BrainChild Bio, Inc. to clinically advance BCB-276, an autologous B7-H3 targeting CAR T-cell therapy for incurable pediatric brain tumors

BrainChild Bio, Inc. to clinically advance BCB-276, an autologous B7-H3 targeting CAR T-cell therapy for incurable pediatric brain tumors The Company has aligned on clinical plan based on a positive FDA meeting, leveraging clinical data from a first-in-human Phase 1 trial published today in Nature Medicine BCB-276 uses repetitive intracerebroventricular dosing to treat diffuse intrinsic pontine glioma (DIPG), a pediatric brain cancer with no approved treatments SEATTLE, WA and Cambridge, MA, January 7, 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 the clinical development plan to advance BCB-276, its lead autologous CAR T-cell therapy targeMng the immune checkpoint B7-H3, for diffuse intrinsic ponMne glioma (DIPG), a type of incurable pediatric brain tumor. The company plans to advance BCB-276 in a single pivotal registraMon trial designed to accelerate the path to submit a Biologics License ApplicaMon for the treatment of children and young adults with DIPG. This clinical plan is based on alignment with the U.S. Food and Drug AdministraMon (FDA) at a Type B meeMng to proceed directly to a mulM-center Phase 2 pivotal clinical trial. This potenMally accelerated clinical path for BCB-276 is supported by the promising preliminary safety and efficacy data for SCRI-CARB7H3(s)1 – the research cell product that derived BCB-276 – from a Phase 1 clinical trial conducted by Sea[le Children’s, published today in Nature Medicine at this paper. “We are very pleased to have a solid path forward for our clinical development of BCB-276 in DIPG, enabling us to conMnue our progress for children and families struggling with this devastaMng brain cancer that currently has no approved treatments,” stated Michael Jensen, MD, Founder and Chief ScienMfic Officer of BrainChild Bio. “We look forward to conMnuing to work with the FDA and to generate the addiMonal data required to support a successful IND submission leading to the iniMaMon of the BCB-276 pivotal trial by the end of 2025.” Ini$al Phase 1 data in DIPG pa$ents suppor$ng BCB-276’s development Sea[le Children’s iniMated BrainChild-03, a single-center, dose-escalaMon Phase 1 clinical trial of repeMMve intracerebroventricular (ICV) dosing of its B7-H3 targeted CAR T therapy, SCRI-CARB7h3(s), in children with recurrent/refractory CNS tumors and DIPG. The clinical data from a subset of 21 DIPG paMents from the Phase 1 study included twelve (12) paMents who began their CAR T treatment ader disease progression and nine (9) paMents who began their CAR T treatment before disease progression. Preliminary safety analyses demonstrated that repeMMve ICV dosing of the B7-H3 targeted CAR T therapy up to 10x107 cells was well tolerated as an outpaMent regimen and without lymphodepleMng chemotherapy . Preliminary efficacy analyses demonstrate a median Mme from diagnosis to death for all 21 paMents treated was 19.8 months. Three (3) paMents, all beginning CAR T treatment prior to disease progression, remained alive at 44.6 months, 45.6 months, and 52.5 months from diagnosis. These data, while preliminary, suggest a meaningful improvement in overall survival for DIPG paMents (for both the pre --progression and post- progession paMent cohorts) as compared to current standard-of-care, which is limited to palliaMve focal radiaMon therapy and has a median Mme of survival from diagnosis of 8-11 months. “This is a Mme of strong momentum for the CAR T discoveries and clinical trials in pediatric brain cancer that were spawned at Sea[le Children’s and are now highlighted in Nature Medicine and serving as a driving force for the clinical progress with a CAR T therapy for DIPG,” said Dr . Jeff Sperring, Chief ExecuMve Officer of Sea[le Children’s. “Our innovaMon model is built to accelerate technology to bring potenMal cures to kids faster, and it is graMfying to see that Sea[le Children’s launch of BrainChild Bio is supporMng the advancement of a CAR T therapy to reach children with an uncurable brain cancer .” About Diffuse Intrinsic Pon$ne Glioma (DIPG) and Applica$on of CAR T-cell Therapies Diffuse intrinsic ponMne glioma (DIPG) is a primary high-grade brain tumor that arises in the pons and is uniformly fatal. DIPG affects 200-300 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 palliaMve focal radiaMon therapy which results in a median overall survival of only 8-11 months from diagnosis.2 Barriers to effecMve therapies for DIPG include the precarious locaMon of the tumor in the brainstem, the infiltraMve growth of the tumor throughout normal brainstem funcMonal anatomy, and the blood brain barrier t hat remains relaMvely intact during tumor progression. The barriers to effecMve therapies for DIPG can be effecMvely 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 potenMal for extensive exposure of the pons to cerebrospinal fluid flow from the ventricular system, thus permikng infused CAR T-cells to directly access the tumor bed. This also allows for repeMMve infusions of CAR T-cells to replenish the tumor bed, offering the potenMal for more durable and sustained efficacy. AddiMonally, with the blood brain barrier intact, this therapeuMc approach can also minimize any on-target, off-tumor toxiciMes resulMng 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, prioriMzing pediatric indicaMons with plans to expand into adult CNS tumors, specifically Glioblastoma and Brain Metastasis. BrainChild Bio is advancing a next- generaMon CAR T-cell therapy plamorm for tumors of the CNS that weaves together syntheMc technologies, including mulMplex targeMng and enhanced potency controls, to enable mulMple 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 ponMne glioma (DIPG), a pediatric cancer that forms in the brainstem which currently has no approved treatments. More informaMon is available at www.brainchildbio.com. _____________________________________________________________ 1. Sea&le Children’s Therapeu4cs BrainChild-03 Phase 1 clinical trial with SCRI-CARB7H3(s), an autologous CAR T cell product designed to specifically target B7-H3 in CNS tumors. 2. DIPG Registry.

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