CT071 / CARsgen - LARVOL DELTA

GPRC5D-Targeted CAR T-Cell Therapy CT071 for the Treatment of Refractory/Relapsed Multiple Myeloma (ASH 2024) - P1 | "Fifteen out of 17 patients achieved MRD negativity at 10−6 threshold. Conclusion Preliminary results from this study of CT071 demonstrate a promising safety profile with compelling clinical response in RRMM patients including those with prior BCMA or BCMA/CD19 CAR T exposure."

CAR T-Cell Therapy • Anemia • Dermatology • Hematological Disorders • Hematological Malignancies • Leukopenia • Multiple Myeloma • Neutropenia • Oncology • Thrombocytopenia

GPRC5D-targeted CAR T-cell therapy (CT071) in patients with relapsed or refractory multiple myeloma: a first-in-human, single-centre, single-arm, phase 1 trial. (PubMed, Lancet Haematol) - P1 | "CT071 demonstrated an encouraging safety profile with compelling activity in patients with relapsed or refractory multiple myeloma."

First-in-human • Journal • P1 data • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Oncology

CARsgen Presents Results on GPRC5D CAR-T CT071 in The Lancet Haematology (PRNewswire) - "With a median follow-up of 10.71 months (IQR 6.13-12.02), the objective response rate (ORR) was 100% (95%CI, 83.2-100). 10 (50%) patients achieved stringent complete response (sCR), 4 (20%) had very good partial response (VGPR) and 6 (30%) had partial response (PR). One patient with a large EMD (125 mm×99 mm at baseline) achieved a 67.6% reduction at month 10 with ongoing PR."

P1 data • Multiple Myeloma

A PHASE I STUDY OF GPRC5D TARGETING CAR T-CELL THERAPY CT071 FOR HIGH-RISK NEWLY DIAGNOSED MULTIPLE MYELOMA (EHA 2025) - "Upon diagnosis, all patients received 2 cycles of VRd (bortezomib, lenalidomide and dexamethasone) prior to study enrollment. Following apheresis and lymphodepletion with fludarabine 30mg/m2/day and cyclophosphamide 300mg/m2/day for 3 days, CT071 was administered as a single infusion... Preliminary results indicate that CT071 induces deep responses in HR NDMM with a favorable safety profile. Further research is warranted to fully evaluate the potential of GPRC5D targeting CAR-T cell therapy in HR NDMM."

CAR T-Cell Therapy • P1 data • Hematological Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Inflammation • Leukopenia • Multiple Myeloma • Neutropenia • Oncology • Rare Diseases • Thrombocytopenia

RESEARCH RESULTS ON CT071 AT 2025 EHA ANNUAL CONGRESS (HKEXnews) - "The board of directors of the Company (the 'Board') announces that the research results of the investigator-initiated trial of CT071 (an autologous CAR T-cell product targeting GPRC5D) for the treatment of newly diagnosed multiple myeloma (NDMM) (NCT06407947) have been presented at the 30th Annual Congress of the European Hematology Association ('EHA')....The research results of IIT of CT071 were presented as a poster at the 30th EHA Annual Congress on June 13, 2025, 18:30 - 19:30 CEST, which was titled 'A phase I study of GPRC5D targeting CAR T-cell therapy CT071 for high-risk newly diagnosed multiple myeloma'."

P1 data • Multiple Myeloma

CT071-CG7001: Study of CT071 Injection in RRMM or PPCL (clinicaltrials.gov) - P1 | N=20 | Active, not recruiting | Sponsor: Shanghai Changzheng Hospital | N=30 ➔ 20 | Trial completion date: Nov 2024 ➔ Jul 2025 | Recruiting ➔ Active, not recruiting | Trial primary completion date: Nov 2024 ➔ Jul 2025

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • Plasma Cell Leukemia

GPRC5D-Targeted CAR T-Cell Therapy CT071 for the Treatment of Refractory/Relapsed Multiple Myeloma (PRNewswire) - P1 | N=30 | NCT05838131 | "CARsgen Therapeutics Holdings Limited...announces that the Company will present the clinical data of...CT071...at the 66th Annual Congress of the American Society of Hematology....In 17 patients who had been dosed with CT071, 11 patients (64.7%) experienced cytokine release syndrome (CRS), all at Grade 1 (n=8) or 2 (n=3). No immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. No dose limiting toxicity (DLT) occurred. The overall response rate (ORR) was 94.1% (16/17), and the stringent complete response (sCR) rate was 52.9% (9/17). Notably, 7 patients achieved complete response or better at week 4. All 4 patients with previous exposure to BCMA or BCMA/CD19 CAR T responded to CT071 (2 with sCR and 2 with partial response)."

P1 data • Multiple Myeloma

CARsgen U.S. Clinical Holds Lifted by FDA (PRNewswire) - "CARsgen Therapeutics Holdings Limited...announces that the U.S. Food and Drug Administration ('FDA') lifted the clinical holds on clinical trials of zevorcabtagene autoleucel (zevor-cel, CT053, an autologous CAR-T product against BCMA), satricabtagene autoleucel (satri-cel, CT041, an autologous CAR-T product against Claudin18.2), and CT071 (an autologous CAR-T product against GPRC5D) in the United States."

FDA event • Hematological Malignancies • Oncology

VOLUNTARY ANNOUNCEMENT TO PRESENT ZEVORCABTAGENE AUTOLEUCEL, CT071 AND CT0590 AT ASH 2024 ANNUAL CONGRESS (HKEXnews) - "The board of directors of the Company...announces that the Company will present the clinical data of zevorcabtagene autoleucel...CT071 (an autologous CAR T-cell therapy candidate targeting GPRC5D), and CT0590 (an allogeneic CAR T-cell product candidate against BCMA) at the 66th Annual Congress of the American Society of Hematology ('ASH'). Abstracts and further details will be announced after November 5, 2024 Eastern Time."

Clinical data • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

CARsgen Submitted Application to Request Lifting Clinical Holds to FDA (CARsgen Press Release) - "CARsgen Therapeutics Holdings Limited...announces that the U.S. Food and Drug Administration ('FDA') recently did a follow-up inspection of the manufacturing site located in Durham, North Carolina. The inspection was positive, and no observation (Form 483) was issued. The Company has submitted the complete response letters to request lifting the clinical holds for zevorcabtagene autoleucel, satricabtagene autoleucel and CT071 to FDA on October 4, 2024. FDA is expected to respond within 30 calendar days."

FDA event • Oncology

CARsgen Announces 2024 Interim Results (PRNewswire) - "CARsgen has been collaborating with Huadong Medicine for the commercialization of zevor-cel in mainland China. As of July 31, 2024, zevor-cel had been included in nearly 20 provincial or municipal government-subsidized insurance programs and private health insurance products; certification and regulatory filings had been completed in over 100 healthcare institutions across 19 provinces or cities nationwide; we had received a total of 52 orders from Huadong Medicine...The Group's revenue was over RMB6 million for the six months ended June 30, 2024 mainly from zevor-cel in which the primary revenue of zevor-cel was calculated on the basis of ex-works price, rather than on the basis of end-of-market prices....Besides, the Company received a milestone payment of RMB75 million from Huadong Medicine for zevor-cel for the six months ended June 30, 2024....Data updates for CT071 and CT0590 are to be disclosed in the second half of 2024."

Clinical data • Commercial • Hematological Malignancies • Multiple Myeloma • Oncology

CARsgen Presents Phase II Results on Zevorcabtagene Autoleucel and First-in-human CT071 Results at EHA 2024 [Google translation] (CARsgen Press Release) - "CARsgen Therapeutics...announces that the updated results of zevorcabtagene autoleucel (zevor-cel; R&D code: CT053, an autologous CAR T-cell product targeting BCMA) and CT071 (an autologous CAR T-cell product targeting GPRC5D) have been presented at the 29th Annual Congress of the European Hematology Association...The results of LUMMICAR STUDY 1 of zevorcabtagene autoleucel were reported as an oral presentation at the 29th EHA Annual Congress on June 15, 2024, 17:30-17:45 CEST....The preliminary results of Phase I of CT071 were presented as a poster at the 29th EHA Annual Congress on June 14, 2024, 18:00 - 19:00 CEST..."

Clinical data • Hematological Malignancies • Multiple Myeloma • Oncology

FIRST-IN-HUMAN STUDY OF GPRC5D-TARGETED CAR T CELLS (CT071) WITH AN ACCELERATED MANUFACTURING PROCESS IN PATIENTS WITH RELAPSED/REFRACTORY MULTIPLE MYELOMA (RRMM) (EHA 2024) - P1 | "In the ongoing exploratory trial, preliminary data from CT071, an autologous fully human anti-GPRC5D CAR T-cell product with expedited manufacturing, demonstrate an acceptable safety profile and encouraging clinicalefficacy in patients with RRMM without the need for bridging therapy, and warrants further clinical evaluation."

CAR T-Cell Therapy • Clinical • P1 data • Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukopenia • Multiple Myeloma • Neutropenia • Oncology • Plasmacytoma • Pneumonia • Respiratory Diseases • Thrombocytopenia • Transplantation

CARsgen Presents First-in-human Results of GPRC5D Targeting CAR-T CT071 at EHA 2024 (PRNewswire) - Early P1 | N=30 | NCT05838131 | "The median follow-up at the time of data cut-off was 4.07 months (range: 2.8-7.4). There were no Grade 3 or higher cytokine release syndrome (CRS) events. No immune effector cell-associated neurotoxicity syndrome (ICANS) was observed. No adverse events of special interest or dose limiting toxicity (DLT) occurred. Four patients experienced treatment-related SAE, including pneumonia (n=1), decreased appetite (n=1) and thrombocytopenia (n=2), and all recovered....The overall response rate was 90%, including 5 patients (50%) with stringent complete response (sCR), 2 patients (20%) with very good partial response (VGPR), and 2 patients (20%) with partial response (PR)."

Clinical data • Multiple Myeloma • Oncology

Study of CT071 Injection in High Risk Newly Diagnosed Multiple Myeloma (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Shanghai Changzheng Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Multiple Myeloma • Oncology

Study of CT071 Injection in RRMM or PPCL (clinicaltrials.gov) - P1 | N=30 | Recruiting | Sponsor: Shanghai Changzheng Hospital | N=18 ➔ 30

Enrollment change • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

Study of CT071 Injection in HRNDMM (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Shanghai Changzheng Hospital

New P1 trial • Hematological Malignancies • Multiple Myeloma • Oncology

Anti-GPRC5D CAR-T Cells (CT071) in Participants With RRMM or RRpPCL (clinicaltrials.gov) - P1/2 | N=166 | Not yet recruiting | Sponsor: CARsgen Therapeutics Co., Ltd.

CAR T-Cell Therapy • New P1/2 trial • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

FDA Puts Clinical Hold on Three CARsgen Therapeutics CAR-T Candidates (BioSpace) - "The FDA has placed a clinical hold on three of CARsgen Therapeutics’ CAR-T cell therapy candidates following an inspection of the Chinese biotech’s manufacturing facility in Durham, North Carolina...CARsgen notified the Hong Kong Stock Exchange that the FDA clinical hold had gone into effect on three of its CAR-T products: CT053, CT071 and CT041. According to CARsgen, the clinical hold on its products are due to chemistry, manufacturing, and controls-related 'questions' that arose due to the FDA inspection....The company said in the notice that it plans to conduct a 'comprehensive review' and improve its Current Good Manufacturing Practices (cGMP) at the facility, which was opened in February 2022."

FDA event • Gastric Cancer • Gastroesophageal Junction Adenocarcinoma • Gastrointestinal Cancer • Hematological Malignancies • Multiple Myeloma • Oncology • Pancreatic Cancer • Solid Tumor

CARsgen's CT071 Received IND Clearance from the FDA for Treating Relapsed/Refractory Multiple Myeloma or Relapsed/Refractory Primary Plasma Cell Leukemia (PRNewswire) - P1 | N=18 | NCT05838131 | "CARsgen Therapeutics Holdings Limited...announces that the U.S. Food and Drug Administration (FDA) has granted Investigational New Drug (IND) clearance for CT071, an autologous CAR T-cell therapy candidate targeting G protein-coupled receptor class C group 5 member D (GPRC5D), for the treatment of patients with relapsed/refractory multiple myeloma (MM) or relapsed/refractory primary plasma cell leukemia (PCL)....An investigator-initiated trial (IIT) is already under way in China to assess the safety and efficacy of CT071 in treating relapsed/refractory MM or PCL (NCT05838131). Preliminary clinical data from the IIT shows an acceptable safety profile with preliminary efficacy."

IND • P1 data • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

Study of CT071 Injection in RRMM or PPCL (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Shanghai Changzheng Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

Study of CT071 Injection in RRMM or PPCL (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Shanghai Changzheng Hospital

New P1 trial • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology