Glioma treatment receives orphan designation in Europe and the US

Biotech company, VAXIMM, has received orphan designation from the European Commission (EC) and the US Food and Drug Administration (FDA) for its lead product candidate, VXM01, for the treatment of glioma.

“We are excited to have received orphan designation from both the EC and the US FDA, important achievements in the development of VXM01 to treat brain tumours, a challenging-to-treat cancer indication,” said Matthias Schroff, PhD, chief executive officer of VAXIMM. “We have seen strong initial results in a Phase I trial of VXM01 in patients with recurrent glioblastoma. In May, we entered into a collaboration with Merck and Pfizer to evaluate, through Phase I/II clinical studies, the combination of VXM01 with avelumab to treat glioblastoma and colorectal cancer. We look forward to continuing the development of our novel immunotherapy to treat these devastating tumour types.”

The Phase I trial evaluated the safety and tolerability of VXM01 in patients with recurrent glioblastoma (a type of glioma) whose disease had progressed after previous treatment with at least radiochemotherapy. The results of the study appeared to demonstrate that the treatment was safe and well tolerated. This data was presented at the annual meeting of the American Society of Clinical Oncology (ASCO).

VXM01 is an oral T-cell immunotherapy based on a live attenuated, bacterial vaccine strain that has been modified to carry vascular endothelial growth factor receptor-2 (VEGFR2) as the target gene. It is designed to activate T-cells to attack the tumour vasculature and in some types of tumour to attack the cancer cells directly.