Oncotelic Therapeutics launches clinical trial for pediatric brain cancer

Oncotelic Therapeutics Inc. has announced the commencement of a multi-center, two-part Phase 1-2 study evaluating its experimental drug OT-101 in pediatric patients diagnosed with diffuse intrinsic pontine glioma (DIPG). DIPG is a rare and aggressive form of brain cancer affecting children, with a particularly poor prognosis.

The study aims to determine the maximum tolerated dose (MTD) or recommended Phase 2 dose (RP2D) of OT-101 and to assess its efficacy in children with DIPG. The administration route will be intrathecal or intraventricular, allowing the drug to bypass the blood-brain barrier and reach therapeutic concentrations directly in the cerebrospinal fluid. This approach is significant as chemotherapy has historically offered limited clinical benefit for DIPG.

OT-101 is designed to target transforming growth factor beta 2 (TGF-β2), a protein implicated in promoting glioma cell growth and creating an immunosuppressive tumor microenvironment. Preclinical and early clinical data suggest OT-101 can reduce TGF-β2 secretion, inhibit tumor cell proliferation and migration, and restore anti-tumor immune responses. The drug has previously shown promising single-agent activity in adult patients with recurrent/refractory high-grade glioma.

The median overall survival for children with DIPG is approximately 10 months following standard radiation therapy, highlighting the urgent need for novel therapeutic strategies. Oncotelic's study seeks to address this unmet need by investigating OT-101 as a post-radiation consolidation therapy. Safety pharmacology studies in rabbits and primates indicated favorable safety profiles for intrathecally delivered OT-101.