Nano24’s inhaled exosome therapy reduced respiratory failure in patients with acute respiratory distress syndrome (ARDS), hitting the main goal of a phase 2b study.
The privately owned Israeli biotech evaluated the therapy, dubbed EXO-CD24, against placebo in the mid-stage trial of 90 patients with ARDS. Patients in the treatment cohort received EXO-CD24 at 1010 particles per dose, twice daily for five days.
At Day 7, 85% of patients who received EXO-CD24 had avoided respiratory failure compared to 33.3% in the placebo arm, hitting the study’s primary endpoint.
Meanwhile, the proportion of patients who died of any cause over 28 days was 6.7% in the EXO-CD24 cohort compared to 30% in the placebo group, Nano24 noted in an Oct. 20 release. This benefit extended to a six-month follow-up, when 13.3% of the treatment cohort had died compared to 43.3% of the placebo cohort.
The mean hospital stay for patients receiving EXO-CD24 was 6.5 days out of 28 days, compared to 14.4 for those on placebo. The biotech suggested that this showed the treatment has “potential to reduce the burden on patients, intensive care units and healthcare systems.”
When it came to safety, EXO-CD24 was well tolerated, with no treatment-related serious adverse events, and fewer serious and non-serious adverse events in the EXO-CD24 arm than in the placebo arm.
“ARDS remains a serious clinical challenge, and patients need additional treatment options,” said Matthew Baldwin, M.D., a pulmonary and critical care physician-scientist, in Nano24’s release. “The findings on the proportion of patients free of respiratory failure at Day 7 and on all-cause mortality are encouraging in this deadly condition.”
EXO-CD24 combines engineered exosomes—tiny, membrane-bound vesicles that act as biological messengers between cells—with the immunomodulatory protein CD24. The inhaled therapy can be delivered directly to the lungs, with the aim of controlling harmful, damage-driven inflammation while preserving the immune system’s ability to recognize and eliminate pathogens.
The plan now is to take EXO-CD24 into a larger, global phase 3 study, Nano24 explained.
ARDS is a life-threatening condition that involves severe lung inflammation and impaired oxygenation, which can lead to respiratory and multi-organ failure. Although scientists’ understanding of ARDS has increased over the years, there are currently no treatments approved specifically for the indication. Instead, clinical management relies on supportive care such as mechanical ventilation, while attempting to treat the underlying triggers, which can include sepsis or pneumonia.
So far, cell-based therapies have proven to be one promising area of treatment R&D. There is also potential to explore specific molecular pathways implicated in ARDS pathogenesis—although this often comes with a risk of off-target effects.
Edesa Biotech’s anti-TLR4 antibody showed promise in a phase 3 study of COVID-linked ARDS last year. Edesa’s drug was selected for inclusion in the Biomedical Advanced Research and Development Authority’s (BARDA) platform trial of potential ARDS treatments in 2024.
Founded in 2020 to take forward research from Tel Aviv Sourasky Medical Center, Nano24 is focused on harnessing exosomes to develop targeted therapies for serious diseases with significant unmet need. The biotech’s scientific board is headed up by Moderna co-founder Robert Langer, and the ARDS program marks the first clinical proof-of-concept for the company’s engineered exosome platform. Nano24 is also exploring EXO-CD24’s potential to treat other inflammatory conditions.
“Exosomes offer a promising foundation for drug delivery because their surfaces and internal cargo can be engineered to perform complementary functions,” Langer explained in this morning’s release.