BrainChild Bio has raised $116 million in Series A funding to advance its CAR T cell therapies for central nervous system tumors, including a pivotal Phase 2 clinical trial of its lead candidate BCB-276 in diffuse intrinsic pontine glioma. The financing was led by an undisclosed private family fund and foundation aligned with BrainChild Bio’s mission. Existing investor Seattle Children’s participated alongside new investor WRF Capital.
Proceeds will primarily support the company’s ILLUMINATE Phase 2 trial of BCB-276, an investigational B7-H3-targeted autologous CAR T cell therapy being developed for diffuse intrinsic pontine glioma, or DIPG.
BrainChild Bio simultaneously announced that it has initiated the pivotal Phase 2 study, which is designed as a registrational trial supporting a potential future Biologics License Application to the U.S. Food and Drug Administration.
The financing will also support development of BCB-214, BrainChild Bio’s triple-targeting CAR T cell therapy being advanced toward initial clinical testing for glioblastoma, along with other pipeline programs for pediatric and adult brain tumors.
BrainChild Bio is a clinical-stage biotechnology company developing CAR T cell therapies specifically for tumors in the central nervous system.
Its platform is based on technology developed at Seattle Children’s and exclusively licensed to BrainChild Bio in December 2023.
Since launching, the company has advanced BCB-276 into what it describes as a registration-stage clinical development program for DIPG.
BrainChild Bio has optimized the therapy for later-stage clinical development and established manufacturing, quality and regulatory infrastructure intended to support a registrational program.
Those efforts led to initiation of the ILLUMINATE Phase 2 trial, NCT07680439.
ILLUMINATE is an open-label, single-arm study evaluating the efficacy and confirming the safety of BCB-276 in children and young adults with newly diagnosed DIPG following initial standard-of-care focal radiation therapy.
BrainChild Bio said the trial design was developed based on alignment with the FDA and is intended to accelerate a potential regulatory filing.
BCB-276 has also received FDA Fast Track designation.
The multicenter trial will be conducted at six pediatric neuro-oncology centers across the U.S., with activation completed or underway at all selected sites.
Patients will receive BCB-276 approximately every two weeks for up to 15 doses over approximately seven to eight months.
The therapy is manufactured using each patient’s own immune cells and administered through locoregional delivery directly into the cerebrospinal fluid using an indwelling reservoir-catheter device.
The study’s primary endpoint is overall survival.
Secondary endpoints include safety and tolerability, progression-free survival and radiographic response.
BCB-276 targets B7-H3, an immune checkpoint target expressed in DIPG and other tumors.
The therapeutic approach builds on a CAR T cell program originally developed by BrainChild Bio founder and Chief Scientific Officer Michael Jensen and his team at Seattle Children’s.
In the earlier FDA-authorized BrainChild-03 Phase 1 study, the underlying CAR T cell therapy demonstrated what the company described as a manageable safety profile in an outpatient administration setting and preliminary evidence of encouraging overall survival outcomes.
Results from that study were published in Nature Medicine.
DIPG is a highly aggressive brain tumor arising in the pons and primarily affects children.
Approximately 300 children per year in the U.S. are diagnosed with the disease, with most diagnoses occurring between ages five and 10.
The current standard of care remains primarily palliative focal radiation therapy, and median overall survival is approximately 11 months from diagnosis.
BrainChild Bio’s locoregional delivery approach is designed to address several challenges associated with treating DIPG, including the tumor’s brainstem location, infiltrative growth pattern and the relatively intact blood-brain barrier.
Direct administration into cerebrospinal fluid is intended to give the CAR T cells access to the tumor bed while enabling repeated dosing and potentially reducing systemic exposure.
Beyond BCB-276, BrainChild Bio is building a broader CAR T platform incorporating technologies such as multiplex tumor targeting, approaches designed to enhance CAR T cell functionality, specialized delivery technologies and streamlined cell therapy manufacturing.
The company’s strategy prioritizes pediatric cancers while also expanding the platform toward adult central nervous system tumors, including glioblastoma.
KEY QUOTES:
“Our path to building BrainChild Bio has led us to secure the support of a unique and committed syndicate of investors who share our mission to prioritize therapeutic innovation for children with cancer, while also rapidly advancing new science that can change the course of brain cancers for a wider population. We believe the time is right to show the breakthrough potential for our CAR T cell therapeutic approach with BCB-276 for DIPG which is a devastating pediatric brain cancer, as well as accelerate our efforts to advance other CAR T cell therapies for pediatric and adult brain tumors.”
Steven Brugger, Chief Executive Officer of BrainChild Bio
“This financing enables us to chart our path forward to serve the children and families afflicted with devastating brain tumors and represents a new paradigm for treating CNS brain tumors in children and adults. Our team at BrainChild Bio is steadfast in its commitment to harness CAR T cell technology in CNS tumors and we are uniquely positioned to do so.”
Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio
“Bringing this pivotal Phase 2 study forward for children battling DIPG has been our steadfast purpose at BrainChild Bio and represents the hard work of our team and the opportunity to advance our mission to combat pediatric brain cancer. We aspire to bring breakthrough medicines to these children, providing hope for cures that are safe and preserve quality of life.”
Michael Jensen, MD, Founder and Chief Scientific Officer of BrainChild Bio
“Children and families facing a diagnosis of DIPG urgently need new treatment options. While we recognize the significant challenges in developing new therapies for DIPG, we are hopeful that our ILLUMINATE study will bring us closer to transforming the treatment landscape and delivering a meaningful therapeutic advance for these children. The pivotal Phase 2 ILLUMINATE study represents an important step in evaluating the potential of BCB-276 for children with newly diagnosed DIPG. We are partnering closely with leading pediatric neuro-oncology investigators and study sites to conduct this trial with the urgency and scientific rigor that this aggressive brain cancer demands.”
Cori Abikoff, MD, Vice President of Clinical Development at BrainChild Bio
