0020; effect size = 0.42; n = 86 for NA-921, n = 87 for placebo).

FAVORABLE SAFETY AND TOLERABILITY
An important focus of the presentation was the safety profile of NA-921.
Comparison with Current Treatments: While FDA-approved DAYBUE™ have shown clinical efficacy, they come with significant safety concerns, including high rates of severe diarrhea, vomiting, and weight loss.(Treatment Management Guide for Healthcare Professionals by Acadia Pharmaceutical, Inc. (https://www.daybuehcp.com/treatment-management-guide.pdf).

A comparison of clinical trial data between NA-921 and DAYBUE revealed the following:
NA-921 exhibited markedly lower incidences of common side effects, including diarrhea (82% for DAYBUE vs 24% for NA-921) and vomiting (29% for DAYBUE vs 11% for NA-921).
Unlike the DAYBUE trial, where 35.7% of patients discontinued treatment due to adverse events, no patients withdrew from the NA-921 trial.

According to the clinical data presented, NA-921 demonstrated favorable tolerability while improving abnormal behaviors and overall health measures in girls and young women with Rett syndrome. These findings highlight NA-921’s potential as a breakthrough treatment with fewer side effects and improved patient retention rates.

PHASE 3 PROGRAM UNDERWAY
Biomed Industries also announced that the company is advancing its global Phase 3 randomized, double-blind, placebo-controlled clinical trial of NA-921 (ClinicalTrials.gov Identifier: NCT06840496).

The Phase 3 study is designed to enroll approximately 210 patients and will further evaluate the efficacy and safety of NA-921 in girls and women with Rett syndrome using the same clinically meaningful endpoints established during earlier studies.

"Our goal is to bring a safe and effective treatment to families affected by Rett syndrome as rapidly as possible," said Dr. Lloyd L. Tran, CEO of Biomed Industries. "The Phase 2/3 data reinforce our confidence in NA-921 and provide a strong foundation for the ongoing Phase 3 program."

EXPANDING GLOBAL ACCESS THROUGH NAMED PATIENT PROGRAMS
Recognizing the significant unmet medical need for Rett syndrome worldwide, Biomed Industries announced that it is establishing an international network of pharmaceutical distributors and commercialization partners throughout the European Union, Japan, China, Russia, Turkey, Brazil, Argentina, and additional international markets.

Subject to applicable local regulations and regulatory approvals, the company intends to make NA-921 available through Named Patient and Compassionate Use programs where permitted, providing physicians with potential access to the investigational therapy for eligible patients who have serious unmet medical needs while the product continues through regulatory development.

Biomed estimates that more than 50,000 children worldwide are living with Rett syndrome and may ultimately benefit from expanded access initiatives if permitted under applicable regulatory frameworks.

ABOUT NA-921 (BIONETIDE™)
NA-921 (Bionetide™) is small molecule drug, and an investigational oral therapy being developed for the treatment of Rett syndrome. Clinical studies have demonstrated encouraging improvements in behavioral symptoms and overall clinical status while maintaining a favorable tolerability profile.

ABOUT BIOMED INDUSTRIES, INC.
Biomed Industries, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing transformative therapies for chronic and complex diseases. The company's investigational pipeline addresses a broad range of unmet medical needs, including:
• Alzheimer's disease
• Major depressive disorder (MDD)
• Obesity and diabetes
• Metabolic dysfunction-associated steatohepatitis (MASH)
• Stroke and alcohol use disorder
• Rare diseases, including Rett syndrome and Fragile X syndrome
For more information, visit www.biomedind.com.

Michael Willis
Biomed Industries, Inc.
+1 800-824-5135
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