A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727, NSC# 820631) and Filgrastim as Maintenance Therapy Post-Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Filgrastim stimulates the production of neutrophils (a type of white blood cell) which can help to prevent infection. Giving ATSX727 and filgrastim may be safe and tolerable in treating children with high risk, recurrent or refractory acute myeloid leukemia who have undergone allogenic hematopoietic stem cell transplantation.

Start Date30 Oct 2026

Sponsor / Collaborator

National Cancer Institute

NCT07257419

/ Not yet recruitingPhase 1IIT

CD45RA-depleted CD19-CAR T Cell Consolidation After TCR??+ T Cell-depleted Haploidentical Hematopoietic Cell Transplantation for Relapsed/Refractory CD19+ ALL and Lymphoma

The purpose of this study is to learn more about newer methods of transplanting blood cells donated by a partially matched family member to children with high-risk CD19 positive leukemia ALL.
Primary Objective:
- To assess the safety and feasibility of combining CD19-CAR(Mem) T cells after TCRαβ+ depleted haploidentical donor transplantation for pediatric patients with relapsed/refractory CD19+ B-cell malignancies.
Secondary Objectives:
* To estimate 1-year post-transplant overall survival, relapse free survival, and GVHD-free relapse-free survival (GRFS).
* To estimate cumulative incidence of engraftment, acute and chronic GVHD, and immune-related adverse events, including CRS and ICANS.

Start Date01 May 2026

Sponsor / Collaborator

St. Jude Children's Research Hospital, Inc.

NCT07278856

/ Not yet recruitingPhase 1IIT

Ruxolitinib in Combination With Chemotherapy for Untreated Nodal T-Follicular Helper (TFH) Cell Lymphomas

This phase I trial tests the safety, side effects and best dose of ruxolitinib in combination with cyclophosphamide, doxorubicin, vincristine, prednisone (CHOP) chemotherapy and how well the combination works in treating patients with untreated nodal T-follicular helper (TFH) cell lymphoma. Ruxolitinib phosphate blocks a protein called janus kinase, which may help keep abnormal blood cells or cancer cells from growing. It may also lower the body's immune response. Ruxolitinib phosphate is a type of tyrosine kinase inhibitor. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill cancer cells. It may also lower the body's immune response. Doxorubicin damages the cell's DNA and may kill cancer cells. It also blocks a certain enzyme needed for cell division and DNA repair. Doxorubicin is a type of anthracycline antitumor antibiotic. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Prednisone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Giving ruxolitinib in combination with CHOP chemotherapy may be safe, tolerable, and/or effective in treating patients with untreated nodal TFH cell lymphoma.

Start Date27 Apr 2026

Sponsor / Collaborator

City of Hope National Medical Center

[+1]

100 Clinical Results associated with Filgrastim (Amgen)

100 Translational Medicine associated with Filgrastim (Amgen)

100 Patents (Medical) associated with Filgrastim (Amgen)

2,534

Literatures (Medical) associated with Filgrastim (Amgen)

01 Mar 2026·Journal of Clinical and Experimental Hepatology

Granulocyte Colony Stimulating Factor and Its Combinations With Anti-inflammatory Therapies in Severe Alcoholic Hepatitis: A Systematic Review and Meta-analysis

Review

Author: Rungta, Sumit ; Singh, Surender ; Mishra, Ajay K ; Goel, Amit ; Sinha, Neeraj ; Sharma, Shaiphali ; Pandey, Shreya ; Kulkarni, Anand V

Background/Aims:

Granulocyte colony-stimulating factor (GCSF) has been used to treat severe alcohol-associated hepatitis (SAH) in different combinations and dosages with variable outcomes. We aimed to evaluate the efficacy of GCSF for the treatment of severe alcoholic hepatitis (AH).

Methods:

We conducted a systematic review and meta-analysis of studies comparing GCSF versus standard medical therapy (SMT) to treat patients with severe AH.

Results:

Eight studies were selected after screening 875 studies with a total of 1241 participants enrolled including 376 female participants. Overall 90-day survival rates were 85.35% and 61.8% in GCSF and SMT groups, respectively. GCSF therapy was associated with a significant increase in 90-day survival, with an odds ratio (OR) of 3.61 (95% confidence interval [CI]: 2.6-5.00) and I² = 7.2%. The 28-day survival between the GCSF therapy and SMT groups were comparable (83.91% and 72.9%, respectively), with an OR of 1.9 (95% CI: 0.87-4.1) and I² = 43.2%. In patients who received anti-inflammatory therapies in combination with GCSF, overall survival was 83.89% and 81.35% in the combination group and 71.84% and 51.45% in the SMT group on days 28 and 90, respectively, with an overall pooled OR of 4.86 (95% CI: 1.63-14.46) at day 90 and 2.53 (95% CI, 1.07-6.03) on day 28 associated low heterogeneity (I² = 20.5% and 24.2%, respectively). The overall rate of infection on day 90 was 20.1% in the GCSF arm and 27.22% in the control arm, with pooled ORs of 0.36 (95% CI: 0.18-0.691). The overall variceal bleeding rates were 5.6% in the GCSF and 14.07% in the control group on day 90. GCSF therapy is associated with a decreased risk of variceal bleeding compared with controls, with an OR of 0.41 (95% CI: 0.25-0.67).

Conclusion:

GCSF therapy is associated with higher survival and lower adverse events in SAH. Combining GCSF with anti-inflammatory therapies can further improve 28- and 90-day survival.

01 Feb 2026·Transplantation and Cellular Therapy

Excellent Outcome of 1-Day Nonmyeloablative Salvage Regimen for Pediatric Patients with Graft Failure following Haploidentical Hematopoietic Stem Cell Transplantation

Article

Author: Lee, Pamela P W ; Leung, Wing ; Chan, Wilson Y K ; Cheuk, Daniel K L

Haploidentical (haplo-) hematopoietic stem cell transplantation (HSCT) has been increasingly used as an alternative transplantation strategy for patients lacking a suitable HLA-matched donor. T cell depletion reduces the risk of graft-versus-host disease (GVHD) but at a cost of increased risk of graft failure, necessitating immediate retransplantation if a cryopreserved autologous graft is not available for rescue. Traditional high-intensity reconditioning with myeloablative and immunoablative regimens of approximately 1 week's duration aimed at further suppressing the recipient-derived immune system capable of rejecting the primary graft, is associated with high morbidity and mortality. A shortened and reduced-intensity conditioning regimen might reduce infection risk and mortality, but sustained engraftment would be a concern with lowered intensity. Here we report the excellent outcomes of 11 pediatric patients who received a 1-day reduced-intensity preparative regimen prior to retransplantation for graft failure following initial myeloablative haplo-HSCT for various malignant and nonmalignant disease conditions. This was a retrospective study conducted at the Hong Kong Children's Hospital, the sole territory-wide pediatric HSCT center in Hong Kong. All pediatric patients who were age ≤18 years at the time of initial haplo-HSCT and subsequently underwent salvage haplo-HSCT with a 1-day nonmyeloablative salvage regimen owing to graft failure between June 1, 2021, and May 31, 2024, were included. The salvage regimen consisted of fludarabine (30 mg/m²), cyclophosphamide (2000 mg/m² or 60 mg/kg), and alemtuzumab (0.3 mg/kg), with or without total body irradiation (2 Gy), all administered 1 day before retransplantation. G-CSF-mobilized peripheral blood stem cells (PBSCs) were collected and transplanted fresh without ex vivo T cell depletion whenever possible, while cryopreserved PBSCs were used if fresh apheresis was not possible. GVHD prophylaxis consisted of cyclosporine, mycophenolate mofetil, tacrolimus, or sirolimus. A total of 11 patients were recruited, including 9 males and 2 females, with a median age of 8.8 years (range, 2.4 to 17.5 years). Underlying diseases included transfusion-dependent anemias (n = 7; beta-thalassemia major = 4, hemoglobin Hammersmith = 1, pyruvate kinase deficiency = 1, severe aplastic anemia = 1), chronic granulomatous disease (n = 1), acute lymphoblastic leukemia (n = 1), post-liver transplant lymphoproliferative disease (n = 1) and neuroblastoma (n = 1). The median interval between haplo-HSCT and administration of the 1-day regimen was 26 days (range, 18 to 50 days). Fresh PBSC grafts were used in 9 patients, and cryopreserved PBSC grafts were used in 2 patients. Median cell viability was 99.6%, with a median stem cell dose of 7.2 × 10⁶ CD34⁺ cells/kg (range, 4.81 to 20.6 × 10⁶ cells/kg) and a median total nucleated cell (TNC) dose of 8.0 × 10⁸/kg (range, 5.17 to 10 × 10⁸/kg). All 9 patients with fresh PBSC grafts demonstrated sustained engraftment and hematopoietic recovery. The median times to neutrophil and platelet engraftment were day +12 (range, days 10 to 19) and day +16 (range, days 10 to 35), respectively. No patient developed grade III/IV acute GVHD or severe chronic GVHD. Both patients with cryopreserved PBSC grafts experienced another graft failure, but 1 of them was successfully salvaged with another 1-day regimen using a fresh PBSC graft, and the other had autologous regeneration and underwent successful haplo-HSCT again from the same donor after full myeloablative conditioning. Overall survival for all 11 patients was 100% at a median follow-up of 35 months (range, 18 to 52 months). To conclude, local experience suggests that the modified 1-day reduced-intensity regimen in combination with fresh but not cryopreserved PBSC grafts is a feasible and promising approach to achieving sustained engraftment and is safe and appropriate for salvaging pediatric patients with graft failure requiring immediate retransplantation.

01 Feb 2026·PROTEIN EXPRESSION AND PURIFICATION

Effect of culture media and fermentation process on the refolding and purification of rh-GCSF

Article

Author: Abolghasemi, Somayeh ; Poureini, Fatemeh ; Farji, Faezeh ; Mofid, Mohammad Reza ; Babaeipour, Valiollah

Human granulocyte-colony stimulating factor (h-GCSF) is used to mitigate neutropenia caused by chemotherapy. E. coli is the most common host for h-GCSF production due to its high yield and versatile culture strategies. However, overproduction of h-GCSF in E. coli often results in the formation of inclusion bodies (IBs) in the cytoplasm. The efficiency of refolding and purifying recombinant h-GCSF (rh-GCSF) produced as IBs depends on the expression strategy, induction, and cell growth conditions. This study investigated how different culture media and fermentation processes affect the refolding of IBs and the purification of rh-GCSF. The purified samples were compared to Neupogen® and PDgrastim reference standards using Western blotting, size exclusion chromatography, and reverse-phase high-performance liquid chromatography. The analysis confirmed that all proteins were correctly refolded and exhibited a purity exceeding 90 % after purification. The highest protein recovery percentage and purity of rh-GCSF with the lowest endotoxin content was achieved using M9 media in fed-batch culture.

143

News (Medical) associated with Filgrastim (Amgen)

17 Feb 2026

·www.biospace.com

/C O R R E C T I O N -- Accord BioPharma/

In the news release, FDA Approves FILKRI™ (filgrastim-laha), Accord BioPharma's Biosimilar to NEUPOGEN® (filgrastim), issued 17-Feb-2026 by Accord BioPharma over PR Newswire, we are advised by the company that changes have been made. The complete, corrected release follows, with additional details at the end: FDA Approves FILKRI™ (filgrastim-laha), Accord BioPharma’s Biosimilar to NEUPOGEN® (filgrastim) FILKRI completes Accord BioPharma's comprehensive granulocyte colony-stimulating factor (G-CSF) portfolio alongside UDENYCA ® (pegfilgrastim-cbqv ) RALEIGH, N.C. , Feb. 17, 2026 /PRNewswire/ -- Accord BioPharma , the U.S. specialty division of Intas Pharmaceuticals, Ltd., focused on the development of oncology, immunology, and critical care therapies, announced that the U.S. Food and Drug Administration (FDA) has approved FILKRI™ (filgrastim-laha), a biosimilar to NEUPOGEN ® (filgrastim), for patients with cancer receiving myelosuppressive chemotherapy; patients with acute myeloid leukemia receiving induction or consolidation chemotherapy; patients with cancer undergoing bone marrow transplantation; patients with severe chronic neutropenia; and patients acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome). 1 The approval of FILKRI marks the sixth product in Accord BioPharma's growing portfolio of FDA-approved biosimilars, following the company's acquisition last year of UDENYCA ® (pegfilgrastim-cbqv), a biosimilar to Neulasta ® (pegfilgrastim), 2 as well as the seventh approved product in the overall portfolio. With the addition of FILKRI, Accord BioPharma now offers physicians in the U.S. a comprehensive granulocyte colony-stimulating factor (G-CSF) portfolio with both long-acting (UDENYCA) and short-acting (FILKRI) biosimilar options to meet the needs of patients, providers, and practices. 1,2 Accord has applied for and expects to receive a permanent Q-code from the U.S. Centers for Medicare & Medicaid Services (CMS), which will standardize and facilitate the billing and reimbursement process across hospital outpatient, ambulatory surgery center, and physician office settings of care. Tackling Vital Healthcare Needs Neutropenia is a common and potentially serious complication of cancer treatment and occurs when white blood cells called neutrophils—which serve as a major line of defense against bacterial and fungal infections—fall below normal levels. This reduction increases a person's risk of developing an infection. Granulocyte colony-stimulating factor, or G-CSF, is a growth factor that stimulates the production and release of neutrophils in the body. By accelerating neutrophil recovery, G-CSF can help reduce the duration of neutropenia. 3,4 FILKRI belongs to the G-CSF class and is a growth factor manufactured by recombinant DNA technology. FILKRI works by regulating the production of neutrophils within the bone marrow. 1 "Cancer patients often face significant challenges with treatment-related neutropenia, which can lead to serious infections, treatment delays, and dose reductions that may compromise therapeutic outcomes," said Chrys Kokino, President, Accord North America. "With FILKRI alongside UDENYCA, the provider-preferred option over Neulasta and all other biosimilars,* we now offer healthcare providers a complete G-CSF portfolio with short- and long-acting biosimilar options. This positions Accord BioPharma as a committed partner in oncology supportive care, expanding access to high-quality biologics." Demonstrated Biosimilarity and Safety Profile FILKRI was approved based on two randomized studies in healthy adults, with pharmacokinetics (PK)/pharmacodynamics (PD) assessed in one study and safety and immunogenicity evaluated in both, compared with reference product NEUPOGEN. These studies demonstrated the biosimilarity in PD and PK parameters between FILKRI and NEUPOGEN and showed overall safety and immunogenicity similar to NEUPOGEN. 5 FILKRI is contraindicated in patients with a history of serious allergic reactions to human granulocyte colony-stimulating factors, such as filgrastim products or pegfilgrastim products. 1 Expanding Access: Accord BioPharma's Biosimilar Vision Accord BioPharma believes deeply in the power of biosimilars to reshape the future of treatment. With the support of Intas, an established global leader with nearly five decades of experience, Accord BioPharma is transforming the conventional treatment landscape across several priority therapeutic areas by introducing biosimilars to the U.S. market. As part of this commitment, Accord BioPharma is rapidly expanding its U.S. portfolio with a strategic goal to launch 20 biosimilar products by 2030, solidifying its position as a reliable partner delivering more affordable, high-quality biologic alternatives. In addition to developing and marketing their own biosimilar products, the company is strategically collaborating with select partners around the world to bring more biosimilars to the U.S. market as swiftly as possible. "This approval of FILKRI demonstrates our steadfast dedication to expanding access to cost-effective biologic treatments in the critically important field of oncology," said Binish Chudgar, Chairman and Managing Director of Intas Pharmaceuticals. "We're proud to have one of the largest biosimilar pipelines within the industry. With Accord BioPharma, we're positioning ourselves as a dependable partner in the United States—one that's deeply committed to understanding stakeholder priorities and revolutionizing patient access." Current Portfolio of Brands The company's current commercial portfolio includes the full UDENYCA franchise, including the UDENYCA prefilled syringe, autoinjector, and on-body injector, IMULDOSA ® (ustekinumab-srlf), a biosimilar to Stelara ® (ustekinumab), HERCESSI™ (trastuzumab-strf), a biosimilar to Herceptin ® (trastuzumab), and CAMCEVI ® (leuprolide) 42 mg injectable emulsion. 6,7 Please refer to the Important Safety Information and full Prescribing Information for these products, and to the Boxed Warning for HERCESSI. Accord BioPharma has received FDA approval on three additional products: CAMCEVI ® (leuprolide) ETM (every three months), OSVYRTI ® (denosumab-desu), a biosimilar to PROLIA ® (denosumab), and JUBEREQ ® (denosumab-desu), a biosimilar to XGEVA ® (denosumab). 8,9 The company plans to announce the commercial launch of these products later this year. Please refer to the Important Safety Information and full Prescribing Information for these products, and to the Boxed Warning for OSVYRTI. Contact: abipr@accordhealthcare.com IMPORTANT SAFETY INFORMATION FILKRI is contraindicated in patients with a history of serious allergic reactions to human granulocyte colony-stimulating factors (G-CSFs) such as filgrastim or pegfilgrastim products. Splenic Rupture: Splenic rupture, including fatal cases, has been reported following the administration of filgrastim products. Evaluate patients who report left upper abdominal or shoulder pain for an enlarged spleen or splenic rupture. Acute Respiratory Distress Syndrome (ARDS): ARDS has been reported in patients receiving filgrastim products . Evaluate patients who develop fever and lung infiltrates or respiratory distress for ARDS . Discontinue FILKRI in patients with ARDS. Serious Allergic Reactions: Serious allergic reactions, including anaphylaxis, have been reported in patients receiving filgrastim products . The majority of reported events occurred upon initial exposure . Provide symptomatic treatment for allergic reactions. Allergic reactions, including anaphylaxis, in patients receiving filgrastim products can recur within days after the discontinuation of initial anti-allergic treatment . Permanently discontinue FILKRI in patients with serious allergic reactions. Sickle Cell Disorders: Severe and sometimes fatal sickle cell crises can occur in patients with sickle cell disorders receiving filgrastim products . Discontinue FILKRI if sickle cell crisis occurs. Glomerulonephritis: Has occurred in patients receiving filgrastim products . Diagnoses were based on azotemia, hematuria, proteinuria, and renal biopsy . Generally, events resolved after dose reduction or discontinuation of filgrastim products . If causality is likely, consider dose-reduction or interruption of FILKRI. Alveolar Hemorrhage and Hemoptysis: Alveolar hemorrhage, manifesting as pulmonary infiltrates and hemoptysis requiring hospitalization, have been reported in FILKRI-treated healthy donors undergoing peripheral blood progenitor cell (PBPC) collection mobilization . Hemoptysis resolved with discontinuation of filgrastim products. The use of FILKRI for PBPC mobilization in healthy donors is not an approved indication. Capillary Leak Syndrome (CLS): CLS has been reported after G-CSF administration, including filgrastim products and is characterized by hypotension, hypoalbuminemia, edema, and hemoconcentration. Episodes vary in frequency, severity, and may be life-threatening if treatment is delayed . Patients with symptoms should be closely monitored and receive appropriate treatment. Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML): Patients with Severe Chronic Neutropenia (SCN ): Confirm the diagnosis of SCN before initiating FILKRI therapy. MDS and AML have been reported to occur in the natural history of congenital neutropenia without cytokine therapy. Cytogenetic abnormalities, transformation to MDS, and AML have also been observed in patients treated with filgrastim products for SCN. Abnormal cytogenetics and MDS have been associated with the eventual development of myeloid leukemia. The effect of filgrastim products on the development of abnormal cytogenetics and the effect of continued filgrastim administration in patients with abnormal cytogenetics or MDS are unknown. Monitor patients for signs and symptoms of MDS/AML in these settings. If a patient with SCN develops abnormal cytogenetics or myelodysplasia‚ the risks and benefits of continuing FILKRI should be carefully considered. Patients with Breast and Lung Cancer : MDS and AML have been associated with the use of filgrastim products in conjunction with chemotherapy and/or radiotherapy in patients with breast and lung cancer. Monitor patients for signs and symptoms of MDS/AML in these settings. Thrombocytopenia: Thrombocytopenia has been reported in patients receiving filgrastim products. Monitor platelet counts. Leukocytosis: Patients with Cancer Receiving Myelosuppressive Chemotherapy : White blood cell counts ≥ 100,000/mm 2 were observed in about 2% of patients who received filgrastim products at dosages above 5 mcg/kg/day. Discontinue FILKRI if the absolute neutrophil count (ANC) surpasses 10,000/mm 2 after the chemotherapy-induced ANC nadir has occurred. Monitor CBCs at least twice weekly during therapy with FILKRI. Discontinuation of filgrastim therapy usually resulted in a 50% decrease in circulating neutrophils within 1 to 2 days‚ with a return to pretreatment levels in 1 to 7 days. Cutaneous Vasculitis: Moderate or severe cases of cutaneous vasculitis has been reported in patients treated with filgrastim products . Most reports involved patients with SCN receiving long-term filgrastim therapy . Hold FILKRI therapy in patients with cutaneous vasculitis . FILKRI dose may be reduced when the symptoms resolve and the ANC has decreased. Potential Effect on Malignant Cells: FILKRI is a growth factor that primarily stimulates neutrophils. The granulocyte colony-stimulating factor (G-CSF) receptor through which FILKRI acts has also been found on tumor cell lines. The possibility that FILKRI acts as a growth factor for any tumor type cannot be excluded. The safety of filgrastim products in chronic myeloid leukemia (CML) and myelodysplasia has not been established. Simultaneous Use with Chemotherapy and Radiation Not Recommended: The safety and efficacy of FILKRI given simultaneously with cytotoxic chemotherapy and radiation have not been established . Do not use FILKRI 24 hours before or after administration of cytotoxic chemotherapy . The safety and efficacy of FILKRI have not been evaluated in patients receiving concurrent radiation therapy . Avoid the simultaneous use of FILKRI with chemotherapy and radiation therapy. Nuclear Imaging: Increased hematopoietic activity of the bone marrow has been associated with transient positive bone-imaging changes on nuclear imaging. Aortitis: Aortitis has been reported in patients receiving filgrastim products . It may occur as early as the first week after start of therapy . Manifestations may include generalized signs and symptoms such as fever, abdominal pain, malaise, back pain, and increased inflammatory markers (e.g., c-reactive protein and white blood cell count) . Consider aortitis in patients who develop these signs and symptoms without known etiology. Discontinue FILKRI if aortitis is suspected. The most common adverse reactions in patients: with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs (≥ 5% difference in incidence compared to placebo) are pyrexia, pain, rash, cough, and dyspnea with AML (≥ 2% difference in incidence) are pain, epistaxis and rash with nonmyeloid malignancies undergoing myeloablative chemotherapy followed by BMT (≥ 5% difference in incidence) is rash with severe chronic neutropenia (SCN) (≥ 5% difference in incidence) are pain, anemia, epistaxis, diarrhea, hypoesthesia and alopecia INDICATIONS FILKRI is a leukocyte growth factor indicated to: Decrease the incidence of infection‚ as manifested by febrile neutropenia‚ in patients with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with a significant incidence of severe neutropenia with fever Reduce the time to neutrophil recovery and the duration of fever, following induction or consolidation chemotherapy treatment of patients with acute myeloid leukemia (AML) Reduce the duration of neutropenia and neutropenia-related clinical sequelae‚ e.g.‚ febrile neutropenia, in patients with nonmyeloid malignancies undergoing myeloablative chemotherapy followed by bone marrow transplantation (BMT) Reduce the incidence and duration of sequelae of severe neutropenia (e.g.‚ fever‚ infections‚ oropharyngeal ulcers) in symptomatic patients with congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia Increase survival in patients acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome) To report SUSPECTED ADVERSE REACTIONS, contact Accord BioPharma Inc at 1-866-941-7875 or FDA at 1-800-FDA-1088 or . FILKRI is supplied as single-dose prefilled syringes for subcutaneous or intravenous use in 300 mcg/0.5 mL and 480 mcg/0.8 mL strengths. For more information, please see the full Prescribing Information . About Accord BioPharma Accord BioPharma, Inc., the U.S. specialty division of Intas Pharmaceuticals, Ltd., seeks to provide affordable, accessible, patient-centric therapies in oncology, immunology, and critical care. With a focus on improving the patient experience, Accord BioPharma goes beyond the biology of medicine to see disease from the patients' perspective and develop high-quality therapies that impact patients' lives. Accord BioPharma believes in the ability of biosimilars to increase access and options for patients and deliver savings to the U.S. healthcare system, and is striving to offer one of the deepest biosimilar portfolios in the industry. For more information, visit AccordBioPharma.com . *Based on cumulative average of prescriptions written from September 2025 - January 2026, according to IQVIA drug distribution data. References: FILKRI ® (filgrastim-laha) injection. Prescribing Information. Accord BioPharma. UDENYCA ® (pegfilgrastim-cbqv). Prescribing Information. Accord BioPharma. Simpson, P. Clinical Implications and Future Research of Neutrophils in Health and Disease. Immunome Research 2024;20(3): 1-2. Link, H. Current state and future opportunities in granulocyte colony‑stimulating factor (G‑CSF). Supportive Care in Cancer 2022;30:7067–7077. Accord BioPharma. Data on file. IMULDOSA ® (ustekinumab-srlf). Prescribing Information. Accord BioPharma. HERCESSI™ (trastuzumab-strf). Prescribing Information. Accord BioPharma. OSVYRTI ® (denosumab-desu). Prescribing Information. Accord BioPharma. JUBEREQ ® (denosumab-desu). Prescribing Information. Accord BioPharma. All trademarks, logos, and brand names are the property of their respective owners. Correction:  An earlier version of this release had the incorrect link to the full Prescribing Information. View original content to download multimedia: SOURCE Accord BioPharma

Drug ApprovalAcquisitionClinical ResultRadiation Therapy

04 Feb 2026

·www.biospace.com

Ayrmid Reports Additional New Real-World Data on Motixafortide for Stem Cell Mobilization in Sickle Cell Disease and Beta-Thalassemia

Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies 73% (11 of 15) of patients were able to collect sufficient HSC to enable progress to gene therapy manufacturing with 5 patients receiving their gene therapy and appropriately engrafted 73% (11 of 15) of patients had previously failed to collect sufficient cells with plerixafor Results support the use of motixafortide as an effective single-agent mobilizer of HSC for people with sickle cell disease and in combination with G-CSF for beta-thalassemia Findings presented at TANDEM 2026 DUBLIN, IE / ACCESS Newswire / February 4, 2026 / Ayrmid, Ltd. ("Ayrmid" or the "Company"), the parent company of Gamida Cell Inc., today announced further encouraging real-world data on the use of motixafortide, a long-acting CXCR4 inhibitor licensed by Ayrmid under the brand name APHEXDA ® , for mobilizing HSCs in patients with sickle cell disease undergoing gene therapy. The results were presented at the 2025 Transplantation & Cellular Therapy meetings of ASTCT ® and CIBMTR ® (TANDEM), taking place from February 4-7, 2026 in Salt Lake City, UT. Motixafortide is currently FDA approved in combination with filgrastim (G-CSF) for stem cell mobilization in multiple myeloma. The product's effectiveness and pharmacologic pro prompted growing interest in its potential use for sickle cell disease, where collecting adequate stem cells remains a significant barrier to gene therapy. Many patients do not mobilize enough cells with standard approaches, limiting access to curative treatment. Researchers from five treatment centers evaluated the real-world use of motixafortide in 15 patients aged 14-50. Each patient underwent one or more collection cycles over two or more days to obtain the number of stem cells required for gene therapy manufacturing. Motixafortide effectively mobilized 11 patients who had previously failed plerixafor, 3 patients mobilized effectively with motixafortide used as first line mobilization therapy. In 11 patients, sufficient cells were achieved to manufacture gene therapy for both sickle cell disease and beta-thalassemia. 5 patients have received their gene therapy and have appropriately engrafted, manufacturing is underway in a further 5 patients. Patients received Casgevy and Lyfgenia, and manufacturing is underway for Casgevy, Lyfgenia and Zynteglo. This data supports that motixafortide mobilized cells can be used across different gene therapy manufacturing platforms. Dr. John Manis,Director Transfusion Medicine Service, Associate Professor of Pathology, Harvard Medical School, commented: "The approval of gene therapies for sickle cell disease has opened the door to transformative outcomes, but inadequate stem cell collection has significantly delayed manufacturing and prevented some patients from starting treatment. These findings suggest that motixafortide may help overcome this challenge, and with more studies, expand access to potentially life-changing therapies." About Sickle Cell Disease Sickle cell disease, also known as sickle cell anemia, is a severe inherited blood disorder caused by a genetic mutation that leads to misshapen, rigid red blood cells. These cells can obstruct blood flow, causing sudden episodes of severe pain, known as pain crises, and leading to life-threatening complications. Ayrmid is dedicated to advancing therapies that help individuals living with sickle cell disease manage their symptoms and improve their quality of life. About Beta-Thalassemia Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin, a component of hemoglobin, which is essential for carrying oxygen in red blood cells. This condition results in anemia, which can cause symptoms such as fatigue, weakness and pallor. About Ayrmid Ltd. and Gamida Cell Ayrmid Ltd. is the parent company of Gamida Cell Inc., a pioneering cell therapy company developing novel treatments designed to turn cells into powerful therapeutics. Gamida Cell Inc. currently has two FDA approved products on the market in the US, namely Omisirge ® (please see the current full Prescribing Information, including boxed warning, here ) and APHEXDA ® (please see the current full Prescribing Information here ). Gamida Cell operates as a wholly owned subsidiary of Ayrmid Limited, a UK entity. For additional information, please visit or follow Gamida Cell on LinkedIn , X , Facebook or Instagram . Contacts: Media, Investors / Business Development: bd@ayrmid.com This information is provided by RNS, the news service of the London Stock Exchange. RNS is approved by the Financial Conduct Authority to act as a Primary Information Provider in the United Kingdom. Terms and conditions relating to the use and distribution of this information may apply. For further information, please contact rns@lseg.com or visit . SOURCE: Ayrmid Pharma Ltd View the original press release on ACCESS Newswire

Gene TherapyDrug ApprovalCell Therapy

09 Jan 2026

·investor.assertiotx.com

Assertio Announces Publication of Rolvedon® Same-Day Dosing Clinical Trial

Peer-reviewed publication in “The Oncologist” is now available online Investigational same-day dosing of Rolvedon demonstrated a clinical response and adverse event profile similar to next-day dosing LAKE FOREST, Ill.--(BUSINESS WIRE)-- Assertio Holdings, Inc. (“Assertio” or the “Company”) (Nasdaq: ASRT) today announced that results of a clinical trial investigating a novel dosing schedule of Rolvedon® (eflapegrastim-xnst) injection have been peer reviewed and published in The Oncologist. In the study of patients with early-stage breast cancer (ESBC), Rolvedon, when administered on the same day (same-day dosing) as TC chemotherapy (Taxotere (docetaxel) and cyclophosphamide) demonstrated an effective neutrophil recovery and an adverse event profile similar to that seen with next-day dosing. Howard Franklin, M.D., Senior Vice President of Medical with Assertio: “We were encouraged to see that same-day dosing of Rolvedon demonstrated efficacy and safety similar to dosing one day after chemotherapy. Assertio is proud to lead this study that provides data that supports a more convenient administration schedule for Rolvedon. Thank you to the investigators and patients who participated in this study.” Lee Schwartzberg, M.D., Professor of Clinical Medicine with Renown Health-Pennington Cancer Institute, Reno, Nevada, and a study author: “Febrile neutropenia (FN) is a significant risk factor among patients with early-stage breast cancer who are undergoing chemotherapy. This important study provides the oncology community with much-needed insight into the potential utility of same-day dosing of GCSF therapy with Rolvedon in these patients.” The Oncologist is the official peer-reviewed medical journal for the Society for Translational Oncology. Results of this study were first presented in December 2024 as part of the San Antonio Breast Cancer Symposium. Study Summary This phase 1, multicenter (13 sites in the U.S.), open-label trial (NCT04187898) evaluated the efficacy and safety of Rolvedon administered on the same day as TC chemotherapy in patients (n = 53) with ESBC. Patients received four cycles of TC chemotherapy with Rolvedon administered at 30 minutes after TC. The primary endpoint was the time to recovery of absolute neutrophil count (ANC) from nadir to ≥1.5 × 109/L in Cycle 1. The ANC is a blood test that measures the number of circulating neutrophils, a type of white blood cell essential for fighting infections. Normal ANC indicates a healthy immune response, while low levels (neutropenia) can increase the risk of infections. Secondary study endpoints included safety, incidence of severe neutropenia, duration of severe neutropenia, incidence of FN, and neutropenic complications. A total of 53 patients enrolled; 49 completed the study. Patients received the recommended 13.2 mg (3.6 mg GCSF) fixed-dose of Rolvedon 30 min following TC chemotherapy. The mean time to ANC recovery was 1.8 days in cycle 1. The incidence of FN was low, reported in only 1 patient during the entire trial. There were no reported neutropenic complications, with no patients requiring hospitalization and/or antibiotic therapy. Same-day administration of Rolvedon was well tolerated and no new safety signals were observed. The benefits of eflapegrastim-xnst given same day as TC have not been evaluated in a randomized controlled trial. About Assertio Assertio is a pharmaceutical company with comprehensive commercial capabilities offering differentiated products designed to address patients’ needs. Our focus is on supporting patients by marketing products in oncology, neurology, and pain management. To learn more about Assertio, visit www.assertiotx.com. About Rolvedon (eflapegrastim-xnst) Injection ROLVEDON (eflapegrastim-xnst) injection is a long-acting granulocyte colony-stimulating factor (G-CSF) with a novel formulation. Rolvedon is indicated to decrease the incidence of infection, as manifested by febrile neutropenia, in adult patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. Assertio has designated Rolvedon as one of the company’s core growth assets. Please see the Important Safety Information below and the full prescribing information for ROLVEDON at https://www.rolvedon.com/pdf/rolvedon-prescribing-information.pdf. Limitations of Use ROLVEDON is not indicated for the mobilization of peripheral blood progenitor cells for hematopoietic stem cell transplantation. Important Safety Information Contraindications ROLVEDON is contraindicated in patients with a history of serious allergic reactions to eflapegrastim, pegfilgrastim or filgrastim products. Reactions may include anaphylaxis. Warnings and Precautions Splenic Rupture Splenic rupture, including fatal cases, can occur following the administration of recombinant human granulocyte colony-stimulating factor (rhG-CSF) products. Evaluate patients who report left upper abdominal or shoulder pain for an enlarged spleen or splenic rupture. Acute Respiratory Distress Syndrome (ARDS) ARDS can occur in patients receiving rhG-CSF products. Evaluate patients who develop fever, lung infiltrates, or respiratory distress. Discontinue ROLVEDON in patients with ARDS. Serious Allergic Reactions Serious allergic reactions, including anaphylaxis, can occur in patients receiving rhG-CSF products. Permanently discontinue ROLVEDON in patients who experience serious allergic reactions. Sickle Cell Crisis in Patients with Sickle Cell Disorders Severe and sometimes fatal sickle cell crises can occur in patients with sickle cell disorders receiving rhG-CSF products. Discontinue ROLVEDON if sickle cell crisis occurs. Glomerulonephritis Glomerulonephritis has occurred in patients receiving rhG-CSF products. The diagnoses were based upon azotemia, hematuria (microscopic and macroscopic), proteinuria, and renal biopsy. Generally, events of glomerulonephritis resolved after dose-reduction or discontinuation. Evaluate and consider dose reduction or interruption of ROLVEDON if causality is likely. Leukocytosis White blood cell (WBC) counts of 100 x 109/L or greater have been observed in patients receiving rhG-CSF products. Monitor complete blood count (CBC) during ROLVEDON therapy. Discontinue ROLVEDON treatment if WBC count of 100 x 109/L or greater occurs. Thrombocytopenia Thrombocytopenia has been reported in patients receiving rhG-CSF products. Monitor platelet counts. Capillary Leak Syndrome Capillary leak syndrome has been reported after administration of rhG-CSF products and is characterized by hypotension, hypoalbuminemia, edema and hemoconcentration. Episodes vary in frequency and severity and may be life-threatening if treatment is delayed. If symptoms develop, closely monitor and give standard symptomatic treatment, which may include a need for intensive care. Potential for Tumor Growth Stimulatory Effects on Malignant Cells The granulocyte colony-stimulating factor (G-CSF) receptor through which ROLVEDON acts has been found on tumor cell lines. The possibility that ROLVEDON acts as a growth factor for any tumor type, including myeloid malignancies and myelodysplasia, diseases for which ROLVEDON is not approved, cannot be excluded. Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML) in Patients with Breast and Lung Cancer MDS and AML have been associated with the use of rhG-CSF products in conjunction with chemotherapy and/or radiotherapy in patients with breast and lung cancer. Monitor patients for signs and symptoms of MDS/AML in these settings. Aortitis Aortitis has been reported in patients receiving rhG-CSF products. It may occur as early as the first week after start of therapy. Consider aortitis in patients who develop generalized signs and symptoms such as fever, abdominal pain, malaise, back pain, and increased inflammatory markers (e.g., c-reactive protein and white blood cell count) without known etiology. Discontinue ROLVEDON if aortitis is suspected. Nuclear Imaging Increased hematopoietic activity of the bone marrow in response to growth factor therapy has been associated with transient positive bone imaging changes. This should be considered when interpreting bone imaging results. Adverse Reactions The most common adverse reactions (≥20%) were fatigue, nausea, diarrhea, bone pain, headache, pyrexia, anemia, rash, myalgia, arthralgia, and back pain. Permanent discontinuation due to an adverse reaction occurred in 4% of patients who received ROLVEDON. The adverse reaction requiring permanent discontinuation in 3 patients who received ROLVEDON was rash. To report SUSPECTED ADVERSE REACTIONS, contact Assertio Holdings, Inc. at 1-866-458-6389 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Forward-Looking Statements Statements in this communication that are not historical facts are forward-looking statements that reflect Assertio’s current expectations, assumptions and estimates of future performance and economic conditions. These forward-looking statements are made in reliance on the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements relate to, among other things, future events or the future performance or operations of Assertio, including risks related to our ability to realize the benefits from our operating model, deliver or execute on our business strategy, including to expand or diversify our asset base and market reach and drive cash flows and growth, successfully integrate new assets, and explore new business development initiatives. All statements other than historical facts may be forward-looking statements and can be identified by words such as “anticipate,” “believe,” “could,” “design,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “imply,” “intend,” “may,” “objective,” “opportunity,” “outlook,” “plan,” “position,” “potential,” “predict,” “project,” “prospective,” “pursue,” “seek,” “should,” “strategy,” “target,” “would,” “will,” “aim” or other similar expressions that convey the uncertainty of future events or outcomes and are used to identify forward-looking statements. Such forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and other factors, some of which are beyond the control of Assertio, including the risks described in Assertio’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the U.S. Securities and Exchange Commission (“SEC”) and in other filings Assertio makes with the SEC from time to time. Investors and potential investors are urged not to place undue reliance on forward-looking statements in this communication, which speak only as of this date. While Assertio may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to update or revise any forward-looking-statements contained in this press release whether as a result of new information or future events, except as may be required by applicable law. View source version on businesswire.com: https://www.businesswire.com/news/home/20260109405279/en/ Investor and Media Contact Longacre Square Partners assertio@longacresquare.com Source: Assertio Holdings, Inc.

Clinical ResultPhase 1ASCO

100 Deals associated with Filgrastim (Amgen)

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KEGG	Wiki	ATC	Drug Bank
D03235	Filgrastim (Amgen)	L03AA02	DB00099

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Approved

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Indication	Country/Location	Organization	Date
Neuroblastoma	Japan	Kyowa Kirin Co., Ltd.	23 Jun 2021
Stem cell mobilisation	Japan	Kyowa Kirin Co., Ltd.	10 Mar 2000
Acute Myeloid Leukemia	United States	Amgen, Inc.	20 Feb 1991
Bone marrow depression	United States	Amgen, Inc.	20 Feb 1991
Neutropenia	United States	Amgen, Inc.	20 Feb 1991

Developing

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Indication	Highest Phase	Country/Location	Organization	Date
Breast Cancer	Phase 3	United Kingdom	Hospira UK Ltd.	23 May 2007
Febrile Neutropenia	Phase 3	United Kingdom	Hospira UK Ltd.	23 May 2007

Clinical Result

Indication

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05115630 (CTgov)	Phase 2	Myeloid Tumor \| Acute Myeloid Leukemia \| Chronic phase chronic myeloid leukemia ... View more	24	TBI+Fludarabine+Melphalan	gaqowmzlyw = mqomzmlmsl kmxzicphaa (ynkgenuxbs, cxudhaskfe - ggxkugssgg) View more	-	19 Dec 2025
NCT00278408 (UNFOLDER, CTgov)	Phase 3	CD20 positive B-Cell Lymphoma	700	R-CHOP-21 (Interventional: 6 R-CHOP-21 for Patients With Radiotherapy Indication)	kwifwwptce = tqvcnezqry aatkmvoljs (lvgqfppcxz, emgknsdrwl - fvqytetfxa) View more	-	26 Aug 2025
NCT00278408 (UNFOLDER, CTgov)	Phase 3	CD20 positive B-Cell Lymphoma	700	R-CHOP-14 (Interventional: 6 R-CHOP-14 for Patients With Radiotherapy Indication)	kwifwwptce = yytwrmiemo aatkmvoljs (lvgqfppcxz, jxokmsyokj - oirobgotlj) View more	-	26 Aug 2025
NCT00878254 (CTgov)	Phase 2	Mantle-Cell Lymphoma	25	Mesna+Etoposide+Cyclophosphamide+Leucovorin+G-CSF+Doxorubicin+ifosfamide+Methotrexate+Rituximab+Vincristine+Cytarabine	olovpopraj(iqyvgvsfdw) = uxgvhfyzjz wgbettnpnf (oydoskygyl, ujgidmflpq - ybyesssnsq) View more	-	23 Jul 2025
ESMO_BC2025	Not Applicable	Early Stage Breast Carcinoma	111	Filgrastim	yswuizivrt(bwztynaupo) = xfycvtbmlx zubarkapqc (tdhnnjtyvv ) View more	Negative	14 May 2025
ESMO_BC2025	Not Applicable	Early Stage Breast Carcinoma	111	Pegfilgrastim	yswuizivrt(bwztynaupo) = mndhqjcqtv zubarkapqc (tdhnnjtyvv ) View more	Negative	14 May 2025
PB2486 (EHA2025)	Phase 2	Refractory acute myeloid leukemia PD-1 \| PD-L1	37	Azacitidine + CAG regimen + Tislelizumab	vheikpauol(bkerkpiupn) = lyufgfevtk yrxkiacnbr (gqaoqzutsc ) View more	Positive	14 May 2025
NCT03944915 (DEPEND, CTgov)	Phase 2	Human Papillomavirus Infection \| Human Papillomavirus-Related Squamous Cell Carcinoma \| Locally Advanced Head and Neck Squamous Cell Carcinoma	35	Nivolumab+Carboplatin+Paclitaxel (Standard Chemotherapy)	ipymyohllw = ojfqcujlay sapkoqrwvs (pmdujvnyuz, wtmlmpjvgz - viopyyowal) View more	-	11 May 2025
NCT03944915 (DEPEND, CTgov)	Phase 2		35	Radiation+Filgrastim Injection+Paclitaxel+Hydroxyurea Pill+cisplatin+5-fluorouracil (De-escalated Chemotherapy)	ipymyohllw = hzuggkjvvh sapkoqrwvs (pmdujvnyuz, wnxnxqoctf - gkjadxhnyq) View more	-	11 May 2025
NCT04514692 (CTgov)	Phase 1/2	Endometrial Carcinoma \| Uterine Cervical Cancer \| Recurrent Endometrial Cancer	7	GCSF (Phase I -Dose Finding, Cohort 1)	elchrugsur = rwpiiynlsh zhzcimqszh (kuvpvughna, jprmtqscol - oeomypxjpe) View more	-	23 Apr 2025
NCT04514692 (CTgov)	Phase 1/2		7	GCSF (Phase I -Dose Finding, Cohort 2)	elchrugsur = zmvewqzvwk zhzcimqszh (kuvpvughna, lzwbdkpafm - nuhrfyhfso) View more	-	23 Apr 2025
NCT04294641 (CTgov)	Phase 2	Chronic graft-versus-host disease	10	Ibrutinib	rjckdtkwic = hqyxbznyzw qjjoyjptzm (iswpuccnwv, jmgmvteopw - mahqwqhqmr) View more	-	22 Jan 2025
ESMO_ASIA2024	Not Applicable	Early Stage Breast Carcinoma Adjuvant	93	Filgrastim	smfvirhfjz(yjquwbdfxv) = mtoaycxnqs fvcqlkxwwt (gfwwxvqxlw ) View more	Positive	07 Dec 2024
NCT02880293 (CTgov)	Not Applicable	Hematologic Neoplasms	44	Mesna+Fludarabine+cyclophosphamide+melphalan+Filgrastim+Tacrolimus+Mycophenolate Mofetil+thiotepa	ciybppuvmu = oqogijofgk ncjtybolgg (fpxdepymei, stpomixkub - irwrdsdwma) View more	-	06 Dec 2024

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