Rimqarto (anbalcabtagene autoleucel) / Curocell - LARVOL DELTA

Gut Microbiome Composition As a Prognostic Marker in CD19-Targeting CAR T-Cell Therapy for Relapsed/Refractory Diffuse Large B-Cell Lymphoma (ASH 2024) - "Methods We analyzed 47 patients with R/R DLBCL who were schedule to undergo anti-CD19 CAR T-cell therapy, including 29 patients receiving tisagenlecleucel (tisa-cel) and 18 receiving anbalcatagene autoleucel (anbal-cel). These findings underscore the potential for microbiome modulation as a strategy to overcome CAR T-cell therapy resistance and improve patient outcomes. We are currently conducting functional studies to explore the interplay between the tumor microenvironment and microbiome."

Biomarker • CAR T-Cell Therapy • B Cell Lymphoma • B Cell Non-Hodgkin Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Curocell Signs CAR-T Raw Material Supply Agreement, Accelerating Commercialization of Rimqarto [Google transkation] (HIT News) - "With the Ministry of Food and Drug Safety (MFDS) currently reviewing the product approval process for 'RIMQARTO,' a next-generation CAR-T treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), the company explained that it has further strengthened its preemptive establishment of a raw materials and supply chain (SCM) to advance to the commercialization stage."

Commercial • Diffuse Large B Cell Lymphoma

Curocell…said it has received approval from the Ministry of Food and Drug Safety (MFDS) to initiate a phase 2 clinical trial of its investigational CAR-T therapy Anbalcel (CRC01) in adult patients with acute lymphoblastic leukemia (ALL) (Korea Biomedical Review) - "The company plans to begin patient enrollment and commence the trial shortly. The newly approved protocol expands the scope of eligible patients and details specific efficacy evaluation methods."

New P2 trial • Acute Lymphocytic Leukemia

A Pilot Study of Anbalcabtagene Autoleucel (Anbal-Cel) in Patients with Relapsed or Refractory Primary or Secondary Central Nervous System Lymphoma (ICBMT 2025) - "Anbalcabtagene autoleucel (Anbal-cel) is a novel second-generation anti-CD19 CAR T-cell product currently under investigation in systemic DLBCL...Following leukapheresis, patients will undergo lymphodepletion with fludarabine and cyclophosphamide for three days, followed by a single intravenous infusion of Anbal-cel at a target dose of 2×10⁶ CAR+ viable T cells/kg... This study is a prospective clinical trial investigating CD19-targeted CAR T-cell therapy in patients with PCNSL or SCNSL using Anbal-cel. Given the poor prognosis and lack of effective salvage options in this population, the study will provide important insights into the safety, feasibility, and potential activity of CAR T-cell therapy in the CNS lymphoma. Keywords: CNS lymphoma, CAR-T"

Clinical • IO biomarker • B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Secondary Central Nervous System Lymphoma

Curocel files IND amendment for Anbalcel for adults with ALL, with the aim of initiating Phase 2 clinical trials. [Google translation] (BioTimes) - "Curocell...announced on the 8th that it has submitted an application for a change to the clinical trial plan (IND) to the Ministry of Food and Drug Safety for the initiation of phase 2 clinical trials of 'Anbalcel (CRC01),' a CAR-T treatment for adult acute lymphoblastic leukemia (ALL). This IND amendment application was submitted to incorporate Phase 1 clinical trial results into the Phase 2 clinical trial design."

New P2 trial • B Acute Lymphoblastic Leukemia

Curocell Receives FDA Approval for Phase 1/2 CAR-T Clinical Trial for SLE [Google translation] (HIT News) - "Curocell...announced on the 1st that the Ministry of Food and Drug Safety approved the phase 1/2 clinical trial plan for systemic lupus erythematosus (SLE) for its CD19-targeting CAR-T treatment 'Anbalcel (CRC01)'....This clinical trial will target patients with severe SLE who have not responded to existing treatments. Phase 1 will evaluate safety and tolerability, while Phase 2 will assess efficacy. Multiple institutions, including Seoul St. Mary's Hospital of the Catholic University of Korea, are expected to participate."

New P1/2 trial • Systemic Lupus Erythematosus

Curocell launches CUROLINK to streamline CAR-T supply chain ahead of Rimqarto rollout (Korea Biomedical Review) - "In advance of the commercialization of its next-generation CAR-T therapy, RIMQARTO, Curocell has developed CUROLINK, an integrated solution that tracks and manages the entire process from prescription to administration in real-time. CUROLINK is a cloud-based cell therapy supply management solution based on a fully licensed Salesforce-based customer relationship management (CRM) platform....Curocell has completed system stabilization and linkage verification and plans to introduce it to major hospitals in Korea in time for the launch of RIMQARTO."

Commercial • Large B Cell Lymphoma

Anbalcabtagene Autoleucel in Relapsed/Refractory CNS Lymphoma (clinicaltrials.gov) - P2 | N=12 | Not yet recruiting | Sponsor: Hyungwoo Cho

New P2 trial • B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Central Nervous System Lymphoma • Secondary Central Nervous System Lymphoma

Comparative Effectiveness of Anbalcabtagene Autoleucel versus Tisagenlecleucel in Patients with Relapsed/Refractory Diffuse Large B-cell Lymphoma. (PubMed, Cancer Res Treat) - "We performed a matching-adjusted indirect comparison (MAIC) using individual patient data from CRC01-01 (NCT#04836507) and aggregate data from JULIET trial (NCT#02445248). After applying MAIC to balance prognostic factors between the groups, adjusted HRs or ORs were 0.47 (0.23-0.95) for OS, 0.59 (0.36-0.96) for PFS, 2.60 (1.04-6.52) for ORR, and 3.00 (1.30-6.92) for CRR. Anbal-cel showed superior effectiveness over tisa-cel, with higher response rates and improved survival outcomes, even after accounting for imbalances in prognostic factors at trial enrollment."

HEOR • Journal • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Large B Cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • PD-1 • TIGIT

Curocell files IND for Anbal-cel trial in sever lupus (Korea Biomedical Review) - "Curocell on Monday filed an Investigational New Drug (IND) application with the Ministry of Food and Drug Safety (MFDS) for its CD19-targeted CAR-T therapy Anbal-cel (CRC01) in systemic lupus erythematosus (SLE)...The study is an open-label, multicenter, single-arm, phase 1/2 trial to evaluate the tolerability, safety, and efficacy of Anbal-cel in patients with severe SLE who have not responded to existing treatments."

New P1/2 trial • Systemic Lupus Erythematosus

ANBALCABTAGENE AUTOLEUCEL (PD-1 AND TIGIT KNOCKDOWN CD19 CAR-T) FOR RELAPSED/REFRACTORY LARGE B-CELL LYMPHOMA (CRC01-01) (ICML 2025) - "Lymphodepletion was performed with cyclophosphamide 500 mg/m2 and fludarabine 30 mg/m2, administered for three days (-5, -4, -3 days). Anbal-cel demonstrated PD-1 and TIGIT knockdown can contribute to long-term response with high CR rate and manageable safety profile."

IO biomarker • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Large B Cell Lymphoma • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Mediastinal Large B-Cell Lymphoma • B3GAT1 • CD38 • ENTPD1 • HAVCR2 • IL7R • PD-1 • TIGIT

Curocell, “Clinical Efficacy and Safety Scientifically Proven” in Final Phase 2 Clinical Trial of ‘anbal-cel’ [Google translation] (BioTimes) - P1/2 | N=91 | NCT04836507 | Sponsor: Curocell Inc. | "A total of 79 patients received anbal-cel, of which 75.3% (55 patients) achieved an objective response rate (ORR) and 67.1% (53 patients) achieved a complete response (CR). The median follow-up period was 8.5 months, and the long-term follow-up results showed that the progression-free survival (PFS) at 12 and 18 months was 41.1% and 35.2%, and the overall survival (OS) was 66.6% and 57.3%, respectively. The median progression-free survival (mPFS) was 6.0 months, which was twice as long as the mPFS (2.9 months) in the Kimria clinical trial."

P2 data • Diffuse Large B Cell Lymphoma

Curocell 'CAR-T', Autoimmune Patient "First Administration.. Positive Initial Response" [Google translation] (Biospectator) - "Curocell announced on the 17th that it recently administered its first CD19 CAR-T 'anbal-cel' to a patient with intractable lupus at the Catholic University of Korea Seoul St. Mary's Hospital. This was conducted based on the Ministry of Food and Drug Safety's therapeutic use approval procedure...According to Curocell, the patient recovered to the point where he could return to his daily life without any acute side effects after administration of Anbalcel, and is showing a positive initial treatment response, such as improvement in lupus-related disease indicators even after discontinuation of immunosuppressants....Curocel plans to submit an Investigational New Drug (IND) application for phase 1/2 clinical trial of Anbalcel targeting patients with systemic lupus erythematosus (SLE) to the Ministry of Food and Drug Safety in the first half of this year."

New P1/2 trial • Trial status • Lupus • Systemic Lupus Erythematosus

Curocell seeks health insurance listing for CAR-T therapy Rimqarto (Korea Biomedical Review) - "Curocell...has officially submitted an application to the Health Insurance Review and Assessment Service (HIRA) for the reimbursement listing of its CAR-T treatment, Rimqarto. Rimqarto is a next-generation CAR-T therapy developed for patients with relapsed or refractory large B-cell lymphoma (LBCL). In its phase 2 clinical trials, Rimqarto demonstrated a complete response rate of 67.1 percent, proving its efficacy and strong safety profile. With this application, Rimqarto has initiated the evaluation process for health insurance coverage. HIRA will assess Rimqarto’s clinical efficacy, cost-effectiveness, and overall therapeutic value before determining its eligibility for reimbursement."

Reimbursement • Large B Cell Lymphoma

Curocell develops Rimqarto, Korea's first CAR-T therapy with superior efficacy [Google translation] (Chosun Biz) - "Curocell applied for the product license for its domestic CAR-T therapy, 'Rimqarto (ingredient name: Anbalcel),' to the Ministry of Food and Drug Safety on the 30th. Curocell is the first pharmaceutical and biotech corporation in South Korea to develop a CAR-T therapy and submit a final approval application...Rimqarto is a treatment for relapsed or refractory large B-cell lymphoma (LBCL), a type of blood cancer...Being selected as a rapid processing candidate allows it to receive approval based solely on the results of phase 2 clinical trials under the 'Advanced Biopharmaceutical Product License Review' regulations....The decision on Rimqarto's domestic approval is expected to be made in the second half of next year."

Korea approval • Korea filing • Diffuse Large B Cell Lymphoma • Large B Cell Lymphoma

Curocell's Anbal-cel wins orphan drug designation for treatment of relapsed or refractory LBCL (Korea Biomedical Review) - "On Tuesday, the Ministry of Food and Drug Safety (MFDS) designated Anbal-cel as an orphan drug in development for the treatment of relapsed or refractory large B-cell lymphoma (LBCL). The MFDS' orphan drug designation facilitates the rapid development and approval of treatments for rare, incurable, and life-threatening diseases....In addition, the period for protecting proprietary data for new drugs under development will be extended from 4-6 years to 10 years....Anbal-cel demonstrated efficacy and safety with a 67.1 percent complete response rate in the final results of its phase 2 clinical trial."

Orphan drug • Diffuse Large B Cell Lymphoma

Curocell's CAR-T therapy picked as 2nd drug for expediated approval and market launch (Korea Biomedical Review) - "Curocell's Anbal-cel (brand name: Rimqarto), a locally developed CAR-T therapy, has been designated by the government for expedited approval and market launch through a simultaneous process of domestic approval, coverage evaluation, and drug price negotiation....Curocell said Tuesday that its next-generation CAR-T therapy, Anbal-cel, has been selected as the second target drug for the Ministry of Health and Welfare's 'Approval application, reimbursement assessment, and drug price negotiation pilot project.'...Through this pilot project, the drug payment process of MFDS approval, HIRA’s reimbursement assessment, and NHIS’ drug price negotiation have been improved so that drug price negotiation is completed at the same time as the MFDS approval. That shortens the process, which previously took more than 300 days, including 120 days for MFDS’ approval, 150 days for HIRA’s reimbursement evaluation, and 60 days for NHIS’ drug price negotiation."

Korea approval • Reimbursement • Large B Cell Lymphoma • Lymphoma

Curocell's CAR-T therapy wins fast-track designation (Korea Biomedical Review) - "Curocell, a chimeric antigen receptor-T (CAR-T) developer, said its CAR-T therapy Anbal-Cel has won the advanced biomedicine expedited approval from the Ministry of Food and Drug Safety...As the first homegrown CAR-T therapy employing next-generation technology, Curocell expects to file for new drug approval for Anbal-Cel this year, and a market launch in the latter half of next year....Anbal-Cel’s designation enables a dedicated review team to prioritize its evaluation over other products."

Fast track • Korea approval • Launch non-US • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

ANBALCABTAGENE AUTOLEUCEL (PD-1 AND TIGIT KNOCK-DOWNED CD19 CAR-T) FOR RELAPSED OR REFRACTORY LARGE B-CELL LYMPHOMA PATIENTS: RESULT OF A PIVOTAL PHASE 2 STUDY. (EHA 2024) - "They received a target dose of 2x106 cells/kg afterlymphodepletion (cyclophosphamide 500mg/m2 and fludarabine 30mg/m2 for 3 days). Anbal-cel demonstrated high objective response rate with durable response. The data showed not only lowincidence of ≥ grade 3 cytokine release syndrome, but also markedly lower rate of neurological eventcompared to other CAR-T products. We will conduct additional analysis to determine how the OVIS platformhas a positive effect on the process of killing tumor cells."

Clinical • P2 data • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • PD-1 • TIGIT

[BIO USA 2024] Curocell considers setting up overseas offshoots (Korea Biomedical Review) - "The BIO International Convention (BIO USA) is the world's largest bio conference, bringing together bio companies, investors, and researchers worldwide to discuss innovative technologies and partnerships. This year's event was held in San Diego from last Monday to Thursday, local time....Korea Biomedical Review met with Curocell CEO Kim Gun-soo and asked him about the company's plans to enter overseas markets and the timeline for applying for the license of its homegrown CAR-T drug CRC01 (anbalcabtagene autolucel or Anbal-cel in general name). In the interview, CEO Kim discussed Curocell's goals, business strategy, and the need to expand the domestic CAR-T therapeutic market."

Clinical • Licensing / partnership • Hematological Malignancies • Leukemia • Lymphoma • Oncology

Curocell ahead of NDA… Target for approval of CAR-T treatment in the first half of next year [Google translation] (HIT News) - "As Curocell...is about to submit a New Drug Application (NDA), the Ministry of Food and Drug Safety will announce the approval of CAR-T treatment 'Anbal-cel (ingredient name) in the first half of 2025. The goal is to obtain product approval for ‘Anbalcabtagene Autoleucel’...The final efficacy of the Phase 2 clinical trial of Anbalcell, the main content of the CSR, was the same as the topline results announced in March...'we will complete the phase 1 clinical trial for adult acute lymphoblastic leukemia (ALL), an additional indication for Anbalcell,' and 'In the first half of next year.'....'We aim to submit a new drug approval application to the Ministry of Food and Drug Safety in September 2024 and receive product approval for CAR-T treatment Anbalcell in 2025.'"

Korea approval • Korea filing • Trial status • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology

Curocell applies to the Ministry of Food and Drug Safety for a rapid processing system for ‘Anbalcell’ advanced biopharmaceutical [Google translation] (HIT News) - "Curocell...announced on the 22nd that it had applied to the Ministry of Food and Drug Safety to designate Anbalcell as a target of the advanced biopharmaceutical expedited processing system....The advanced biopharmaceutical expedited processing system is a law designed to quickly launch highly innovative medicines, such as treatments for life-threatening diseases or serious diseases, to the market and support rapid supply to patients. Through this application, Curocell plans to proceed with new drug approval after being designated as an expedited processing target for Anbalcell within the third quarter of this year."

Non-US regulatory • Hematological Malignancies • Oncology

Curocell receives government funding to expand CAR-T treatment into lupus (Korea Biomedical Review) - "Curocell...said it will start developing a next-generation anti-CD19 CAR-T therapy for treatment-resistant systemic lupus erythematosus (SLE) as part of the 2024 Multi-Ministerial Regenerative Medicine Technology Development Program...This initiative, driven by the Ministry of Science and ICT along with the Ministry of Health and Welfare...The company has received funding of 1.07 billion won ($789,959) to support clinical trials for systemic lupus using its advanced CAR-T therapy, Anbal-cell..."

Financing • Systemic Lupus Erythematosus

Kim Geon-soo, CEO of Curocell, 'Commercialization of domestic CAR-T treatment is not far away' [CEO met on-site] [Google translation] (Korea Economic TV) - "Curocell is the company that is speeding up the development of domestic CAR-T treatments. The market's attention is focused on the recent positive results from phase 2 clinical trials....Curocell plans to complete the application for conditional approval of Anvalcel to the Ministry of Food and Drug Safety in September and commercialize it in 2025."

Launch non-US • Non-US regulatory • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Curocell's CAR-T therapy effective to treat DLBCL in phase 2 study (Korea Biomedical Review) - P1/2 | N=91 | NCT04836507 | Sponsor: Curocell Inc. | "Curocell, a Kosdaq-listed biotech company, said it received the topline data from its phase 2 clinical trial for its CAR-T (chimeric antigen receptor-T) therapy, Anbal-cel, to treat diffuse large B-cell lymphoma (DLBCL)....The analysis of the phase 2 clinical trial topline data revealed a complete response rate (CRR) of 67.1 percent among 73 participants, indicating that Anbal-cel completely eradicated cancer cells in these patients....Additionally, the objective response rate (ORR), a primary evaluation metric indicating the proportion of patients who showed a response to the drug, was 75.3 percent....Following these promising results, Curocell plans to apply for domestic drug approval in the latter half of this year."

P2 data • Regulatory • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology