BrainChild Bio, Inc., a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the central nervous system (CNS), today announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Breakthrough Therapy designation (BTD) by the U.S Food and Drug Administration (FDA) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This FDA designation was based on the promising overall survival benefit in patients with brain tumors treated with an autologous B7-H3 CAR T-cell therapy observed in the BrainChild-03 Phase 1 trial (NCT04185038), conducted by BrainChild Bio’s academic partner, Seattle Children’s, and recently published in Nature Medicine.
“Breakthrough Therapy designation gives us the possibility to accelerate the development path for BCB-276 as a CAR T-cell therapy that can potentially transform the treatment of DIPG,” stated Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio. “This designation is a major milestone for the children and families aflicted 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.”
FDA grants Breakthrough Therapy designation to investigational medicines that demonstrate the potential to treat a serious or life-threatening condition and show preliminary clinical evidence that the drug may show substantial clinical improvement over available therapies. Investigational medicines with BTD are provided early and more frequent interactions with the FDA to discuss the product candidate’s development plan in addition to being eligible for rolling submission and priority review of the marketing application.
“This designation is an important milestone for Seattle Children’s and demonstrates our continued momentum in pediatric brain cancer research,” said Dr. Jeff Sperring, Chief Executive Officer of Seattle Children’s. “We harness the power of research to bring potential cures to kids faster, and we’re excited by the early promise shown by our work with BrainChild Bio to advance a potential CAR T therapy.”
BrainChild Bio is preparing to advance BCB-276 in a Phase 2 multi-center, pivotal registration trial to support a potential Biologics License Application (BLA) to the FDA for the treatment of children and young adults with DIPG. This clinical plan is based on alignment between BrainChild Bio and FDA at a Type B meeting in late 2024.
