Oncurious NV, an emerging oncology company focused on the development of innovative orphan drugs for the treatment of pediatric cancers, has initiated a phase I/IIa study that will evaluate the safety and tolerability and explore the preliminary efficacy of TB-403 for the treatment of relapsed or refractory medulloblastoma, a rare, life-threatening brain tumor that mainly affects children.

Today’s study initiation follows the earlier announced partnership between Oncurious, its TB-403 project partner BioInvent, and the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC) in the US. NMTRC is a collaboration of 25 US academic medical centers, teaching hospitals and other entities, with the purpose of facilitating and conducting collaborative research activities and investigations of new treatments for neuroblastoma, medulloblastoma and other pediatric cancers.

Headquartered at the Helen DeVos Children’s Hospital in Grand Rapids, MI, USA, NMTRC is the key clinical trial partner for this Phase I/IIa study. The study aims at recruiting a minimum of 27 patients, with first results expected to be reported in 2017.

TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF) which is expressed in several types of cancer, including medulloblastoma. A paper in Cell in February 2013 (Cell, 152, 1065-76, 2013), highlighted for the first time that PlGF plays a role in the growth of medulloblastoma. The paper was based on pre-clinical research conducted by Prof Rakesh Jain from the Massachusetts General Hospital at Harvard (Boston) and the team of Prof Peter Carmeliet from the VIB/ KU Leuven.

Treatment with TB-403 in pre-clinical models for medulloblastoma has demonstrated beneficial effects on tumor growth and survival. TB-403 has been shown to have a favorable safety profile in previous clinical trials in healthy volunteers and adult patients with various types of solid tumors.

BioInvent International AB is Oncurious’ co-development partner for the TB-403 clinical trial.

Patrik De Haes, MD, executive chairman of Oncurious, commented, “We are pleased to announce the initiation of this important phase I/IIa study with TB-403, a key corporate milestone for Oncurious. Given the encouraging pre-clinical data generated with TB-403 in Leuven and Boston, we are hopeful that this novel antibody, targeting placental growth factor, will provide an improved treatment option for children afflicted with this lethal brain tumor.”

Michael Oredsson, CEO of BioInvent commented, “The initiation of this study is a key step in the development of this new targeted potential treatment for children with devastating brain cancers. We are looking forward to be working with NMTRC’s experienced investigator network to complete this study in a timely manner.”

Giselle Sholler, MD MSC, Chair of NMTRC, “We are delighted to be starting this study, which is very much in line with our mission to bring forward new therapies that could improve the quality of life and survival of children with medulloblastoma. We believe that with its unique mode of action TB-403 offer could offer an important solution to address this tremendous unmet medical need.”