Clinical Studies

The phase 1/2 trial (ClinicalTrials.gov ID NCT06279741) aims to evaluate the safety and efficacy of EXOB-001 consisting of extracellular vesicles derived from umbilical cord mesenchymal stromal cells in the prevention of bronchopulmonary dysplasia (BPD) in extremely premature neonates. The study population includes babies born between 23 and 28 (27 + 6 days) weeks of gestational age and body weight between 500g and 1,500 g. Thirty-six subjects will receive one or three administrations of the three doses of EXOB-001 via the endotracheal route in phase 1. In phase 2, two dosages based on the results of phase 1 will be selected and a total of 203 subjects will be randomised to receive either EXOB-001 or placebo (saline solution). Infants will be followed up to 2 years of corrected age (end of study).

Giovanni Migliaccio, Scientific Director at CVBF, member of BoD of EPTRI, participated in the preclinical phase and the preparation of the dossier for CTIS. He was also part of the group of Scientific Advisors for the product development.

BIOGENERA has officially signed an agreement with CVBF (Consortium for Biological and Pharmacological Evaluations) for the monitoring and execution of the Phase I clinical trial for the new orphan drug BGA002, intended for the treatment of aggressive pediatric tumors associated with alterations in the MYCN tumor gene. BGA002 has received orphan drug designation for both SCLC (from the FDA, orphan register: DRU-18- 6260) and NB (from the FDA, orphan register: DRU-17-6085 and the European Medicines Agency (EMA), orphan drug application: EMA/OD/020/12), certifying its potential as a novel therapeutic approach for SCLC and NB.

CVBF is part of various European research networks focused on knowledge transfer and scientific excellence, including EPTRI-AISBL which is dedicated to advancing the development of safe and effective new drugs for children.

O.Della Pasqua (UCL), member of the Board of Directors at EPTRI, has been involved in the project development phase specifically for the dose rationale and design of a first-time-in-children protocol for the evaluation of MYCN inhibition BGA002 in neuroblastoma (more info here).

The primary goal of the collaboration between BIOGENERA and CVBF is to initiate the Phase I clinical trial of the anti-tumor drug BGA002 in early 2025, leveraging the benefits of its Orphan Drug designation from both the European Medicines Agency and the U.S. FDA.

The GAPP project, sponsored by CVBF, aims to evaluate the efficacy, pharmacokinetics, and safety of gabapentin in children aged 3 months to under 18 years who suffer from moderate to severe chronic neuropathic or mixed pain. Currently, Gabapentin is used off-label in these clinical conditions due to the lack of robust paediatric-specific evidence.

This represents a significant unmet need for adequate pharmacological treatments for neuropathic chronic and mixed pain in children, highlighting the necessity for therapies with a favourable benefit/risk profile (and age-appropriate formulation) to be considered as first-line options.

O.Della Pasqua (UCL), member of the Board of Directors at EPTRI, contributed to the dose rationale for gabapentin using as tools pharmacokinetic (PK) modelling and clinical trial simulations (CTSs) to optimize doses and protocol design for a prospective investigation in paediatric patients with chronic pain. Giorgio Reggiardo (Head of Biostatistic Unit at CVBF) contributed to the implementation on an innovative study design with the advantage to include prospectively planned opportunities for modifying study design elements and hypotheses based upon interim data analyses. CVBF Regulatory office for PIP (PIP number: EMEA-001310-PIP01-12-M03) preparation and submission.

More information available here.