HONG KONG, June 10, 2021 /PRNewswire/ -- CBMG Holdings (or the "Company"), a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, today announced updated clinical data for C-CAR039, a novel CD19/CD20 bi-specific CAR-T cell product in relapsed or refractory (r/r) B-cell non-Hodgkin lymphoma (B-NHL). This work was presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, by the Principal Investigator (PI) of the study, Aibin Liang, M.D., Professor of Department of Hematology, Shanghai Tongji Hospital, Tongji University School of Medicine.
Additionally, the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted the Company an orphan drug designation (ODD) to C-CAR039, for the treatment of follicular lymphoma, an indolent form of Non-Hodgkin's Lymphoma.
"C-CAR039 preliminary data has demonstrated an early favorable safety profile and encouraging efficacy in clinical trial in patients with r/r B-NHL," commented Tony (Bizuo) Liu, Chairman and CEO of CBMG Holdings. "The high response rate, especially the CR rate in r/r DLBCL is likely to allow the asset to differentiate from existing therapies. The clinical efficacy data compares favorably to any existing anti-CD19 CAR-T therapies. We are also pleased that the FDA has recognized C-CAR039's potential as a treatment option for follicular lymphoma in granting it ODD. This designation speaks to our therapy already showing promise in treating such serious illness and marks another significant regulatory milestone for our C-CAR039 product. We are excited to accelerate the development of C-CAR039 and to bring its potential therapeutic benefits to patients all over the world."
About the Study
C-CAR039 has been developed as a novel 2nd generation 4-1BB bi-specific CAR-T targeting both CD19 and CD20 antigens with an optimized bi-specific antigen binding domain. C-CAR039 can eradicate CD19/CD20 single or double positive tumor cells in vitro and in vivo. GMP manufacturing of C-CAR039 was carried out in a serum free and fully closed semi-automatic system. Dose escalation and expansion studies were conducted to evaluate the safety and efficacy of C-CAR039 in r/r B-NHL patients.
In the Phase I clinical trials in China (NCT04317885, NCT04655677, NCT04696432, NCT04693676), dose escalation and expansion studies were conducted to evaluate the safety and efficacy of C-CAR039 in r/r B-NHL patients. C-CAR039 was administered as a single intravenous dose after a 3-day cyclophosphamide (300mg/m2x3d) plus fludarabine (30mg/m2x3d) conditioning regimen. The median manufacturing time was 6 days and the median vein to vein time was 19 days.
As of April 20, 2021, 34 patients were infused with C-CAR039. Among them, 28 (DLBCL, n = 25; PMBCL, n = 1; tFL, n=1; FL, n=1) patients had more than 1 month safety data and 27 were evaluable for efficacy at dose ranges of 1.0 x 106 to 5.0x106 CAR-T cells/kg (1 patient did not have evaluable disease on the day of C-CAR039 infusion). The median age was 55.5 (range, 28-71) years, median number of prior lines of therapy was 3 (range, 1-5), 75% (21/28) of patients were in Ann Arbor Stage III/IV, and 28.6% (8/28) never achieved CR to their prior treatments. 5 patients (17.9%) received bridging therapy.
Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were graded according to ASTCT 2019 criteria. 92.9% (26/28) of patients experienced CRS. 25 of 26 were grade 1 or 2. Only 1 patient experienced grade 3 CRS. All CRS are reversible. 2 patients experienced a grade 1 ICANS. Grade≥3 neutropenia, anemia, thrombocytopenia and infection were reported in 89.3%, 32.1%, 25% and 3.6% of patients, respectively.
At a median follow-up of 7.0 months, the best overall response rate was 92.6% (25/27). The complete response (CR) rate was 85.2% (23/25). Of the 24 DLBCL patients, 20 (83.3%) had complete response. The median time to response was 1.0 month (range, 0.9-1.6). The Kaplan Meyer estimation of PFS at 6 months was 83.2% (95% CI, 69.1 to 100.0). The median duration of response has not been reached.
The Company plans to submit an IND to the US FDA later this year and initiate a Phase 1b study in the first half of 2022 based on communication with FDA.
About CBMG Holdings
CBMG Holdings develops proprietary cell therapies for the treatment of cancer and degenerative diseases. The Company operates a state-of-the-art facility in Rockville, Maryland with five GMP rooms in order to augment its global research and development capabilities and to support clinical development of multiple cell therapy platform technologies in the United States. The company conducts immuno-oncology and stem cell clinical trials in China using products from its integrated GMP laboratory. The Company's GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. The Company currently has ongoing CAR-T Phase I clinical trials in China. The China NMPA (formerly CFDA) approved the Company's IND application for a Phase II trial for AlloJoin®, its "Off-the-Shelf" allogenic haMPC therapy for the treatment of Knee Osteoarthritis (KOA), and has accepted the Company's IND application for a Phase II trial for ReJoin® autologous haMPC therapy for the treatment of KOA.
Statements in this communication relating to plans, strategies, specific activities, and other statements that are not descriptions of historical facts are forward-looking statements. Forward-looking information is inherently subject to risks and uncertainties, and actual results could differ materially from those currently anticipated due to a number of factors, which include any risks detailed from time to time in CBMG Holding's reports, including risks relating to the impact of the COVID-19 pandemic on our operations, including risks associated with the evolving COVID-19 pandemic and actions taken in response to it. Such statements are based on the current beliefs and expectations of the management of the Company and are subject to significant risks and uncertainties outside of the Company's control. Given these uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as otherwise required by law, CBMG Holdings does not undertake any obligation, and expressly disclaims any obligation, to update, alter or otherwise revise any forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future events or otherwise.
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SOURCE CBMG Holdings