FDA grants Breakthrough Therapy Designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable pediatric brain tumors

FDA grants Breakthrough Therapy Designation for BrainChild Bio’s B7-H3 CAR T-cell therapy for incurable pediatric brain tumors Breakthrough Therapy Designation is based on the encouraging survival data from the Phase 1 BrainChild-03 trial in children and young adults with diffuse intrinsic pontine glioma (DIPG) BrainChild Bio advancing BCB-276 towards BLA submission with a single pivotal Phase 2 trial planned to commence in Q4 2025 SEATTLE, WA and Cambridge, MA, April 22, 2025 – 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 invesJgaJonal B7-H3 targeJng autologous CAR T-cell therapy has been granted Breakthrough Therapy designaJon (BTD) by the U.S Food and Drug AdministraJon (FDA) for the treatment of diffuse intrinsic ponJne glioma (DIPG), an incurable pediatric brain tumor . This FDA designaJon was based on the promising overall survival benefit in paJents 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, Sea\le 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 ScienJfic Officer of BrainChild Bio. “This designaJon is a major milestone for the children and families afflicted with these devastaJng brain tumors and represents a new paradigm for treaJng CNS brain tumors in children and adults, including a large number of paJents 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 designaJon is an important milestone for Seattle Children’s and demonstrates our conJnued momentum in pediatric brain cancer research,” said Dr . Jeff Sperring, Chief ExecuJve Officer of Sea\le Children’s. “We harness the power of research to bring potenJal cures to kids faster, and we’re excited by the early promise shown by our work with BrainChild Bio to advance a potenJal CAR T therapy.” BrainChild Bio is preparing to advance BCB-276 in a Phase 2 mulJ-center, pivotal registraJon trial to support a potenJal Biologics License ApplicaJon (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 meeJng in late 2024. About Diffuse Intrinsic Pon1ne Glioma (DIPG) and Applica1on of CAR T-cell Therapies Diffuse intrinsic ponJne glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects approximately 300 children per year in the U.S. with the majority of diagnoses made in children between 5 and 10 years of age. Current standard -of-care treatment remains limited to palliaJve focal radiaJon therapy which results in a median overall survival of only about 11 months from diagnosis.1 Barriers to effecJve therapies for DIPG include the precarious locaJon of the tumor in the brainstem, the infiltraJve growth of the tumor throughout normal brainstem funcJonal anatomy, and the blood brain barrier that remains relaJvely intact during tumor progression prevenJng therapies from gaining access to the cancer . The barriers to effecJve therapies for DIPG can be effecJvely overcome by the locoregional delivery of appropriately targeted CAR T-cells directly into the cerebrospinal fluid via intracerebroventricular (ICV) dosing with an indwelling reservoir-catheter device. This enables the potenJal for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permiing infused CAR T-cells to directly access the tumor bed. This also allows for repeJJve infusions of CAR T-cells to replenish the tumor bed, offering the potenJal for more durable and sustained efficacy. AddiJonally, with the blood brain barrier intact, this therapeuJc approach can also minimize any on-target, off-tumor toxiciJes resulJng from systemic exposure of CAR T cells. About BrainChild Bio BrainChild Bio, Inc., is a kids-first, clinical-stage biotechnology company harnessing the power of CAR T-cell technology to treat tumors in the central nervous system, prioriJzing pediatric indicaJons with plans to expand into adult CNS tumors, specifically Glioblastoma and brain metastasis. BrainChild Bio was launched out of Sea\le Children's TherapeuJcs program and founded on the work of Dr . Michael Jensen, a pioneer in the cancer immunotherapy field and previous Chief TherapeuJcs Officer at Sea\le Children's. BrainChild Bio is advancing a next-generaJon CAR T-cell therapy plalorm for tumors of the CNS that weaves together syntheJc technologies, including mulJplex targeJng and enhanced potency controls, to enable mulJple targets in a single CAR T-cell therapy, novel transgenes to increase potency, delivery technology for durable efficacy, and streamlined CAR T-cell design and manufacturing. BrainChild Bio’s lead drug candidate is BCB-276, an autologous CAR T-cell therapy that targets the immune checkpoint B7-H3, that is advancing in clinical trials for the treatment of diffuse intrinsic ponJne glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. More informaJon is available at www.brainchildbio.com. _____________________________________________________________ 1 DIPG Registry.

The release as fetched from its publisher. brainchildbio.com ↗