Autolus announced positive CHMP opinion for obe-cel for adults with R/R B-ALL

CLINICAL AND REGULATORY 

Autolus announced positive CHMP opinion for obe-cel for adults with R/R B-ALL

Autolus announced that the EMA’s CHMP has recommended European Commission approval of obe-cel for the treatment of adult patients, 26+, with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. The CHMP recommendation was based on the results of the FELIX study, an open-label, multicenter, single arm study in adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia. The results were published in the New England Journal of Medicine in November 2024. In the pivotal cohort of patients, (cohort IIA (n=94)), the Complete Response/Complete Response with Incomplete Hematological Recovery (CR/CRi) for patients who received at least one infusion of obecabtagene autoleucel was 76.6%. Median response duration for all infused patients was 21.2 months. Median event-free survival (EFS) was 11.9 months and the estimated 6- and 12-month event-free survival rates were 65.4% and 49.5%, respectively. The most common non-laboratory Grade 3 or higher adverse reactions were infections-pathogen unspecified (32%), febrile neutropenia (24%) and bacterial infectious disorders (11%). Cytokine release syndrome developed in 87 of the 127 patients (68.5%), with events of grade 3 or higher in three patients (2.4%). Immune effector cell-associated neurotoxicity syndrome developed in 29 of the 127 patients (22.8%), with grade 3 or higher occurring in nine patients (7%).

FDA grants RMAT designation for BrainChild Bio’s B7-H3 CAR-T therapy for pediatric brain tumors

BrainChild Bio, a clinical-stage biotechnology company developing CAR T-cell therapies to treat tumors in the CNS, announced that the investigational B7-H3 targeting autologous CAR T-cell therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDA for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. The use of a regenerative medicine, specifically a CAR T-cell therapy, offers the potential to overcome barriers for other drug modalities to be effective in addressing DIPG, including the precarious location of the DIPG tumor in the brainstem, the infiltrative growth of the tumor throughout normal brainstem functional anatomy, and the blood brain barrier that remains intact during tumor progression. BrainChild Bio has designed BCB-276 to be administered by locoregional delivery of targeted CAR T-cells directly into the cerebrospinal fluid, permitting infused CAR T-cells to directly access the tumor bed, using an indwelling reservoir-catheter device. This approach to administering an autologous B7-H3 CAR T-cell therapy has been successfully implemented and resulted in the promising overall survival benefit in patients with brain tumors observed in the BrainChild-03 Phase 1 trial, conducted by BrainChild Bio’s academic partner, Seattle Children’s Research Institute.

DEALS AND FINANCING 

Cellipont and Optieum partner to advance cGMP manufacturing of CAR-T therapy for glioblastoma

Cellipont Bioservices, a cell therapy CDMO, and Optieum Biotechnologies, a preclinical stage company dedicated to the discovery and development of innovative CAR T cell therapies, announced a partnership for cGMP manufacturing of OPTF01, Optieum's novel CAR-T therapy for glioblastoma treatment, a product derived from their proprietary Eumbody System. OPTF01 specifically targets Fibroblast activation protein-alpha (FAPα) a protein expressed on both tumor cells and the surrounding pericytes and Cancer-associated Fibroblasts (CAFs). Hence, OPTF01 can potentially disrupt the immunosuppressive microenvironment around the tumor while simultaneously attacking the malignant cells within the tumor. Successful development of this therapeutic approach would address a critical unmet medical need for patients with refractory glioblastoma who currently face limited treatment options with poor prognoses, as well as various other solid tumor indications. Cellipont will provide technology transfer, process development, and cGMP manufacturing of Optieum's novel OPTF01 CAR-T product.