MDC-CAR-BCMA001 / German Cancer Research Center, Dresden University of Technology - LARVOL DELTA

PATIENTS WITH RELAPSED/REFRACTORY SYSTEMIC LIGHT CHAIN AMYLOIDOSIS ACHIEVE DEEP HEMATOLOGIC AND ORGAN RESPONSES WITH THE NOVEL MDC-CAR-BCMA001 (EBMT 2026) - " Seven patients were treated with a fixed dose of 800 x 106 BCMA-directed CAR T-cells after lymphodepleting chemotherapy with fludarabine/cyclophosphamide (4/7) or bendamustine (3/7 – in cases of neuropathy or severe nephropathy)...grade 3), rapidly resolving after tocilizumab and/or dexamethasone... MDC-CAR-BCMA001 shows a manageable toxicity profile with high efficacy even in heavily pretreated patients, making it a suitable construct for treating the vulnerable cohort of AL amyloidosis patients. Long-lasting, deep hematologic responses translate into excellent organ responses even in patients with advanced organ disease. These findings warrant further prospective evaluation, as planned in the phase I CLEAR AL trial (EU CT No."

Clinical • Amyloidosis • Hematological Malignancies • Inflammation • Monoclonal Gammopathy • Multiple Myeloma • Myositis • Renal Disease • Smoldering Multiple Myeloma

PATIENTS WITH RELAPSED/REFRACTORY SYSTEMIC LIGHT CHAIN AMYLOIDOSIS ACHIEVE DEEP HEMATOLOGIC AND ORGAN RESPONSES WITH THE NOVEL MDC-CAR-BCMA001 (EBMT 2026) - " Seven patients were treated with a fixed dose of 800 x 106 BCMA-directed CAR T-cells after lymphodepleting chemotherapy with fludarabine/cyclophosphamide (4/7) or bendamustine (3/7 – in cases of neuropathy or severe nephropathy)...grade 3), rapidly resolving after tocilizumab and/or dexamethasone... MDC-CAR-BCMA001 shows a manageable toxicity profile with high efficacy even in heavily pretreated patients, making it a suitable construct for treating the vulnerable cohort of AL amyloidosis patients. Long-lasting, deep hematologic responses translate into excellent organ responses even in patients with advanced organ disease. These findings warrant further prospective evaluation, as planned in the phase I CLEAR AL trial (EU CT No."

Clinical • Amyloidosis • Hematological Malignancies • Inflammation • Monoclonal Gammopathy • Multiple Myeloma • Myositis • Renal Disease • Smoldering Multiple Myeloma

Successful Treatment of Patients with Relapsed/Refractory Systemic Light Chain Amyloidosis with the Novel MDC-CAR-BCMA001 (EHA-EBMT-CART 2026) - "Methods : Seven patients were treated with a fixed dose of 800 x 10 6 BCMA-directed CAR T-cells after lymphodepleting chemotherapy with fludarabine/cyclophosphamide (4/7) or bendamustine (3/7 – in cases of neuropathy or severe nephropathy)...All patients were daratumumab-refractory (6/7 proteasome inhibitor- and 6/7 immunomodulatory drug-exposed) and 6/7 had active disease prior to CAR T-cell infusion (4/6 progressive disease, 1/6 relapsed disease, 1/6 no response to prior treatment)...grade 3), rapidly resolving after tocilizumab and/or dexamethasone. Neither immune effector cell-associated neurotoxicity nor higher-grade late hematotoxicity were observed. Conclusions : MDC-CAR-BCMA001 showed a favorable safety profile with high efficacy, making it a suitable construct for treating the vulnerable cohort of AL amyloidosis patients with preexisting organ insufficiencies, albeit an extended observation period is warranted."

Clinical • Amyloidosis • Hematological Malignancies • Inflammation • Monoclonal Gammopathy • Multiple Myeloma • Myositis • Renal Disease • Smoldering Multiple Myeloma

Safety, tolerability and early efficacy of mdc-CAR-BCMA001 – a high affinity BCMA targeting CAR T-cell therapy in patients with multiple myeloma and diffuse large B-cell lymphoma (ASH 2025) - P1 | "Patients receive fludarabine (30 mg/m2/d) and cyclophosphamide (500 mg/m2/d) on days -5to -3 followed by a single infusion of MDC-BCMA-CAR001 at 3 pre-defined dose levels (DL) ranging from0.5 to 9.0 x106 CAR T-cells/kg...There was no needfor the administration of tocilizumab or steroids.Circulating CAR-T cell levels peaked at day 14 post infusion and remained detectable up to last time pointcurrently available (150 days)...BCMA expression was readily detectable by IHC in all assessed DLBCL samples. Expression levels by qPCRwere considerably lower in DLBCL than in MM but, on average, 3-fold higher than in non-tumor controls,suggesting feasibility of targeting BCMA with a high affinity CAR T-cell product in DLBCL.As of July 1st, 2025, 12 patients have been consented for this phase I trial of whom 10 underwentleukapheresis and 6 received treatment with MDC-CAR-BCMA001. Patients were heavily pretreated with 9prior lines in rrDLBCL and a median of 6 prior lines in rrMM."

CAR T-Cell Therapy • Clinical • IO biomarker • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma

Successful treatment of patients with relapsed/refractory systemic light chain amyloidosis with the novel MDC-CAR-BCMA001 (DGHO 2025) - "We initiated compassionate use of the in-house manufactured 2nd-generation BCMA-directed MDC-CAR-BCMA001 in patients with relapsed/refractory AL amyloidosis and report our initial experience in this challenging patient group. Five patients were treated with a fixed dose of 800 x 106 BCMA-directed CAR T-cells after lymphodepleting chemotherapy with fludarabine/cyclophosphamide (3/5) or bendamustine (2/5 – in cases of neuropathy or severe nephropathy). MDC-CAR-BCMA001 seems to comprise a favorable safety profile with high efficacy, making it a suitable construct for treating the vulnerable cohort of AL amyloidosis patients with preexisting organ insufficiencies."

Clinical • Amyloidosis • Hematological Malignancies • Inflammation • Monoclonal Gammopathy • Multiple Myeloma • Renal Disease • Smoldering Multiple Myeloma

CARLOTTA01: A Phase I Trial to Establish the Safety and Maximum Tolerated Dose of High-affinity Autologous BCMA-targeting Chimeric Antigen Receptor (CAR) T-cells in Patients With Relapsed and Refractory B-cell Malignancies (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Technische Universität Dresden | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Jun 2025 ➔ Jun 2027

Trial completion date • Trial primary completion date • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • CD31 • SLAMF7

MDC-CAR-BCMA001 Shows Efficacy, Safety as Compassionate Use in R/R Multiple Myeloma and AL Amyloidosis (OncLive) - "The novel second-generation BCMA-directed CAR T-cell construct MDC-CAR-BCMA001 demonstrated activity and a favorable safety profile with no incidence of neurotoxicity in the compassionate use setting for heavily pretreated patients with relapsed/refractory multiple myeloma and systemic light chain (AL) amyloidosis who displayed preexisting organ insufficiencies, according to findings presented at the 51st Annual EBMT Meeting. At a median follow-up of 13 months, 5 of the 6 patients treated with MDC-CAR-BCMA001 achieved an overall response, 4 of which were complete responses (CRs) and 1 of which was a very good partial response (VGPR). Additionally, all CRs translated in minimal residual disease negativity. The 12-month overall survival and event-free survival rates were 83% and 50%, respectively. Notably, 4 patients achieved sustained remission without a need for subsequent therapy."

Clinical data • Amyloidosis • Multiple Myeloma

CARLOTTA01: A Phase I Trial to Establish the Safety and Maximum Tolerated Dose of High-affinity Autologous BCMA-targeting Chimeric Antigen Receptor (CAR) T-cells in Patients With Relapsed and Refractory B-cell Malignancies (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Technische Universität Dresden | Not yet recruiting ➔ Recruiting

CAR T-Cell Therapy • Enrollment open • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • CD31 • SLAMF7

CARLOTTA01: A Phase I Trial to Establish the Safety and Maximum Tolerated Dose of High-affinity Autologous BCMA-targeting Chimeric Antigen Receptor (CAR) T-cells in Patients With Relapsed and Refractory B-cell Malignancies (clinicaltrials.gov) - P1 | N=16 | Not yet recruiting | Sponsor: Technische Universität Dresden | Initiation date: Jul 2023 ➔ Dec 2023

CAR T-Cell Therapy • Trial initiation date • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • CD31 • SLAMF7

CARLOTTA01: A Phase I Trial to Establish the Safety and Maximum Tolerated Dose of High-affinity Autologous BCMA-targeting CAR T-cells in Patients With Relapsed and Refractory B-cell Malignancies (clinicaltrials.gov) - P1 | N=16 | Not yet recruiting | Sponsor: Technische Universität Dresden

CAR T-Cell Therapy • New P1 trial • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • SLAMF7