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AbelZeta Receives FDA Clearance of Registrational Phase II Trial for C-CAR168 in Refractory Lupus Nephritis

ROCKVILLE, MD and SHANGHAI July 27, 2026 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced that the Company received the U.S. Food and Drug Administration (FDA) clearance under its Regenerative Medicine Advanced Therapy (RMAT) designation regarding the multiple-center registrational Phase II clinical trial for C-CAR168, an anti-CD20/BCMA bispecific CAR-T, in the treatment of patients with Lupus Nephritis (LN) refractory to standard therapy. The Phase II trial will further evaluate the safety and efficacy of C-CAR168 in this patient population with significant unmet medical needs.

“The FDA clearance to proceed with this multiple-center registrational Phase II trial for C-CAR168 in LN represents an important step forward in the development of this promising new modality for patients with severe, treatment-refractory lupus. This is a population with severe unmet medical needs,” said Tony (Bizuo) Liu, Chairman and Chief Executive Officer of AbelZeta. “Together with the RMAT and PRIority MEdicines (PRIME) designations granted by the FDA and European Medicines Agency (EMA), respectively, this regulatory progress underscores the tremendous potential of C-CAR168 to address a significant unmet need for autoimmune diseases. We look forward to effectively advancing the program globally. We hope this drug can deliver deep and durable clinical benefits for LN patients. We are also actively and aggressively evaluating the potential of C-CAR168 in other major disease indications.”

About C-CAR168
C-CAR168 is a novel autologous bi-specific CAR-T therapy targeting both CD20 and BCMA. C-CAR168 is designed to support deep depletion of disease-driving B cells and plasma cells, with the goal of inducing an immune reset in patients with severe autoimmune diseases, including refractory Systemic Lupus Erythematosus (SLE) and LN, progressive Multiple Sclerosis (MS) and others. The FDA and the EMA have granted RMAT and PRIME designations, respectively, to C-CAR168 for the treatment of refractory SLE including LN.

About SLE and LN
SLE is a chronic autoimmune disease characterized by systemic inflammation and multi-organ involvement. LN is a serious manifestation of SLE involving inflammation of the kidneys and can lead to progressive kidney damage and kidney failure. Despite available standards of care, patients with refractory disease continue to face significant unmet medical needs, including persistent disease activity, risk of organ damage, and cumulative toxicity associated with long-term immunosuppressive treatment.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies, inflammatory and immunological diseases and solid tumors. AbelZeta advances research and development in its own GMP facilities at its centers of excellence, with a pipeline comprised of multiple CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

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AbelZeta announces publication in Nature journal highlighting clinical findings for an armored autologous GPC3 CAR-T therapy in patients with advanced hepatocellular carcinoma

ROCKVILLE, MD and SHANGHAI, July 23, 2026 – AbelZeta Pharma, Inc. (“AbelZeta” or “the Company”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced a publication in Nature sharing the results of the first-in-human clinical study evaluating the safety and efficacy of C-CAR031, a novel glypican-3 (GPC3)-targeted CAR-T that AbelZeta and AstraZeneca previously partnered to develop in China, and which is now being developed solely by AstraZeneca as AZD7003. The publication provides an update to the preliminary safety and efficacy data presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. The investigator-initiated trial (IIT) reported in the Nature publication was led by The First Affiliated Hospital, Zhejiang University School of Medicine’s Dr. Tingbo Liang and Dr. Qi Zhang with the support of AbelZeta.

C-CAR031/AZD7003 is an autologous glypican 3 (GPC3)-targeting CAR-T therapy, designed by AstraZeneca using their dominant-negative transforming growth factor-beta receptor II (dnTGFβRII) armoring. The dnTGFβRII armoring platform is designed to overcome the immunosuppressive tumor microenvironment.  

The publication reports clinical findings that indicate a manageable safety profile and encouraging antitumor activity for C-CAR031/AZD7003 in 36 patients with heavily pretreated advanced hepatocellular carcinoma (HCC) [median four lines of prior treatment].

Efficacy

  • Tumor regression was observed in 32 / 36 patients, with a median best tumour reduction from baseline of 41.6% (range: 3.4–94.4%) in target lesions.
  • Objective response rate (ORR) was 44.4%, and the median duration of response (DOR) was 4.4 months (95% confidence interval: 2.9–7.4).
  • Median progression-free survival (PFS) was2 months (95% confidence interval: 2.9–4.8).
  • Median overall survival (OS) was2 months (95% confidence interval: 10.1 to not evaluable).

Safety

  • 2 patients had cytokine release syndrome of grade 3.
  • 9 patients had non-hematological adverse events of grade 3 or higher.

“We would like to express our deepest appreciation to The First Affiliated Hospital, Zhejiang University School of Medicine’s Dr. Liang and Dr. Zhang, as well as the outstanding clinical team for conducting this landmark study with exceptional scientific rigor,” said Tony (Bizuo) Liu, Chairman and Chief Executive Officer of AbelZeta, “We are especially grateful to the patients and their families who participated in this trial, whose courage and trust made this important research possible.”

“We are delighted to have collaborated with AstraZeneca on C-CAR031/AZD7003 and are honored to see the clinical data in hepatocellular carcinoma published in Nature.  Reaching this milestone is testament to our complementary cell therapy capabilities.”

In December 2023, AbelZeta announced an agreement with AstraZeneca to co-develop C-CAR031 in China. In January 2026, AbelZeta announced entering into an agreement where AstraZeneca would acquire AbelZeta’s remaining China development and commercialization rights to C-CAR031, such that AstraZeneca now solely leads the global development and commercialization of the investigational product, now known as AZD7003.

References
The Nature publication, titled, “GPC3-specific dnTGFβRII–armoured CAR-T cells for hepatocellular carcinoma” can be accessed at https://www.nature.com/articles/s41586-026-10786-z .

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies, inflammatory and immunological diseases and solid tumors. AbelZeta advances research and development in its own GMP facilities at its centers of excellence, with a pipeline comprised of multiple CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

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AbelZeta Receives EMA PRIME Designation for C-CAR168 in Lupus Nephritis and Systemic Lupus Erythematosus

ROCKVILLE, Md. and SHANGHAI, July 20, 2026 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted PRIority MEdicines (PRIME) designation for C-CAR168, the Company’s novel autologous anti-CD20/BCMA CAR-T product candidate, for the treatment of refractory Systemic Lupus Erythematosus (SLE), with or without Lupus Nephritis (LN).

The granting of EMA PRIME designation marks one of the first major regulatory recognitions of a CAR-T therapy for SLE, highlighting both the significant unmet need faced by lupus patients and the potential of this approach to transform treatment. Building on the clinical promise demonstrated by earlier CAR-T therapies, AbelZeta has applied extensive expertise in developing dual-target CAR-T technology to engineer its dual anti-CD20/BCMA-targeted CAR-T. This next-generation construct has been rationally designed to more effectively address the underlying immunopathology of lupus by targeting both autoreactive B cells and antibody-producing plasma cells, with the goal of achieving deeper, more durable remissions while maintaining a favorable safety profile.

“The PRIME designation represents another significant milestone for AbelZeta’s scientific capabilities and further demonstrates that well-designed early clinical trials can generate highly clinically meaningful results that translate into regulatory recognition and inform global drug development. This recognition reflects the strength of our scientific platform, the dedication of our team, and the significant progress we have made in expanding cell therapy well beyond oncology,” said Tony (Bizuo) Liu, Chairman and Chief Executive Officer of AbelZeta. “It provides important momentum as we advance C-CAR168 through global development in multiple disease indications and further establishes AbelZeta as a leader in cell therapy for autoimmune diseases.”

In May 2025, the U.S. Food and Drug Administration (FDA) also granted Regenerative Medicine Advanced Therapy (RMAT) designation to C-CAR168 for the treatment of refractory SLE, including LN.

About EMA PRIME
PRIority MEdicines, or PRIME, is a scheme established by the EMA, granted to promising medicines that may address significant unmet medical needs and that provides enhanced regulatory support, including early dialogue and scientific advice, to help optimize development and potentially accelerate evaluation.

About C-CAR168
C-CAR168 is a novel autologous bi-specific CAR-T therapy targeting both CD20 and BCMA. C-CAR168 is designed to support deep depletion of disease-driving B cells and plasma cells, with the goal of inducing an immune reset in patients with severe autoimmune diseases, including refractory SLE and LN, progressive Multiple Sclerosis (MS) and others.

About SLE and LN
SLE is a chronic autoimmune disease characterized by systemic inflammation and multi-organ involvement. LN is a serious manifestation of SLE involving inflammation of the kidneys and can lead to progressive kidney damage and kidney failure. Despite available standards of care, patients with refractory disease continue to face significant unmet medical needs, including persistent disease activity, risk of organ damage, and cumulative toxicity associated with long-term immunosuppressive treatment.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies, inflammatory and immunological diseases and solid tumors. AbelZeta advances research and development in its own GMP facilities at its centers of excellence, with a pipeline comprised of multiple CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

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AbelZeta Announces Remaining China Rights to GPC3 Armored CAR-T Therapy to be Acquired by AstraZeneca

ROCKVILLE, MD and SHANGHAI, CHINA January 18, 2026 – AbelZeta Pharma, Inc. (“AbelZeta”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, announced that AstraZeneca has agreed to acquire AbelZeta’s 50% share of the China development and commercialization rights to C-CAR031, such that AstraZeneca acquires the sole right to develop, manufacture and commercialize C-CAR031 globally. Under the terms of the agreement, AbelZeta will be entitled to receive up to $630 million from AstraZeneca including an upfront payment, and development, regulatory and sales milestone payments for the GPC3 program in China.

Under the terms of a prior agreement with AbelZeta, AstraZeneca owns the development, manufacturing and commercialization rights to C-CAR031 in rest of world, outside of China. AbelZeta is also eligible to receive additional milestone payments and royalties for rest of world development.

“This transaction reflects our commitment to leverage our platform technology to develop novel cell therapies in solid tumors of high unmet medical need, including Hepatocellular carcinoma (HCC), and provides the opportunity to maximize C-CAR031’s global reach” said Tony (Bizuo) Liu, Chairman and CEO of AbelZeta.

C-CAR031 is an autologous, Glypican 3 (GPC3)-targeting chimeric antigen receptor T-Cell (CAR-T) therapy, designed using AstraZeneca’s dominant negative transforming growth factor-beta receptor II armoring platform, and is currently being investigated for the treatment of HCC and other solid tumors.

About HCC
Liver cancer is the third-leading cause of cancer death and the sixth most commonly diagnosed cancer worldwide. Several types of primary liver cancer occur in adults, HCC being the most common form. About 75% of all primary liver cancers in adults are HCC. Liver cancers are categorized based on the originating cell type. HCC begins in the liver as either a single tumor or several small nodules and at this local stage, it can be treated by locally targeted or surgical methods. However, most patients are diagnosed at advanced-stage, or their disease progresses to advanced-stage HCC when the prognosis is poor, with a 5-year survival rate of only 7% and a median survival of approximately 20 months. According to Frost & Sullivan, the incidence of HCC in China has been growing steadily to approximately 344,500 cases in 2024.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies, inflammatory and immunological diseases and solid tumors. AbelZeta advances research and development in its own GMP facilities at its centers of excellence, with a pipeline comprised of multiple CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

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AbelZeta to Attend 44th Annual J.P. Morgan Healthcare Conference and Join Panel Discussion

ROCKVILLE, MD, January 6, 2026 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, will attend  the 44th Annual J.P. Morgan Healthcare Conference on January 12-15, 2026 in San Francisco, CA, and host meetings showcasing the Company’s recent accomplishments and sharing strategic plans moving forward. The Company’s Chairman and CEO, Tony (Bizuo) Liu, has also been invited as a panel speaker at the Conference.

Session:            Emerging Biotechs from China
Time & Date:    January 14, 2026, 16:30 – 17:15 PST
Location:          Pacific Hall, Convene, 40 O’Farrell St, San Francisco
Description:     China is emerging as a hub of global biotech innovation. Leveraging on strong capability in early discovery and clinical resources, China’s biotech companies are joining the global innovation in the next wave of oncology and autoimmune assets. In this panel, J.P. Morgan invited CEOs with lead assets in cell therapy and other modalities and will discuss differentiation of their assets and strategy for business developments.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies, inflammatory and immunological diseases and solid tumors. AbelZeta advances research and development in its own GMP facilities at its centers of excellence, with a pipeline comprised of multiple CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

Download PDF

AbelZeta Announces Late-Breaking Poster Presentation at ACR Convergence 2025 of First-in-Human Phase I Trial of anti-CD20/BCMA Bispecific CAR-T Therapy in Patients with Refractory Autoimmune Diseases

ROCKVILLE, MD, October 20, 2025 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on discovery and development of innovative and proprietary cell-based therapeutic products, today announced that clinical data from the first-in-human Phase I trial (NCT06249438) of C-CAR168 in patients with refractory autoimmune diseases will be presented as a late-breaking poster presentation at American College of Rheumatology (ACR) Convergence 2025, being held October 24-29, 2025, at McCormick Place in Chicago, Illinois. C-CAR168 is an autologous anti-CD20/BCMA bispecific Chimeric Antigen Receptor T-cell (CAR-T) therapy designed to simultaneously target autoantibody-producing plasma cells and their B-cell precursors, aiming to achieve “immune reset”. 

Presentation Type: Poster
Session: Late-Breaking Posters
Display Dates: October 26-28, 2025
Presentation Date & Time: Tuesday, October 28 , 10:30 AM – 12:30 PM CT
Location: Hall F1, McCormick Place
Abstract ID: 2211304
Abstract Title: Anti-CD20/BCMA Bispecific CAR-T Cell Therapy Promotes Immune Reset and Sustained Drug Free Remission in Refractory Autoimmune Diseases

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T therapies.

Forward-Looking Statements

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 numerous factors. Forward looking statements are based on the current beliefs and management’s expectations and are subject to significant risks and uncertainties outside of AbelZeta Pharma, Inc.’s (“AbelZeta” or the “Company”) 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, AbelZeta 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

Download PDF

AbelZeta Announces Promising Long-Term Outcomes for C-CAR039, a Novel CD19/CD20 Bi-Specific CAR-T Therapy, in Patients with R/R B NHL at EHA 2025

MILAN, June 16, 2025 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on discovery and development of innovative and proprietary cell-based therapeutic products, presented promising four-year clinical outcomes from 48 relapsed or refractory B-cell non-Hodgkin lymphoma (R/R B-NHL) patients treated with C-CAR039 (also known as Prizloncabtagene Autoleucel) in greater China, at the European Hematology Association (EHA) 2025 Congress.

The study evaluated the safety and efficacy of C-CAR039, a novel anti-CD19/CD20 bi-specific CAR-T therapy. The dual-targeting approach to both CD20 and CD19 antigens aims to enhance therapeutic efficacy and reduce the likelihood of antigen escape, a common mechanism of resistance in B-cell malignancies.

Key Findings:

  • The overall response rate (ORR) and complete response (CR) rate were 91.5% and 85.1%, respectively for all patients
  • Among the 43 LBCL pts, ORR and CR were 90.7% and 86.0%, respectively
  • With a median follow-up of 45.5 months (range, 3.1–62.8), the median duration of response (DOR), progression-free survival (PFS), and overall survival (OS) were not reached
  • KM estimates of 48-m PFS rate are 52.5% for all patients and 53.4% for LBCL patients, respectively
  • KM estimates of 48-m OS rate are 65.4% for all patients and 66.7% for LBCL patients, respectively

Safety Highlights:

  • CRS of any grade:8% | Grade ≥3: 2.1%
  • ICANS of any grade:3% | No severe cases (Grade ≥3) reported
  • No new safety signals were observed with longer follow-up

C-CAR039 has continued to show an excellent safety profile and deep and durable responses in R/R B-NHL patients, especially in those with R/R LBCL. These results continue to provide strong support for the curative potential of C-CAR039 as a next-generation therapy for R/R B-NHL. Late-stage clinical development is ongoing.

“The long-term clinical outcomes data for C-CAR039 are highly encouraging, demonstrating sustained responses in patients with relapsed or refractory B-cell non-Hodgkin lymphoma,” said Tony (Bizuo) Liu, Chairman and Chief Executive Officer of AbelZeta. “These results underscore our commitment and corporate goal to develop transformative therapies that address significant unmet medical needs in cancer.”

Dr. Yihong Yao, Chief Scientific Officer of AbelZeta, commented, “The strong clinical results of C-CAR039 demonstrate the added value of targeting CD20 in addition to CD19 in aggressive B-NHL. By addressing the limitations of single-antigen therapies – such as CD19 antigen escape – C-CAR039 enables deeper tissue penetration and more complete B-cell depletion. The durable responses observed support the continued advancement of C-CAR039 as a promising new treatment for patients with aggressive B-cell malignancies.”

For more information on the study and access to the poster presentation, please visit the EHA Library poster here.

AbelZeta previously presented 30-month Phase 1 clinical trial results at the 65th American Society of Hematology (ASH) Annual Meeting in December 2023, through oral presentation #1025, as well as at EHA 2024, through poster presentation #P1475. The data with 30 months follow up was published in Blood 2025 Apr 3;145(14):1526-1535.

AbelZeta Inc. (formerly known as Cellular Biomedicine Group, Inc.), entered into a worldwide collaboration and licensing agreement with Johnson & Johnson* in 2023 to develop and commercialize next-generation CAR-T cell therapies (excluding Greater China). The Phase 1 study for C-CAR039 was conducted by AbelZeta in China for the treatment of patients with B-cell non-Hodgkin lymphoma. Johnson & Johnson is evaluating the safety and efficacy of this asset (known as JNJ-90014496 outside of Greater China) through a separate study involving a global patient population. Phase 1b data for JNJ-90014496 were presented at EHA 2025 on Friday, June 13, informing the recommended Phase 2 dose and an initial assessment of safety, efficacy and pharmacokinetics in R/R LBCL patients. For more information, please visit Johnson and Johnson website: https://www.jnj.com/media-center/press-releases/johnson-johnsons-dual-targeting-car-t-cell-therapy-shows-encouraging-first-results-in-large-b-cell-lymphoma.

*The legal entity to the worldwide collaboration and license agreement is Janssen Biotech, Inc., a Johnson & Johnson company.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T therapies.

Forward-Looking Statements

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 the Company’s reports. Such statements are based on the management’s current beliefs and expectations and are subject to significant risks and uncertainties outside of management and 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, the Company 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

Download PDF

AbelZeta Receives RMAT Designation for C-CAR168 for the Treatment of Refractory SLE, Including LN

ROCKVILLE, MD, May 27, 2025 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on discovery and development of innovative and proprietary cell-based therapeutic products, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational therapy, C-CAR168, for the treatment of refractory Systemic Lupus Erythematosus (SLE), including Lupus Nephritis (LN).

C-CAR168 is a novel autologous bi-specific CAR-T therapy targeting both CD20 and B-cell maturation antigens (BCMA) in the treatment of patients with autoimmune diseases including autoimmune neurological diseases. The RMAT designation, granted under Investigational New Drug (IND) 30283, is based on promising early clinical data demonstrating the potential of C-CAR168 to address serious unmet medical needs in patients with treatment-resistant forms of autoimmune disease. Early clinical results from the Phase 1, first-in-human investigator-initiated trial (IIT) of C-CAR168 in patients with refractory autoimmune disease (NCT06249438) were shared as a podium presentation at the 16th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), in Toronto, Canada on May 22, 2025. The presentation can be viewed on the Company website page “Publications & Presentations”.

“We are pleased to receive the RMAT designation from the FDA for C-CAR168, a recognition made possible by the unwavering efforts of our dedicated team,” said Tony (Bizuo) Liu, AbelZeta’s Chairman and CEO. “This designation represents a significant milestone highlighting the promise that C-CAR168 holds for patients suffering from severe, treatment-resistant lupus and lupus nephritis. It also enables us to work more closely with the FDA, allowing us to accelerate development of this innovative therapy and bring hope and the potential for drug free disease remission to patients around the world who currently face limited treatment options.”

The RMAT designation enables sponsors to have early and frequent interactions with the FDA, including obtaining guidance on the development plan and the potential eligibility for priority review and accelerated approval. AbelZeta plans to request a multidisciplinary type B meeting with the FDA to discuss the next steps in the clinical and manufacturing development of C-CAR168.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T therapies.

Forward-Looking Statements

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 the Company’s reports. Such statements are based on the management’s current beliefs and expectations and are subject to significant risks and uncertainties outside of management and 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, the Company 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

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AbelZeta Presents New Data on C-CAR168 for the Treatment of Autoimmune Diseases at LUPUS 2025

  • Novel Anti-CD20/BCMA Bispecific Autologous CAR-T Therapy Targeting Lupus Nephritis , Systemic Lupus Erythematosus and Multiple Sclerosis
  • Phase 1, First-in-Human Investigator-Initiated Trial (IIT)

ROCKVILLE, MD, May 22, 2025 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on the discovery and development of innovative and proprietary cell-based therapeutic products, today announced that data from its Phase 1 first-in-human investigator-initiated trial (IIT) of C-CAR168 study (NCT06249438, the “Study”) has been published in an abstract shared in a podium presentation at the 16th International Congress on Systemic Lupus Erythematosus (“LUPUS 2025”) in Toronto, Canada. The Principal Investigator (PI) of the Study, Professor Nan Shen from Department of Rheumatology, Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine, presented the early clinical results in patients with refractory autoimmune disease.

Key Highlights:

  • Overall, 10 patients have been treated with C-CAR168 including 7 LN patients, 1 Secondary Progressive Multiple Sclerosis (SPMS), 1 Neuromyelitis Optica Spectrum Disorder (NMOSD) and 1 Immune-Mediated Necrotizing Myopathy (IMNM) patient as of February 28, 2025;
  • Early clinical data showed that C-CAR168 was well tolerated. Four LN patients and one SPMS patient experienced low-grade 1-2 CRS with median time to onset of 2 days post C-CAR168 treatment. Neither ICANS nor severe infection was observed.

Mechanism of Action and Efficacy in LN Patients and a SPMS Patient:

  • Robust C-CAR168 expansion and rapid, complete depletion of B cells, CD20dim T cells, and plasma cells in peripheral blood was observed. Complete elimination of B cells, CD19+– and CD19– long-lived plasma cells in bone marrow was also demonstrated in the only patient in whom bone marrow was examined;
  • Four LN patients treated with C-CAR168 reached the 6-month evaluation timepoint. All 4 achieved and remained in SRI (4), 2 patients achieved CR, and 1 PR based on Kidney Disease: Improving Global Outcomes (KDIGO) 2024 LN Response Criteria;
  • All patients discontinued IS/biologics after lymphodepletion, and most patients steroid-free after C-CAR168 infusion;
  • One LN patient flared before 6-month post treatment;
  • Evidence of C-CAR168-induced immune reset was demonstrated through peripheral blood flow cytometry and RNA sequencing analysis in LN patients;
  • The SPMS patient treated showed very promising early efficacy signal, including improvement in gait and orbital movement, significant reduction of brain inflammation and lesion size, improvement in EDSS, 9-HPT, T25-FW, and biomarker level.

“The early clinical results for C-CAR168 mark a significant step forward in the treatment of patients suffering from LN and SPMS with severe manifestations and few effective treatment options,” said Tony (Bizuo) Liu, the Company’s Chairman and CEO. “These findings support the extension of our bi-specific CAR-T platform, including anti CD20/19 bispecific CAR-T for NHL and now adding C-CAR168, anti-CD20/BCMA, for autoimmune diseases. Success in treatment with C-CAR168 would enable patients to discontinue immunosuppressive therapy and substantially reduce disease activity. We will work closely with the FDA and the scientific community to move forward with Phase 1b and Phase 2 studies.”

About the Study (NCT06249438)

This is an investigator-initiated, multicenter, open-label study of C-CAR168 for the treatment of adult patients with autoimmune diseases and neurological diseases, such as refractory LN, SLE and progressive Multiple Sclerosis (MS), resistant and refractory to standard therapy. As of February 28, 2025, 7 patients with refractory LN received C-CAR168 therapy, with 4 dosed at 0.75×106 cells/kg and 3 at 1.5×106 cells/kg. The treated population had long-standing refractory disease (median SLE duration 9 years, LN 5 years) with exposure to a median of 4 IS or biologic agents.

About C-CAR168

C-CAR168 is a novel autologous bi-specific CAR-T therapy targeting both CD20 and B-cell maturation antigens (BCMA). The Food and Drug Administration (FDA) has granted clearance of the Investigational New Drug (IND) application of C-CAR168 and preclinical data was presented at the American College of Rheumatology (ACR) Convergence 2024 held in Washington, DC, in November 2024.

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T and TIL therapies.

Forward-Looking Statements

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 the Company’s reports. Such statements are based on the management’s current beliefs and expectations and are subject to significant risks and uncertainties outside of management and 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, the Company 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

Download PDF

AbelZeta Announces Upcoming Oral Presentations at LUPUS 2025

ROCKVILLE, MD, May 16, 2025 – AbelZeta Pharma, Inc. (“AbelZeta” or the “Company”), a global clinical-stage biopharmaceutical company focused on discovery and development of innovative and proprietary cell-based therapeutic products, today announced that the abstract presenting early clinical results from the Phase 1, first-in-human investigator-initiated trial (IIT) of C-CAR168 in Chinese patients with refractory autoimmune disease (NCT06249438) has been selected for a podium presentation at the 16th International Congress on Systemic Lupus Erythematosus (“LUPUS 2025”), to be held in Toronto, Canada on May 21-24, 2025.

C-CAR168 is a novel autologous bi-specific CAR-T therapy targeting both CD20 and B-cell maturation antigens (BCMA) in the treatment of patients with autoimmune diseases and neurological diseases, such as resistant and refractory Lupus Nephritis (LN), Systemic Lupus Erythematosus (SLE), and progressive Multiple Sclerosis (MS). The Food and Drug Administration (FDA) has granted clearance of the Investigational New Drug (IND) application of C-CAR168 and the preclinical data was presented at the American College of Rheumatology (ACR) Convergence 2024 held in Washington, DC, in November 2024.

Details of the oral presentation are as follows:

  • Abstract: https://cslide.ctimeetingtech.com/lupus25/attendee/confcal/show/session/23
  • Abstract Title: Clinical Impact of C-CAR168, A Novel Anti-CD20/BCMA Composite Autologous CAR-T Therapy, in Refractory Lupus Nephritis
  • Session Title: Abstract Concurrent Session 04: Advancing Lupus Therapies and Insights
  • Presentation Time and Date:  May 22, 2025 / 14:10 – 14:20
  • Presenter: Nan Shen, M.D., PhD, Principal Investigator (PI), Department of Rheumatology, Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine
  • Location: Westin Harbour Castle, Toronto, Canada
  • Room: Queens Quay

In addition, the Company’s Vice President of Clinical Development, Barbara Mittleman, M.D, is an invited speaker at the congress in the following Scientific Session:

  • Session Name: Cell Therapy in Lupus
  • Session Time and Date: May 23, 2025 / 11:35 – 12:20
  • Room: Pier 2&3
  • The session will cover the following topics:
  1. Cell Therapy in SLE: Where We Are and how We Got Here
  2. Considerations, Challenges and Opportunities

About AbelZeta Pharma, Inc.

AbelZeta is a global clinical-stage biopharmaceutical company with centers of excellence in Rockville, Maryland and Shanghai, China. AbelZeta is focusing on developing innovative and proprietary cell-based therapeutic products and is committed to ushering in bespoke treatments that harness the body’s own immune system to fight against hematological malignancies and solid tumors, as well as inflammatory and immunological diseases. AbelZeta advances research and development in its own GMP facilities at its centers of excellence for early-stage clinical studies, with a pipeline comprised of CAR-T and TIL therapies.

Forward-Looking Statements

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 the Company’s reports. Such statements are based on the management’s current beliefs and expectations and are subject to significant risks and uncertainties outside of management and 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, the Company 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.

Company Contact:

Sarah Kelly
Director of Communications
AbelZeta Pharma Inc.
Phone: +1 (240) 552 5870
Email: [email protected]

Download PDF

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