Neuroblastoma - Pipeline Insight, 2025

This “Neuroblastoma - Pipeline Insight, 2025” report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Neuroblastoma pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Neuroblastoma: Understanding

Neuroblastoma: Overview

Neuroblastoma is a rare type of cancer that mostly affects babies and young children. It develops from specialised nerve cells (neuroblasts) left behind from a baby's development in the womb. Neuroblastoma most commonly occurs in 1 of the adrenal glands situated above the kidneys, or in the nerve tissue that runs alongside the spinal cord in the neck, chest, tummy or pelvis. It can spread to other organs, such as the bone marrow, bone, lymph nodes, liver and skin. Neuroblastoma is a type of cancer that primarily affects young children and arises from immature nerve cells. Common signs and symptoms include a lump or mass in the abdomen, chest, or neck, often accompanied by pain or swelling. Other symptoms can include fatigue, loss of appetite, weight loss, and fever. In advanced stages, neuroblastoma may cause bone pain, difficulty breathing, and changes in bowel or bladder habits. Some children may also exhibit unusual eye movements or dark circles under the eyes. Symptoms often depend on the tumor's size and location.

Neuroblastoma originates from neural crest cells, which are precursors to the sympathetic nervous system, typically affecting the adrenal glands or nerve tissue along the spine. Pathologically, these tumors are composed of small, round, blue cells with varying degrees of differentiation. They can range from well-differentiated, more benign ganglioneuromas to poorly differentiated, aggressive neuroblastomas. The tumor often shows areas of necrosis and calcification. Genetic mutations, including amplifications of the MYCN oncogene, are associated with more aggressive forms of the disease, while chromosomal aberrations, such as loss of heterozygosity, also play a role in its development and progression.

The diagnosis of neuroblastoma typically involves a combination of clinical evaluation, imaging studies, and laboratory tests. Common imaging techniques include ultrasound, MRI, and CT scans to detect the location and size of the tumor, while a metaiodobenzylguanidine (MIBG) scan helps identify the spread to other areas of the body. Blood and urine tests are conducted to detect elevated levels of catecholamines, which are often produced by neuroblastoma cells. Treatment for neuroblastoma depends on the risk category of the disease, which ranges from low to high risk. Low-risk cases may be treated successfully with surgery alone to remove the tumor, while intermediate-risk cases often require a combination of surgery and chemotherapy to shrink the tumor before removal. High-risk neuroblastoma typically involves more intensive treatment, including high-dose chemotherapy, surgery, radiation therapy, stem cell transplantation, and immunotherapy, such as the use of anti-GD2 antibodies. In some cases, targeted therapy, like ALK inhibitors for tumors with ALK mutations, may be used. Treatment strategies aim to control the tumor locally, prevent recurrence, and address metastasis.

'Neuroblastoma- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Neuroblastoma pipeline landscape is provided which includes the disease overview and Neuroblastoma treatment guidelines. The assessment part of the report embraces, in depth Neuroblastoma commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Neuroblastoma collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Neuroblastoma R&D. The therapies under development are focused on novel approaches to treat/improve Neuroblastoma.

Neuroblastoma Emerging Drugs Chapters

This segment of the Neuroblastoma report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, Phase II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Neuroblastoma Emerging Drugs

Hu14.18: Essential Pharma

Hu14.18K322A, commonly referred to as Hu14.18, is a humanized anti-GD2 monoclonal antibody developed by Renaissance Pharma for the treatment of high-risk neuroblastoma (HRNB). In April 2025, Essential Pharma announced that it had completed the acquisition of the Renaissance Pharma Ltd. with its lead asset being Hu14.18K322A (Hu14.18). Currently the drug is in Phase II stage of its clinical trial for the treatment of neuroblastoma.

67-Cu-SARTATE: Clarity Pharmaceuticals

67Cu-SARTATE is an emerging theranostic radiopharmaceutical specifically designed for the diagnosis and treatment of high-risk neuroblastoma, a particularly aggressive childhood cancer. This compound utilizes copper-67 (67Cu) as a therapeutic agent, complementing its diagnostic counterpart, copper-64 (64Cu), which is used for imaging. The drug has been granted Rare Pediatric Disease Designation and Orphan Drug Designation for the clinical management of neuroblastoma. Currently the drug is in Phase I/II stage of its clinical trial for the treatment of neuroblastoma.

Alrizomadlin (APG-115): Ascentage Pharma

APG-115 is a potent, orally bioavailable MDM2 inhibitor that binds to human recombinant MDM2 protein with high affinity. Mechanistically, APG-115 interferes the binding of MDM2 oncoprotein with the tumor suppressor P53 protein, leads to increased P53 and P21 protein expression and trigger P53-mediated apoptosis. In addition to its direct tumor-targeting activity, APG-115 plays an important role in immune modulation through its effects on immune cells, tumor cells and multiple cytokines. Alrizomadlin has been granted Orphan Drug Designations by the U.S. FDA for the treatment of gastric cancer, soft-tissue sarcoma, AML, Retinoblastoma, IIB-IV melanoma and Neuroblastoma. Currently the drug is in Phase I stage of its clinical trial for the treatment of Neuroblastoma.

Neuroblastoma: Therapeutic Assessment

This segment of the report provides insights about the different Neuroblastoma drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Neuroblastoma

• There are approx. 20+ key companies which are developing the therapies for Neuroblastoma. The companies which have their Neuroblastoma drug candidates in the most advanced stage, i.e. Phase II include, Essential Pharma.

Phases

The report covers around 25+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Neuroblastoma pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Neuroblastoma: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Neuroblastoma therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Neuroblastoma drugs.

Neuroblastoma Report Insights

• Neuroblastoma Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Neuroblastoma Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Neuroblastoma drugs?
• How many Neuroblastoma drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Neuroblastoma?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Neuroblastoma therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Neuroblastoma and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Tmunity Therapeutics
• Valent Technologies
• Ohara Pharmaceutical
• Panbela Therapeutics
• Clarity Pharmaceuticals
• Ascentage Pharma

Key Products

• GPC2 CAR T cells
• VAL-413
• Tamibarotene
• Eflornithine oral
• 67-Cu-SARTATE
• Alrizomadlin (APG-115)

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