TL;DR
A phase IIb trial of inhaled EXO-CD24, an exosome-based immune modulator, found more patients free of respiratory failure at day 7 and shorter hospital stays than with placebo. Deaths were also less frequent in the treatment group at 28 days and six months, but the 90-patient findings are preliminary and need confirmation in a larger phase III trial.
In a 90-patient phase IIb trial, inhaled EXO-CD24 was associated with a higher proportion of patients free of respiratory failure at day 7 than placebo, according to results presented at the CHEST annual meeting. The study also reported fewer deaths in the treatment group, but the findings are preliminary and require testing in a larger trial before the treatment’s benefits are established.
By day 7, 85% of patients receiving EXO-CD24 were free of respiratory failure, compared with 33.3% in the placebo group (P<0.0001), Shiran Shapira, PhD, of Nano24 and Tel-Aviv Medical Center, reported. Average hospital stay was 6.5 days with the inhaled agent and 14.4 days with placebo (P=0.0001).
The report also gave mortality figures favoring EXO-CD24: 6.7% versus 30% through day 28, and 13.3% versus 43.3% at six months. For the six-month comparison, the reported hazard ratio was 0.28 (95% CI 0.084-0.902; P=0.033). These results come from a small phase IIb study; they do not establish that the drug reduces mortality across the broader ARDS population.
Participants were randomly assigned to aerosolized saline or 10¹⁰ particles of EXO-CD24 in 1.5 mL of saline, given through a nebulizer for five minutes twice daily from days 1 to 5, for up to 10 doses. Patients discharged from intensive care before day 5 could finish treatment at home. The study monitored safety through day 28, with a final safety check at six months.
Potential Gains in ARDS Recovery
The trial reported differences in the proportion of patients free of respiratory failure at day 7 and in average hospital stay between the EXO-CD24 and placebo groups. It also reported fewer deaths in the treatment group, although a phase IIb trial of this size cannot establish whether the treatment improves survival.
Alice Gallo De Moraes, a Mayo Clinic pulmonologist and critical care specialist who was not involved in the research, said it had been a while since a medication was found that could help ARDS. She described the safety and efficacy results as promising while qualifying her view: “If this would pan out, it will be very exciting.” Her comment reflects potential, not confirmation that the treatment works in routine care.
Further research would be needed to determine whether the results are confirmed in a larger trial.
inhaled immune modulator for respiratory support
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How EXO-CD24 Is Designed
EXO-CD24 uses exosomes, membrane-bound vesicles that naturally carry proteins, lipids and nucleic acids between cells. The investigational product is engineered to express the protein CD24. Shapira said CD24 helps distinguish self from foreign tissue, regulating inflammation associated with tissue damage while preserving pathogen recognition.
Shapira described the inhaled delivery as a way to bring the agent directly to affected tissue, limiting systemic exposure. She said the approach seeks to influence signaling involved in the production of cytokines and chemokines. Those are explanations of the proposed mechanism; the trial’s clinical results do not by themselves prove how the treatment works in patients.
The phase IIb study adds to earlier clinical experience with the product. The report said 90 patients in the phase IIb trial and 178 patients in prior phases had received the treatment, with no treatment-related or dose-related serious adverse events reported across that accumulated experience. That is an early safety record, not evidence that uncommon or longer-term harms have been ruled out.
““If this would pan out, it will be very exciting.””
— Alice Gallo De Moraes, MD, Mayo Clinic, who was not involved in the study
exosome-based therapy for respiratory failure
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Questions Before Phase III Evidence
The results have been reported from 90 participants, and the excerpt does not include details on the patients’ underlying causes of ARDS, baseline characteristics, or how those factors may have affected the group comparisons. The investigator described the findings as proof of concept, and a larger phase III trial is planned.
The report described safety monitoring through day 28 and a final safety check at six months. It also said no treatment-related or dose-related serious adverse events were reported in the accumulated clinical experience described.
The findings were presented at a medical meeting and described by the investigator as proof of concept. A larger international phase III trial is planned.
acute respiratory distress syndrome treatment devices
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Larger Trial Plans Ahead
Shapira said the research team plans to advance EXO-CD24 into a larger international phase III trial. That study will test whether the recovery and mortality differences seen in phase IIb are reproducible. The reported material does not specify further design details.
The company also plans to explore other indications for its exosome carrier, but those potential uses are separate from establishing the treatment’s effectiveness for ARDS.
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Key Questions
What is EXO-CD24?
EXO-CD24 is an investigational inhaled treatment that uses engineered exosomes expressing the immune-regulating protein CD24. It is being studied as a way to affect inflammation associated with ARDS.
What did the phase IIb trial find?
The trial reported that 85% of patients receiving EXO-CD24 were free of respiratory failure at day 7, compared with 33.3% receiving placebo. Average hospital stay and reported mortality were also lower in the treatment group, but the study was small and the results need confirmation.
Did the study establish that EXO-CD24 prevents death?
No. The study reported lower death rates in the treatment group at 28 days and six months, but a 90-patient phase IIb trial cannot establish a reliable survival benefit. A larger trial is needed to test that finding.
Was EXO-CD24 reported to cause serious side effects?
The report said there were no treatment-related or dose-related serious adverse events among the patients in the phase IIb trial and earlier study phases. This does not rule out uncommon or longer-term risks.
What happens next for the treatment?
The research team plans a larger international phase III trial. Its timing, design and enrollment details have not been provided in the reported material.
Source: rss
