EXO Biologics doses first cohort in MSC-based exosome clinical trial

Premature baby in hospital

EXO Biologics has completed dosing of the first cohort of patients in its EVENEW study, the first European Medicines Agency (EMA) authorised clinical trial involving mesenchymal stromal cell (MSC)-based exosomes.

The Phase I/II clinical trial is testing the company’s lead candidate EXOB-001, developed to prevent bronchopulmonary dysplasia (BPD), the most common cause of death in preterm newborns.

EXO Biologics has received Orphan Drug Designation for BPD from both the EMA and FDA.

BPD primarily affects extremely pre-mature infants (under 28 weeks gestational age) who require prolonged oxygen therapy to support their breathing. Despite improved perinatal care in developed countries, 40% of extremely pre-term babies develop BPD.

“BPD has severe health implications for considerable numbers of pre-term babies, and is the most common cause of morbidity for this patient group. BPD patients also currently have no specific therapeutic alternative,” said Dr Beatrice De Vos, CMO, EXO Biologics. “The completion of dosing of the first cohort in this major study is a significant step in EXO Biologics’ efforts to provide potentially enhanced therapies for pre-term babies who face long-lasting health challenges due to BPD.”

The Phase I part of the trial will be run as a single-arm, dose-escalating study to assess the safety of the intratracheal administration of EXOB-001, enriched with a historical nested case-control group to compare clinical outcomes for further evaluation.

In the dose escalation part of the trial, up to 40 patients will be enrolled for up to three dose levels and two regimes.

The Phase II part, triggered by a positive interim analysis, is a double-blind, randomised, placebo-controlled dose-finding study with efficacy evaluation of EXOB-001 on reducing BPD severity grade. It will aim to recruit up to 225 subjects.

Diana Spencer, Senior Digital Content Editor, DDW

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