
The European Medicines Agency (EMA) recently approved the EVENEW clinical trial. EVENEW is an adaptive, seamless phase 1/2 trial that will evaluate the safety and efficacy of intratracheal administration of EXOB-001 in preventing severe cases of bronchopulmonary dysplasia (BPD) in preterm newborns.
Belgian biotech company EXO Biologics pioneered the study, which is the first mesenchymal stromal cell (MSC)-derived extracellular vesicles (EVs) trial the EMA has approved. EXO Biologics develops biopharmaceuticals that use EVs to treat rare diseases with high unmet medical needs, such as BPD, which is the most common and adverse result of infants who are born prematurely (<28 gestational weeks). BPD is a chronic lung disease that often leads to severe symptoms and high medical costs.
The EMA approved EVENEW on July 11, 2023. Phase 1 of the trial features a single-arm dose escalation that will assess intratracheal administration of EXOB-001. The case-control group will include a maximum of 40 patients, who will receive up to three dose levels and two regimes.
Phase 2 is a double-blind, randomized, placebo-controlled, dose-finding study that will be triggered by a positive interim analysis. This part of the clinical trial will evaluate the efficacy of EXOB-001 on reducing the severity of BPD. Researchers will monitor patients in hospital and outpatient settings until they reach the corrected age of two years old.
EXOB-001 is a new EV-based drug candidate produced by EXO Biologics. EVs are derived from human umbilical cord MSCs (hMSCs) but are believed to be safer that MSCs. Without a nucleus, an EV is expected to remove the risk of malignant transformation and ectopic colonization.
Maurizio Muraca, MD, PhD, is codirector at the Institute for Pediatric Research at the University of Padova in Italy. He has been involved with the development and use of EVs as intracellular communicators for more than 15 years.
“Seeing our groundbreaking discovery evolving into a potentially life-saving therapy is a great result of the intensive cooperation with EXO Biologics,” said Dr. Muraca. “Furthermore, the therapeutic potential of EVs as loaded cargo represents an exciting avenue to explore to benefit patients worldwide.”