About Cellular Biomedicine Group

Cellular Biomedicine Group, Inc. operates as a clinical-stage biopharmaceutical company that focuses on developing immunotherapies for the treatment of cancer and stem cell therapies for the treatment of degenerative diseases. The company provides integrated research and manufacturing services throughout the discovery, development and manufacturing spectrum for cell-based technologies. The company has two major components to its global strategy. First, the company intends on developing its own internal pipeline, focusing on immune cell therapy, regenerative medicine, as well as other biotechnology modalities. Second, the company plans to partner with companies to monetize its technologies in markets where it does not has a presence. The company’s platform enables discovery, development and manufacturing of cell-based therapies from concept to commercial manufacturing. The manufacturing and delivery of T cell therapies involve complex, integrated processes, comprised of isolating T-cells from patients, T cell enrichment, activation, viral vector transduction, expansion, harvest and fill-finish. Its in-house cell therapy manufacturing includes a semi-automated, fully closed system and could manufacture plasmids, and serum-free reagents, as well as viral vectors for its immuno-oncology cell therapy products. The company’s technologies include two major cell platforms, Immune Cell Therapy for treatment of a range of cancer indications, including Hematological Cancer-Chimeric Antigen Receptor modified T-cells (CAR-T) and Solid Tumors, including T-cells with genetically modified, tumor antigen-specific T-Cell Receptors (TCRs); and next-generation neoantigen-reactive, bio-markers based Tumor Infiltrating lymphocytes (TILs); and regenerative Medicines using human adipose-derived mesenchymal progenitor cells (haMPC) for treatment of joint diseases, such as knee osteoarthritis (Autologous and Allogeneic); and other degenerative and dermatologic diseases (assessing the feasibility). The company’s initial target market is China, where its cell-based therapies would be able to help patients with significant unmet medical needs. For hematological cancer it has finished enrolling patients for the second cohort in a Phase I dose escalation clinical study for its anti-B cell maturation antigen(anti-BCMA) CAR-T therapy for the treatment of multiple myeloma in China. The company is enrolling patients for the third cohort in China. The company has submitted its Investigational New Drug dossier to the New Medical Products Administration for approval for the Phase Ib study and are waiting for feedback. The company has initiated first-in-human non-Hodgkin lymphoma clinical trials in China for its CD19 and CD20 bi-specific CAR-T products; and achieved the first-in-human milestone and continued patient recruitment in China for its Phase I investigator initiated clinical trial of anti-CD20 CAR-T targeting anti-CD19 antibody or CAR-T treated, relapsed diffuse large B-cell lymphoma and small B-cell lymphoma patients. For solid tumors the clinical trial of Alpha-fetoprotein T-cell receptor targeting hepatocellular carcinoma (HCC) has been approved by Zhongshan Hospital in Shanghai and its first HCC patient has been infused with the company’s Human Alpha Fetoprotein-Specific T Cell Receptor modified T-cells (AFP TCR-T-cells) in December 2019; and it plans to launch non-small cell lung cancer (NSCLC) TIL clinical trials in the U.S. in the first half of 2021. Targeted Indications and Potential Therapies Immuno-oncology The company’s CAR-T platform is built on lenti-virial vector and second-generation CAR design, which is used by majority of trials and studies. The company selects the patient population for each asset and indication to allow the optimal path forward for potential regulatory approval. The company integrates the translational medicine effort into each clinical study to aid in dose selection, to investigate the mechanism of action and proof-of-concept, and to attempt to identify the optimal targeting patient population. The company has developed a serial of CAR-Ts to treat hematological malignancies, including CD20, CD22 and BCMA CAR-Ts, which have been proved to be potent in treating hematology tumors in the early phase of clinical studies. CD20 CAR CD20 is broadly overexpressed in a serial of B cell malignant tumors. In the patients relapsed after CD19 CAR-T treatment, the expression of CD20 on target tumor cells is relatively stable. It is proven to be an optimal target for treating CD19 CAR-T relapsing patients. The company has developed a CD20 CAR-Ts clinical lead product, which demonstrated strong anti-tumor activity in both in vitro assays and in vivo animal studies. The company has filed a patent application in China and have initiated a first in human investigator initiated trial with CD19 CAR-T relapsed NHL patients. CD22 CAR CD22 is another surface marker highly expressed in B cell malignancies especially in hairy cell leukemia. It also expresses in the patients relapsed after CD19 CAR-T treatment. The company has developed a CD22 CARs clinical lead, which displayed anti-tumor activity in in vitro cytotoxicity assays. The company plans to initiate an investigator initiated trial with CD19 CAR-T relapsing ALL patients and hairy cell leukemia. BCMA CAR BCMA is a member of the TNF receptor superfamily, universally expressed in multiple myeloma (MM) cells. It is not detectable in normal tissues except plasma and mature B cells. It is a safer target for treating refractory MM patients in various clinical trials. The company has developed BCMA CARs. Its BCMA CAR clinical lead exhibits potent anti-tumor activity both in vitro and in vivo. The company has filed a patent for BCMA CAR in China and begun an investigator initiated trial in refractory MM patients in January 2019. NKG2D CAR The company focuses on developing novel second generation CAR-Ts using NKG2D extracellular fragment as antigen binding domain. These CARs could recognize target tumor cells expressing NKG2D ligands. The company plans to initiate a first in human investigator initiated trial with relapsed or refractory acute myeloid leukemia patients. AFP TCR The company licenses the AFP-TCR technology from Augusta University. The company is continuing its evaluation on the efficacy and specificity of the AFP TCRs to identity the most appropriate candidate for a first time in human study. The company plans to redirect Human T-cells with the AFP TCRs and evaluate their anti-tumor activity on in vitro cytokine release and cytotoxicity assays; and potential on/off-target toxicity, including allo-reactivity, as well as in vivo efficacy tests in animal models. TIL Augmented by the U.S. National Cancer Institute technology license, the company’s wholly owned subsidiary, Cellular Biomedicine Group VAX, Inc. is developing neoantigen reactive TIL therapies to treat immunogenic cancers. In the early stages of cancer, lymphocytes infiltrate into the tumor, specifically recognizing the tumor targets and mediating anti-tumor response. These cells are known as TIL. TIL-based therapies have shown encouraging clinical results in early development. In Phase-2 clinical studies in patients with metastatic melanoma performed, TIL therapy demonstrated efficacy in patients with metastatic melanoma with objective response rates of 56% and complete response rates of 24%. The company has started its development with NSCLC, and plans to expand into other cancer indications. Knee Osteoarthritis (KOA) The company is pursuing two primary therapies for the treatment of KOA: ReJoin therapy and AlloJoin therapy. The company completed the Phase I/IIa clinical trial for the treatment of KOA. The trial tested the safety and efficacy of intra-articular injections of autologous haMPCs in order to reduce inflammation and repair damaged joint cartilage. The six-month follow-up clinical data showed ReJoin therapy to be both safe and effective. In 2014, the company completed patient enrollment for the Phase IIb clinical trial of ReJoin for KOA. The multi-center study has enrolled 53 patients to participate in a randomized, single blind trial. The company announced positive Phase IIb 48-week follow-up data in 2016, with statistical significant evidence that ReJoin cartilage regeneration, which concluded the planned phase IIb trial. Strategy The company intends to use its first-mover advantage in China, against a backdrop of enhanced regulation by the central government, to differentiate ourselves from the competition and establish a leading position in the China cell therapeutic market. The company intends to invest and expand its clinical research capabilities by building drug development and manufacturing infrastructure in China and in the U.S., expanding its clinical research platform, hiring new talent and enhancing its existing coverage. Regulations The company’s business operations in China are subject to customary regulation and licensing requirements under regulatory agencies, including the local Administration for Industry and Commerce, General Administration of Quality Supervision, Inspection and Quarantine, and the State Administration of Taxation, for each of its business locations. Additionally, the company’s clean room facilities and the use of reagents are also regulated by local branches of the Ministry of Environmental Protection. The company complies with current Good Tissue Practices and Good Manufacturing Practices guidelines that apply to biological products. Competition The company’s primary competitors in the field of cancer immune cell therapies include pharmaceutical, biotechnology companies, such as Eureka Therapeutics, Inc., Iovance Biotherapeutics Inc., Juno Therapeutics, Inc. (acquired by Celgene), Kite Pharma, Inc. (acquired by Gilead), CARSgen, Sorrento Therapeutics, Inc. and others. Among its competitors, the ones based in and operating in Greater China are CARsgen, Hrain Biotechnology, Nanjing Legend Biotechnology Galaxy Biomed, Persongen, Anke Biotechnology, Shanghai Minju Biotechnology, Unicar Therapy (Cooperated with Terumo BCT), Wuxi Biologics, Junshi Pharma, BeiGene, Immuno China Biotech, Chongqing Precision Biotech, Innovative Cellular Therapeutics and China Oncology Focus Limited. The company’s primary competitors in the field of stem cell therapy for osteoarthritis and other indications include Mesoblast Ltd., Caladrius Biosciences, Inc. and others. Additionally, in the general area of cell-based therapies for knee osteoarthritis ailments, the company potentially competes with various companies, from big pharma to specialty medical products or biotechnology companies. Some of these companies include Abbvie, Merck KGaA, Sanofi, Teva, GlaxosmithKline, Baxter, Johnson & Johnson, Sanumed, Medtronic and Miltenyi Biotech. As cell-based products are emerging as viable medical therapies, majority of the company’s more direct competitors are smaller biotechnology and specialty medical products companies that include Vericel Corporation, Regeneus Ltd., Advanced Cell Technology, Inc., Nuo Therapeutics, Inc., ISTO technologies, Inc., Ember Therapeutics, Athersys, Inc., Bioheart, Inc., Mesoblast, Pluristem, Inc., Medipost Co. Ltd. and others. History Cellular Biomedicine Group, Inc. was incorporated in 2001.