IASO Bio Announces U.S. FDA Approval of Clinical Trial Application for BCMA CAR-T CT103A for Relapsed/Refractory Multiple Myeloma

SHANGHAI and NANJING,China and SAN JOSE,Calif.,Dec. 22,2022 -- IASO Biotherapeutics (IASO Bio),a clinical-stage biopharmaceutical company engaged in discovering,developing,and manufacturing innovative cell therapies and antibody products,today announced that the Investigational New Drug (IND) application for its BCMA CAR-T CT103A (equecabtagene autoleucel)has been approved by the U.S. Food and Drug Administration (FDA) for use in U.S. clinical trials for relapsed/refractory multiple myeloma (R/R MM).

Multiple myeloma is a deadly blood cancer that often infiltrates the bone marrow causing anemia,kidney failure,immune problems,and bone fractures. For multiple myeloma patients,common first-line drug treatments include proteasome inhibitors,immunomodulatory drugs,and alkylating agents. While treatment may result in remission,most patients will inevitably enter the relapsed or refractory stage as there's currently no cure. As a result,there is a significant unmet need for patients with relapsed/refractory multiple myeloma. In the United States,MM accounts for nearly 2% of new cancer cases,and more than 2% of all cancer-related deaths.

According to Frost & Sullivan,the number of new MM cases in the United States rose from 30,300 in 2016 to 32,300 in 2020 and is expected to increase to 37,800 by 2025. The total number of patients diagnosed with MM in the United States increased from 132,200 in 2016 to 144,900 in 2020 and is expected to rise to 162,300 by 2025.

CT103A,a Chimeric Antigen Receptor (CAR) T-Cell Therapy targeting the B-cell maturation antigen (BCMA),contains a fully human single-chain variable fragment (scFv),allowing it to bypass potential anti-CAR immunogenicity of the host while retaining anti-tumor activity. Results from a clinical phase I/II (NCT05066646) study in China showed excellent safety and efficacy of CT103A.

The data presented at the European Hematology Association (EHA) Research Conference 2022 showed that as of January 21,2022,79 subjects have received CT103A infusion at the recommended Phase II dose (RP2D) of 1.0×106 CAR-T cells/kg. The median follow-up time for the 79 subjects was 9 months (1.2,19.6),and the objective response rate (ORR)was 94.9%. The median time to response (mTTR) was only 16 days. 73 (92.4%) subjects achieved at least once negative minimal residual disease (MRD) status after cell infusion. All subjects who achieved complete response (CR) or better achieved MRD-negative. Subjects who had previously received CAR-T therapy continued to benefit from CT103A infusion. Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) that occurred after cell infusion were only grade 1~2. No subject developed grade ≥3 CRS or ICANS,and all CRS and ICANS resolved after supportive treatment.

CT103A's regulatory milestones:

In February 2021,CT103A was granted the "breakthrough therapy designation (BTD)" by China's National Medical Products Administration (NMPA) for the treatment of relapsed/refractory multiple myeloma.

In February 2022,CT103A was granted the "orphan drug designation (ODD)" by the FDA for the treatment of multiple myeloma.

In June 2022,CT103A was accepted by the NMPA for the NDA application for the treatment of relapsed/refractory multiple myeloma and included in the priority review.

In August 2022,a new IND was approved by NMPA for CT103A with an expanded indication of AQP4-IgG-positive neuromyelitis optica spectrum disorder (NMOSD).

Dr. Wen (Maxwell) Wang,Chief Executive Officer of IASO Bio,said,"The IND approval of CT103A in the U.S. is an important milestone of IASO Bio and a new starting point outside China. IASO Bio will accelerate its overseas clinical trials and the development and implementation of cellular immunotherapy drugs to benefit more patients globally."

About CT103A

Equecabtagene autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection,a lentiviral vector containing a CAR structure with a fully human scFv,CD8a hinger and transmembrane,4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening,utilizing a proprietary in-house optimization platform,and integrated in-house manufacturing process improvement,the construct of the BCMA CAR-T is potent and equecabtagene autoleucel shows prolonged persistency in patients. The NMPA accepted the New Drug Application for equecabtagene autoleucel for the treatment of relapsed/refractory multiple myeloma (R/R MM). Equecabtagene autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD) by the U.S. FDA in February 2022. In addition to multiple myeloma,the NMPA has received IND application of equecabtagene autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).IASO Bio and Innovent Biologics,Inc. (1801.HK) are jointly developing CT103A for the treatment of RRMM in mainland China.

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 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 includes a diversified portfolio of over 10 novel pipeline products,including IASO's leading asset,equecabtagene autoleucel (CT103A),a fully human BCMA chimeric antigen receptor autologous T cell injection. Equecabtagene autoleucel received New Drug Application (NDA) acceptance from China's National Medical Products Administration (NMPA) for the treatment of relapsed/refractory multiple myeloma (r/r MM). It also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD) by the U.S. FDA in February 2022. In addition to multiple myeloma,NMPA has accepted its Investigational New Drug (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 the treatment of 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 U.S. Food and Drug Administration in October 2021. 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.comor www.linkedin.com/company/iasobiotherapeutics.

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