Based in Seattle, Massachusetts, BrainChild Bio specializes in the development of CAR T cell therapies that target tumors found in the central nervous system (CNS). Its lead drug candidate, BCB-276, is an autologous CAR T cell therapy for the treatment of diffuse intrinsic pontine glioma (DIPG), a pediatric cancer for which there are currently no approved treatments.
In a previous phase 1 clinical trial conducted by the Seattle Children’s hospital, BCB-276 showed promising preliminary safety and efficacy data in 21 patients treated with repeated doses of the cell therapy. Based on this data, the FDA has supported the start of a multi-center phase 2 pivotal clinical trial, designed to accelerate the submission of a biologics license application (BLA) for the treatment of children and young adults with DIPG.
“We are very pleased to have a solid path forward for our clinical development of BCB-276 in DIPG, enabling us to continue our progress for children and families struggling with this devastating brain cancer that currently has no approved treatments,” stated Michael Jensen, Founder and Chief Scientific Officer of BrainChild Bio. “We look forward to continuing to work with the FDA and to generate the additional data required to support (...) the initiation of the BCB-276 pivotal trial by the end of 2025.”
Preliminary efficacy analysis from the phase 1 trial initiated by Seattle Children’s showed a median time of 19.8 months from diagnosis to death for all 21 DIPG patients, of which 12 began their CAR T treatment after disease progression and 9 began it before. Three of the patients who began the treatment prior to disease progression remained alive at 44.6 months, 45.6 months, and 52.5 months from diagnosis.
According to a statement by BranChild Bio, “These data, while preliminary, suggest a meaningful improvement in overall survival for DIPG patients (...) as compared to current standard-of-care, which is limited to palliative focal radiation therapy and has a median time of survival from diagnosis of 8-11 months.”
“This is a time of strong momentum for the CAR T discoveries and clinical trials in pediatric brain cancer,” said Jeff Sperring, Chief Executive Officer of Seattle Children’s. “Our innovation model is built to accelerate technology to bring potential cures to kids faster, and it is gratifying to see that Seattle Children’s launch of BrainChild Bio is supporting the advancement of a CAR T therapy to reach children with an incurable brain cancer.”
