On March 31, 2023, Cabaletta Bio, Inc., one of IASO Biotherapeutics ("IASO Bio") partners, announced that the Company’s Investigational New Drug (IND) application for CABA-201 has been cleared by the U.S. Food and Drug Administration (FDA). Cabaletta plans to initiate a Phase 1/2 clinical trial of CABA-201 for the treatment of systemic lupus erythematosus (SLE) in patients with active lupus nephritis (LN) or active SLE without renal involvement. CABA-201 is a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, whose CD19 binder is licensed exclusively from IASO Bio.
SLE is a chronic, potentially severe, autoimmune disease, most commonly impacting young women between the ages of 15 and 40, where the immune system attacks healthy tissue throughout the body. It is characterized by abnormal B cell function and autoantibody production resulting in a range of clinical manifestations including end organ damage and an increased risk of death. It affects an estimated 160,000-320,000 patients in the U.S. in total and up to 1 million patients in China. LN is the most common end-organ manifestation of SLE, affecting approximately 40% of SLE patients. Among these patients, the risk of end-stage renal disease is approximately 17% and the risk of death is approximately 12%, each within 10 years of diagnosis.
October, 2022, IASO Bio and Cabaletta Bio entered into an agreement pursuant to which Cabaletta obtained from IASO Bio an exclusive, worldwide license to develop, manufacture and commercialize a clinically validated fully-human CD19 binder for use in product that is designed to modify T cells in treatment of autoimmune diseases. IASO Bio is entitled to receive up to approximately $162 million in aggregate payments, including an upfront payment and potential development and sales milestone payments across up to two products, as well as royalties. IASO Bio has the right of first negotiation to develop and commercialize Cabaletta’s products using the licensed sequence in the Greater China region.
About IASO Bio
IASO Bio is a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and autoimmune diseases. Leveraging its proprietary fully human antibody discovery platform (IMARS), high-throughput chimeric antigen receptor T-cell (CAR-T) drug screening platform, and proprietary manufacturing processes, IASO Bio is developing a rich clinical-stage pipeline of multiple autologous and allogeneic CAR-T and biologics product candidates. This pipeline includes a diversified portfolio of over 10 novel products, including IASO's leading asset, Equecabtagene Autoleucel (CT103A), a fully human BCMA CAR-T injection. Equecabtagene Autoleucel received New Drug Application (NDA) acceptance from as China's National Medical Products Administration (NMPA) for the treatment of RRMM and obtained the U.S. FDA IND approval. The company also received Breakthrough Therapy Designation (BTD) from the NMPA in February 2021 and Orphan Drug Designation (ODD) in February 2022 and Regenerative Medicine Advanced Therapy (RMAT) and Fast Track (FT) Designations in February 2023 from the FDA. In addition to multiple myeloma, NMPA has accepted its IND application for the new extended indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).
Additionally, the company's in-house developed fully human CD19/CD22 dual-targeted CAR-T cell therapy has received two IND clearances for treating relapsed/refractory B-cell non-Hodgkin's lymphoma (r/r B-NHL) and relapsed/refractory acute B-lymphoblastic leukemia (r/r B-ALL). It is currently in Phase I clinical trial for r/r B-NHL. It was also granted ODD by the FDA in October 2021. In the approximately 20 patients who have been dosed to date in the investigator-initiated trial, there was no ICANS, or immune effector cell-associated neurotoxicity syndrome, of any grade observed in any patient, and a grade 3 cytokine release syndrome rate of less than 5%, with the remainder of patients experiencing no CRS or less than grade 3 cytokine release syndrome.
Leveraging its strong management team, innovative product pipeline, integrated manufactural and clinical capabilities, IASO aims to deliver transformative, curable and affordable therapies that fulfil unmet medical needs to the patients in China as well as around the world. For more information, please visit www.iasobio.com or www.linkedin.com/company/iasobiotherapeutics.