GDA-201 / Ayrmid - LARVOL DELTA

Evaluation of Safety and Efficacy of Allo GDA-201 NK Cells in Patients With Relapsed/Refractory B Cell NHL (clinicaltrials.gov) - P1/2 | N=13 | Terminated | Sponsor: Gamida Cell ltd | N=99 ➔ 13 | Trial completion date: Mar 2025 ➔ Apr 2024 | Recruiting ➔ Terminated | Trial primary completion date: Aug 2024 ➔ Apr 2024; Study terminated by Sponsor.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

A Phase I/II Study of GDA-201, Cryopreserved Nicotinamide-Enhanced Allogeneic Natural Killer Cells, in Patients with Relapsed/Refractory B-Cell Lymphoma (TCT-ASTCT-CIBMTR 2024) - P1/2 | "GDA-201 was administered with rituximab after fludarabine/cyclophosphamide lymphodepletion, followed by low dose IL-2. Cohort 4, at the target GDA-201 dose level of 2x108 cells/kg, is currently enrolling. Conclusion GDA-201 with rituximab was well tolerated in doses up to 1x108 cells/kg and demonstrated evidence of clinical efficacy in pts with R/R B-cell NHL including in the post-autologous CAR-T setting."

Clinical • IO biomarker • P1/2 data • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

Gamida Cell to Present at the 2024 Tandem Meetings of ASTCT and CIBMTR (Gamida Cell Press Release) - P1 | N=99 | NCT05296525 | Sponsor: Gamida Cell ltd | "Abstract highlights: This is an ongoing multicenter Phase 1 study of allogeneic cryopreserved NK cell therapy candidate GDA-201 in patients with relapsed/refractory B-cell CD20 positive non-Hodgkin lymphoma. Preliminary data for the 10 patients enrolled in the first three of four total cohorts, who were heavily pretreated with a median of six prior lines of therapy including CAR-T cell therapy and hematopoietic stem cell transplant, showed no infusion reactions, dose limiting toxicities or related serious adverse events reported in patients treated with doses up to 1x10⁸ cells/kg GDA-201 in combination with rituximab....There were no reported cases of immune effector cell associated neurotoxicity syndrome or graft versus host disease."

P1 data • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Evaluation of Safety and Efficacy of Allo GDA-201 NK Cells in Patients With Relapsed/Refractory B Cell NHL (clinicaltrials.gov) - P1/2 | N=99 | Recruiting | Sponsor: Gamida Cell ltd | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Gamida Cell Reports Third Quarter 2023 Financial Results and Provides Company Update (GlobeNewswire) - "Upcoming participation in the American Society of Hematology (ASH) Annual Meeting and Exposition: Members of the Gamida Cell team will attend the upcoming ASH Annual Meeting December 9-12 in San Diego, California. A product theater will be presented on Omisirge....Net Revenue for the third quarter 2023 was $0.7 million, resulting from the delivery of two units of Omisirge....Research and development expenses were $4.2 million in the third quarter of 2023, compared to $9.9 million in the same quarter in 2022. The $5.7 million decrease was primarily due to the aforementioned reporting transition, along with reduced omidubicel clinical spend relating to the Phase 3 clinical trial....Solely for financial reasons, we do not plan to conduct the Phase 2 portion of the GDA-201 Phase 1/2 study."

Clinical data • Commercial • Trial status • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

GDA-201, nicotinamide (NAM) expanded NK cells derived from peripheral apheresis, show unique culture kinetics and increased expansion (SITC 2023) - P1/2 | "Examination of cell identity revealed that NAM prolonged the survival of feeder cells on the first days of expansion (45% on day 2–3 compared to number seeded), while feeder cells survival decreased dramatically in the absence of NAM (13% on day 2–3 compared to number seeded). Conclusions These data provide further evidence for the unique characteristics of NAM-NK, resulting in increased cell proliferation and robustness of the manufacturing process."

Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Gamida Cell Presents New Data on Allogeneic Stem Cell Therapy Omidubicel and Natural Killer (NK) Cell Therapy Candidate GDA-201 at Society for Immunotherapy of Cancer (SITC) Annual Meeting (GlobeNewswire) - "During the first days of expansion, NAM increased the survival of the feeder cells (45% on day 2-3 vs 13% on day 2-3) and prolonged their support in NK cell expansion, resulting in higher fold expansion persisting up to 21 days. In addition, NK cells expanded using NAM showed significantly higher cytotoxicity against leukemia cells (from a killing cytotoxicity function of 62.54% without NAM to 81.84% with NAM), and preservation of the NK cells in a partially mature state with low expression of CD57....A multicenter Phase 1 study of GDA-201 for the treatment of non-Hodgkin lymphoma is ongoing (NCT05296525). Results are expected in Q1 2024."

P1 data • Preclinical • Hematological Malignancies • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Gamida Cell Reports Preliminary Data from Phase 1 Study of Natural Killer (NK) Cell Therapy Candidate GDA-201 (Businesswire) - P1/2 | N=88 | NCT05296525 | Sponsor: Gamida Cell ltd | "Preliminary results showed marked shrinkage of target lesions in five patients; efficacy evaluation showed two patients with complete response, two with partial response, and one with stable disease. No dose-limiting toxicities were reported in the 10 patients treated with doses up to 1x10⁸ cells/kg GDA-201 in combination with rituximab....Activity appears to be dose dependent with two of the three patients in Cohort 3 responding. The fourth and final cohort of the study, at the target dose level of 2x10⁸ cells/kg, is currently enrolling....We look forward to continuing to follow these patients and completing enrollment in our next cohort, and anticipate sharing the full Phase I data in early 2024.'"

Enrollment status • P1 data • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology

Gamida Cell to Present at Society for Immunotherapy of Cancer’s (SITC) 38th Annual Meeting (Businesswire) - "Gamida Cell Ltd...today announced that it will be presenting at the Society for Immunotherapy of Cancer’s 38th Annual Meeting (SITC 2023) taking place in San Diego, CA and virtually November 1-5, 2023."

Preclinical • Oncology

Gamida Cell Reports Second Quarter and First Half 2023 Financial Results and Provides Company Update (Businesswire) - "Gamida Cell’s GDA-201, an intrinsic natural killer (NK) cell therapy candidate being investigated for the treatment of hematologic malignancies, is being evaluated in an ongoing Phase 1 study for treatment of non-Hodgkin lymphoma. The study is continuing to enroll patients at six sites in the U.S. Data are expected in the first quarter of 2024....Research and development expenses were $8.7 million in the second quarter of 2023, compared to $10.6 million in the same quarter in 2022. The decrease of $1.9 million was primarily due to a $1.6 million reduction associated with the discontinuation of development of our engineered NK cell therapy pipeline, and $0.7 million in lower Omisirge Phase 3 spend, including a decrease in payments for manufacturing services, partially offset by a decrease of $0.4 million in IIA (Israeli Innovation Authority) income."

Commercial • P1 data • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

Nicotinamide enhances natural killer cell function and yields remissions in patients with non-Hodgkin lymphoma. (PubMed, Sci Transl Med) - P1 | "Cellular therapy with GDA-201 and rituximab was well tolerated and yielded an overall response rate of 74% in 19 patients with advanced NHL. GDA-201 cells were detected for up to 14 days in blood, bone marrow, and tumor tissues and maintained a favorable metabolic profile. The safety and efficacy of GDA-201 in this study support further development as a cancer therapy."

Journal • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • IL15

Responses after Allogeneic NK Cell Therapy for Lymphoma: correlative analysis revealed impact of host monocytes and robust T cells tumor trafficking (ICML 2023) - P1 | " R/R NHL patients (10 follicular lymphoma, 8 DLBCL, 1 MCL) received lymphodepleting chemotherapy followed by 2 infusions of GDA-201 (days 0, 2) combined with rituximab and low dose IL2. Overall, our findings showed that responses after allogeneic NK cell therapy are influenced by levels of pre-treatment monocytes and PMN MDSCs. Our results suggest that immune microenvironment changes after allogeneic NK therapy stimulate influx of host T cells into tumor sites and support cross-talk between innate and the adaptive arms to enhance lymphoma elimination."

IO biomarker • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • CCR4 • CD4 • CD69 • CD8 • CXCR3 • CXCR4 • PD-1 • TIGIT

Data To Be Presented on Gamida Cell Natural Killer (NK) Cell Therapy Candidate GDA-201 at the International Society for Cell & Gene Therapy 2023 Annual Meeting (Businesswire) - "Gamida Cell Ltd...announced that an oral presentation highlighting Gamida Cell’s investigational natural killer (NK) cell therapy candidate GDA-201 will be shared at the International Society for Cell and Gene Therapy (ISCT) 2023 Annual Meeting. The meeting takes place May 31-June 3 in Paris, France. Additionally, a poster will be presented with data from Gamida Cell’s pre-clinical NK cell therapy candidate GDA-501, an engineered intrinsic NK cell with a CAR modification targeting the HER2 protein...Batch-to-batch variability of 18 batches of cryopreserved formulation of GDA-201 from 18 donors demonstrated an overall variability of ≤25% in critical parameters including viability, phenotyping and cytotoxicity...Compared with non-engineered NK cells cultured with NAM, GDA-501 cells displayed increased cytotoxicity against HER2+ tumor cells. When TRAIL was neutralized on GDA-501, a decrease in cytotoxicity was observed."

Clinical • Preclinical • Oncology • Solid Tumor

Tumor Microenvironment Spatial Analysis after Adoptive NK Cell Therapy for Lymphoma Revealed Cross-Talk with Adaptive T-Cell Immunity (TCT-ASTCT-CIBMTR 2023) - P1 | " All patients received lymphodepleting chemotherapy followed by 2 infusions of GDA-201 at days 0 and 2 combined with rituximab and low dose IL2. Overall, our findings from multiplexed spatial profiling support a model in which adoptive NK cell infusions trigger profound immune microenvironment changes which support the influx of host T cells. This occurs early post GDA-201 infusion concurrent with limited blood compartment persistence. Our findings suggest cross-talk with the adaptive arm of the immune system and effective tumor elimination."

Biomarker • IO biomarker • Tumor microenvironment • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • CD4 • CD8 • IL15 • IL2RA • IL7

Gamida Cell Announces Pricing of $23 Million Public Offering of Securities (Businesswire) - "Gamida Cell Ltd...announced the pricing of an underwritten public offering of 17,500,000 ordinary shares and accompanying warrants to purchase 17,500,000 ordinary shares at a public offering price of $1.30 per ordinary share and accompanying warrant, for gross proceeds of approximately $22.8 million, before deducting underwriting discounts and commissions and estimated offering expenses....Gamida Cell intends to use the net proceeds from this offering, together with its existing cash and cash equivalents and trading financial assets, to fund: (i) commercialization activities to support the launch of Omisirge^®; (ii) the continued clinical development of GDA-201; and (iii) general corporate purposes, including general and administrative expenses and working capital."

Financing • Oncology

Gamida Cell Reports Full Year 2022 Financial Results and Provides Company Update (Businesswire) - "The company highlighted positive data, productive regulatory interactions and progress on commercial readiness activities supporting its lead product candidate, omidubicel, an advanced cell therapy candidate for allogeneic stem cell transplant, as it advances toward its May 1, 2023 target Prescription Drug User Fee Act (PDUFA) action date The company also announced a strategic restructuring of its operations to prioritize launch of omidubicel to ensure that, if approved, patients who may potentially benefit will have access to therapy....Continue its GDA-201 Phase 1 study: The company will continue to enroll patients in its GDA-201 Phase 1 dose escalation study."

Commercial • PDUFA date • Oncology

[VIRTUAL] Results of a Phase 1 Trial of Gda-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma (ASH 2020) - "Pts with R/R B-cell NHL or MM received lymphodepleting (LD) therapy with cyclophosphamide (400mg/m2 IV x 3d) and fludarabine (30 mg/m2 /d IV x 3d), followed by GDA-201 (days 0 and 2) and low-dose IL-2 (6 million units sc x 3 doses). Pts with NHL or MM received rituximab (375 mg/m2) or elotuzumab (10 mg/kg), respectively, x 3 weekly infusions... Cellular therapy using GDA-201 with monoclonal antibodies to enhance ADCC was well-tolerated, and demonstrated significant clinical activity in heavily pretreated pts with advanced NHL. Data support the future testing of multiple infusions to potentially enhance anti-tumor effect. The omission of lymphodepleting chemotherapy is feasible and contributes to safety of this approach."

Clinical • IO biomarker • P1 data • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hypertension • Immunology • Indolent Lymphoma • Infectious Disease • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Septic Shock • Thrombocytopenia • Transplantation • IL15

Data Presented on Gamida Cell’s Omidubicel, GDA-201 at the 2023 Tandem Meetings of ASTCT and CIBMTR (Businesswire) - P1 | N=39 | NCT03019666 | "Gamida Cell Ltd...announced that two oral presentations and a poster presentation highlighting Gamida Cell’s investigational product candidates, omidubicel and GDA-201, were shared at the 2023 Tandem Meetings, Transplantation & Cellular Therapy (TCT) Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT), and the Center for International Blood and Marrow Transplant Research (CIBMTR)....'The data presented at Tandem demonstrate Gamida Cell’s expertise in developing potent, potentially curative cell therapies for patients with hematologic malignancies,' said Ronit Simantov, M.D., chief medical and scientific officer of Gamida Cell."

P1 data • Preclinical • Hematological Malignancies • Leukemia • Non-Hodgkin’s Lymphoma • Oncology

"#tandem23 #celltx Bachanova: GDA-201 (Gamidacel) is an allo NK cell product cultured w nicotinamide and IL15 for enhanced effector fxn. ORR 74% in NHL (N=19). Several remain in CR beyond 18mo." (@hemedoc)

IL15

Gda-201: A Cryopreserved, Readily Available Formulation of Nicotinamide-Enabled Natural Killer Cells, Shows Increased Potency and Enhanced Cytotoxicity (TCT-ASTCT-CIBMTR 2023) - "Cells maintained expression of CD56 and CD16 and expressed high levels of the homing and retention marker CD62L. Cryopreserved GDA-201 showed high cytotoxicity and enhanced potency as detected by intracellular secretion of IFN-"

IO biomarker • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • NCAM1

Gamida Cell Announces Closing of $25 Million Financing With Highbridge (Businesswire) - "Gamida Cell Ltd...announced the closing of a senior secured convertible term loan of $25 million with certain funds managed by Highbridge Capital Management, LLC (collectively, 'Highbridge'). Pursuant to the loan agreement, Gamida Cell’s wholly-owned subsidiary, as borrower, will draw down $25 million from the facility with a maturity date of December 12, 2024...The proceeds from the term loan, together with the net proceeds from Gamida Cell’s $20 million public offering of ordinary shares announced on September 27, 2022 and its existing cash and cash equivalents and trading financial assets, are expected to (i) fund commercial readiness and initial launch activities to support launch of omidubicel, if approved; (ii) fund the continued development of its NK product pipeline, including clinical stage asset GDA-201; and (iii) be used for general corporate purposes, including general and administrative expenses and working capital."

Financing • Hematological Malignancies • Oncology • Solid Tumor

Gda-201, a Novel Metabolically Enhanced Allogeneic Natural Killer (NK) Cell Product Yields High Remission Rates in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma (NHL): 2-Year Survival and Correlation with Cytokine IL7 (ASH 2021) - P1 | "Pts with R/R B-cell NHL received lymphodepleting (LD) chemotherapy with cyclophosphamide (400mg/m 2 IV x 3d) and fludarabine (30 mg/m 2 /d IV x 3d), followed by GDA-201 (days 0 and 2) and low-dose IL-2 (6 million units sc x 3 doses at days 0,2,4). Cellular therapy using GDA-201 with rituximab is well-tolerated, and demonstrated significant clinical activity in heavily pre-treated pts with advanced NHL. A cytokine surge following LD chemotherapy appears to be associated with clinical activity. Phase II studies in aggressive and indolent NHL cohorts are planned."

Clinical • IO biomarker • Anemia • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Febrile Neutropenia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hypertension • Immunology • Indolent Lymphoma • Infectious Disease • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Oncology • Septic Shock • Thrombocytopenia • Transplantation • IL15 • IL7 • SELL

GDA-501: Engineered NAM-NK cells with HER2-CAR expression demonstrate increased cytotoxicity against HER2-expressing solid tumors (SITC 2022) - "Methods HER2-CAR-NK cells were developed based on a single-chain variable fragment (scFv) of the widely used humanized monoclonal antibody trastuzumab. Informed consent has been approved by the ECs. The Israeli template of informed consent is in used and it includes study specific information (e.g. study goal, design, method, duration, risks, etc.)."

IO biomarker • Gastric Cancer • Oncology • Ovarian Cancer • Solid Tumor • HER-2 • LAMP1 • TNFA

Gamida Cell to Present Corporate Highlights at the Jefferies London Healthcare Conference (Businesswire) - "Gamida Cell Ltd...will present its corporate highlights at the Jefferies London Healthcare Conference, November 17, 2022 with a presentation at 9:10 a.m. GMT....Ms. Jenkins will discuss 2022 catalysts and potential milestones including the U.S. market opportunity for omidubicel upon potential U.S. Food and Drug Administration (FDA) approval. Additional topics include, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy candidate, GDA-201, as a potential new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically-engineered NK cells."

Commercial • Pipeline update • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology

Ph I Trial of NAM NK Cells and IL-2 for Adult Pts With MM and NHL (clinicaltrials.gov) - P1 | N=39 | Completed | Sponsor: Masonic Cancer Center, University of Minnesota | Recruiting ➔ Completed | N=24 ➔ 39

Enrollment change • Trial completion • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Indolent Lymphoma • Lymphoma • Lymphoplasmacytic Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Waldenstrom Macroglobulinemia • CD20