The BioPharm Brief: Lung Injury Survival, Alzheimer Prevention, and Longer Dosing Intervals

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. We’re looking at survival data for a deadly lung condition, a regulatory boost for a vaccine designed to prevent Alzheimer disease, and an antibody that could mean fewer doses for people with inflammatory bowel disease.

First, Nano24’s inhaled EXO-CD24 has reduced mortality in a phase 2b trial in patients with acute respiratory distress syndrome, or ARDS. The therapy uses engineered exosomes, tiny cell-derived particles, to deliver an immune-regulating protein directly to the lungs. In the 90-patient study, 85% of patients receiving EXO-CD24 were free of respiratory failure at day 7, compared with 33.3% of patients on placebo. At 6 months, 13.3% of treated patients had died, compared with 43.3% in the placebo group. Hospital stays were also shorter. The results have not yet been peer reviewed, and Nano24 plans a larger international phase 3 trial.

Next, the FDA has granted fast track designation to Nuravax’s AV-1959R, an investigational vaccine designed to prevent Alzheimer disease. The vaccine is intended for people who have no symptoms but show biomarker evidence of amyloid buildup in the brain. In a small phase 1 study, all 12 people who received the vaccine developed antibodies against amyloid-beta. Nuravax plans to test the vaccine in a 160-person phase 2 trial that will track safety, immune response, and changes in amyloid and tau. No preventive therapy is currently approved for this population.

And Bionyra Pharma’s long-acting antibody BYN-002 has shown an extended half-life in an early-stage study. The antibody targets T-L-1-A, a protein that drives inflammation in inflammatory bowel disease. In interim phase 1 results from healthy volunteers, the antibody’s half-life reached roughly 78 to 82 days in the repeat-dosing groups, and its target remained suppressed at every dose tested. No treatment-related serious side effects were reported. Bionyra says the profile could support maintenance dosing every 3 to 6 months, which could ease the treatment burden for patients.

That’s today’s BioPharm Brief. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways

• ARDS survival: Inhaled EXO-CD24 lowered mortality and kept more patients free of respiratory failure in a phase 2b trial.
• Alzheimer prevention: AV-1959R earned FDA fast track designation ahead of a 160-person phase 2 prevention study.
• Longer dosing intervals: BYN-002’s roughly 80-day half-life could support maintenance dosing every 3 to 6 months in IBD.