Cell Medica successfully doses first patient with CMD-501 targeting pediatric neuroblastoma

Cell Medica announces the treatment of the first patient world-wide to receive CMD-501, an autologous CAR-NKT therapy targeting pediatric neuroblastoma. This is the first time an engineered NKT cell therapy has been used in humans. Cell Medica is a clinical-stage biopharmaceutical company that is transforming the treatment of solid and hematological cancer by developing the next generation of CAR therapies.

This open-label Phase 1 study, GINAKIT2, is being carried out in collaboration with both Baylor College of Medicine (BCM) and Texas Children's Hospital.

Dr. Andras Heczey, Principal Investigator, Assistant Professor, Pediatrics-Oncology at Baylor College of Medicine and Physician-Scientist, Texas Children's Cancer Center commented: "Dosing the first patient with this novel CAR-NKT therapy is an important milestone for all pediatric patients with neuroblastoma. CAR-NKTs may offer an exciting new therapeutic option for these patients and potentially for others with solid and hematological cancers. I am extremely grateful to the patients and families participating in this ground-breaking study."

Chris Nowers, Cell Medica's CEO, said: "We believe that our CAR-NKT platform has a unique profile, with a potential to target solid and hematological tumors, as well as the possibility of a subsequent allogeneic "off the shelf" CAR-NKT therapy that could address some challenges of current autologous CAR-T therapies. This study marks an important step forward for Cell Medica and we are proud to be leading the development of this innovative class of next generation CAR therapies with our colleagues at BCM and Texas Children's."

Innovative CAR-NKT Platform
CMD-501 is based on Cell Medica's novel CAR-NKT platform, a next-generation technology of engineered immune cells with enhanced functions for the treatment of hematological and solid tumors, utilizing the unique properties of NKT cells, a specialized type of innate lymphocytes, sharing properties of T and NK cells. CMD-501 is the initial study from Cell Medica's CAR-NKT pipeline and utilizes an autologous approach. The patient's own NKT cells are genetically engineered with a CAR targeting GD2, a molecule expressed on the surface of nearly all neuroblastoma cells. In collaboration with its partners at BCM and Texas Children's, Cell Medica designed this CAR-NKT cell therapy to also secrete the cytokine IL-15, which has been shown in pre-clinical studies to increase the persistence of CAR-NKT cells and improve their efficacy within the immunosuppressive tumor microenvironment.

Dr. Leonid Metelitsa, Professor of Pediatrics, Hematology-Oncology, Baylor College of Medicine and Co-Director, Neuroblastoma Program, Texas Children's Cancer Center added: "It has been a great pleasure leading the multi-disciplinary team in the development of this versatile CAR-NKT platform. NKT cells effectively traffic to the tumor site, so expressing tumor-specific CARs in these cells ensures delivery to the site of disease for maximum efficacy. We're now exploiting another natural feature of NKT cells, their lack of allo-reactivity and we are developing allogeneic, "off the shelf", therapies that will further harness the unique advantages of NKT cells."

First-in-Human Study
GINAKIT2 is a first-in-human, dose escalation evaluation of CMD-501 in children with relapsed or refractory (R/R) high-risk neuroblastoma, (NCT03294954). Neuroblastomas occur primarily in children and account for 7-10 percent of all pediatric cancers. Ninety percent of patients are younger than 5 years at diagnosis. R/R high risk neuroblastoma is one of the deadliest types of childhood cancer and the current median survival is around 1-3 years. Almost all neuroblastomas express GD2, which is targeted by CMD-501. This study is supported by a grant from Alex's Lemonade Stand Foundation (ALSF), awarded to BCM investigators, Drs. Heczey and Metelitsa.

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