Precision NeuroMed (PNM), a clinical-stage biotechnology company developing advanced drug delivery technologies for central nervous system (CNS) diseases, announced on Tuesday that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational therapy for glioblastoma (GBM).

PNM says that it is reintroducing cintredekin besudotox (IL13-PE38QQR), a potent cytotoxic protein designed to target and kill cells that express the IL-13 alpha-2 receptor (IL13aR), uniquely present on tumour cells such as glioblastoma. The protein's selective expression allows it to precisely attack cancer cells while minimising damage to normal brain tissue.

Despite advancements in understanding the genetics of glioblastoma and identifying novel drug targets, the blood–brain barrier (BBB) remains a significant obstacle, severely limiting the effectiveness of many potential therapies delivered through the bloodstream. According to PNM, its platform uses convection-enhanced delivery (CED) to bypass the BBB and enable direct, targeted administration of nanoparticles, including proteins, liposomes, and gene therapy at therapeutic concentrations to the brain.

PNM is developing a personalised approach that targets both molecular and regional aspects of tumour cells within the brain. By improving drug delivery at the site of disease, the company aims to extend survival and enhance quality of life for patients with glioblastoma in a meaningful way.