This is an open label, Phase 1b, multiple ascending dose, and dose-expansion study of IDP-023 administered in combination with interleukin-2 (IL-2) and ocrelizumab to evaluate the safety, tolerability, and biologic activity on autoreactive immune cells in patients with refractory progressive multiple sclerosis.

Start Date30 Jun 2026

Sponsor / Collaborator

Indapta Therapeutics, Inc.

NCT06815003

/ Not yet recruitingPhase 2IIT

Phase-2 Study of Vedolizumab Plus Post-Transplant Cyclophosphamide and Short Course Tacrolimus for Graft-versus-Host Disease Prevention After Reduced Intensity Conditioning Peripheral Blood Stem Cell Allogeneic Hematopoietic Cell Transplantation

This phase II trial studies how well vedolizumab plus post-transplant cyclophosphamide (PTCy) and short course tacrolimus work for the prevention of graft versus host disease (GVHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT) after reduced intensity conditioning. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a donor. Giving reduced conditioning chemotherapy before an allogeneic HCT helps kill cancer cells in the body and helps make room in the patient's bone marrow for new stem cells to grow using less than standard doses of chemotherapy. Sometimes, the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Vedolizumab is a monoclonal antibody, which is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). It may reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus suppresses the immune system by preventing the activation of certain types of immune cells. Giving vedolizumab plus PTCy and short course tacrolimus may be effective at preventing GVHD after allogeneic HCT.

Start Date08 Jan 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

NCT05521087

/ WithdrawnPhase 1

A Phase I/Ib Study of JNJ-75276617 in Combination With Conventional Chemotherapy for Pediatric and Young Adult Participants With Relapsed/Refractory Acute Leukemias Harboring KMT2A, NPM1, or Nucleoporin Gene Alterations

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2Ds) of JNJ-75276617 in combination with a conventional chemotherapy backbone in pediatric and young adult participants with relapsed/refractory acute leukemia harboring histone-lysine N-methyltransferase 2A1 ([KMT2A1], nucleophosmin 1 gene (NPM1), or nucleoporin alterations in Part 1 (Dose Escalation) and to further evaluate safety at the RP2D(s) of JNJ-75276617 in combination with chemotherapy in pediatric and young adult participants with relapsed/refractory acute leukemia harboring KMT2A1, NPM1, or nucleoporin alterations and safety at the RP2D(s) of JNJ-75276617 as monotherapy in a select low burden of disease cohort in Part 2 (Dose Expansion).

Start Date26 Dec 2025

Sponsor / Collaborator

Janssen Research & Development LLC

100 Clinical Results associated with Fludarabine Phosphate

100 Translational Medicine associated with Fludarabine Phosphate

100 Patents (Medical) associated with Fludarabine Phosphate

8,278

Literatures (Medical) associated with Fludarabine Phosphate

31 Dec 2025·JOURNAL OF MEDICAL ECONOMICS

US consumer and healthcare professional preferences for combination COVID-19 and influenza vaccines

Article

Author: Ghaswalla, Parinaz ; Kent, Cannon ; Poulos, Christine ; Rudin, Deborah ; Mehta, Darshan ; Buck, Philip O.

AIMS:

To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines.

MATERIALS AND METHODS:

This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine.

RESULTS:

Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18-64 with ≥1 risk factor over the standalone influenza vaccine.

LIMITATIONS:

Results are subject to potential hypothetical, responder, selection, and information biases.

CONCLUSIONS:

Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

31 Dec 2025·ANNALS OF MEDICINE

Efficacy and safety of low-dose TBI combined MAC regimen for HSCT in high-risk AML patients with active disease

Article

Author: Chen, Can ; Qian, Shenxian ; Tan, Junfeng ; Shi, Pengfei ; Huang, Xilian ; Chen, Kuang ; Gao, Daquan ; Xu, Ying ; Liu, Lirong ; Xie, Yaping ; Fan, Yang

BACKGROUND:

The management of high-risk acute myeloid leukaemia (AML) remains challenging, highlighting the need for innovative conditioning strategies beyond current regimens.

METHODS:

In the present single-arm study, a FACT regimen comprised of low-dose total body irradiation (TBI) with fludarabine, cytarabine and cyclophosphamide was employed to treat cytogenetically high-risk AML patients exhibiting pre-transplant active disease. This clinical trial is registered in the Chinese Clinical Trial Registry with the registration number ChiCTR2000035111.

RESULTS:

In this study, 21 high-risk AML patients with pre-transplant disease statuses including primary induction failure, relapse and measurable residual disease positivity, were enrolled to undergo FACT conditioning. The FACT group demonstrated a 1-year non-relapse mortality (NRM) rate of 9.5%, indicating a similar level of safety and tolerability among the conditioning regimens. The estimated cumulative incidence of grade 2-4 acute graft-versus-host disease (GVHD) at one year was 30.7%. Additionally, the cumulative incidence of chronic GVHD was 36.0% at one year and increased to 43.0% at two years.

CONCLUSIONS:

The FACT regimen is an effective myeloablative conditioning (MAC) strategy for high-risk AML patients, potentially reducing relapse risk without increasing NRM, warranting further research.

01 Dec 2025·JOURNAL OF CLINICAL IMMUNOLOGY

Early Haploidentical Hematopoietic Stem Cell Transplantation Provides Rapid Leukocyte and Immune Reconstitution in AK2 Patient Identified by TREC Newborn Screening

Article

Author: Meisel, Roland ; Hoenig, Manfred ; Schuster, Friedhelm R ; Boyle, Janel O ; Cicek, Alphan ; Ghosh, Sujal

Abstract:

Reticular dysgenesis (RD) is a rare inborn error of immune cell formation defined by severe combined immunodeficiency, agranulocytosis and sensorineural deafness. We report a case of successful haploidentical maternal hematopoietic stem cell transplantation (HSCT) in a boy with RD detected by TREC newborn screening. The patient was admitted to our hospital at 2 weeks of age and was kept in laminar-air flow / hepa-filtered isolation until HSCT was performed at 8 weeks of age with a busulfan, fludarabine conditioning regime. Except few episodes of acute skin graft-versus-host disease (aGVHD) the peritransplant period was uneventful. The patient was discharged 7 weeks post-HSCT. At 18 months of age cochlear implants were placed. The patient was thriving well, showed full donor chimerism and a T cell count > 1000 TCRab + CD3 + cells/µl after one year. Our case highlights that severely immune-compromised patients with RD benefit from early diagnosis by newborn screening, immediate isolation to prevent infections, and early haploidentical HSCT to overcome neonatal neutropenia and establish protective immunity.

355

News (Medical) associated with Fludarabine Phosphate

21 hours ago

·www.globenewswire.com

Artiva Biotherapeutics Reports First Quarter 2025 Financial Results, Recent Business Highlights

IND clearance and initiation of global basket trial exploring AlloNK® + rituximab in refractory rheumatoid arthritis, Sjögren’s disease, idiopathic inflammatory myopathies and systemic sclerosis First trial to explore an allogeneic cell therapy in refractory rheumatoid arthritis and Sjögren’s disease in the U.S. Initial safety, translational data, and lead indication selection to be presented by year-end 2025; initial clinical response data in the lead indication to be presented in 1H2026 Longer-term clinical data from Phase 1/2 trial exploring AlloNK + rituximab in B-NHL in heavily pretreated CAR-T naïve patients to be presented at ASGCT, demonstrating complete response rates and median duration of responses in line with approved auto-CAR-T therapies, support deep B-cell depletion Cash runway extension into Q2 2027, with cash, cash equivalents and investments of $166.0 million as of March 31, 2025 SAN DIEGO, May 08, 2025 (GLOBE NEWSWIRE) -- Artiva Biotherapeutics, Inc. (Nasdaq: ARTV) (Artiva), a clinical-stage biotechnology company whose mission is to develop effective, safe, and accessible cell therapies for patients with devastating autoimmune diseases and cancers, today announced financial results for the first quarter ended March 31, 2025, and highlighted recent progress. “We are encouraged by our early experience with AlloNK® in autoimmune disease. We have initiated regulatory submissions globally for our company-sponsored basket trial in refractory rheumatoid arthritis, Sjögren’s disease, myositis and scleroderma. Notably, we are first to announce a company-sponsored allogeneic cell therapy trial in rheumatoid arthritis and Sjögren’s disease in the U.S.; two sizeable indications that could benefit from AlloNK’s potential ease of use and well-tolerated safety profile in the community setting,” said Fred Aslan, M.D., Chief Executive Officer of Artiva. “Since the IPO last July, we have transformed our development team and now have a deep bench of autoimmune expertise to expand and refine the clinical trial infrastructure and development strategy for AlloNK in autoimmune disease. By the end of 2025, we plan on sharing initial safety and translational data supporting AlloNK’s mechanism of action and tolerability profile across multiple autoimmune diseases, as well as announcing our lead indication. In the first half of 2026, we expect to share clinical response data in our lead indication with longer follow-up to substantiate AlloNK’s emerging target product profile, and to have initial insights on registrational approaches.” Dr. Aslan continued, “Our longer-term aggressive B-NHL data is maturing into a leading data set, in line with approved auto-CAR-T therapies, providing proof of concept of AlloNK’s ability to deplete B-cells deeply and durably. We remain well capitalized to generate meaningful data sets and to differentiate ourselves over time from other therapeutic approaches in areas of significant unmet need in the community setting.” Recent Business Highlights AlloNK® (also known as AB-101) Updates Autoimmune clinical program updates: Company-sponsored clinical trials: Phase 2a company-sponsored basket clinical trial: Following Investigational New Drug application clearance by the U.S. Food and Drug Administration, initiating global Phase 2a company-sponsored basket clinical trial for AlloNK + rituximab for refractory rheumatoid arthritis (RA), Sjögren’s disease, idiopathic inflammatory myopathies (myositis, or IIM) and systemic sclerosis (scleroderma, or SSc), with site initiation underwayFirst company-sponsored trial exploring an allogeneic cell therapy in refractory RA and Sjögren’s disease in the U.S.Protocol allows for continuous enrollment with no stagger within dose levels and no hospitalization/inpatient requirement for patients dosed with AlloNKInitiation of regulatory submissions across multiple geographic regions Phase 1/1b company-sponsored trial in systemic lupus erythematosus (SLE) with or without lupus nephritis (LN): Treatment of patients in combination cohort with AlloNK + monoclonal antibodies (mAb) initiated in late 2024Prioritization of patient enrollment in AlloNK + obinutuzumab cohort Currently exploring 1 billion cells per AlloNK dose x 3 weekly doses in company-sponsored trials with plan to escalate to 4 billion cells per AlloNK dose x 3 weekly doses across both trials Investigator-initiated trial (IIT): Ongoing IIT Basket Trial: IIT assessing the safety, tolerability, and clinical activity of AlloNK + rituximab in patients with RA, pemphigus vulgaris, granulomatosis with polyangiitis/microscopic polyangiitis, and SLE. The trial is being conducted by Integral Rheumatology & Immunology Specialists (IRIS), in an outpatient setting at a community rheumatology clinic. Initial tolerability supports prioritization of community sites for clinical development of AlloNK AlloNK + rituximab in B-cell non-Hodgkin lymphoma (B-NHL) updates: Longer-term clinical data from Phase 1/2 trial exploring AlloNK + rituximab in patients with relapsed/refractory B-NHL to be presented at The American Society of Gene & Cell Therapy (ASGCT) 28th annual meeting on May 13, 2025. Abstracts are currently available on the ASGCT websiteUpdated clinical data in heavily pre-treated, CAR-T naïve patients with B-NHL shows prolonged duration of response, with complete response rates and median duration of response in line with approved auto-CAR-T therapies in aggressive B-NHL patients with multiple prior lines of treatment Other program updates: Preclinical data on the ablation of SLE donor B-cells with AlloNK in combination with either an anti-CD19 or anti-CD20 monoclonal antibody presented at the 2025 Pan-American League of Rheumatology Congress (PANLAR) and to be presented at the 16th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), being held May 21-24, 2025, in Toronto, CanadaPoster presentation featuring the scalability and consistency of the manufacturing process for AlloNK to be presented at the ASGCT 28th annual meeting on May 15, 2025 Data highlights consistent and high expression of CD16, uniform expansion across greater than 40 manufacturing lots, and high viability and cytotoxicity after 4 years of shelf life for AlloNK Corporate Updates Appointment of Subhashis Banerjee, M.D., as Chief Medical Officer announced on April 8, 2025, culminating Artiva’s efforts to build a seasoned development team with strong expertise in autoimmune disease and cell therapy Upcoming Milestones By Year-End 2025: Initial safety and translational data for AlloNK + mAb across multiple autoimmune indications from ongoing clinical trials and disclosure of lead autoimmune indication for further development Mechanistic and translational data for AlloNK in autoimmune indicationsInsights into tolerability of AlloNK + mAb, and the patient journey in community rheumatology sites, including the potential ease of use of conditioning regimens with cyclophosphamide and fludarabineDisclosure of lead indication for AlloNK development in autoimmune diseases 1H 2026: Initial clinical response data in the lead autoimmune indication from ongoing clinical trials with longer follow-up to inform registrational strategy First Quarter 2025 Financial Results Cash, Cash Equivalents and Investments. As of March 31, 2025, Artiva had cash, cash equivalents, and investments of $166.0 million which is expected to fund operations into Q2 2027.Research and Development Expenses. Research and development expenses were $17.1 million for the three months ended March 31, 2025, compared to $11.2 million for the three months ended March 31, 2024.General and Administrative Expenses. General and administrative expenses were $5.1 million for the three months ended March 31, 2025, compared to $3.6 million for the three months ended March 31, 2024.Other Income, net. Other income, net, was $1.9 million for the three months ended March 31, 2025, compared to other income, net, of $0.5 million for the three months ended March 31, 2024.Net Loss. Net loss totaled $20.3 million for the three months ending March 31, 2025, as compared to net loss of $14.0 million for the three months ending March 31, 2024, with non-cash stock-based compensation expense of $2.1 million and $1.4 million for the three months ended March 31, 2025 and 2024, respectively. About Artiva Biotherapeutics Artiva is a clinical-stage biotechnology company whose mission is to develop effective, safe and accessible cell therapies for patients with devastating autoimmune diseases and cancers. Artiva’s lead program, AlloNK® (also known as AB-101), is an allogeneic, off-the-shelf, non-genetically modified, cryopreserved NK cell therapy candidate designed to enhance the antibody-dependent cellular cytotoxicity effect of monoclonal antibodies to drive B-cell depletion. AlloNK is currently being evaluated in three ongoing clinical trials for the treatment of B-cell driven autoimmune diseases. This includes two company-sponsored trials, one in systemic lupus erythematosus for patients with or without lupus nephritis, and a basket trial across autoimmune diseases including rheumatoid arthritis and Sjögren’s disease, as well as an investigator-initiated basket trial in B-cell driven autoimmune diseases. Artiva’s pipeline also includes CAR-NK candidates targeting both solid and hematologic cancers. Artiva was founded in 2019 as a spin out of GC Cell, formerly GC Lab Cell Corporation, a leading healthcare company in the Republic of Korea, pursuant to a strategic partnership granting Artiva exclusive worldwide rights (excluding Asia, Australia and New Zealand) to GC Cell’s NK cell manufacturing technology and programs. Artiva is headquartered in San Diego, California. For more information, please visit www.artivabio.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Statements in this press release that are not statements of historical fact are forward-looking statements. Such forward-looking statements include, without limitation, statements regarding: expectations of Artiva Biotherapeutics, Inc. (the “Company”) regarding the potential benefits, accessibility, ease of use, effectiveness and safety of AlloNK; the Company’s ability to advance AlloNK in autoimmune disease; the Company’s ability to demonstrate progress and clinical validation of its approach; the belief that of Phase 1/2 trial data in B-NHL provides support for B-cell depletion as a MoA and its comparison to approved auto-CAR-T therapies; the Company’s expectations regarding design, timing and availability of data from the Company’s clinical trials or the basket IIT; the timing related to the selection of a lead autoimmune indication; the timing, likelihood or success of the Company's business strategy, as well as plans and objectives of management for future operations; and the Company’s future results of operations and financial position, including cash runway. These forward-looking statements are based on the beliefs of the management of the Company as well as assumptions made by and information currently available to the Company. Such statements reflect the current views of the Company with respect to future events and are subject to known and unknown risks and uncertainties. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. These and other factors that may cause the Company’s actual results to differ from current expectations are discussed in the Company’s filings with the Securities and Exchange Commission (the “SEC”), including the section titled “Risk Factors” in the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this press release is given. Except as required by law, the Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise. Artiva Biotherapeutics, Inc.Condensed Balance Sheets(Unaudited)(in thousands) March 31, 2025 December 31, 2024Assets Cash, cash equivalents and investments$165,963 $185,428 Property and equipment, net 7,057 6,370 Operating and financing lease right-of-use assets 13,728 14,055 Other assets 4,515 3,728 Total assets$191,263 $209,581 Liabilities and stockholders' equity Accounts payable and accrued expenses$8,411 $8,513 Operating and financing lease liabilities 13,996 14,354 Other liabilities 73 73 Total liabilities 22,480 22,940 Stockholders' equity 168,783 186,641 Total liabilities and stockholders' equity$191,263 $209,581 Artiva Biotherapeutics, Inc.Condensed Statements of Operation and Comprehensive Loss(Unaudited)(in thousands, except share and per share data) Three Months Ended March 31, 2025 2024License and development support revenue$- $251 Operating expenses: Research and development 17,052 11,156 General and administrative 5,119 3,587 Total operating expenses 22,171 14,743 Loss from operations (22,171) (14,492)Other income, net Interest income 1,864 650 Change in fair value of SAFEs — (268)Other income (expense) (4) 147 Total other income, net 1,860 529 Net loss$(20,311) $(13,963)Net loss per share, basic and diluted$(0.83) $(17.24)Weighted-average common shares outstanding, basic and diluted 24,341,978 809,758 Comprehensive loss: Net loss$(20,311) $(13,963)Other comprehensive income (loss) 129 (101)Comprehensive loss$(20,182) $(14,064) ContactsInvestors: Neha Krishnamohan, Artiva Biotherapeutics, ir@artivabio.comMedia: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com, +1.858.344.8091 Source: Artiva Biotherapeutics, Inc.

Cell TherapyFinancial StatementLicense out/inPhase 2ASH

06 May 2025

·www.prnewswire.com

Actinium Announces Enrollment of First Patient in the Iomab-ACT Commercial CAR-T Trial at the University of Texas Southwestern Medical Center

- Initial clinical data expected in the second half of 2025 from this first-of-its-kind trial to administer a targeted radiotherapy conditioning agent with a commercial CAR-T therapy - Iomab-ACT supported by results of NIH funded trial with MSK showing effective lymphodepletion of targeted immune cells resulting in negligible rates of CAR-T toxicities ICANS and CRS and CAR T-cell persistence with a novel CD19 CAR-T therapy - Iomab-ACT has the potential to increase the addressable market for CAR-T therapies, which generated $4 billion in sales in 2024, by enabling improved access and better patient outcomes compared to current chemotherapy conditioning agents NEW YORK, May 6, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced that the first patient was enrolled on the trial studying Iomab-ACT targeted conditioning with a commercial CAR-T therapy at the University of Texas Southwestern Medical Center (UTSW) (NCT06768905). Initial clinical data from this trial is expected in the second half of 2025. Actinium is developing Iomab-ACT as a targeted radiotherapy conditioning agent intended to replace non-targeted chemotherapeutic conditioning agents such as Fludarabine and Cyclophosphamide (Flu/Cy) to address serious CAR-T related toxicities including immune effector cell-associated neurotoxicity (ICANS) and cytokine release syndrome (CRS), to potentially improve patient access and outcomes. Currently, there are seven CAR-T therapies approved for certain leukemias and lymphomas and multiple myeloma, that over 150,000 patients are diagnosed with annually. In 2024, the seven approved CAR-T therapies generated over $4 billion in sales and CAR-T therapies are forecasted to reach $12 billion in annual sales in 2030. Dr. Farrukh Awan, Professor of Medicine, Division of Hematology Oncology at UTSW said, "We are thrilled to initiate patient enrollment to study Iomab-ACT targeted radiotherapy conditioning with a commercial CAR-T therapy. Iomab-ACT is supported by compelling preclinical and clinical data, and we believe it has immense potential to eliminate the need for chemotherapy-based conditioning, which is a major barrier for many patients seeking CAR-T treatment. Despite the positive impact CAR-T therapy has had on patient outcomes, there is still significant room for improvement. We are optimistic that Iomab-ACT can transform CAR-T therapy conditioning if this trial demonstrates it has the ability to increase patients access and reduce the rates and severity of ICANS and CRS and also potentially improve patient outcomes. We are excited to begin treating patients with Iomab-ACT and eager to present our preliminary findings later this year." Iomab-ACT targets CD45, a cell surface marker expressed on immune cells relevant to CAR-T therapy including lymphocytes and is the only clinical stage conditioning agent targeting CD45. Preclinical data demonstrated that Iomab-ACT can selectively target immune cells implicated in CAR-T toxicities, while sparing bone marrow stem cells, red blood cells and platelets. Preclinical and clinical data also showed that Iomab-ACT produces transient lymphodepletion that aligns with the CAR-T treatment process. This data supported the first clinical trial of Iomab-ACT with a novel CD19 CAR-T therapy in collaboration with Memorial Sloan Kettering Cancer Center (MSK) in patients heavily pretreated with relapsed and refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) or Diffuse Large B-cell Lymphoma (DLBCL). In this study, no patients (0/4) developed ICANS of any grade, and minimal CRS. Iomab-ACT also demonstrated transient depletion of peripheral blood lymphocytes and monocytes, persistence of CAR T-cells up to 8 weeks and minimal non-hematologic toxicities. These positive findings supported the continued advancement of Iomab-ACT and the initiation of the commercial CAR-T trial at UTSW. Sandesh Seth, Actinium's Chairman and CEO, stated, "This is a pivotal moment for our Iomab-ACT CD45 targeted radiotherapy conditioning program. Iomab-ACT is a highly differentiated conditioning agent that has produced promising initial clinical results where multiple targeted conditioning approaches including monoclonal antibodies and antibody drug conjugates directed against a variety of targets have not achieved clinical success to date. Based on the promising initial outcomes from the pilot study of Iomab-ACT with a novel CD19 CAR-T, we are incredibly excited by the potential of this commercial CAR-T trial and future development path. With initial clinical data expected beginning in the second half of this year, we are making strong progress to achieving our goal of establishing Iomab-ACT as a universal targeted conditioning regimen for CAR-T and other cellular therapies." Targeted Radiotherapy CAR-T Conditioning Opportunity A multi-billion-dollar market opportunity exists for better conditioning in other areas of cellular therapy, such as CAR-T. Currently, there are seven CAR T-cell therapies targeting CD19 for lymphoma and leukemia and BCMA for multiple myeloma that are approved by the FDA with total sales of over $4.0 billion in 2024. The pipeline of CAR-T therapies in development has rapidly expanded, with the addressable patient population expected to nearly double and reach approximately 93,000 patients in the U.S. by 2030 based on the current pipeline of cellular therapies. The addressable market for Iomab-ACT is in line with the patient population for cellular therapies that is approximately150,000 patients annually across the indications in which CAR-T therapies are approved, as all patients receive conditioning of some type. We believe a potential blockbuster revenue opportunity exists for Iomab-ACT assuming it can provide clinical benefits related to adverse events related to CAR-T, longer duration of response or improved survival outcomes. About Actinium Pharmaceuticals, Inc. Actinium is a pioneer in the development of targeted radiotherapies intended to meaningfully improve patient outcomes. Actinium is advancing its lead product candidate Actimab-A, a CD33 targeting therapeutic, as potential backbone therapy in acute myeloid leukemia (AML) and other myeloid malignancies leveraging the mutation agnostic alpha-emitter radioisotope payload Actinium-225 (Ac-225). Actimab-A has demonstrated potential activity in relapsed and refractory acute myeloid leukemia (r/r AML) patients in combination with the chemotherapy CLAG-M including high rates of Complete Remissions (CR) and measurable residual disease (MRD) negativity leading to improved survival outcomes and is being advanced to a pivotal Phase 2/3 trial. In addition, Actinium is engaged with the National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) for development of Actimab-A in AML and other myeloid malignancies. The first clinical trial under the CRADA will evaluate the triplet combination comprised of Actimab-A, Venetoclax (Abbvie/Roche) an oral Bcl-2 inhibitor and ASTX-727 (Taiho Oncology, an Otsuka holdings company) a novel oral hypomethylating agent (HMA) in frontline acute myeloid leukemia (AML) patients. Additionally, Actinium is developing Actimab-A as a potential pan tumor therapy in combination with PD-1 checkpoint inhibitors including KEYTRUDA® and OPDIVO® by depleting myeloid derived suppressor cells (MDSCs), which represents a potential multi-billion-dollar addressable market. ATNM-400 is Actinium's novel non-PSMA targeting Ac-225 radiotherapy for prostate cancer, which is supported by preclinical data demonstrating higher efficacy than Pluvicto (PSMA-617-Lutetium-177) and potent efficacy in Pluvicto resistant prostate cancer models. Iomab-ACT, Actinium's next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with r/r AML, which Actinium is seeking a potential strategic partner for the U.S. In addition, the company's R&D efforts are primarily focused on advancing several preclinical programs for solid tumor indications. Actinium holds 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron. For more information, please visit: Forward-Looking Statements This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time. Investors: [email protected] SOURCE Actinium Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyCell TherapyClinical Study

29 Apr 2025

·www.astrazeneca.com

Fixed-duration Calquence-based regimens recommended for approval in the EU by CHMP for 1st-line chronic lymphocytic leukaemia

A fixed-duration regimen of AstraZeneca’s Calquence (acalabrutinib) in combination with venetoclax, with or without obinutuzumab, has been recommended for approval in the European Union (EU) for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) based its positive opinion on results from the AMPLIFY Phase III trial, which were presented at the American Society of Haematology (ASH) 2024 Annual Meeting and published in The New England Journal of Medicine.1 Results showed Calquence plus venetoclax reduced the risk of disease progression or death by 35% compared to standard-of-care chemoimmunotherapy (investigator’s choice of fludarabine-cyclophosphamide-rituximab or bendamustine-rituximab; hazard ratio [HR] 0.65; 95% confidence interval [CI] 0.49-0.87; p=0.0038). Calquence plus venetoclax with obinutuzumab demonstrated a 58% reduction in the risk of disease progression or death compared to standard-of-care chemoimmunotherapy (HR 0.42; 95% CI 0.30-0.59; p<0.0001).2 At three years, 77% of patients treated with Calquence plus venetoclax and 83% of patients treated with Calquence plus venetoclax and obinutuzumab were progression free, versus 67% of patients treated with chemoimmunotherapy.1 Median progression-free survival (PFS) was not reached for either experimental arm versus 47.6 months for chemoimmunotherapy.1 Wojciech Jurczak, MD, Maria Sklodowska-Curie National Institute of Oncology, Kraków, Poland and investigator for the trial, said: “Chronic lymphocytic leukaemia is an incurable cancer which means patients live with the disease and stay on treatment for many years, which can have long-term effects. The fixed-duration Calquence regimens will allow patients to take breaks from their treatment, reducing the risk of long-term adverse events and drug resistance.” Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: “With this recommendation, Calquence plus venetoclax can potentially be the only all-oral second-generation BTK inhibitor option approved in Europe for patients with previously untreated chronic lymphocytic leukaemia. Calquence has demonstrated efficacy and safety in fixed-duration and treat-to-progression regimens providing patients and their doctors more treatment flexibility.” CLL is the most common type of leukaemia in adults, with an estimated 27,000 patients diagnosed in the UK, France, Germany, Spain and Italy in 2024.3 The safety and tolerability of Calquence was consistent with its known safety profile, and no new safety signals were identified. Regulatory applications for Calquence plus venetoclax, with or without obinutuzumab, in this setting are currently under review in several countries based on the AMPLIFY results. Notes Chronic lymphocytic leukaemia (CLL) CLL is the most prevalent type of leukaemia in adults, with over 117,000 new cases globally in 2021.4 Although some people with CLL may not experience any symptoms at diagnosis, others may experience symptoms, such as weakness, fatigue, weight loss, chills, fever, night sweats, swollen lymph nodes and abdominal pain.5 In CLL, there is an accumulation of abnormal lymphocytes within the blood, bone marrow and lymph nodes. As the number of abnormal cells increases, there is less room within the marrow for the production of normal white blood cells, red blood cells and platelets.6 This could result in infection, anaemia and bleeding. B-cell receptor signalling through BTK is one of the essential growth pathways for CLL. AMPLIFY AMPLIFY is a randomised, global, multi-centre, open-label Phase III trial evaluating the efficacy and safety of Calquence in combination with venetoclax, with or without obinutuzumab, compared to investigator's choice of chemoimmunotherapy (fludarabine-cyclophosphamide-rituximab or bendamustine-rituximab) in adult patients with previously untreated CLL without del(17p) or TP53 mutation.7 Patients were randomised 1:1:1 to receive either Calquence plus venetoclax, Calquence plus venetoclax with obinutuzumab for a fixed duration or standard-of-care chemoimmunotherapy.7 Both the Calquence containing arms were administered for a fixed duration of 14 cycles (each 28 days), and the standard-of-care chemoimmunotherapy was for 6 cycles.7 The primary endpoint is PFS in the Calquence and venetoclax arm as assessed by an Independent Review Committee and PFS in the Calquence plus venetoclax with obinutuzumab is a key secondary endpoint.7 Other key secondary endpoints include OS and undetectable measurable residual disease.7 The trial includes 27 countries across North and South America, Europe, Asia and Oceania.7 The AMPLIFY trial enrolled patients from 2019 to 2021, continuing through the COVID-19 pandemic.7 Patients with blood cancer remain at a disproportionately high risk of severe outcomes from COVID-19, including hospitalisation and death compared to the general population.8 Calquence Calquence (acalabrutinib) is a second-generation, selective inhibitor of Bruton’s tyrosine kinase (BTK). Calquence binds covalently to BTK, thereby inhibiting its activity.9 In B-cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion. Calquence is approved for the treatment of chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) in the US, Japan and China, approved for CLL in the EU and many other countries worldwide. Calquence is also approved for the treatment of adult patients with previously untreated MCL in the US and other countries. It is also approved for the treatment of adult patients with MCL who have received at least one prior therapy in China and several other countries. Calquence is not currently approved for the treatment of MCL in Japan. As part of an extensive clinical development programme, Calquence is currently being evaluated as a single treatment and in combination with standard-of-care chemoimmunotherapy for patients with multiple B-cell blood cancers, including CLL, MCL and diffuse large B-cell lymphoma. AstraZeneca in haematology AstraZeneca is pushing the boundaries of science to redefine care in haematology. Our goal is to help transform the lives of patients living with malignant, rare and other related haematologic diseases through innovative medicines and approaches that are shaped by insights from patients, caregivers and physicians. In addition to our marketed products, we are spearheading the development of novel therapies designed to target underlying drivers of disease across multiple scientific platforms. Our acquisitions of Alexion, with expertise in rare, non-malignant blood disorders, and Gracell Biotechnologies Inc., pioneers of autologous cell therapies, expand our haematology pipeline and enable us to reach more patients with high unmet needs through the end-to-end discovery, development and delivery of novel therapies. AstraZeneca in oncology AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients. The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience. AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death. AstraZeneca AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Social Media @AstraZeneca. Contacts For details on how to contact the Investor Relations Team, please click here. For Media contacts, click here. References 1. Brown J, et al. Fixed-Duration Acalabrutinib Combinations in Untreated Chronic Lymphocytic Leukemia. NEJM. 2025;392:748-762. 2. Brown, J et al. Fixed-Duration Acalabrutinib plus Venetoclax with or without Obinutuzumab versus Chemoimmunotherapy for First-Line Treatment of Chronic Lymphocytic Leukemia: Interim Analysis of the Multicenter, Open-label, Randomized, Phase 3 AMPLIFY Trial. Presented at ASH 2024. Abstract 1009. 2024. 3. AstraZeneca 2024. Full Year and Q4 2024 Financial Results Epidemiology Spreadsheet. Available at: https://www.astrazeneca.com/investor-relations.html. Accessed April 2025. 4. Global Burden of Disease Collaborative Network's "Global Burden of Disease Study 2021 (GBD 2021)" published by the Institute for Health Metrics and Evaluation (IHME) in Seattle, United States, in 2024. Accessed April 2025. 5. American Cancer Society. Signs and Symptoms of Chronic Lymphocytic Leukemia. Available at: https://www.cancer.org/cancer/types/chronic-lymphocytic-leukemia/detection-diagnosis-staging/signs-symptoms.html. Accessed April 2025. 6. National Cancer Institute. Chronic lymphocytic leukemia treatment (PDQ®)–Patient version. Available at: https://www.cancer.gov/types/leukemia/patient/cll-treatment-pdq. Accessed April 2025. 7. ClinicalTrials.gov. Study of Acalabrutinib (ACP-196) in Combination With Venetoclax (ABT-199), With and Without Obinutuzumab (GA101) Versus Chemoimmunotherapy for Previously Untreated CLL (AMPLIFY). Available at: https://clinicaltrials.gov/study/NCT03836261. Accessed April 2025. 8. Dube S, et al. Continued Increased Risk of COVID-19 Hospitalisation and Death in Immunocompromised Individuals Despite Receipt of ≥4 Vaccine Doses: Updated 2023 Results from INFORM, a Retrospective Health Database Study in England. Poster P0409 at ECCMID 2024. 9. Wu J, et al. Acalabrutinib (ACP-196): a selective second-generation BTK inhibitor. J Hematol Oncol. 2016;9(21). Adrian Kemp Company Secretary AstraZeneca PLC Oncology Corporate and financial

Clinical ResultDrug ApprovalImmunotherapyPhase 3Acquisition

100 Deals associated with Fludarabine Phosphate

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KEGG	Wiki	ATC	Drug Bank
D01907	Fludarabine Phosphate	L01BB05	DB01073

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Lymphoma	Japan	Sanofi KK	26 Mar 2019
Multiple Myeloma	Japan	Sanofi KK	30 Jun 2015
Myelodysplastic Syndromes	Japan	Sanofi KK	30 Jun 2015
Philadelphia chromosome positive chronic myelogenous leukemia	Japan	Sanofi KK	30 Jun 2015
Mantle-Cell Lymphoma	Japan	Sanofi KK	06 Nov 2009
Non-Hodgkin Lymphoma	Japan	Sanofi KK	06 Nov 2009
Hematopoietic stem cell transplantation	Japan	Sanofi KK	20 May 2008
B-Cell Lymphoma	Japan	Sanofi KK	26 Jan 2007
Thrombocytopenia	Japan	Sanofi KK	29 Sep 1999
Chronic Lymphocytic Leukemia	United States	Genzyme Corp.	18 Apr 1991

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Pulmonary Disease, Chronic Obstructive	Phase 3	Australia	Novartis Pharmaceuticals Corp.	01 Apr 2012
Pulmonary Disease, Chronic Obstructive	Phase 3	New Zealand	Novartis Pharmaceuticals Corp.	01 Apr 2012
Disorder related to transplantation	Phase 3	United States	Sanofi	02 Nov 2011
Infectious Diseases	Phase 3	United States	Sanofi	02 Nov 2011
Acute Graft Versus Host Disease	Phase 3	United States	National Cancer Institute	01 Nov 2010
Acute Graft Versus Host Disease	Phase 3	Denmark	National Cancer Institute	01 Nov 2010
Acute Graft Versus Host Disease	Phase 3	Germany	National Cancer Institute	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	United States	National Cancer Institute	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	Denmark	National Cancer Institute	01 Nov 2010
Recurrent Multiple Myeloma	Phase 3	Germany	National Cancer Institute	01 Nov 2010

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03615105 (CTgov)	Phase 2	Hodgkin's Lymphoma \| Ph-Like Acute Lymphoblastic Leukemia \| Acute Myeloid Leukemia ... View more	9	Hyperfractionated total body irradiation+Cyclophosphamide+Rituximab+Thiotepa (Radiation, Thiotepa & Cyclophosphamide)	aswgcrnzph = bxkrdimwzn wcwmygkjso (turpdpemev, jehavepoeu - stwwslswqf) View more	-	06 Apr 2025
				HPC(A) stem cell allograft+Fludarabine+Busulfan+Melphalan+Rituximab (Busulfan, Fludarabine & Melphalan)	aswgcrnzph = tdcldkgdqj wcwmygkjso (turpdpemev, tflbwvywfm - aaikwjbbps) View more
NCT05108805 (Prospective Clinical Trial of Outpatient Administration of Axicabtagene Ciloleucel, CTgov)	Phase 4	Diffuse Large B-Cell Lymphoma	25	Telemedicine Visit+Axicabtagene Ciloleucel+Fludarabine+Cyclophosphamide	nrxacrsmur = bkgmupfrti ekaaecbcgi (uthkzvpwzv, zanapkpdef - afnscqxdba) View more	-	27 Mar 2025
NCT02566304 (CTgov)	Phase 2	Anemia, Refractory, With Excess of Blasts \| Refractory cytopenia with multilineage dysplasia and ringed sideroblasts \| Relapsing acute myeloid leukemia ... View more	35	Peripheral Blood Stem Cell Transplantation+Fludarabine+Cyclophosphamide+Tacrolimus+Mycophenolate mofetil	ezmrepryrj = rmeanfmyuy gpjbdbumxq (oedrtppwzq, rnrpkizgak - rjjgiwysym) View more	-	20 Mar 2025
NCT04191187 (Phase II Trial of Reduced-Intensity Fludarabine, Melphalan 70 Mg/m 2 , and Total Body Irradiation Conditioning \| 4900, CTgov)	Phase 2	Marginal zone lymphoma recurrent \| Acute Myeloid Leukemia \| Recurrent Follicular Lymphoma ... View more	34	Total Body Irradiation+Fludarabine+Melphalan	txbivcbugx = kzfxzegdmn hucdyoywvd (vuerpjjwbk, oioqvscrtl - ysfvxqowbz) View more	-	07 Mar 2025
NCT04432506 (CTgov)	Phase 2	Primary mediastinal large B-cell lymphoma refractory \| Refractory High-Grade B-Cell Lymphoma \| Primary mediastinal large B-cell lymphoma recurrent ... View more	22	Anakinra (Cohort 1)	fyqgxoivdh = hlevdthmbg vnlippwuml (ghcbxdczcx, owmzrrwilc - ehexndekar) View more	-	28 Jan 2025
				Anakinra (Cohort 2)	fyqgxoivdh = gsykmlhadx vnlippwuml (ghcbxdczcx, nqpkptqsqd - xlmalvqhxe) View more
ASH2024	Not Applicable	Hematopoietic stem cell transplantation	-	Fludarabine 30mg/m2 + Thiotepa 5mg/kg	jnpuvjafbw(eqmggbmgaa) = pionkrtptv otmnoriefr (bxfsbdnjol ) View more	-	09 Dec 2024
				Fludarabine 30mg/m2 + Busulfan 3.2mg/kg	yoonouqfoj(xelsparrjo) = vyxhyuhctc haqkmkvxwn (wbqypepjok ) View more
NCT04339101 (1043, ASH2024)	Phase 2	Myelofibrosis \| acute leukemia \| Myelodysplastic Syndromes HGF \| IL17 \| TNFaR ... View more	59	Itacitinib + Tacrolimus/Sirolimus	fqgwsxlxbs(glgvihtuox) = kstfvrtmbw iyuwuzjwma (ammgzjsllq, 41 - 66) View more	Positive	09 Dec 2024
				Tacrolimus/Sirolimus	fqgwsxlxbs(glgvihtuox) = oxvohfcqct iyuwuzjwma (ammgzjsllq )
NCT05322733 (cwCLL-001, ASH2024) Manual	Phase 2	Chronic Lymphocytic Leukemia First line	25	Orelabrutinib+Fludarabine+Cyclophosphamide+Obinutuzumab	avtjytffof(pxxxttxhzc) = tznxwgqlxy rirknsnsiy (keetgnyrmo ) View more	Positive	08 Dec 2024
ASH2024	Not Applicable	Peripheral Stem Cell Transplantation	-	3-day fludarabine	dnykqvduwu(nrvlncseua) = gwnnlxhfyf mkeqwwaxpk (mmslzxdczk ) View more	-	08 Dec 2024
				5-day fludarabine	dnykqvduwu(nrvlncseua) = ecqexqinsu mkeqwwaxpk (mmslzxdczk ) View more
ASH2024	Not Applicable	Myelofibrosis	-	Bu 80 mg/m2	hjnmjofvde(inxknzoded) = dizsqtroru jgdqynuwxz (gzndvlqcbu ) View more	-	08 Dec 2024
				Thiotepa 5 mg/kg	hjnmjofvde(inxknzoded) = kzfrbujqao jgdqynuwxz (gzndvlqcbu ) View more

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