News

• Study to target Bronchopulmonary Dysplasia, the most common cause of fatality in pre-natal birth.

Liège, Belgium, June 24, 2024 – EXO Biologics, a clinical-stage biotech and a pioneer in developing exosome-based therapies, today announces it has completed dosing of the first cohort of patients in EVENEW. This EVENEW study is the first European Medicines Agency (EMA) authorized clinical trial involving mesenchymal stromal cell (MSC)-based exosomes.

This EVENEW study is a Phase 1/2 European clinical trial using EXO Biologics’ lead candidate EXOB-001. EXO Biologics is initially developing EXOB-001 to prevent Bronchopulmonary Dysplasia (BPD), the most common cause of death in preterm newborns. 

BPD, the most common and severe consequence of preterm birth

Bronchopulmonary dysplasia (BPD) is a severe lung disease. 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. There are over 80,000 babies born extremely prematurely in the US, UK and EU alone. 30,000 to 35,000 develop BPD each year. As a result, BPD is the most common and severe consequence of pre-term birth and the most common cause of morbidity of pre-maturity[1]. BPD persists throughout life. Patients show reduced lung function and respiratory symptoms during childhood and young adulthood[2]. In addition, BPD patients are at higher risk of many other complications related to pre-maturity[3] and a higher risk of mortality for cardiovascular causes[4]. Consequently, the healthcare system costs are much higher during and after the perinatal period for BPD patients.

“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. EXO Biologics selected this indication as it is dedicated to using its leading position in developing exosome therapies to advance medical treatments that could improve the lives of those most at risk,” 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.”

EVENEW Study

EXO Biologics’ EVENEW study is an adaptive, seamless Phase 1/2 trial. EVENEW is assessing the safety and efficacy of the intratracheal administration of EXO Biologics’ lead MSC-based exosome candidate EXOB-001. The study will target the prevention of Bronchopulmonary Dysplasia (BPD) in preterm newborns.

The Phase 1 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 this dose escalation part of the trial, up to 40 patients will be enrolled for up to three dose levels and two regimes.  The Phase 2 part, triggered by a positive interim analysis, is a double-blind, randomized, placebo-controlled dose-finding study with efficacy evaluation of EXOB-001 on reducing BPD severity grade. It will aim to recruit up to 225 babies subjects.

“EXO Biologics is now progressing quickly with the first trial approved by the EMA with an MSC-based exosome. As the clear leader in this potentially-transformative field, EXO Biologics has a responsibility to select indications that could improve patient care in indications with no satisfactory alternative therapeutic treatments. This is why we decided to initially target BPD, the most common consequence and morbidity of premature birth,” said Hugues Wallemacq, CEO, EXO Biologics. “The completion of the first milestone of this trial is a demonstration of EXO Biologics’ ability to translate pioneering, cutting-edge research into the clinical phase through its proprietary technology and expertise.”

About EXOB-001     

EXO Biologics has developed EXOB-001, a novel exosome-based drug candidate produced with ExoPulseTM. Exosomes, also known as extracellular vesicles (or EVs), are derived from cultured human umbilical cord mesenchymal stromal cells (hMSCs). Human MSCs are characterized by their ability to secrete growth factors and cytokines that modulate the immune system and promote tissue repair and regeneration. Exosomes are nanoparticles naturally released from these cells. Exosomes are expected to be a safer therapeutic alternative to MSCs as they eliminate the risk of emboli and an immune response. Since exosomes are nuclei-free, they eliminate the risk of malignant transformation and ectopic colonization. EXO Biologics has been granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA and ODD by EMA for EXOB-001 to prevent Bronchopulmonary Dysplasia (BPD) in preterm newborns.

About ENEVEW

EVENEW is a multi-center adaptive, seamless Phase 1/2 trial conducted by EXO Biologics. The trial assesses the safety and efficacy of intratracheal administration of EXOB-001 in preventing severe forms of Bronchopulmonary Dysplasia (BPD) in preterm newborns. Phase 1 is 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 this dose escalation part of the trial, a maximum of 40 patients will be enrolled for up to three dose levels and two regimes. The second phase, triggered by a positive interim analysis, is a double-blind, randomized, placebo-controlled dose-finding study with efficacy evaluation of EXOB-001 on reducing BPD severity grade. Patients will be monitored up to the corrected age of two years (in hospital and outpatient).

About EXO Biologics

EXO Biologics is a biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with unmet medical needs. Partnering with leading academic researchers, EXO Biologics aims to set the stage for future nanomedicines.  The Company’s development strategy focuses on novel drug candidates for therapeutic applications in respiratory diseases, inflammatory bowel diseases, neurology, and oncology. The research was partly financed by the Walloon Region.

For additional information, please visit www.exobio.be and follow us on LinkedIn. 

[1] Shah PS, Sankaran K, Aziz K, Allen AC, Seshia M, Ohlsson A, et al. Outcomes of preterm infants <29 weeks gestation over a 10-year period in Canada: a cause for concern? J Perinatol. (2012) (https://pubmed.ncbi.nlm.nih.gov/21593814/)

[2] Moschino, Bonadies, and Baraldi 2021

[3] Jobe and Bancalari 2001

[4] Crump et al. 2019