NEW YORK – Seattle-based BrainChild Bio announced Friday that it is advancing its leading drug candidate, a CAR T-cell therapy targeting the immune checkpoint B7-H3, to a registrational trial to support the treatment's accelerated approval by the US Food and Drug Administration.
The BCB-276 treatment for diffuse intrinsic pontine glioma (DIPG), a highly aggressive pediatric brain tumor, was granted breakthrough therapy designation following promising survival benefits for patients in a Phase I trial conducted in partnership with Seattle Children's.
"This designation is a major milestone for the children and families afflicted with these devastating brain tumors and represents a new paradigm for treating CNS brain tumors in children and adults, including a large number of patients suffering with glioblastomas and brain metastases," said Michael Jensen, BrainChild's founder and chief scientific officer.
DIPG affects roughly 300 children each year in the United States, most between the ages of 5 and 10. It's difficult to treat, since the tumor develops within the pons, a part of the brainstem, and is shielded by the blood brain barrier that prevents drug delivery. With existing radiation treatments, the median survival from diagnosis for DIPG is just 11 months.
BCB-276 targets CAR T-cells directly into the cerebrospinal fluid, allowing the calls to access the tumor. In the Phase I trial, BCB-276 raised median survival to 19.8 months.
BrainChild met with the FDA in late 2024, the company said, to develop a clinical plan for the Phase II trial, which will begin in late 2025.