Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Market: Competitive Landscape, Pipeline, and Market Analysis 2023

Granulocyte-macrophage colony-stimulating factor (GM-CSF), also known as colony-stimulating factor 2 (CSF2), is a monomeric glycoprotein secreted by macrophages, T cells, mast cells, natural killer cells, endothelial cells, and fibroblasts that functions as a cytokine. GM-CSF is a monomeric glycoprotein that functions as a cytokine. Monocytes exit the circulation and migrate into the tissue, whereupon they mature into macrophages and dendritic cells. Thus, it is part of the immune/inflammatory cascade, a process crucial for fighting infection. The pharmaceutical analogs of naturally occurring GM-CSF are sargramostim and molgramostim. The mechanism of action (MOA) of GMCSF analogs is they bind to receptor proteins on the surfaces of hematopoietic stem cells, thereby activating intracellular signaling pathways that can cause the cells to proliferate and differentiate into a specific kind of blood cell, usually white blood cells and stimulates monocytes and macrophages to produce pro-inflammatory cytokines, including CCL17. Elevated GM-CSF has been shown to contribute to inflammation in inflammatory arthritis, osteoarthritis, colitis asthma, obesity, and COVID-19.

GMCSF analogs are used in the treatment of respiratory diseases, adenocarcinoma, gastrointestinal diseases, autoimmune diseases, endocrine gland neoplasm, lung neoplasm, thoracic neoplasms, leukemia, asthma, hematologic diseases, and kidney cancer among others. Increased prevalence of autoimmune disorders and various cancers are the key drivers for the GMCSF market. For instance, according to the World Health Organization 2022, around 1.9 million new cancer cases were diagnosed in the USA. Breast and lung cancers are most prevalent cancers in adults worldwide. The introduction of newer products by market players significantly enhances the revenue of GMCSF market over the forecast years. For instance, Amgen’s Neupogen (Filgrastim) launch for the treatment of chemotherapy-induced neutropenia.  Moreover, to address the difficulties in therapy, numerous market participants are developing innovative compounds. For instance, Kiniksa’s Mavrilimumab (KPL-301) for the indication of severe COVID infection is under the various stages of clinical studies.

Key Market Developments:

• In March 2018, Partner Therapeutics Leukine (Sargramostim) received FDA’s approval for the additional use 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]).
• In December 2019, Savara’s Molgramostim received grant of the FDA as Breakthrough Therapy Designation in an autoimmune pulmonary alveolar proteinosis (aPAP) based on Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial.

Approved Drug Molecules and Brand Names for GMCSF:

• Neupogen (Filgrastim)
• Leukine (Sargramostim)

Drugs under the Pipeline for GMCSF:

• Imlygic (Talimogene Laherparepvec)
• Pexa-Vec (Pexastimogene Devacirepvec)
• GVAX Pancreas (Allogeneic GM-CSF-Secreting Tumor Cells)
• Molgradex (Molgramostim)
• Vigil (Gemogenovatucel-T)
• Otilimab (GSK3196165)
• Namilumab (IZN-101)
• OH2
• Lenzilumab (KB003)
• Oncos-102
• Stingvax (CG8123)
• Cretostimogene Grenadenorepvec (CG0070)
• ASM8
• Orienx010
• Gimsilumab (KIN-1901)
• GVAX Leukemia Vaccine
• KB002
• Plonmarlimab (TJM2)
• Leucomax (Molgramostim)
• MVX-Onco-1
• Prostatac (PSA
• Topleucon (Molgramostim Biosimilar)
• Allogeneic Myeloma GM-CSF Vaccine
• Neustim (Molgramostim Biosimilar)
• Trabikibart (CSL311)  

Clinical Activity and Developments of GMCSF:

As of June 2023, there were about 35 companies with over 37 compounds that focus on various autoimmune and cancer diseases. For these molecules, more than hundreds of clinical trials are being conducted and the majority are in phase-2, phase-3 clinical. For instance,

• In June 2023, Bayer and Partner Therapeutics has conducting a phase 1, open-label study for validating the treatment-induced biomarkers following Sargramostim treatment in Parkinson’s disease.
• In June 2023, Merck has conducted a phase 3, randomized, double-blind study to evaluate Pembrolizumab plus chemotherapy having Filgrastim versus Placebo Plus chemotherapy as neoadjuvant therapy for Triple Negative Breast Cancer (TNBC).