A Phase I Safety and Feasibility Study of Cellular Immunotherapy for Extensive Stage Small Cell Neuroendocrine Prostate Cancer Using Autologous T Cells Lentivirally Transduced to Express L1CAM-Specific Chimeric Antigen Receptor

This phase I trial studies the side effects and best dose of autologous CD8+ and CD4+ lentivirally transduced to express L1CAM-specific chimeric antigen receptor (CAR) and EGFRt mutation specific T cells and to see how well they work in treating patients with small cell neuroendocrine prostate cancer (SCNPC) that has spread to nearby tissue or lymph nodes (locally advanced) and cannot be removed by surgery (unresectable) or has spread from where it first started (primary site) to other places in the body (metastatic). CAR T-cell therapy is a type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack tumor cells. T cells are taken from a patient's blood. Then the gene for a special receptor that binds to a certain protein on the patient's tumor cells is added to the T cells in the laboratory. Some solid tumor cells have an L1CAM protein on their surface, and T cells can be modified with a receptor, called a chimeric antigen receptor (CAR), to help recognize this protein and kill these tumor cells. Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion for treatment of certain cancers. These L1CAM mutation specific T cells may help the body's immune system identify and kill L1CAM locally advanced and unresectable or metastatic small cell neuroendocrine prostate cancers' tumor cells.

开始日期2025-04-15

申办/合作机构

Fred Hutchinson Cancer Research Center

[+1]

NCT06561347 / Not yet recruiting临床2期IIT

A Phase 2 Multicenter Study of the Combination Zanubrutinib, Bendamustine, and Rituximab in Previously Untreated Waldenström Macroglobulinemia (ZEBRA Trial)

The purpose of this study is to determine the very good partial response (VGPR) or better rate in participants with Waldenström macroglobulinemia (WM).
The names of the study drugs involved in this study are as follows: zanubrutinib, bendamustine, and rituximab.

开始日期2025-01-01

申办/合作机构

The General Hospital Corp.

[+1]

NCT06557330 / Not yet recruiting临床2期IIT

HM-225: Measurable Residual Disease (MRD) Guided De-intensification of Bendamustine/Rituximab (BR) for Indolent Non-Hodgkin Lymphoma

This is a phase II pilot, single arm, open label study designed to assess the efficacy, safety, and feasibility of MRD adapted duration of BR for untreated or R/R iNHL.

开始日期2024-12-01

申办/合作机构

The Fox Chase Cancer Center

[+1]

100 项与盐酸苯达莫司汀相关的临床结果

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100 项与盐酸苯达莫司汀相关的转化医学

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100 项与盐酸苯达莫司汀相关的专利（医药）

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2,804

项与盐酸苯达莫司汀相关的文献（医药）

2024-12-31·JOURNAL OF MEDICAL ECONOMICS

Treatment patterns, healthcare resource utilization, and costs in Medicare patients with diffuse large B-cell lymphoma: a retrospective claims analysis (2015–2020)

Article

作者: Wang, Anthony ; Chhibber, Anindit ; Mutebi, Alex ; Kalsekar, Anupama ; Liang, Chenxue ; Keshishian, Allison ; He, Jing ; Jun, Monika P. ; Wang, Tongsheng ; Rivas Navarro, Fernando

AIMS:

To understand treatment patterns, healthcare resource utilization (HCRU), and the economic burden of diffuse large B-cell lymphoma (DLBCL) in elderly adults in the US.

MATERIALS AND METHODS:

This retrospective database analysis utilized US Centers for Medicare and Medicaid Services Medicare fee-for-service administrative claims data from 2015 to 2020 to describe DLBCL patient characteristics, treatment patterns, HCRU, and costs among patients aged ≥66 years. Patients were indexed at DLBCL diagnosis and required to have continuous enrollment from 12 months pre-index until 3 months post-index. HCRU and costs (USD 2022) are reported as per-patient per-month (PPPM) estimates.

RESULTS:

A total of 11,893 patients received ≥1-line (L) therapy; 1,633 and 391 received ≥2 L and ≥3 L therapies, respectively. Median (Q1, Q3) age at 1 L, 2 L, and 3 L initiation, respectively, was 76 (71, 81), 77 (72, 82), and 77 (72, 82) years. The most common therapy was R-CHOP (70.9%) for 1 L and bendamustine ± rituximab for 2 L (18.7%) and 3 L (17.4%). CAR T was used by 14.8% of patients in 3 L. Overall, 39.6% (1 L), 42.1% (2 L), and 47.8% (3 L) of patients had all-cause hospitalizations. All-cause mean (median [Q1-Q3]) costs PPPM during each line were $22,060 ($20,121 [$16,676-$24,597]) in 1 L, $30,027 ($20,868 [$13,416-$31,016]) in 2 L, and $47,064 ($25,689 [$15,555-$44,149]) in 3 L, with increasing costs driven primarily by inpatient expenses. Total all-cause 3 L mean (median [Q1-Q3]) costs PPPM for patients with and without CAR T were $153,847 ($100,768 [$26,534-$253,630]) and $28,466 ($23,696 [$15,466-$39,107]), respectively.

CONCLUSIONS:

No clear standard of care exists in 3 L therapy for older adults with relapsed/refractory DLBCL. The economic burden of DLBCL intensifies with each progressing line of therapy, thus underscoring the need for additional therapeutic options.

2024-11-01·BRITISH JOURNAL OF HAEMATOLOGY

Targeting CD25+ lymphoma cells with the antibody–drug conjugate camidanlumab tesirine as a single agent or in combination with targeted agents

Article

作者: Bertoni, Francesco ; Van Berkel, Patrick H. ; Scalise, Lorenzo ; Spriano, Filippo ; Zammarchi, Francesca ; Zucca, Emanuele ; Cacciapuoti, Maria Teresa ; Golino, Gaetanina ; Inghirami, Giorgio ; Gaudio, Eugenio ; Cascione, Luciano ; Tarantelli, Chiara ; Stathis, Anastasios

Summary:

Camidanlumab tesirine (ADCT‐301) is a CD25‐specific antibody‐drug conjugate (ADC) employing SG3199, a highly cytotoxic DNA minor groove cross‐linking pyrrolobenzodiazepine dimer. The ADC has shown early clinical antitumour activity in various cancers, including B‐ and T‐cell lymphomas. We assessed its preclinical activity as a single agent in 57 lymphoma cell lines and in combination with selected drugs in T‐cell lymphoma‐derived cell lines. Cells were exposed to increasing concentrations of the ADC or SG3199 for 96 h, followed by an MTT proliferation assay. CD25 expression was measured at cell surface and RNA levels. Experiments with PDX‐derived cell lines were used for validation studies. Camidanlumab tesirine presented more potent single agent in vitro cytotoxic activity in T‐ than B‐cell lymphomas. In vitro activity was correlated with CD25 cell surface and RNA expression. In vitro activity was correlated with CD25 cell surface and RNA expression. When camidanlumab tesirine‐containing combinations were evaluated in four T‐cell lymphoma models, the most active partners were everolimus, copanlisib, venetoclax, vorinostat, and pralatrexate, followed by bortezomib, romidepsin, bendamustine, and 5‐azacytidine. The strong camidanlumab tesirine single‐agent anti‐lymphoma activity and the in vitro synergisms with targeted agents identify potential combination partners for future clinical studies.

2024-11-01·ANNALS OF HEMATOLOGY

Lymphocyte recovery after bendamustine therapy in patients with mantle cell lymphoma. Results of a retrospective analysis and prognostic impact in the CAR-T era

Article

作者: Di Rocco, A ; Di Palma, M. ; Antonacci, M ; Donzelli, Livia ; Antonacci, M. ; Di Rocco, A. ; Zhdanovskaya, N ; Zhdanovskaya, N. ; Martelli, M. ; Martelli, M ; Di Palma, M ; Petrucci, L. ; Petrucci, L

Bendamustine in combination with rituximab (BR) or with rituximab and cytarabine (R-BAC) is the standard first-line immunochemotherapy in mantle cell lymphoma (MCL) for elderly patients and patients ineligible for intensive regimens or autologous transplantation. As bendamustine causes prolonged lymphopenia and the literature lacks evidence of its persistence in patients with MCL, this retrospective analysis aims to estimate the lymphocyte recovery time, also in view of potential immunotherapy with CAR-T cells. Data were collected from 44 consecutive MCL patients who received bendamustine (BR or R-BAC) as first-line therapy at the Hematology Unit of Sapienza University Hospital between May 2011 and April 2022. Twenty patients (45%) were treated with R-BAC and 24 (55%) with BR. At baseline, the median lymphocyte count was 1795/µl (range: 370-11730/µL). One month after the end of therapy, it was 450/µl (range: 50-3300/µl) and 3 months after 768/µl (range: 260-1650/µl). After 6 and 9 months, we observed a gradual increase in median lymphocyte count of 900/µl (range: 370-2560/µl and 130-2770/µl, respectively). After 12 months median lymphocyte count was 1256/µl (range: 240-4140/µl). Median lymphocyte count at 1, 3, 6, and 9 months post-treatment was significantly lower than baseline but showed recovery by the 12 months. This finding is crucial for MCL patients considering CAR-T cell therapy, suggesting a minimum 9-month interval between bendamustine administration and leukapheresis.

398

项与盐酸苯达莫司汀相关的新闻（医药）

2024-11-18

·GlobeNewswire-Health Care

Fate Therapeutics Presents 6-Month Follow-up Data on First Patient Treated in Phase 1 Autoimmunity Study with Fludarabine-free Conditioning and FT819 Off-the-shelf, 1XX CAR T-cell Product Candidate at ACR Convergence

27-year-old African American-Asian Woman with Active Lupus Nephritis Achieved DORIS Clinical Remission; Patient Remains On-study, in Clinical Remission, and Free of All Immunosuppressive Therapies Patient Treated with Fludarabine-free Conditioning and Single-dose FT819; Favorable Safety Profile with No Grade ≥3 Adverse Events and No Events of CRS, ICANS, or GvHD Reconstituted B Cell Compartment Predominantly Consists of Naïve, Non-class Switched B Cells with Deep Depletion of Aberrant B Cells and Plasmablasts, Indicative of Immune Reset Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning Chemotherapy Opened for Enrollment SAN DIEGO, Nov. 18, 2024 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders, today presented initial clinical and translational data from the first patient treated in its FT819 Phase 1 Autoimmunity study for moderate-to-severe systemic lupus erythematosus (SLE) at the American College of Rheumatology (ACR) Convergence being held in Washington, D.C. The patient, a 27-year-old African American-Asian woman diagnosed with lupus nephritis (LN) over ten years ago, received fludarabine-free conditioning followed by a single dose of FT819. The patient achieved DORIS (definition of remission in SLE) clinical remission and LLDAS (low lupus disease activity state) as of Month 6 follow-up. The patient continues on-study, in clinical remission, and free of all immunosuppressive therapies as of a data cutoff date of November 11, 2024. FT819 is the Company’s off-the-shelf, CD19-targeted, 1XX CAR T-cell product candidate comprised of CD8αβ+ T cells with a memory phenotype and high CXCR4 expression to promote tissue trafficking. “These initial results are incredibly promising. Our patient not only went into drug-free clinical remission but also had resolution of fatigue, something that we as rheumatologists struggle to improve with our treatments,” said Jennifer Medlin, M.D., and Principal Investigator at the University of Nebraska Medical Center. “If we continue to see similar results in other patients with an acceptable safety profile, off-the-shelf CAR T-cell therapy could be a complete game-changer for our sickest lupus patients. This gives me hope for a future where we can make a great impact on these patients with devastating disease.” Patient 1 Case Study The patient presented with active LN and severe disease, which was marked by renal BILAG A (British Isles Lupus Assessment Group) disease activity score based on biopsy, SLEDAI-2K (Systemic Lupus Erythematosus Disease Activity Index) score of 20, FACIT-Fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue) score of 33 (range 0-52, where a score of 52 indicates no fatigue) and PGA (Physician Global Assessment) score of 2.5 (where a score of 3 indicates most severe activity). Following administration of fludarabine-free conditioning and treatment with a single dose of FT819 at 360 million cells, the patient was discharged from the hospital without notable adverse events (AEs) after a protocol-required three-day stay. As of the data cutoff date, the patient has experienced no Grade ≥3 AEs, no serious adverse events (SAEs), and no events of any grade of cytokine release syndrome (CRS), immune effector-cell associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD). The patient reported that her debilitating fatigue had entirely resolved without further treatment, and treatment with methylprednisolone was discontinued at Month 3. The patient achieved DORIS clinical remission, including with resolution of arthritis and active urinary sediment and with a substantial reduction in proteinuria, as of Month 6 follow-up. As of the data cutoff date, the patient continues in DORIS clinical remission and remains free of all immunosuppressive therapy. Rapid elimination of CD19+ B cells in the periphery was observed following fludarabine-free conditioning and treatment with a single dose of FT819. B-cell recovery by Month 3 was predominantly comprised of naïve, non-class switched B cells with near-complete elimination of switched memory B cells and deep depletion of plasmablasts, indicative of an immune reset. Notably, the unique double-negative (DN) B-cell subset, previously described and associated with severe SLE, was nearly eliminated. “These seminal data with fludarabine-free conditioning and off-the-shelf CAR T-cell therapy in autoimmunity are exciting, and we are very pleased that the first patient with active lupus nephritis had a favorable clinical experience, achieved drug-free clinical remission, and continues on-study free of all immunosuppressive therapy,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “We believe our highly-differentiated therapeutic approach has the potential to transform outcomes for patients with autoimmune diseases without requiring patient apheresis, discontinuation of maintenance therapy, intense conditioning chemotherapy, and extended hospitalization.” FT819 Phase 1 Autoimmunity Study The ongoing multi-center, Phase 1 clinical trial for patients with moderate-to-severe SLE is designed to evaluate the safety, pharmacokinetics, and anti-B cell activity of FT819 (NCT06308978). The first three patients, all of whom presented with active LN despite having been treated with multiple standard-of-care therapies, received fludarabine-free conditioning consisting of either cyclophosphamide alone or bendamustine alone, followed by a single dose of FT819 at 360 million cells. All three patients remain on-study, and there have been no dose-limiting toxicities (DLTs) and no events of any grade of CRS, ICANS, or GvHD. The Company plans to present clinical and translational data from the first three patients at the American Society of Hematology (ASH) Annual Meeting being held in San Diego, CA on December 7-10. The Company is also enrolling a second treatment arm under the FT819 Phase 1 Autoimmunity study to assess the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 as an add-on to maintenance therapy without conditioning chemotherapy in patients with SLE. This new arm is being conducted in parallel with the study’s conditioning arm. About Fate Therapeutics’ iPSC Product Platform Human induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be combined and administered with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the manufacture of cell therapies using patient- or donor-sourced cells. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications. About Fate Therapeutics, Inc. Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived natural killer (NK) cell and T-cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com. Forward-Looking Statements This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Company’s iPSC-derived CAR T-cell product candidates, including FT819, the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, including the expected clinical development plans for FT819, the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the timing and availability of data from the Company’s clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, the Company’s clinical and product development strategy, and the Company’s expectations regarding progress, plans, and timelines. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties or delays in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise. Contact: Christina TartagliaPrecision AQ212.362.1200christina.tartaglia@precisionaq.com

临床结果免疫疗法细胞疗法临床1期ASH会议

2024-11-12

·GlobeNewswire-Health Care

Fate Therapeutics Reports Third Quarter 2024 Financial Results and Business Updates

Initial Clinical Data from Phase 1 Autoimmunity Study of FT819 1XX CAR T-cell Product Candidate to be Presented in 4Q24; Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning Chemotherapy Opened for Enrollment FT522 Multi-indication IND Application for Conditioning-free Treatment of B Cell-mediated Autoimmune Diseases Allowed by FDA; Initial Phase 1 Clinical Data in B-cell Lymphoma to be Presented at ACR Convergence Initial Low-Dose Cohort of FT825 / ONO-8250 Shows Favorable Safety Profile in Phase 1 Solid Tumor Study; Peripheral Blood from First Three Patients Demonstrates CAR T-cell Expansion with Maintenance of Activated State $331 Million in Cash, Cash Equivalents and Investments with Projected Operating Runway through YE26 SAN DIEGO, Nov. 12, 2024 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, today reported business highlights and financial results for the third quarter ended September 30, 2024. “We look forward to sharing initial Phase 1 clinical data of our off-the-shelf FT819 CAR T-cell product candidate in systemic lupus erythematosus at ACR Convergence and ASH. We continue to make great strides in our pursuit of therapeutic differentiation for patients with B cell-mediated autoimmune diseases, and patient enrollment is ongoing assessing FT819 with fludarabine-free conditioning as well as in a new treatment arm as add-on to maintenance therapy without conditioning chemotherapy,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “In addition, we are very pleased with the Phase 1 study design allowed by the FDA for our FT522 ADR-armed CAR NK cell product candidate in autoimmunity, which is designed to assess multiple doses of FT522 without conditioning chemotherapy across a basket of B cell-mediated autoimmune diseases, and we look forward to presenting an initial look at the clinical and translational data of FT522 in B-cell lymphoma at ACR.” FT819 iPSC-derived 1XX CAR T-cell Program Three Patients Treated in Phase 1 Autoimmunity Study using Fludarabine-free Conditioning. The ongoing multi-center, Phase 1 clinical trial for patients with moderate-to-severe systemic lupus erythematosus (SLE) is designed to evaluate the safety, pharmacokinetics, and anti-B cell activity of FT819, the Company’s off-the-shelf CD8αβ+ T-cell product candidate that incorporates a CD19-targeted chimeric antigen receptor (CAR) with a novel 1XX costimulatory domain into the T-cell receptor alpha constant (TRAC) locus (NCT06308978). The first three patients, all of whom presented with active lupus nephritis (LN) despite having been treated with multiple standard-of-care therapies, received fludarabine-free conditioning consisting of either bendamustine alone or cyclophosphamide alone, followed by a single dose of FT819 at 360 million cells (Regimen A). All three patients remain on-study, and there have been no dose-limiting toxicities (DLTs) and no events of any grade of cytokine release syndrome (CRS), immune effector-cell associated neurotoxicity syndrome (ICANS), or graft-versus-host disease (GvHD). The Company plans to present clinical and translational data from the first three patients at the American Society of Hematology (ASH) Annual Meeting being held in San Diego, CA on December 7-10.First SLE Patient Case Study to be Presented at ACR Convergence. The first patient treated in the ongoing FT819 Phase 1 Autoimmunity study was a 27 year-old woman diagnosed with LN over ten years ago who received fludarabine-free conditioning followed by a single dose of FT819 at 360 million cells. The first patient has completed six-month follow-up and remains on-study. The Company plans to present clinical and translational data from the first patient at the American College of Rheumatology (ACR) Convergence being held in Washington, D.C. on November 16-19.Initiated Second Treatment Arm Adding FT819 to Maintenance Therapy without Conditioning. The Company amended the clinical protocol of its FT819 Phase 1 Autoimmunity study to include a second treatment arm (Regimen B) to assess the safety, pharmacokinetics, and anti-B cell activity of a single dose of FT819 as an add-on to maintenance therapy without conditioning chemotherapy in patients with SLE. The new arm is open for enrollment at a starting cell dose of 360 million cells, and is being conducted in parallel with Regimen A. FT825 / ONO-8250 iPSC-derived CAR T-cell Program Initial Phase 1 Clinical Data Presented at 2024 SITC. Under its collaboration with Ono Pharmaceutical Co., Ltd. (Ono), the Company is conducting a multi-center, Phase 1 study to assess the safety, pharmacokinetics, and activity of FT825 / ONO-8250, a multiplexed-engineered CAR T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2), in patients with advanced solid tumors (NCT06241456). At the 2024 Society of Immunotherapy of Cancer (SITC) 39th Annual Meeting, the Company presented initial clinical data from three heavily pre-treated patients, all of whom were previously treated with at least five prior lines of therapy including HER2-targeted therapy. Each patient was administered conditioning chemotherapy and a single dose of FT825 / ONO-8250 at the first dose level of 100 million cells. As of a data cutoff date of October 25, 2024, FT825 / ONO-8250 demonstrated a favorable safety profile with no DLTs and no events of any grade of CRS, ICANS, or GvHD. In addition, at Day 8 following treatment, peak CAR T-cell expansion was observed and phenotyping of FT825 / ONO-8250 sourced from the patients’ peripheral blood was indicative of an activated state (as evidenced by high levels of Granzyme B expression and maintenance of CAR expression) with no evidence of exhaustion (as evidenced by low levels of PD-1 and TIM3 expression). Enrollment is currently ongoing at the second dose level of 300 million cells as monotherapy and at the first dose level of 100 million cells in combination with epidermal growth factor receptor (EGFR)-targeted monoclonal antibody therapy.New Preclinical Data Demonstrates Cancer-selectivity of Novel H2CasMab-2 CAR. FT825 / ONO-8250 incorporates seven synthetic controls of cell function including a novel cancer-selective H2CasMab-2 CAR, which has exhibited similar potency with greater specificity for cancer cells expressing HER2 compared to trastuzumab in preclinical studies. New preclinical data presented at SITC demonstrated potent HER2-specific, anti-tumor activity in both in vitro and in vivo settings with limited cytolytic targeting of HER2+ normal cells. The on-tumor selectivity of FT825 / ONO-8250 was attributed to its incorporation of a novel HER2-targeted antigen binding domain, which was derived from a cancer-specific monoclonal antibody H2CasMab-2 (Kaneko et al., 2024), that was shown to differentially and preferentially recognize both locally misfolded HER2 and p95 truncation variants of HER2 as compared to trastuzumab. FT522 iPSC-derived CAR NK Cell Program Initial Clinical Data from Phase 1 BCL Study to be Presented at ACR Convergence. FT522 is the Company’s off-the-shelf, CD19-targeted CAR NK cell product candidate and its first to incorporate Alloimmune Defense Receptor (ADR) technology, which is designed to reduce or eliminate the need for administration of conditioning chemotherapy to patients receiving cell therapies. In its ongoing multi-center, Phase 1 clinical trial of FT522 in patients with relapsed / refractory B-cell lymphoma (BCL) (NCT05950334), the Company is currently enrolling patients in the second three-dose cohort at 900 million cells per dose with conditioning chemotherapy (Regimen A) and in the first three-dose cohort at 300 million cells per dose without conditioning chemotherapy (Regimen B). No DLTs, and no events of any grade of CRS, ICANS, or GvHD, have been reported in the Phase 1 study. The Company plans to present initial clinical and translational data from the Phase 1 BCL study at ACR Convergence.IND Application for Phase 1 Basket Study in Autoimmunity Allowed by FDA. The U.S. Food and Drug Administration (FDA) has allowed the Company’s Investigational New Drug (IND) application to assess the safety, pharmacokinetics, and activity of FT522 across a basket of B cell-mediated autoimmune diseases. The Phase 1 study is intended to treat patients with multiple doses of FT522 without conditioning chemotherapy as an add-on to rituximab induction therapy (Regimen A) and as an add-on to maintenance therapy in combination with rituximab (Regimen B). Dose escalation is expected to commence at 900 million cells per dose. The Company previously presented preclinical data from a novel re-challenge assay using peripheral blood mononuclear cells (PBMCs) from unmatched SLE donors, showing that FT522 uniquely drove rapid and deep CD19+ B cell depletion, maintained functional persistence, and eliminated alloreactive T cells, indicating that FT522 has the potential to function effectively in the presence of an unmatched host immune system. Third Quarter 2024 Financial Results Cash & Investment Position: Cash, cash equivalents, and investments as of September 30, 2024 were $330.5 million.Total Revenue: Revenue was $3.1 million for the third quarter of 2024, which was derived from the conduct of preclinical development activities for a second collaboration candidate targeting an undisclosed solid tumor antigen under its collaboration with Ono.Total Operating Expenses: Total operating expenses were $55.5 million for the third quarter of 2024, including research and development expenses of $34.7 million and general and administrative expenses of $20.8 million. Such amounts included $11.8 million of non-cash stock-based compensation expense.Shares Outstanding: As of September 30, 2024, common shares outstanding were 113.9 million, pre-funded warrants outstanding were 3.9 million, and preferred shares outstanding were 2.8 million. Each preferred share is convertible into five common shares. About Fate Therapeutics’ iPSC Product PlatformHuman induced pluripotent stem cells (iPSCs) possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s proprietary iPSC product platform combines multiplexed-engineering of human iPSCs with single-cell selection to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a starting cell source to manufacture engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be combined and administered with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the manufacture of cell therapies using patient- or donor-sourced cells. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 500 issued patents and 500 pending patent applications. About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases. Using its proprietary iPSC product platform, the Company has established a leadership position in creating multiplexed-engineered master iPSC lines and in the manufacture and clinical development of off-the-shelf, iPSC-derived cell products. The Company’s pipeline includes iPSC-derived natural killer (NK) cell and T-cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit www.fatetherapeutics.com. Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Company’s results of operations, financial condition, anticipated operating expenses and cash runway, and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to the Company's product candidates, clinical studies and preclinical research and development programs, the Company’s progress, plans and timelines for the clinical investigation of its product candidates, including the initiation and continuation of enrollment in the Company’s clinical trials, the initiation of additional clinical trials and additional dose cohorts in ongoing clinical trials of the Company’s product candidates, the availability of data from the Company’s clinical trials, the therapeutic and market potential of the Company’s research and development programs and product candidates, the Company’s clinical and product development strategy, and the Company’s expectations regarding progress and timelines, and the objectives, plans and goals of its collaboration with Ono. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Company’s research and development programs and product candidates, including those product candidates in clinical investigation, may not demonstrate the requisite safety, efficacy, or other attributes to warrant further development or to achieve regulatory approval, the risk that results observed in prior studies of the Company’s product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Company’s product candidates or in the initiation and conduct of, or enrollment of patients in, any clinical trials, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, changes in the therapeutic, regulatory, or competitive landscape for which the Company’s product candidates are being developed, the amount and type of data to be generated or otherwise to support regulatory approval, difficulties or delays in patient enrollment and continuation in the Company’s ongoing and planned clinical trials, difficulties in manufacturing or supplying the Company’s product candidates for clinical testing, failure to demonstrate that a product candidate has the requisite safety, efficacy, or other attributes to warrant further development, and any adverse events or other negative results that may be observed during preclinical or clinical development), the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects, the risk that the Company may not comply with its obligations under and otherwise maintain its collaboration agreement with Ono, the risk that research funding and milestone payments received by the Company under its collaboration may be less than expected, and the risk that the Company may incur operating expenses in amounts greater than anticipated. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Company’s periodic filings with the Securities and Exchange Commission, including but not limited to the Company’s most recently filed periodic report, and from time to time in the Company’s press releases and other investor communications. Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise. Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(unaudited) Three Months Ended Nine Months Ended September 30, September 30, 2024 2023 2024 2023 Collaboration revenue$3,074 $1,944 $11,771 $61,857 Operating expenses: Research and development 34,650 34,275 101,392 140,780 General and administrative 20,801 18,948 58,907 63,513 Total operating expenses 55,451 53,223 160,299 204,293 Loss from operations$(52,377) $(51,279) $(148,528) $(142,436)Other income (expense): Interest income 4,438 4,697 13,414 12,772 Change in fair value of stock price appreciation milestones (13) 1,049 149 3,160 Other Income 274 363 856 9,698 Total other income, net 4,699 6,109 14,419 25,630 Net loss$(47,678) $(45,170) $(134, 109) $(116,806)Other comprehensive income: Unrealized gain on available-for-sale securities, net 1,257 88 820 1,355 Comprehensive loss$(46,421) $(45,082) $(133,289) $(115,451)Net loss per common share, basic and diluted$(0.40) $(0.46) $(1.19) $(1.19)Weighted–average common shares used to compute basic and diluted net loss per share 117,768,161 98,568,012 112,305,430 98,342,898 Condensed Consolidated Balance Sheets(in thousands)(unaudited) September 30, December 31, 2024 2023 Assets Current assets: Cash and cash equivalents$37,909 $41,870 Accounts receivable 4,127 1,826 Short-term investments 259,014 273,305 Prepaid expenses and other current assets 9,244 14,539 Total current assets$310,294 $331,540 Long-term investments 33,607 980 Operating lease right-of-use asset 58,441 61,675 Other long-term assets 92,628 112,022 Total assets$494,970 $506,217 Liabilities and stockholders’ equity Current liabilities: Accounts payable and accrued expenses$29,929 $32,233 Deferred revenue 600 685 Operating lease liability, current portion 6,893 6,176 Total current liabilities$37,422 $39,094 CIRM award liability 1,940 --- Operating lease liability, net of current portion 92,085 97,360 Stock price appreciation milestones 1,197 1,346 Stockholders’ equity 362,326 368,417 Total liabilities and stockholders’ equity$494,970 $506,217 Contact:Christina TartagliaPrecision AQ212.362.1200christina.tartaglia@precisionaq.com

临床1期细胞疗法免疫疗法ASH会议财报

2024-11-12

·健识局

单药年销售有望突破10亿！诺诚健华又行了

诺诚健华成功翻盘。 11月11日，诺诚健华发布三季报：前三季度实现营收6.98亿元，同比增长近30%；第三季度单季实现营收2.77亿元，同比大增73.96%，环比也有9%的增长。去年第三季度，公司营收同比下降18%，环比下降15%，遭遇增速放缓的质疑。但今年，BTK抑制剂奥布替尼销售增长很快：前三季度，奥布替尼收入达6.93亿元，同比增长45%，第三季度达到2.76亿元，是奥布替尼上市以来单季最高收入。公司表示，这一业绩表现主要归功于奥布替尼是中国首个且唯一针对边缘区淋巴瘤(MZL适应症)获批上市的BTK抑制剂。这一新适应症2023年纳入医保，今年实现快速放量。诺诚健华再次上调了全年的业绩指引，从去年年报的25%提升到35%。业内预测，今年奥布替尼收入就将突破10亿元大关，BTK抑制剂领域又一款“重磅炸弹药物”呼之欲出。 MZL领域弯道超车奥布替尼2021年底进入医保，2022年开始放量。 2022年，百济神州的BTK抑制剂泽布替尼在中国市场销售额超过10亿元，迈入“重磅炸弹药物”序列。奥布替尼则稍显逊色，2022年、2023年销售额分别只有5.66亿元、6.71亿元。直到今年，奥布替尼销售收入才有了明显起色。诺诚健华表示，这一成绩主要归因于奥布替尼在MZL上抢先上市并进入医保。MZL属于惰性淋巴瘤，是中国第二常见的B细胞型非霍奇金淋巴瘤，约占所有淋巴瘤的8.3%。华中科技大学同济医学院附属协和医院院长胡豫曾表示，MZL病程长、易反复复发，而且药物可选性低。近几年国内没有新药在MZL上面获批上市。包括利妥昔单抗和苯达莫司汀等常用于淋巴瘤治疗的药物在MZL上也没有适应症注册。 2017年，伊布替尼曾获FDA加速批准用于治疗MZL，但由于上市后在验证性III期临床中数据表现不佳，没有达到主要终点。去年4月，强生和艾伯维申请撤回在这一适应症的批准；2021年起，泽布替尼基于两项2期单臂试验，相继拿下美国FDA、欧盟的二线R/R MZL批准，但在国内却迟迟不见动静。于是，奥布替尼就成了中国首个且唯一能用于MZL治疗的BTK药物，是CSCO诊疗指南中推荐的二线治疗I级药物。今年2月，诺诚健华引入近30年医药从业经验的老将陈少峰，打造商业化团队，销售成绩提升明显。自免领域布局奥布替尼在自免领域还有不少潜力，今年最大的进展来自于多发性硬化症。目前，全球范围内尚无BTK抑制剂在这一领域获批。去年2月，渤健公司退回了奥布替尼多发性硬化的全球独家权利，一度引发诺诚健华股价大跌。这次诺诚健华透露，FDA已同意启动奥布替尼治疗原发进展型多发性硬化（PPMS）的III期临床研究，同时建议启动治疗继发进展型多发性硬化（SPMS）的III期临床研究。同时，奥布替尼治疗特发性血小板减少性紫癜，预计明年上半年完成全部患者入组；治疗红斑狼疮的IIb期临床试验已完成患者入组。值得一提的是，奥布替尼也是全球首个在红斑狼疮II期临床试验中显示出疗效的BTK抑制剂。除此之外，公司还在开发BCL2抑制剂ICP-248等管线与奥布替尼的联用方案，试图进一步拓宽商业前景。今年前三季度，诺诚健华研发费用同比增加11.9%，达到6.15亿元。截至第三季度，诺诚健华账上余有78亿元，这些钱能砸出一款大药吗？市场拭目以待。撰稿丨李傲编辑丨江芸贾亭运营｜韩瑾睿插图｜视觉中国声明：健识局原创内容，未经许可请勿转载药品追溯码上传率不达标，134家药店被医保“清退” 巡视一个月后，医科大学原副校长被带走新事 | 一家药企被暂停采购资格

CSCO会议上市批准临床3期财报临床2期

100 项与盐酸苯达莫司汀相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D07085	盐酸苯达莫司汀	L01AA09	DB06769

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
弥漫性大B细胞淋巴瘤	日本	SymBio Pharmaceuticals Ltd.	2021-04-28
B细胞淋巴瘤	中国	Cephalon, Inc.	2018-12-17
B细胞淋巴瘤	中国	Roche Holding AG	2018-12-17
淋巴细胞白血病	日本	SymBio Pharmaceuticals Ltd.	2016-08-26
惰性B细胞非霍奇金淋巴瘤	美国	Eagle Pharmaceuticals, Inc.	2015-12-07
套细胞淋巴瘤	日本	SymBio Pharmaceuticals Ltd.	2010-10-27
慢性淋巴细胞白血病	美国	Cephalon, Inc.	2008-03-20
难治性转化惰性 B 细胞非霍奇金淋巴瘤	加拿大	Dr. Reddy's Laboratories Ltd.	-

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
难治性B细胞淋巴瘤	临床3期	中国	山东蓝金生物工程有限公司	2010-04-01
惰性非霍奇金淋巴瘤	临床3期	美国	Cephalon, Inc.	2005-10-01
惰性非霍奇金淋巴瘤	临床3期	加拿大	Cephalon, Inc.	2005-10-01
惰性非霍奇金淋巴瘤	临床3期	波多黎各	Cephalon, Inc.	2005-10-01
典型霍奇金淋巴瘤	临床2期	美国	Bristol Myers Squibb Co.	2018-11-26
难治性经典霍奇金淋巴瘤	临床2期	美国	Bristol Myers Squibb Co.	2017-03-28
难治性经典霍奇金淋巴瘤	临床2期	加拿大	Bristol Myers Squibb Co.	2017-03-28
难治性经典霍奇金淋巴瘤	临床2期	捷克	Bristol Myers Squibb Co.	2017-03-28
难治性经典霍奇金淋巴瘤	临床2期	法国	Bristol Myers Squibb Co.	2017-03-28
难治性经典霍奇金淋巴瘤	临床2期	德国	Bristol Myers Squibb Co.	2017-03-28

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临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASH2024	N/A	套细胞淋巴瘤	-	Bendamustine/Rituximab (BR)	糧衊築夢餘顧選製壓艱(餘鹹範醖膚繭觸夢衊膚) = 餘願願鹽窪鬱齋簾衊獵淵廠鬱鹽壓網醖製艱齋 (餘築簾膚簾齋繭鹽衊獵, 59.8 ~ 79.7) 更多	-	2024-12-09
ASH2024	N/A	滤泡性淋巴瘤	-	Tazemetostat 800mg BID	願衊鹽遞壓構廠製醖憲(襯壓鑰壓壓襯選鹹鬱繭) = leukopenia (n=2), neutropenia (n=1), anemia (n=1), atrial fibrillation (n=1), bone pain (n=1), infection (n=2), and infusion related reaction (n=1) 蓋選壓積襯齋鏇夢鬱窪 (範醖鬱鏇繭構憲夢鹹觸 ) 更多	-	2024-12-09
ASH2024	N/A	惰性B细胞非霍奇金淋巴瘤	-	Bendamustine Rituximab	憲廠衊網願憲窪鏇鹹鹽(鹽蓋鹹顧壓繭衊鹹築鏇) = 廠選顧糧鹹蓋鑰餘窪餘構廠衊齋鑰艱憲蓋網築 (獵觸鹹壓蓋壓廠獵鑰蓋, 8.6 ~ 10.4)	-	2024-12-08
				Placebo	憲廠衊網願憲窪鏇鹹鹽(鹽蓋鹹顧壓繭衊鹹築鏇) = 製淵鏇範壓觸願積鹹獵構廠衊齋鑰艱憲蓋網築 (獵觸鹹壓蓋壓廠獵鑰蓋, 5.9 ~ 9.4)
ASH2024	N/A	套细胞淋巴瘤	-	Acalabrutinib plus bendamustine-rituximab (ABR)	窪襯鑰簾觸齋構積廠鑰(鬱襯夢糧簾壓壓艱齋鹹) = 窪顧艱鑰蓋範淵積窪範壓鏇齋膚繭襯積蓋艱製 (積膚選鹽鏇艱鹽餘廠鏇 ) 更多	-	2024-12-07
				Placebo plus bendamustine-rituximab (PBR)	窪襯鑰簾觸齋構積廠鑰(鬱襯夢糧簾壓壓艱齋鹹) = 願艱夢蓋範顧顧壓壓膚壓鏇齋膚繭襯積蓋艱製 (積膚選鹽鏇艱鹽餘廠鏇 ) 更多
NCT03311126 (CTgov)	临床2期	套细胞淋巴瘤	21	Bendamustine+Obinutuzumab	蓋製鑰齋齋願選艱遞淵(簾簾膚選糧願憲廠醖鏇) = 壓構願積窪簾齋夢艱窪顧鬱獵窪範蓋築憲憲蓋 (鏇壓構製鹽膚鹽鏇鏇憲, 鏇繭餘網築衊醖顧繭鏇 ~ 窪遞範衊製鹹膚獵簾願) 更多	-	2024-10-24
NCT02002598 (CTgov)	临床1/2期	多发性骨髓瘤	23	Bendamustine+Carfilzomib+Dexamethasone	鬱繭鏇構襯衊廠糧顧顧(淵鬱膚築壓願積簾壓膚) = 鹹遞鹽簾淵夢餘鹹蓋齋積鑰範網糧選膚廠齋鹽 (膚鹽醖繭築簾鹽獵製鏇, 積簾廠醖壓願顧範鑰簾 ~ 繭選蓋醖衊積積鏇範顧) 更多	-	2024-09-19
SOHO2024	N/A	难治性B细胞淋巴瘤	-	Obinutuzumab+Bendamustine+Lenalidomide	壓構鑰築獵壓積鹽積範(獵蓋艱觸觸膚觸願鏇遞) = 40% 網淵簾範餘範糧繭構鑰 (簾鬱網鑰憲艱鹹壓壓壓 ) 更多	-	2024-09-04
NCT00974233 (Pubmed)	临床2期	慢性淋巴细胞白血病 \| 小淋巴细胞淋巴瘤维持	-	Bendamustine + Rituximab	窪觸襯齋鑰範衊廠餘獵(膚蓋憲廠觸積獵顧衊膚) = 範鹽選餘齋餘遞淵積壓淵築積醖齋鑰選構積餘 (糧衊簾廠製構遞淵膚憲 ) 更多	积极	2024-06-10
				Lenalidomide + Rituximab	窪觸襯齋鑰範衊廠餘獵(膚蓋憲廠觸積獵顧衊膚) = 顧膚鏇衊壓鑰艱顧製願淵築積醖齋鑰選構積餘 (糧衊簾廠製構遞淵膚憲 )
NCT01415752 (E1411, Pubmed)	临床2期	套细胞淋巴瘤维持	373	Bendamustine-Rituximab (BR)	簾獵簾夢選廠繭襯鹽網(積鏇窪糧觸遞簾艱襯製) = 窪夢廠顧鑰糧願願範齋鹽願鏇繭齋窪蓋醖構膚 (網艱鑰顧積壓蓋繭築襯 )	不佳	2024-05-31
				Bortezomib + Bendamustine-Rituximab (BVR)	簾獵簾夢選廠繭襯鹽網(積鏇窪糧觸遞簾艱襯製) = 範窪選憲觸蓋窪鏇選醖鹽願鏇繭齋窪蓋醖構膚 (網艱鑰顧積壓蓋繭築襯 )
MAINTAIN (ASCO2024)	N/A	套细胞淋巴瘤维持	796	Rituximab maintenance	糧鏇顧獵範遞網願夢網(選簾夢鏇憲鏇鬱遞膚觸) = 鑰衊製壓淵遞製壓襯衊簾齋夢醖糧範蓋顧淵網 (鑰觸鹹鏇觸遞夢衊憲構 ) 更多	积极	2024-05-24
				No rituximab maintenance	糧鏇顧獵範遞網願夢網(選簾夢鏇憲鏇鬱遞膚觸) = 醖齋獵廠願遞遞齋願糧簾齋夢醖糧範蓋顧淵網 (鑰觸鹹鏇觸遞夢衊憲構 ) 更多