On December 13, 2021 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer, reported that positive interim Phase 1 data from the Company’s FT596 program for patients with relapsed / refractory B-cell lymphoma (BCL) at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (Press release, Fate Therapeutics, DEC 13, 2021, View Source [SID1234597016]). FT596 is the Company’s off-the-shelf, multi-antigen targeted, iPSC-derived natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology that targets B-cell antigen CD19; a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor that has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"The interim dose-escalation clinical data from our FT596 program in relapsed / refractory B-cell lymphoma demonstrate that off-the-shelf, iPSC-derived CAR NK cells can bring substantial therapeutic benefit to heavily pre-treated patients in urgent need of therapy, with high response rates and meaningful duration of responses," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "We are particularly pleased with the therapeutic profile that has emerged with FT596 in combination with rituximab, where over half of the patients treated with a single dose of FT596 at higher dose levels achieved a complete response with a favorable safety profile that is clearly differentiated from CAR T-cell therapy. We look forward to assessing a two-dose treatment schedule for FT596 to further define its potential best-in-class therapeutic profile and ability to reach more patients, including those earlier in care."

The ongoing Phase 1 study in relapsed / refractory BCL is assessing a single dose of FT596 as monotherapy (Monotherapy Arm) and in combination with a single dose of rituximab (375 mg/m2) (Combination Arm) following three days of conditioning chemotherapy (500 mg/m2 of cyclophosphamide and 30 mg/m2 of fludarabine). Certain patients are eligible for re-treatment with a second, single-dose cycle.

The ASH (Free ASH Whitepaper) presentation (Session 704—Cellular Immunotherapies: Expanding Targets and Cellular Sources for Immunotherapies, Abstract 823) includes clinical data from 25 evaluable patients for safety (n=12 in Monotherapy Arm; n=13 in Combination Arm) in the first, second, and third single-dose cohorts of 30 million, 90 million, and 300 million cells, respectively, of which 24 patients were also evaluable for efficacy (n=12 in Monotherapy Arm; n=12 in Combination Arm), as of the data cutoff date of October 11, 2021. These 25 patients had received a median of four prior lines of therapy and a median of two prior lines containing CD20-targeted therapy. Of the 25 patients, 15 patients (60%) had aggressive B-cell lymphoma, 15 patients (60%) were refractory to most recent prior therapy, and 8 patients (32%) were previously treated with autologous CD19-targeted CAR T-cell therapy. Subsequent to the data cutoff date for the ASH (Free ASH Whitepaper) presentation, an additional patient in the third single-dose cohort of the Combination Arm was evaluable for initial anti-tumor response, and seven patients in the fourth single-dose cohort of 900 million cells (n=1 in Monotherapy Arm; n=6 in Combination Arm) were evaluable for safety and initial anti-tumor response.

Single-dose, Single-cycle Response Data

In the second, third, and fourth dose cohorts of the Monotherapy and Combination Arms comprising a total of 26 patients, 18 patients (69%) achieved an objective response, including 12 patients (46%) that achieved a complete response, on Day 29 following a single dose of FT596 (see Table 1). Nine of these 26 patients were previously treated with autologous CD19-targeted CAR T-cell therapy and, of these nine patients, six achieved an objective response (67%) on Day 29 following a single dose of FT596. Notably, in the third and fourth dose cohorts of the Combination Arm comprising a total of 12 patients, nine patients (75%) achieved an objective response, including seven patients (58%) that achieved a complete response, on Day 29 following a single dose of FT596.

Durability of Response Data

The ASH (Free ASH Whitepaper) presentation includes durability of response data from 13 responding patients in the second and third single-dose cohorts of 90 million cells and 300 million cells (n=9 in Monotherapy Arm; n=10 in Combination Arm). As of the data cutoff date of October 11, 2021, 10 patients continued in ongoing response, including three patients in ongoing complete response at least six months from initiation of treatment; two patients reached six months in complete response and subsequently had disease progression; and one patient had disease progression prior to six months. Of these 13 responding patients:

Monotherapy Arm (n=7 responding patients). Five patients, all of whom were treated with a second FT596 single-dose cycle with the consent of the U.S. Food and Drug Administration (FDA), continued in ongoing response at a median follow-up of 4.1 months, including one patient in ongoing complete response at 8.1 months; one patient, who was treated with only one FT596 single-dose cycle, reached six months in complete response and subsequently had disease progression at 6.5 months; and one patient, who was treated with only one FT596 single-dose cycle, had disease progression at 1.7 months.
Combination Arm (n=6 responding patients). Five patients, all of whom were treated with a second FT596 single-dose cycle with the consent of the FDA, continued in ongoing response at a median follow-up of 4.6 months, including two patients in ongoing complete response at 6.0 and 10.8 months; and one patient, who was treated with a second FT596 single-dose cycle with the consent of the FDA, reached six months in complete response and subsequently had disease progression at 6.7 months.
Table 1. FT596 Interim Phase 1 Data – Day 29 Response Assessment 1
1 Dose x 1 Cycle Monotherapy
(n=13) Combination
(n=19)
Single-dose Level Cohorts (Cells) OR CR OR CR
30M 1/3 (33%) 0 0/3 (0%) 0
90M 3/4 (75%) 2 2/4 (50%) 2
300M 2 4/5 (80%) 1 4/6 (67%) 3
900M 2 0/1 (0%) 0 5/6 (83%) 4
aCD19 History (≥90M Cells) n=10 n=16
Naïve 7/9 (78%) 3 5/8 (63%) 4
Prior 0/1 (0%) 0 6/8 (75%) 5
Disease Histology (≥90M Cells) n=10 n=16
Aggressive 1/3 (33%) 0 6/11 (55%) 4
Mantle cell 0/1 (0%) 0 2/2 (100%) 2
Indolent 6/6 (100%) 3 3/3 (100%) 3
aCD19 = autologous CD19-targeted CAR T-cell therapy; Aggressive = diffuse large B-cell lymphoma, Grade 3b follicular lymphoma, Richter’s transformation, and high-grade B-cell lymphoma; CR = complete response; Indolent = splenic diffuse red pulp small B-cell lymphoma, non-Grade 3b follicular lymphoma, Waldenstrom’s macroglobulinemia, and small lymphocytic lymphoma; M = million; OR = objective response
1 As of data cutoff date of October 11, 2021, unless otherwise noted. Objective response and complete response are based on Cycle 1 Day 29 protocol-defined response assessment per Lugano 2014 criteria. Data subject to source document verification.
2 Cycle 1 Day 29 protocol-defined response assessment completed subsequent to data cutoff date for one patient in the third single-dose cohort of 300 million cells in the Combination Arm and seven patients in the fourth single-dose cohort of 900 million cells (n=1 in Monotherapy Arm; n=6 in Combination Arm).

Safety Data

The FT596 treatment regimens were well tolerated, including in those patients treated with a second, single-dose cycle. No dose-limiting toxicities, and no treatment-emergent adverse events (TEAEs) of any grade of immune effector cell-associated neurotoxicity syndrome (ICANS) or graft-versus-host disease (GvHD) were observed. Three low-grade adverse events (two Grade 1, one Grade 2) of cytokine release syndrome (CRS) were reported, which were of limited duration and resolved without intensive care treatment (see Table 2).

The Company has initiated enrollment of a two-dose treatment schedule in the Combination Arm, with FT596 administered on Day 1 and Day 15 at 900 million cells per dose. Patients with clinical benefit following administration of the first two-dose cycle are eligible for re-treatment with a second two-dose cycle. Additionally, patients with clinical response are eligible for re-treatment following disease progression.

Table 2. FT596 Interim Phase 1 Data – TEAEs of Interest
n (%)
Monotherapy
(n=13) Combination
(n=19)
All Grade Grade 3+ All Grade Grade 3+
CRS 1 (8%) — 2 (11%) —
ICANS — — — —
GvHD — — — —
Infections 6 (46%) 3 (23%) 6 (32%) 3 (16%)
FT596-related SAEs — — 1 (5%) a —
CRS = Cytokine Release Syndrome; GvHD = Graft vs. Host Disease; ICANS = Immune Cell-Associated Neurotoxicity Syndrome; TEAE = Treatment-Emergent Adverse Event; SAE = Severe Adverse Events
a Grade 2 CRS

Investor Event Webcast

The Company will host a live audio webcast on Tuesday, December 14, 2021 at 8:00 a.m. ET to highlight interim Phase 1 clinical data from the Company’s FT516 and FT596 programs for the treatment of relapsed / refractory B-cell lymphoma. The live webcast can be accessed under "Events & Presentations" in the Investors section of the Company’s website at www.fatetherapeutics.com. The archived webcast will be available on the Company’s website beginning approximately two hours after the event.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT596
FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that augments NK cell activity. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and hnCD16 targeting receptors enhances cytotoxic activity, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a humanized mouse model of lymphoma, FT596 in combination with the anti-CD20 monoclonal antibody rituximab showed enhanced killing of tumor cells in vivo as compared to rituximab alone. FT596 is being investigated in a multi-center Phase 1 clinical trial for the treatment of relapsed / refractory B-cell lymphoma as a monotherapy and in combination with rituximab, and for the treatment of relapsed / refractory chronic lymphocytic leukemia (CLL) as a monotherapy and in combination with obinutuzumab (NCT04245722).

On December 13, 2021 Biosion, Inc. ("Biosion"), a global, clinical stage biotechnology company, reported that Biosion and OBI Pharma Inc. (4174.TWO) ("OBI Pharma") have signed an exclusive license agreement under which OBI Pharma will be granted a global exclusive license to Biosion’s proprietary anti-Trop2 humanized monoclonal antibody, BSI04702, for developing next generation biologics (Press release, Biosion, DEC 13, 2021, View Source;and-other-derivative-products-301442963.html [SID1234597014]). The license agreement enables OBI Pharma to conduct further preclinical and clinical development, registration, and commercialization of BSI04702 as an Antibody Drug Conjugate and other derivative products. Under the terms of the agreement, OBI Pharma will pay license fees to Biosion, including an upfront payment, future development milestones and net sales royalties. The specific terms of the agreement were not disclosed.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Biosion has been driving innovation to deliver breakthrough therapies for cures. The licensing of our anti-TROP-2 mAb to OBI Pharma for global development further exhibits the strength of our discovery engine and proprietary SynTracerTM HT-endocytosis platform to identify superior mAbs, ideal as ADCs" said Dr. Hugh Davis, Chief Operating Officer of Biosion, Inc. and President of Biosion USA. "We are looking forward to partnering with OBI Pharma to advance BSI-04702 into the clinical stage as fast as possible and making a difference for patients worldwide."

BSI04702 was created through Biosion’s proprietary SynTracerTM HT-endocytosis platform, a high-throughput endocytosis screening application that can identify antibody candidates with high internalization rates- a critical parameter for lead antibody success in next generation ADC development.

On December 13, 2021 Bionomics Limited (Bionomics or Company), a clinical-stage biopharmaceutical company, reported the launch of its initial public offering (the Offering) of 1,620,000 American Depositary Shares (ADSs), each representing 180 ordinary shares, in the United States (Press release, Bionomics, DEC 13, 2021, View Source [SID1234596999]). The target size of the of the Offering is US$25.0 million in gross proceeds.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

All ADSs to be sold in the Offering will be sold by Bionomics. In addition, Bionomics expects to grant the underwriters an option to purchase up to an additional 243,000 ADSs within 30 days from the date of the final prospectus at the initial public offering price, less underwriting discounts and commissions.

Bionomics has applied to have its ADSs listed on the Nasdaq Global Market under the symbol "BNOX." Bionomics’ ordinary shares are currently traded on the Australian Securities Exchange (ASX) under the symbol "BNO."

Evercore ISI and William Blair are acting as lead book-running managers for the Offering. Cantor, Berenberg and H.C. Wainwright & Co. are acting as book-running managers for the Offering.

The Offering will be made only by means of a prospectus under the U.S. Securities Act of 1933. When available, copies of the preliminary prospectus relating to and describing the terms of the Offering may be obtained from (i) Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, New York 10055, or by telephone at (888) 474-0200, or by email at [email protected] ; or (ii) William Blair & Company, L.L.C., Attention: Prospectus Department, 150 North Riverside Plaza, Chicago, Illinois 60606, or by telephone at (800) 621-0687, or by email at [email protected] . Australian investors are only eligible to invest under the prospectus if they are exempt from disclosure as sophisticated or professional investors under the Corporations Act 2001 (Cth).

A registration statement relating to these securities has been filed with the U.S. Securities and Exchange Commission but has not yet become effective. These securities may not be sold, nor may offers to buy these securities be accepted, prior to the time the registration statement becomes effective. This press release shall not constitute an offer to sell or a solicitation of an offer to buy the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such an offer, solicitation, or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. As disclosed in the registration statement, the underwriters may purchase and sell ADSs in the open market, including to cover over-allotments.

Released on authority of the Company Secretary.

On December 13, 2021 Nektar Therapeutics (NASDAQ:NKTR) reported two data presentations from the dose-escalation portion of its ongoing Phase 1 study of NKTR-255 in patients with relapsed/refractory hematologic malignancies at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Nektar Therapeutics, DEC 13, 2021, View Source [SID1234596998]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Initial clinical results from the Phase 1 study of NKTR-255 in patients with relapsed/refractory (R/R) hematologic malignancies were presented by Nina Shah, M.D., Associate Professor, Department of Medicine, at the University of California San Francisco on Monday, December 13th. A pharmacodynamic analysis of CAR-T cell persistence in patients treated in the Phase 1 study who had received prior treatment with CAR-T therapy was also presented by Alexandre V. Hirayama, M.D., Fred Hutchison Cancer Research Center on Sunday, December 12th.

"The data presented at this year’s ASH (Free ASH Whitepaper) meeting underscore the potential of NKTR-255 and provide clinical evidence of its unique ability to trigger the induction of natural killer and CD8+ T cells and highlight its potential role in rescuing and enhancing CAR-T cell persistence," said Jonathan Zalevsky, Ph.D., Head of Research and Development at Nektar. "Engaging the full spectrum of IL-15 biology, NKTR-255 can be combined with multiple mechanisms to potentially improve their efficacy. The clinical data presented at ASH (Free ASH Whitepaper) support the development of NKTR-255 in combination with anticancer agents that induce antibody dependent cellular toxicity as well as CAR-T therapies. We look forward to completing the dose escalation portion of this Phase 1 study in the first half of 2022 and further investigating NKTR-255 in combination with rituximab or daratumumab for patients with non-Hodgkin’s lymphoma or multiple myeloma."

2021 ASH (Free ASH Whitepaper) presentations are available for download at View Source

Highlights from the presentations are as follows:

Abstract #3134: "Safety, Tolerability, PK/PD, and Preliminary Efficacy of NKTR-255, a Novel IL-15 Receptor Agonist, in Patients with Relapsed/Refractory Hematologic Malignancies," Shah, N., et al.

NKTR-255 was well tolerated and early evidence of clinical activity was observed in this heavily pre-treated and highly refractory patient population with hematologic malignancies. Treatment-related adverse events were generally low-grade, transient and easily managed.
Evidence of on-target biological activity was observed despite highly compromised bone marrow hematopoietic capacity; NKTR-255 led to expansion and proliferation of natural killer (NK) and CD8+ T cells.
Among the 15 response-evaluable patients, 8 (53%) reported disease stabilization (5/8 [63%] patients with multiple myeloma (MM); 3/7 [43%] patients with non-Hodgkin lymphoma (NHL)).
One patient with NHL (with 3 prior lines of therapy) was treated at the 1.5 µg/kg dose and experienced a metabolic response in a splenic target lesion at cycle 5.1
Two patients with MM (with 3 prior lines of therapy) were treated at the 1.5 µg/kg and the 4.5 µg/kg doses and experienced disease stabilization for greater than 180 days.
The maximum tolerated dose/recommended Phase 2 dose has not been reached and dose escalation of NKTR-255 is ongoing.
Abstract #2815: "Pharmacodynamic Analysis of CAR-T Cell Persistence in Patients with Hematologic Malignancies Treated with NKTR-255, an IL-15 Receptor Agonist That Enhances CD8+ T cells: Preliminary results from a Phase 1 Study," Hirayama, A., et al.

This is the first clinical safety and pharmacodynamic assessment of the effects of any IL-15 agent on CAR-T cell counts in relapsed/refractory NHL or MM patients who had progressed or relapsed after CAR-T therapy.
Of the patients with detectable CAR-T cells in blood at baseline, 100% (4/4) showed an increase of CD3+ CAR-T cells following NKTR-255 treatment. NKTR-255 induced proliferation of CD8+ T cells and an increase of the total CD8+ cell fraction in all patients with CAR-T cells at baseline.
These preliminary data suggest that NKTR-255 administration represents a potentially novel means of CAR-T augmentation through enhancement and persistence of CD8+ T cells via generation of long-term memory CD8+ and provides promising evidence of CAR-T cell rescue.
Results support planned evaluation of NKTR-255 in combination with CAR-T therapy as a potential strategy to enhance the efficacy of CAR-T therapy.
NKTR-255 is currently being evaluated in dose-escalation in a Phase 1 study in patients with R/R non-Hodgkin’s lymphoma and R/R multiple myeloma (NCT04136756) and in a Phase 1b/2 trial in combination with ERBITUX for the treatment of R/R colorectal cancer and R/R squamous cell carcinoma of the head and neck (NCT04616196). In collaboration with Merck KGaA, Darmstadt, Germany, Nektar will also be investigating NKTR-255 in combination with BAVENCIO, a PD-L1 inhibitor, in patients with locally advanced or metastatic urothelial carcinoma in the randomized Phase II JAVELIN Bladder Medley study.

About NKTR-255
NKTR-255 is a novel polyethylene glycol-conjugate of recombinant human interleukin-15, which was designed to retain all known receptor binding interactions of the IL-15 molecule. The investigational candidate is uniquely designed to overcome known challenges of recombinant IL-15 and other IL-15 agonists, which are rapidly cleared from the body and have shown diminishing response to successive doses. Through an extended circulating half-life and optimal engagement of the IL-15Rα/IL-2Rβγ receptor complex, NKTR-255 enhances functional natural killer cell populations and formation of long-term CD8+ mediated immunological memory, which may lead to sustained anti-tumor immune response.

On December 13, 2021 Cellular Biomedicine Group Inc. (CBMG or the "Company"), a biopharmaceutical company developing innovative cellular immunotherapies for the treatment of cancer, reported that the Food and Drug Administration (FDA) granted clearance of the Investigational New Drug (IND) application to proceed with the Phase 1b clinical development of its chimeric antigen receptor (CAR) T-cell therapy targeting CD19 and CD20 (C-CAR039) (Press release, Cellular Biomedicine Group, DEC 13, 2021, View Source [SID1234596995]). C-CAR039 is a novel autologous bi-specific CAR-T therapy targeting both CD19 and CD20 antigens in the treatment of patients with relapsed or refractory non-Hodgkin lymphoma (r/r B-cell NHL).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"This is fantastic news for us as we believe we may potentially have best-in-class therapeutics for patients with poor prognosis. The largest subtype of NHL is DLBCL, and those refractory patients whose treatment has failed have limited options. We look forward to initiating the C-CAR039 trial soon," said Tony (Bizuo) Liu, Chairman and CEO. "The FDA’s C-CAR039 IND application clearance is a significant milestone for CBMG. We are fond of our manufacturing, quality control, and clinical development teams for their dedication to our mission of ‘Saving Lives and Revitalizing Lives’ and bringing to market innovative targeted therapies to address cancers."

Yihong Yao, PhD, Chief Scientific Officer of CBMG, commented, "We are excited about the potential of C-CAR039 as a best-in-class treatment in r/r B-NHL. C-CAR039 clearly showed potential superior efficacy and favorable safety results for patients with r/r B-cell NHL. Our C-CAR039 trial is a testament of the teams’ translational medicine capability. We look forward to bringing more potentially promising development to clinical trials."

About C-CAR039
Early clinical results of C-CAR039 from an investigator-initiated trial (IIT) conducted across multiple sites in China demonstrate exciting efficacy and favorable safety data of C-CAR039 in r/r B-cell NHL. As of April 20, 2021, a total of 34 patients received C-CAR039 cell therapies, with 28 patients evaluable for safety analyses and 27 patients evaluable for efficacy analyses. Patients’ median age was 55.5 years, and 75% had cancer of Ann Arbor stage III/IV. Patients had a median of three prior lines of therapy. Bridging therapy had been given to 17.9% of patients. The best overall response rate (ORR) was reported to be 92.6%, with a complete response rate (CRR) of 85.2%. Patients had a median time to response of 1.0 month, and at a median follow-up of 7 months, 74.1% of patients continued to be in complete remission. The 6-month estimated progression-free survival rate was 83.2% (95% CI, 69.1%-100.0%). Cytokine release syndrome (CRS) occurred in 96% of patients. 92% of CRS was of grade 1/2 and only 1 patient had grade 3 CSR. Immune effector cell-associated neurotoxicity syndrome occurred at grade 1 in 2 patients and no ≥grade 2 neurologic events reported in the study. CBMG will continue to evaluate patients with longer follow-up (ClinicalTrials.gov Identifiers: NCT04317885, NCT04655677, NCT04696432, NCT04693676).

Separately, in June 2021, the FDA Office of Orphan Products Development granted CBMG an Orphan Drug Designation to C-CAR039 for the treatment of Follicular Lymphoma, an indolent form of non-Hodgkin lymphoma.

About CBMG
Cellular Biomedicine Group Inc. (CBMG) is a wholly owned subsidiary of CBMG Holdings. CBMG Holdings ("Holdings") develops proprietary cell therapies for the treatment of cancer and degenerative diseases. CBMG 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. Holdings conducts immuno-oncology and stem cell clinical trials in China using products from its integrated GMP laboratory. Holdings’ GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. Holdings currently conducts ongoing studies in China, for CAR-T therapies targeting blood cancers, including C-CAR039, an anti-CD19, CD20 BiCAR treatment for non-Hodgkin lymphoma (NHL) and CAR088, an anti-BCMA treatment for Multiple Myeloma, in addition to T cell receptor (TCR-T) and tumor-infiltrating lymphocytes (TIL) therapies targeting solid tumors. Holdings has completed patient treatment in a Phase II trial for AlloJoin, its "Off-the-Shelf" allogenic haMPC therapy for the treatment of Knee Osteoarthritis (KOA), and a Phase II trial for ReJoin autologous haMPC therapy for the treatment of KOA.