New Publication Highlights GB13 as a Promising Therapeutic Strategy for DIPG

A new study demonstrating the potential of GB13 as an effective treatment strategy for diffuse intrinsic pontine glioma (DIPG) has been published in Nature Communications Biology. Titled Radiotherapy plus neoadjuvant and concomitant IL-13Rα2-directed immunotoxin therapy for diffuse intrinsic pontine glioma,” the paper by Rechberger et al. represents the second publication resulting from the collaboration between Dr. David Daniels’ laboratory at Mayo Clinic (Rochester, MN) and Targepeutics.

In this study, GB13 was evaluated across multiple animal models of DIPG. When administered directly into tumors via convection-enhanced delivery (CED), GB13 significantly reduced tumor volume and improved overall survival. Importantly, the findings support a clinical strategy to enhance standard-of-care radiation therapy. Animals treated with GB13 prior to radiation exhibited smaller tumors and longer survival compared with radiation or GB13 treatment alone. Notably, curative outcomes were observed in a human-derived DIPG model.

The authors also identified a mechanistic basis for these effects. Neoadjuvant GB13 treatment impaired tumor cells’ ability to repair radiation-induced DNA damage, leading to more rapid and effective tumor cell death.

Clinically, these data support Targepeutics’ development strategy for GB13 and inform the company’s upcoming IND submission and first-in-human clinical trial for DIPG. The planned Phase I trial, with an expansion Phase II component, will evaluate neoadjuvant versus adjuvant GB13 administration in combination with radiation therapy.

 

 

https://www.nature.com/articles/s42003-025-09155-9