Last update 06 Nov 2024

Sargramostim

Last update 06 Nov 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Colony-stimulating factors

Synonyms

23-L-Leucinecolony-Stimulating Factor 2, Leukine Liquid, Prokine

+ [19]

Target

CSF-2R

Mechanism

CSF-2R stimulants(Granulocyte-macrophage colony-stimulating factor receptor stimulants)

Therapeutic Areas

NeoplasmsImmune System DiseasesCardiovascular Diseases+ [5]

Active Indication

Pulmonary Alveolar Proteinosis, AcquiredAcute Radiation SyndromeHematopoietic stem cell transplantation+ [4]

Inactive Indication

Acute Myeloid LeukemiaBladder CancerCastration-Resistant Prostatic Cancer+ [19]

Originator Organization

Amgen, Inc.

Active Organization

Berlex Laboratories, Inc.

Partner Therapeutics, Inc.Startup

Nobelpharma Co., Ltd.

Inactive Organization

Bayer AG

Genzyme Corp.Nihon Schering KK

+ [3]

Drug Highest PhaseApproved

First Approval Date

US (05 Mar 1991),

Hematopoietic stem cell transplantationMyeloid LeukemiaPrimary Graft Dysfunction

RegulationOrphan Drug (US), Orphan Drug (EU)

Gene Sequence

Sequence Code 18441095

Source: *****

316

Clinical Trials associated with Sargramostim

NCT06202066 / Not yet recruitingPhase 2IIT

A Phase II Study of Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) in Patients With Progressing Metastatic Neuroendocrine Tumors (NETs)

This phase II trial compares the safety and effect of temozolomide combined with survivin long peptide vaccine (SurVaxM) to temozolomide alone in patients with neuroendocrine tumors (NET) that has spread from where it first started (primary site) to other places in the body (metastatic) and is growing, spreading or getting worse (progressing). Temozolomide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill tumor cells and slow down or stop tumor growth. Survivin, a protein, is expressed in 50% of patients that have neuroendocrine tumors and, is associated with poor outcomes. SVN53-67/M57-KLH peptide vaccine (SurVaxM) is a vaccine that has been shown to produce an immune system response against cancer cells that express a survivin and may block the growth of new tumor cells. Giving temozolomide with SurVaxM may kill more tumor cells in patients with progressing metastatic neuroendocrine tumors.

Start Date15 Dec 2024

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

NCT06450041 / RecruitingPhase 2IIT

Phase II Study of Ex-Vivo Expanded Allogeneic Universal Donor TGFβi NK Cell Infusions in Combination With Temozolomide, Irinotecan, Dinutuximab, and Sargramostim in Patients With Relapsed or Refractory Neuroblastoma The STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial

This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.

Start Date01 Nov 2024

Sponsor / Collaborator

United Therapeutics Corp.

[+1]

NCT06626516 / Not yet recruitingPhase 1/2IIT

A Phase I/II Study of Tebentafusp-tebn in Combination With Liver-Directed Therapies for the Treatment of Metastatic Uveal Melanoma

We will conduct a multicenter, open label phase I/ II trial to assess the safety and clinical efficacy of tebentafusp-tebn in combination with liver-directed therapies in HLA-A*0201 positive patients with metastatic uveal melanoma. In Part 1 of the study, we will investigate the safety and efficacy of tebentafusp-tebn in combination with hepatic IE in patients with a low to moderate hepatic disease burden. In Part 2, we will investigate the efficacy of tebentafusp-tebn in combination with TACE in patients with bulky hepatic disease.

Start Date31 Oct 2024

Sponsor / Collaborator

Thomas Jefferson University

[+1]

100 Clinical Results associated with Sargramostim

100 Translational Medicine associated with Sargramostim

100 Patents (Medical) associated with Sargramostim

1,686

Literatures (Medical) associated with Sargramostim

03 Oct 2024·Microbiology Spectrum

Multicenter study of the performance of NTM Elite agar for the detection of nontuberculous mycobacteria from patients with cystic fibrosis

Article

Author: Kahl, Barbara C. ; Van Bleyenbergh, Pascal ; Dupont, Lieven ; Perry, John D. ; Beuselinck, Kurt ; Lorent, Natalie ; André, Emmanuel ; Gafsi, Johanne ; Laenen, Lies ; Deiwick, Susanne ; Raymaekers, Lise

ABSTRACT:

The performance of a novel selective agar was evaluated against the performance of conventional mycobacterial cultures, i.e., a combination of the mycobacterial growth indicator tube (MGIT) with Löwenstein-Jensen (LJ), for the detection of nontuberculous mycobacteria (NTM) in sputum samples from people with cystic fibrosis (pwCF). Two hundred eighty-three sputum samples (231 fresh sputum and 52 spiked sputum) from 143 pwCF were collected. They were inoculated without prior decontamination on NTM Elite agar (30°C ± 2°C for 28 days) and inoculated on both MGIT and LJ (35°C–37°C for 6–8 weeks) after N-acetyl-L-cysteine-2% sodium hydroxide decontamination. NTM were identified by Matrix-Assisted Laser Desorption Ionization/Time of Flight Mass Spectrometry and/or PCR, and whole-genome sequencing. A total of 67 NTM were recovered overall by the combination of all culture media. NTM Elite agar allowed the recovery of 65 NTM (97%), compared to 22 for the conventional MGIT and LJ media combination (32.8%), including 22 NTM for MGIT (32.8%) and 3 NTM with the LJ medium (4.5%). For Mycobacterium abscessus complex, the sensitivity of NTM Elite agar was 95% compared with a sensitivity of 30% for the conventional MGIT and LJ media combination. Overall, 17.3% of cultures on NTM Elite agar were contaminated with other micro-organisms vs 46.3% on MGIT and 77% on LJ. This study shows that the novel selective agar (NTM Elite agar) significantly outperforms the conventional MGIT and LJ media combination in terms of sensitivity, selectivity, and ease of culture, without the requirement of an L3 laboratory.

IMPORTANCE:

Nontuberculous mycobacteria (NTM) are significant pulmonary pathogens in patients with pre-existing structural lung conditions such as cystic fibrosis, bronchiectasis, or chronic obstructive pulmonary disease. Mycobacterium avium complex and Mycobacterium abscessus complex (MABSC) are the most frequently isolated organisms. Compared to the recommended culture method for NTM, which combines solid and liquid culture media, NTM Elite agar enables a faster/easier diagnosis and speeds up identification and susceptibility testing as the final reading is at 28 days instead of 6–8 weeks for the conventional mycobacterial cultures. In addition, for the NTM Elite agar, no decontamination stage before inoculation is necessary, unlike the conventional mycobacterial cultures. NTM Elite agar is derived from a formulation of medium adapted to rapidly growing mycobacteria (RGM). The medium enables the growth of RGM while suppressing other flora. It is supported with published clinical data showing the benefits of this medium.

06 Aug 2024·Microbiology Spectrum

Anti-biofilm activities and antibiotic synergy of naturally occurring compounds against drug-resistant rapidly growing mycobacteria

Article

Author: Yen, Chun-Lun ; Huang, Ya-Ling ; Huang, Yu-Chieh ; Huang, Chen-Hsiu ; Hsu, Chun-Ru

ABSTRACT:

Some naturally occurring compounds, known for their antimicrobial activities, have been employed as food additives. However, their efficacy in treating infections caused by antibiotic-resistant bacteria is yet to be fully explored. Rapidly growing mycobacteria (RGM), a category within nontuberculous mycobacteria (NTM), are prevalent in various environments and can lead to infections in humans. The rise of antimicrobial resistance within RGM is a documented concern. In this study, we reported that four specific natural compounds effectively inhibited the growth and biofilm formation of three key RGM pathogens M. abscessus , M. fortuitum , and M. chelonae . We screened 12 natural compounds for their effectiveness against antibiotic-resistant clinical strains of RGM. Four compounds showed significant inhibitory effects from the most effective to least: trans -cinnamaldehyde, carvacrol, gentisaldehyde, and phloroglucinaldehyde. In the analysis of time-killing kinetics, gentisaldehyde and phloroglucinaldehyde displayed bactericidal activity while trans -cinnamaldehyde and carvacrol exhibited bacteriostatic effects. At 1× minimal inhibition concentrations, these compounds significantly reduced biofilm formation in all three RGM species to levels between 2.9% and 20.5% relative to controls. Checkerboard assays indicated synergistic interactions between these four compounds and antibiotics such as amikacin, clarithromycin, and linezolid. Of these 12 compound-antibiotic combinations, the pairs of carvacrol-linezolid, carvacrol-amikacin, and gentisaldehyde-clarithromycin demonstrated the most synergy against multiple RGM strains. Moreover, two other compounds citral and geraniol showed synergism with all three test antibiotics. Time-killing assays further confirmed most of synergistic combinations identified in the checkerboard tests. Our research suggests the potential of these essential oils and phenolic aldehydes, both individually and in combination with antibiotics, in treating RGM infections. In addition, this work illuminates applications of these natural compounds in environmental remediation to mitigate bacterial persistence for the control of infectious diseases.

IMPORTANCE:

The emergence of antimicrobial resistance within rapidly growing mycobacteria (RGM) poses a significant threat to public health. This study investigates the potential of naturally occurring compounds to combat infections caused by antibiotic-resistant RGM including M. abscessus , M. fortuitum , and M. chelonae . We identified four specific natural compounds showing impressive inhibitory effects against antibiotic-resistant clinical strains. These compounds not only inhibited the growth and biofilm formation but also exhibited synergistic interactions with antibiotics against key RGM pathogens. Our findings highlight the alternative treatment strategies for RGM infections and potential environmental applications of these natural compounds in mitigating microbial persistence and controlling infectious diseases.

01 Aug 2024·ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY

Successful subcutaneous desensitization to sargramostim in a pediatric patient

Article

Author: Retif, Hope M ; Wisner, Elizabeth L ; Abreo, Andrew ; Qayum, Sarah ; LeBlanc, Zachary

News (Medical) associated with Sargramostim

01 Jun 2024

·www.globenewswire.com

Y-mAbs Announces New Interim Analysis of Phase 2 Data for Naxitamab at 2024 ASCO Annual Meeting

NEW YORK, June 01, 2024 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel radioimmunotherapy and antibody-based therapeutic products for the treatment of cancer, today announced new interim data from the Phase 2 Trial 201 of naxitamab in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”) in patients with relapsed or refractory high-risk neuroblastoma with residual disease limited to bone and/or bone marrow. The results are summarized in poster presentations scheduled to be presented today, June 1, 2024, at the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting in Chicago, IL. Improved patient outcomes with extended naxitamab therapy: A poster titled “Patterns of improvement following initial response in patients treated with naxitamab for relapsed/refractory high-risk neuroblastoma” (poster #10033) demonstrates that continued treatment with naxitamab can further reduce disease following the initial response to naxitamab therapy. The Trial 201 prespecified interim analysis achieved a 50% overall response rate (“ORR”) in 26 out of 52 patients. Nearly a quarter of patients achieved their first complete response (“CR”) or partial response (“PR”) after three or more cycles of naxitamab therapy. Among these patients, most had initial stable disease within specific bone or bone marrow compartments prior to achieving a CR or PR. These results support the rationale for extended naxitamab therapy in patients who do not achieve a CR or PR at first assessment. Evolution of adverse events over the course of treatment: A poster titled “Naxitamab-related adverse events within and across treatment cycles in patients with relapsed/refractory high-risk neuroblastoma” (poster #10032) demonstrates the frequency and patterns of adverse events (“AEs”) based on the interim analysis of Trial 201. In this analysis, 81% of naxitamab-related AEs were Grade 1 or 2, while Grade ≥3 AEs reported in ≥10% of patients included hypotension (60% of patients), pain (58%), urticaria (19%), bronchospasm (18%), and abdominal pain (16%). None resulted in treatment discontinuation. Notably, the frequency of naxitamab-related pain Grade 3 AEs decreased from the first cycle (53%) to the second (37%), generally stabilizing thereafter, with frequencies consistent across infusions. Similarly, the frequency of hypotension showed marked reductions across cycles and infusions. “We are encouraged to see that some patients who did not demonstrate an objective response after initial assessment went on to achieve a complete or partial response after continued naxitamab treatment. In addition, these data provide a practical understanding of the temporal patterns of treatment-related adverse events,” said Vignesh Rajah, MBBS, DCH, MRCP (UK), Chief Medical Officer. In addition, Dr. Jaume Mora and researchers from the Sant Joan de Déu Barcelona Children’s Hospital in Spain will present results from an independent retrospective analysis of patients treated with naxitamab in combination with chemotherapeutics in a poster titled “Naxitamab chemo-immunotherapy regimens other than with irinotecan/temozolomide for patients with relapsed/refractory high-risk neuroblastoma” (poster #10037). In this study, 29 patients with mostly chemo-immunotherapy refractory disease received compassionate use of naxitamab plus sargramostim in combination with alternative chemotherapeutics. Importantly, prior sensitivity to chemo-immunotherapy predicted better responses with the new combinations. The safety profiles of the alternative naxitamab chemo-immunotherapy regimens were considered manageable. Y-mAbs will be available for comment at booth #35151 on the Exhibition Floor of McCormick Place. Researchers at Memorial Sloan Kettering Cancer Center (“MSK”) developed DANYELZA® (naxitamab-gqgk), which is exclusively licensed by MSK to Y-mAbs. MSK has institutional financial interests in the compound and Y-mAbs. About DANYELZA® (naxitamab-gqgk)DANYELZA® (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor ("GM-CSF"), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA® includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information for complete Boxed Warning and other important safety information. About Y-mAbs Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, radioimmunotherapy and antibody-based therapeutic cancer products. The Company’s technologies include its investigational Self-Assembly DisAssembly (“SADA”) Pretargeted Radioimmunotherapy Platform (“PRIT”) and bispecific antibodies generated using the Y-BiClone platform. The Company’s broad and advanced product pipeline includes the anti-GD2 therapy DANYELZA® (naxitamab-gqgk), the first FDA-approved treatment for patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow after a partial response, minor response, or stable disease to prior therapy. Forward-Looking StatementsStatements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements include, but are not limited to, statements about our business model, including financial outlook for 2023 and beyond, including estimated operating expenses, cash burn and DANYELZA product revenue and sufficiency of cash resources and related assumptions; implied and express statements regarding the future of the Company’s business, including with respect to expansion and its goals; the Company’s plans and strategies, development, commercialization and product distribution plans, including potential partnerships; expectations with respect to the Company’s products and product candidates, including potential territory and label expansion of DANYELZA and the potential market opportunity related thereto and potential benefits thereof, and the potential of the SADA Technology and potential benefits and applications thereof; statements with respect to DANYELZA as a growing commercial product and SADA as a differentiated radioimmunotherapy platform positioning the Company on a path to potentially transform the treatment paradigm for a variety of cancers and improve patients’ lives; expectations relating to key anticipated development milestones, including potential expansion of international commercialization efforts with respect to DANYELZA development efforts and the SADA Technology, including potential indications and applications, and the timing thereof; expectations with respect to current and future clinical and pre-clinical studies and the Company’s research and development programs, including with respect to timing and results; expectations related to the timing of the initiation and completion of regulatory submissions; additional product candidates and technologies; expectations regarding collaborations or strategic partnerships and the potential benefits thereof; expectations related to the use of cash and cash equivalents, and the need for, timing and amount of any future financing transaction; expectations with respect to the Company’s future financial performance; and other statements that are not historical facts. Words such as ‘‘anticipate,’’ ‘‘believe,’’ “contemplate,” ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ “hope,” ‘‘intend,’’ ‘‘may,’’ ‘‘might,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘should,’’ ‘‘target,’’ “will”, ‘‘would’’, “guidance,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Our product candidates and related technologies are novel approaches to cancer treatment that present significant challenges. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors, including but not limited to: risks associated with the Company’s financial condition and need for additional capital; the risks that actual results of the Company’s restructuring plan and revised business plan will not be as expected; risks associated with the Company’s development work; cost and success of the Company’s product development activities and clinical trials; the risks of delay in the timing of the Company’s regulatory submissions or failure to receive approval of its drug candidates; the risks related to commercializing any approved pharmaceutical product including the rate and degree of market acceptance of product candidates; development of sales and marketing capabilities and risks associated with failure to obtain sufficient reimbursement for products; the risks related to the Company’s dependence on third parties including for conduct of clinical testing and product manufacture; the Company’s inability to enter into partnerships; the risks related to government regulation; risks related to market approval, risks associated with protection of the Company’s intellectual property rights; risks related to employee matters and managing growth; risks related to the Company’s common stock, risks associated with macroeconomic conditions, including the conflict between Russia and Ukraine and sanctions related thereto, the state of war between Israel and Hamas and the related risk of a larger regional conflict, inflation, increased interest rates, uncertain global credit and capital markets and disruptions in banking systems; and other risks and uncertainties affecting the Company including those described in the "Risk Factors" section included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 and future filings and reports by the Company. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. DANYELZA®, and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc. Investor Contact:Courtney DuganVP, Head of Investor Relationscdu@ymabs.com

Clinical ResultImmunotherapyASCOPhase 2Drug Approval

18 Apr 2024

·www.prnewswire.com

Comprehensive Review Highlights Potential Benefits of Leukine® in Management of Gastrointestinal (GI) Adverse Events Associated with Immune Checkpoint Inhibitors (ICIs) in the Treatment of Cancer

NCI sponsored, ECOG-ACRIN Phase 3 Study of Leukine in Combination with Ipilimumab and Nivolumab in Front Line Treatment of Melanoma Heads to Last Stages of Enrollment LEXINGTON, Mass., April 18, 2024 /PRNewswire/ -- Partner Therapeutics, Inc. (PTx) announced publication of a comprehensive review by Dougan et al.1 in Cancers summarizing the mechanistic rationale and pre-clinical and clinical data regarding the use of Leukine (sargramostim; glycosylated, yeast-derived recombinant human granulocyte-macrophage colony-stimulating factor [rhu GM-CSF]) to manage gastrointestinal (GI) immune-related adverse events (irAEs) for patients taking immune checkpoint inhibitors (ICIs) for the treatment of cancer. Leukine is not FDA approved for treatment of irAEs or melanoma. Continue Reading Addition of sargramostim (recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF)) to ipilimumab significantly lowered grade 3-5 adverse events compared to the ipilimumab-alone arm in a multi-center, randomized clinical trial for advanced melanoma. (Figure and caption from Dougan 20241 illustrating results from Hodi 20142) The authors describe the mechanisms underlying GI irAEs and their similarities to inflammatory bowel disease and review the available clinical data supporting a potential role for Leukine in both settings. In the prospective, randomized study E1608, Hodi and colleagues2 observed that advanced melanoma patients who received Leukine plus ipilimumab had a reduction in GI irAEs (p=0.05) (see figure below) as well as a significant improvement in overall survival (p=0.01) compared to those who received ipilimumab alone. "Immune checkpoint inhibitors have meaningfully improved prognoses for cancer patients in a number of cancers," said Michael Dougan MD, PhD, Associate Professor of Medicine at Massachusetts General Hospital. "Unfortunately, these drugs also can cause the immune system to attack healthy cells creating unwanted GI side effects including some that lead to discontinuation of treatment and more severe outcomes. The potential Leukine has shown in mitigating GI irAEs and improving patient outcomes, including survival, are encouraging." Ongoing Phase 3 Study of Leukine with Ipilimumab and Nivolumab in Front Line Metastatic Melanoma Leukine continues to be studied in combination with ICIs to assess differences in the rate of serious adverse events as well as its impact on overall survival. EA6141 (NCT02339571) is a randomized, controlled phase 2/3 study of Leukine in combination with ipilimumab and nivolumab for the front line treatment of patients with unresectable stage III or stage IV melanoma that is sponsored by the National Cancer Institute (NCI) and being conducted by ECOG-ACRIN Cancer Research Group.3 It is led by Study Chair F. Stephen Hodi Jr., MD, Director of the Center for Immuno-Oncology at Dana-Farber Cancer Institute, and Study Co-Chair Ahmad A. Tarhini, MD, PhD, Professor of Oncologic Sciences and Director of Cutaneous and Clinical Translational Research at H. Lee Moffitt Cancer Center and Research Institute. Both are members of the ECOG-ACRIN Melanoma Committee. "The phase 2 portion of study EA6141 was completed previously, and after a successful planned interim analysis, advanced to the phase 3 portion, which is now in the last stages of enrollment," said Dr. Hodi in a recent statement. "The primary objective is to compare the overall survival rates of patients who receive nivolumab/ipilimumab/rhu GM-CSF versus those who receive only nivolumab/ipilimumab." REFERENCES Dougan M, Nguyen LH, Buchbinder EI, Lazarus HM. Sargramostim for Prophylactic Management of Gastrointestinal Immune-Related Adverse Events of Immune Checkpoint Inhibitor Therapy for Cancer. Cancers. 2024; 16(3):501. doi.org/10.3390/cancers16030501 Hodi FS, Lee S, McDermott DF, et al. Ipilimumab Plus Sargramostim vs Ipilimumab Alone for Treatment of Metastatic Melanoma: A Randomized Clinical Trial. JAMA. 2014; 312(17):1744-1753. doi:10.1001/jama.2014.13943 National Cancer Institute. A Phase II/III Trial of Nivolumab, ipilimumab, and GM-CSF in patients with advanced melanoma. Available at: . NLM identifier: NCT02339571. Accessed April 16, 2024. ABOUT MELANOMA Melanoma is the most aggressive form of skin cancer and rates of melanoma have been rising for the past 30 years. The American Cancer Society estimates 96,480 new melanoma cases will be diagnosed in the US and 7,230 people will die from the disease in 2019. The FDA grants orphan drug designation to promote the development of promising treatments for conditions that affect 200,000 or fewer U.S. patients annually. ABOUT LEUKINE LEUKINE (sargramostim) is a glycosylated recombinant human granulocyte-macrophage colony-stimulating factor (rhu GM-CSF) produced by recombinant DNA technology in yeast. The product is commercially available in the United States and accessible through a named patient program operated by Tanner Pharma Group outside of the United States. LEUKINE is indicated: To shorten time to neutrophil recovery and to reduce the incidence of severe and life-threatening infections and infections resulting in death following induction chemotherapy in adult patients 55 years and older with acute myeloid leukemia (AML). For the mobilization of hematopoietic progenitor cells into peripheral blood for collection by leukapheresis and autologous transplantation in adult patients. For the acceleration of myeloid reconstitution following autologous bone marrow or peripheral blood progenitor cell transplantation in adult and pediatric patients 2 years of age and older. For the acceleration of myeloid reconstitution following allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older. For treatment of delayed neutrophil recovery or graft failure after autologous or allogeneic bone marrow transplantation in adult and pediatric patients 2 years of age and older. To increase survival in adult and pediatric patients from birth to 17 years of age acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [H-ARS]). Important Safety Information for Leukine (sargramostim) Contraindications Do not administer LEUKINE to patients with a history of serious allergic reaction, including anaphylaxis, to human granulocyte-macrophage colony-stimulating factor, sargramostim, yeast-derived products, or any other component of LEUKINE. Warnings and Precautions Serious hypersensitivity reactions, including anaphylactic reactions, have been reported with LEUKINE. If a serious allergic or anaphylactic reaction occurs, immediately discontinue LEUKINE therapy, and institute medical management. Discontinue LEUKINE permanently for patients with serious allergic reactions. LEUKINE can cause infusion-related reactions that may be characterized by respiratory distress, hypoxia, flushing, hypotension, syncope, and/or tachycardia. Observe closely during infusion, particularly in patients with preexisting lung disease; dose adjustment or discontinuation may be needed. LEUKINE should not be administered simultaneously with or within 24 hours preceding cytotoxic chemotherapy or radiotherapy or within 24 hours following chemotherapy. Edema, capillary leak syndrome, and pleural or pericardial effusions have been reported in patients after LEUKINE administration. LEUKINE should be used with caution in patients with preexisting fluid retention, pulmonary infiltrates, or congestive heart failure. Such patients should be monitored. Supraventricular arrhythmia has been reported in uncontrolled studies during LEUKINE administration, particularly in patients with a history of cardiac arrhythmia. Use LEUKINE with caution in patients with preexisting cardiac disease. If absolute neutrophil count (ANC) > 20,000 cells/mm3 or if white blood cell (WBC) counts > 50,000/mm3, LEUKINE administration should be interrupted, or the dose reduced by half. Monitor complete blood counts (CBC) with differential twice per week. Discontinue LEUKINE therapy if tumor progression, particularly in myeloid malignancies, is detected during LEUKINE treatment. Treatment with LEUKINE may induce neutralizing anti-drug antibodies. Use LEUKINE for the shortest duration needed. Avoid administration of solutions containing benzyl alcohol (including LEUKINE for injection reconstituted with Bacteriostatic Water for Injection, USP [0.9 % benzyl alcohol]) to neonates and low birth weight infants. Drug Interactions Avoid the concomitant use of LEUKINE and products that induce myeloproliferation. Monitor for clinical and laboratory signs of excess myeloproliferative effects. Adverse Reactions Adverse events occurring in >10% of patients receiving LEUKINE in controlled clinical trials and reported at a higher frequency than in placebo patients are: In recipients of autologous bone marrow transplantation (BMT)–asthenia, malaise, diarrhea, rash, peripheral edema, urinary tract disorder In recipients of allogeneic BMT–abdominal pain, chills, chest pain, diarrhea, nausea, vomiting, hematemesis, dysphagia, GI hemorrhage, pruritus, bone pain, arthralgia, eye hemorrhage, hypertension, tachycardia, bilirubinemia, hyperglycemia, increased creatinine, hypomagnesemia, edema, pharyngitis, epistaxis, dyspnea, insomnia, anxiety, high glucose, low albumin In patients with AML–fever, weight loss, nausea, vomiting, anorexia, skin reactions, metabolic laboratory abnormalities, edema ABOUT PARTNER THERAPEUTICS Partner Therapeutics, Inc. (PTx), an integrated biotechnology company, focuses on development and commercialization of late-stage therapeutics to improve health outcomes in treatment of cancer and other serious diseases. The company believes in delivering products and supporting medical teams with the purpose of achieving superior outcomes for patients and their families. Visit . SOURCE Partner Therapeutics, Inc.

ImmunotherapyClinical ResultPhase 3Phase 2Orphan Drug

15 Apr 2024

·www.prnewswire.com

Norgine B.V. submits Marketing Authorisation Application via Project Orbis for eflornithine (difluoromethylornithine [DFMO]) in high-risk neuroblastoma

AMSTERDAM, April 15, 2024 /PRNewswire/ -- Norgine B.V. today announced its first marketing authorisation application submissions on 10 April 2024, seeking approval for eflornithine in high-risk neuroblastoma (HRNB), via Project Orbis in Australia, Switzerland and the United Kingdom. This milestone supports Norgine's efforts to deliver patient access to eflornithine and bring a further treatment option in the field of paediatric oncology. Norgine and USWM, LLC (dba US WorldMeds), a Kentucky-based specialty pharmaceutical company, have an exclusive licensing agreement by which Norgine will register and commercialise eflornithine, also referred to as DFMO, in Europe, Australia and New Zealand. On 13 December 2023, the US Food and Drug Administration (FDA) approved eflornithine as the first oral maintenance therapy for high-risk neuroblastoma (HRNB), indicated to reduce the risk of relapse in adult and paediatric patients who have received certain prior therapies.1 The approval decision was based on findings from a trial comparing outcomes from Study 3b (NCT02395666; investigational arm)2,4 and Study ANBL0032 (NCT00026312; clinical-trial-derived external control arm)3,4, where use of eflornithine resulted in improved event-free survival and overall survival when compared to outcomes for patients with high-risk neuroblastoma treated with the standard of care (SoC) without the drug.1 Dr David Gillen, Chief Medical Officer at Norgine, added, "These submissions via Project Orbis represent an important first step in the regulatory process for eflornithine and re-emphasise Norgine's passion and commitment in attempting to secure additional treatment options for patients living with HRNB, a condition with a high level of unmet medical need." Follow @norgine Notes to Editors: Project Orbis Project Orbis is an initiative (since May 2019) of the US FDA Oncology Center of Excellence (OCE) and provides a framework for concurrent submission and collaborative review of innovative oncology products among international regulatory authorities. It was created with the overarching goal to speed worldwide patient access to innovative cancer therapies. Project Orbis is coordinated by the FDA, and its partners include United Kingdom Medicines and Healthcare Products regulatory Agency (UK MHRA), Australia Therapeutic Goods Administration (TGA), Canada (Health Canada), Singapore (Health Sciences Authority (HSA), Switzerland (Swissmedic), Brazil (Agência Nacional de Vigilância Sanitária (ANVISA), Israel (Ministry of Health). HRNB background Children diagnosed with HRNB undergo an intense SoC regimen that still leaves them vulnerable to relapse and death, a risk that is particularly acute during the first two years.5 Approximately 30% of patients who attain remission following upfront therapy will relapse, resulting in a poor prognosis and low likelihood of long-term survival (e.g. estimates as low as 15% of patients will live for five years after relapsing).6 Avoiding relapse is key to long-term survival, yet outside of the United States there are no approved therapies for sustaining remission following SoC treatment. The data with eflornithine demonstrate using it as maintenance therapy extends remission and reduces risk of relapse in patients with HRNB. About Norgine Norgine is a leading European specialist pharmaceutical company that has been bringing transformative medicines to patients for over a century. Our commitment to transforming people's lives drives everything we do, and our European experience, fully integrated infrastructure and exceptional partnership approach enable us to quickly apply creative solutions to bring life-changing medicines to patients that they may not otherwise be able to access. Norgine is proud to have helped more than 25 million patients around the world in 2022 and generated €530 million in net product sales, a growth of nearly 5% over 2021. Norgine has a direct presence in 16 European countries, as well as Australia and New Zealand. We also have a strong global network of partnerships in non-Norgine markets. We are a flexible and fully integrated pharmaceutical business, with manufacturing (Hengoed, Wales and Dreux, France), third party supply networks and significant product development capabilities, in addition to our sales and marketing infrastructure. This enables us to acquire, develop and commercialise specialist and innovative products that make a real difference to the lives of patients around the world. NORGINE and the sail logo are trademarks of the Norgine group of companies. References FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma. FDA. News release. 13 December 2023. Available from: FDA approves eflornithine for adult and pediatric patients with high-risk neuroblastoma | FDA Clinical Trial NCT02395666; Preventative trial of difluoromethylornithine (DFMO) in high risk patients with neuroblastoma that is in remission. Available from: Study Details | Preventative Trial of Difluoromethylornithine (DFMO) in High Risk Patients With Neuroblastoma That is in Remission | ClinicalTrials.gov. Clinical Trial NCT00026312; Isotretinoin with or without dinutuximab, aldesleukin, and sargramostim following stem cell transplant in treating patients with neuroblastoma. Available from: Study Details | Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma | ClinicalTrials.gov Oesterheld J, Ferguson W, Kraveka JM, Bergendahl G, Clinch T, Lorenzi E et al. Eflornithine as Postimmunotherapy Maintenance in High-Risk Neuroblastoma: Externally Controlled, Propensity Score-Matched Survival Outcome Comparisons. 2024; 42 (1): 90-102. Basta NO, Halliday GC, Makin G, Birch J, Feltbower R, Bown N, et al. Factors associated with recurrence and survival length following relapse in patients with neuroblastoma. Br J Cancer. 2016;115:1048-57. London WB, Bagatell R, Weigel BJ, Fox E, Guo D, Van Ryn C, et al. Historical time to disease progression and progression-free survival in patients with recurrent/refractory neuroblastoma treated in the modern era on Children's Oncology Group early-phase trials. Cancer. 2017;123:4914-23. Logo -

License out/inDrug ApprovalClinical ResultImmunotherapy

100 Deals associated with Sargramostim

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KEGG	Wiki	ATC	Drug Bank
D05803	Sargramostim	L03AA09	DB00020

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Pulmonary Alveolar Proteinosis, Acquired	JP	Nobelpharma Co., Ltd.	26 Mar 2024
Acute Radiation Syndrome	US	Berlex Laboratories, Inc.	29 Mar 2018
Hematopoietic stem cell transplantation	US	Berlex Laboratories, Inc.	05 Mar 1991
Myeloid Leukemia	US	Berlex Laboratories, Inc.	05 Mar 1991
Primary Graft Dysfunction	US	Berlex Laboratories, Inc.	05 Mar 1991

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
COVID-19	Phase 3	JP	Nobelpharma Co., Ltd.	17 Dec 2020
stage IIB melanoma	Phase 3	US	Genzyme Corp.	05 Aug 2011
Bladder Cancer	Phase 3	US	Bayer AG	01 Nov 2005
Metastatic urothelial carcinoma	Phase 3	US	Bayer AG	01 Nov 2005
Transitional Cell Carcinoma	Phase 3	US	Bayer AG	01 Nov 2005
Melanoma	Phase 3	US	Bayer AG	21 Sep 2005
Crohn Disease	Phase 3	US	Berlex Pharmaceuticals, Inc.	30 Jun 2004
Sepsis	Phase 3	US	Immunex Corp.	22 Oct 2001
Acute Myeloid Leukemia	Phase 2	-	Sanofi	01 Feb 2017
Alzheimer Disease	Phase 2	US	Sanofi	01 Nov 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04013672 (CTgov)	Phase 2	Recurrent Glioblastoma	41	Montanide ISA 51+Sargramostim+pembrolizumab (Arm A - Have Not Received Immunotherapy)	bdyvehcwot(vqwoihrypf) = hsubqjtyxf jvkkxntlba (hjssgeiijv, tgkgngjupq - cerlmpkwek) View more	-	28 Aug 2024
				Montanide ISA 51+Sargramostim+pembrolizumab (Arm B - Have Failed Prior Anti-PD1 Therapy)	bcmcmuvhfn(dfaxtpadpl) = zcjkobytmd hwryaoymml (lhaztslnbn, usujwyrzbg - nuavrekqgl) View more
NCT04642950 (CTgov)	Phase 2/3	COVID-19	70	Sargramostim (NPC-26)	fnzhakenqq(cojffphjqe) = emnagxcqvj dpeytwntxe (waogodsjzt, qoaymzmiau - fsfyjuhsgq) View more	-	19 Jul 2024
				Placebo (NP-26 Placebo)	fnzhakenqq(cojffphjqe) = vadqeqhfte dpeytwntxe (waogodsjzt, nqowjzqnkk - eidwdproaz) View more
NCT00630565 (CTgov)	Phase 2/3	Acute Myeloid Leukemia	12	total-body irradiation+etoposide+CYCLOPHOSPHAMIDE+sargramostim+Dexamethasone	iiiyuqfcdd(lttmqkkrsh) = gcewldjvzp ipmwionqsx (nglxwosifa, nowhwifczo - kvgwhjamfj) View more	-	29 Jan 2024
Literature Manual	Phase 2	COVID-19 FCGR2B	600	Sargramostim 250 mcg	azzdrdjlkz(ixxiqdzwjq) = lklcfoeite ikcuydomqp (yddexkgifd )	Positive	27 Nov 2023
				placebo	azzdrdjlkz(ixxiqdzwjq) = lbzxbdbafp ikcuydomqp (yddexkgifd )
NCT04707664 (SCOPE, CTgov)	Phase 2	COVID-19	600	Sargramostim (Sargramostim Arm)	vdkspkugew(vzzinpfbud) = kulrwkrwcz wnjhhcwcjf (owwsfsvcsi, wfhgpknnqk - cqdkruixzk) View more	-	06 Feb 2023
				Placebo (Placebo Arm)	vdkspkugew(vzzinpfbud) = haffeayjdg wnjhhcwcjf (owwsfsvcsi, vucxoxpxfa - ydvzpjtsoe) View more
NCT02593123 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Recurrent Multiple Myeloma \| Acute Lymphoblastic Leukemia ... View more	31	Sargramostim+mycophenolate mofetil (Arm I (MMF-15, Sargramostim))	phibiosyme(smzixrjyvh) = ddatjxmqcr iuvgwxjwyx (rewffliiwv, ukvpseptuk - daewedmmqa) View more	-	23 Jan 2023
				Filgrastim+mycophenolate mofetil (Arm II (MMF-30, Filgrastim))	phibiosyme(smzixrjyvh) = oalkhtslbb iuvgwxjwyx (rewffliiwv, awoypjdmev - ericmrpaax) View more
iLeukPulm (Pubmed)	Phase 2	COVID-19	122	Inhaled Sargramostim	nftwaiuykp(vaqojzvsfb) = tznhxeeyra cbvdzfzggf (vrdwsopypc ) View more	-	02 Dec 2022
				Sargramostim	nftwaiuykp(vaqojzvsfb) = hvlxbszbia cbvdzfzggf (vrdwsopypc ) View more
NCT04326920 (SARPAC, CTgov)	Phase 4	Hypoxic respiratory failure \| COVID-19	87	Sargramostim (Active Sargramostim Treatment Group)	oyxudzhicu(mgqhjsyhak) = dznoqdvkxe bgnxwyaxdn (kovxdtylem, dozctqmxhf - wvsdmongkr) View more	-	04 Nov 2022
				Control+Sargramostim (Control Group)	oyxudzhicu(mgqhjsyhak) = vlphgwkmbz bgnxwyaxdn (kovxdtylem, efjrsaemni - zydaymfeyg) View more
NCT04411680 (iLeukPulm, CTgov)	Phase 2	COVID-19	123	Standard of care+Sargramostim (Sargramostim Arm)	nkgkiplryg(bodwejaylz) = lhveojzyga dxfqdiukyi (clzwbsoctt, qhbcghbnav - ibgcuaiwrz) View more	-	18 Feb 2022
				Standard of care (Control Arm)	nkgkiplryg(bodwejaylz) = tlbhyvkupn dxfqdiukyi (clzwbsoctt, yiozvyrgqv - wfdqtocanp) View more
NCT01700673 (CTgov)	Phase 2	Acute Myeloid Leukemia \| Myelodysplastic Syndromes	25	Sargramostim+Azacitidine (Non-myeloablative BMT)	wrzdglnpti(dukzazxtkc) = rrrphravkw exblyznnga (crionkobeh, kylpuyyilo - nishoephnk) View more	-	02 Sep 2021
				Sargramostim+Azacitidine (Myeloablative BMT)	wrzdglnpti(dukzazxtkc) = xxvbwieakv exblyznnga (crionkobeh, uvlgbzmmdg - sinlohtcmq) View more

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