Targepeutics Receives Rare Pediatric Disease Designation for GB13

Targepeutics Inc is proud to announce that the U.S. Food and Drug Administration (FDA) has granted Pediatric Rare Disease Designation (RPDD) for its investigational immunotoxin therapy, GB13 (IL13-E13K-PE4E). This breakthrough therapy is aimed at treating pediatric diffuse high-grade glioma, an aggressive and life-threatening brain tumor that primarily affects children and adolescents. The designation is based on FDA findings that (1) pediatric diffuse high-grade gliomas is a serious or life-threatening disease in which the serious or life-threatening manifestations, such as mortality, primarily affect individuals aged from birth to 18 years, and (2) pediatric diffuse high-grade glioma meets the definition of “rare disease or condition” at section 526 of the FD&C Act.

The RPDD designation acknowledges the significant need for innovative treatments addressing rare, life-threatening diseases that affect children. With this designation, Targepeutics Inc becomes eligible for a priority review voucher (PRV) upon potential FDA approval of GB13. This voucher can expedite the review of a subsequent therapy or be sold to another company.

About GB13 for Treatment of Pediatric Diffuse High-Grade Glioma

Pediatric diffuse high-grade glioma is an aggressive form of pediatric gliomas that rapidly proliferate, have a poor overall survival and can be classified by molecular signatures. The three subtypes include diffuse midline glioma (DMG) with H3K27 alterations, diffuse hemispheric glioma (DHG) with H3G34 mutations and diffuse high-grade gliomas with wild type H3 and IDH.

RPDD was based on the strength of preclinical data that demonstrated the ability of GB13 to potently kill pediatric diffuse glioma tumor cells that correlates to expression of the tumor restricted target, IL13Rα2.  GB13 decreases tumor volume and increases animal survival up to 200% when administered via convection enhanced delivery (CED).

A Milestone for Targepeutics Inc

“We are honored to receive this designation, which underscores the potential of GB13 to make a meaningful impact for patients and families affected by this devastating disease,” said Sil Lutkewitte, President of Targepeutics. “This milestone reinforces our commitment to addressing unmet medical needs in rare pediatric populations.”

Targepeutics Inc is currently finalizing GMP manufacturing and final investigational new drug (IND)-enabling studies.  The first-in-human Phase 1, with expansion to Phase 2, clinical trial is planned for patient enrollment in Q1 2026.