BrainChild Bio granted FDA RMAT designation for its CAR T-cell therapy for incurable pediatric brain tumors
The company plans on moving to Phase 2 later this year, hopefully supporting a BLA in the near future.
BrainChild Bio announced that its investigational B7-H3-targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA.
This designation covers the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric brain tumor that currently has no cure.
To those unaware, CAR T-cell therapy is a type of regenerative medicine where they engineer a patient’s own immune cells (T-cells) to recognize and attack specific tumor cells. The approach may help address a handful of significant challenges in treating DIPG, including the tumor’s sensitive location (brainstem), its tendency to infiltrate surrounding healthy brain tissue, and the blood-brain barrier issue, which limits many systemic therapies.
BrainChild Bio’s lead candidate, BCB-276, is designed for local delivery through an indwelling catheter device directly into the cerebrospinal fluid (CSF). The goal of this locoregional administration is to enable the CAR T-cells to directly access the tumor bed, potentially overcoming limitations posed by traditional intravenous treatments.
They’ve evaluated it in their Phase 1 trial with their partner Seattle Children’s Research, found here:
https://clinicaltrials.gov/study/NCT04185038
Along with the accompanying Nature publication found here:
https://www.nature.com/articles/s41591-024-03451-3
Which reported favorable overall survival outcomes for these patients.
BrainChild Bio says it’s preparing to advance BCB-276 into a Phase 2 in the fourth quarter of 2025, which will hopefully support a potential Biologics License Application (BLA).
“We are very pleased to now also receive RMAT designation, less than one month after being granted Breakthrough Therapy designation from FDA for our lead CAR T therapy, BCB-276, for the treatment of DIPG. Receiving designations from two independent reviews within FDA further validates the positive CAR-T clinical results achieved by our team to date and the urgent need for a treatment for DIPG,” stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. “Our team is keenly focused on initiating the pivotal Phase 2 trial by the end of this year and look forward to continuing to work with the FDA on an accelerated path forward to bring potential new CAR-T treatments for CNS brain tumors in children and adults.”
