DelveInsight’s 2025 report on the Neuroblastoma Pipeline provides detailed insights into over 20 companies and more than 25 drugs in development. It features profiles of pipeline therapies across clinical and non-clinical stages, evaluations by product category, development phase, administration route, and molecular type. The report also covers inactive projects in this therapeutic area.

Interested in the newest developments in the Neuroblastoma Pipeline? Discover the treatments and studies gaining attention in the Neuroblastoma Pipeline Outlook Report

Key Highlights from the Neuroblastoma Pipeline Report

• On October 31, 2025, Milton S. Hershey Medical Center initiated a study to assess the investigational oral medication AMXT 1501 combined with oral eflornithine (DFMO). This drug is not yet approved by the FDA or other global regulators for standalone or combined use in treating the condition.

• On October 28, 2025, Novartis Pharmaceuticals announced a study to evaluate long-term outcomes in pediatric patients treated with dabrafenib and/or trametinib.

• DelveInsight’s Neuroblastoma Pipeline analysis reveals a vibrant sector with over 20 active organizations developing more than 25 therapies for Neuroblastoma management.

• Leading companies in Neuroblastoma include INSYS Therapeutics Inc., Hoffmann-La Roche, Endeavor Biomedicines Inc., Novartis Pharmaceuticals, Daiichi Sankyo, Celgene, Cancer Research UK, Cardiff Oncology, Elicio Therapeutics, Merck, Eli Lilly and Company, Pfizer, Adaptimmune, and others.

• Promising therapies include Eflornithine, Temozolomide, Irinotecan, DFMO, Racotumomab, Bevacizumab, Temozolomide, Topotecan hydrochloride, and more.

Want to identify which organizations are driving progress in Neuroblastoma? Explore the complete pipeline details in the Neuroblastoma Clinical Trials Assessment

The Neuroblastoma Pipeline Report offers a comprehensive overview of the disease, the current pipeline landscape, and evaluations of major therapies. It also addresses unmet needs in Neuroblastoma care.

Understanding Neuroblastoma

Neuroblastoma is a rare cancer that primarily impacts infants and young children, originating from leftover nerve cells (neuroblasts) from fetal development. It typically arises in the adrenal glands above the kidneys or nerve tissues along the spinal cord in the neck, chest, abdomen, or pelvis. The disease can metastasize to areas like bone marrow, bones, lymph nodes, liver, and skin. As a cancer affecting immature nerve cells, it often presents with abdominal, chest, or neck masses, pain, or swelling, alongside symptoms such as fatigue, reduced appetite, weight loss, and fever.

Profiles of Emerging Neuroblastoma Drugs

• Hu14.18: Essential Pharma
  Hu14.18K322A, often called Hu14.18, is a humanized monoclonal antibody targeting GD2 for high-risk neuroblastoma (HRNB). In April 2025, Essential Pharma finalized the acquisition of Renaissance Pharma Ltd., including this key asset. The therapy is currently in Phase II trials for Neuroblastoma.

• 67-Cu-SARTATE: Clarity Pharmaceuticals
  67Cu-SARTATE is a novel theranostic radiopharmaceutical for diagnosing and treating high-risk neuroblastoma, a severe pediatric cancer. It employs copper-67 (67Cu) for therapy, paired with copper-64 (64Cu) for imaging. It has received Rare Pediatric Disease Designation and Orphan Drug Designation for Neuroblastoma management. The drug is in Phase I/II trials for Neuroblastoma.

• Alrizomadlin (APG-115): Ascentage Pharma
  APG-115 is a potent, orally available MDM2 inhibitor that strongly binds to MDM2 protein. It disrupts MDM2's interaction with the tumor suppressor P53, boosting P53 and P21 levels to induce apoptosis. Beyond direct anti-tumor effects, it modulates immunity via impacts on immune cells, tumor cells, and cytokines. It holds Orphan Drug Designations from the U.S. FDA for gastric cancer, soft-tissue sarcoma, AML, retinoblastoma, stage IIB-IV melanoma, and Neuroblastoma. The drug is in Phase I trials for Neuroblastoma.

If you're monitoring active Neuroblastoma Clinical Trials, this update is essential. Learn about the innovations in Neuroblastoma Treatment Drugs

The Neuroblastoma Pipeline report provides insights into:

• Detailed profiles of companies developing Neuroblastoma therapies, including the total treatments per company.

• Categorization of therapeutic candidates into early, mid, and late development stages for Neuroblastoma treatment.

• Companies engaged in targeted drug development, with information on active and inactive (dormant or discontinued) projects.

• Drugs in progress, analyzed by stage, administration route, target receptor, monotherapy or combination, mechanism of action, and molecular type.

• Thorough examination of partnerships (company-to-company and company-academia), licensing arrangements, and funding to advance the Neuroblastoma market.

Neuroblastoma Companies

Key players include INSYS Therapeutics Inc., Hoffmann-La Roche, Endeavor Biomedicines Inc., Novartis Pharmaceuticals, Daiichi Sankyo, Celgene, Cancer Research UK, Cardiff Oncology, Elicio Therapeutics, Merck, Eli Lilly and Company, Pfizer, Adaptimmune, and others.

Neuroblastoma Therapeutic Assessment by Route of Administration

The report evaluates pipeline drugs based on delivery methods, grouped into categories such as:

• Oral

• Intravenous

• Subcutaneous

• Parenteral

• Topical

Neuroblastoma Products by Molecule Type

Products are classified by molecular structures, including:

• Recombinant fusion proteins

• Small molecules

• Monoclonal antibodies

• Peptides

• Polymers

• Gene therapies

From promising new candidates to market intelligence, the Neuroblastoma Pipeline Report covers everything—review it at Neuroblastoma Market Drivers and Barriers, and Future Perspectives

Scope of the Neuroblastoma Pipeline Report

• Coverage: Global

• Neuroblastoma Companies: INSYS Therapeutics Inc., Hoffmann-La Roche, Endeavor Biomedicines Inc., Novartis Pharmaceuticals, Daiichi Sankyo, Celgene, Cancer Research UK, Cardiff Oncology, Elicio Therapeutics, Merck, Eli Lilly and Company, Pfizer, Adaptimmune, and others.

• Neuroblastoma Therapies: Eflornithine, Temozolomide, Irinotecan, DFMO, Racotumomab, Bevacizumab, Temozolomide, Topotecan hydrochloride, and others.

• Therapeutic Assessment by Product Type: Monotherapy, Combination Therapy, or Both.

• Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III.

Stay informed in healthcare research—learn about future trends in the Neuroblastoma Treatment landscape through this comprehensive analysis at Neuroblastoma Emerging Drugs and Major Players

Table of Contents

* Introduction

* Executive Summary

* Neuroblastoma: Overview

* Neuroblastoma Pipeline Therapeutics

* Neuroblastoma Therapeutic Assessment

* Neuroblastoma- DelveInsight's Analytical Perspective

* Late Stage Products (Phase III)

* Drug name: Company name

* Drug profiles in the detailed report.....

* Mid Stage Products (Phase II)

* Hu14.18: Essential Pharma

* Drug profiles in the detailed report.....

* Early Stage Products (Phase I)

* Alrizomadlin (APG-115): Ascentage Pharma

* Drug profiles in the detailed report.....

* Preclinical and Discovery Stage Products

* Drug name: Company name

* Drug profiles in the detailed report.....

* Inactive Products

* Neuroblastoma Key Companies

* Neuroblastoma Key Products

* Neuroblastoma- Unmet Needs

* Neuroblastoma- Market Drivers and Barriers

* Neuroblastoma- Future Perspectives and Conclusion

* Neuroblastoma Analyst Views

* Neuroblastoma Key Companies

* Appendix

About DelveInsight

DelveInsight is a leading market research and consulting firm specializing in healthcare, providing high-quality intelligence and analysis to support strategic decisions. Our team of industry veterans, with deep expertise in life sciences and healthcare, offers customized solutions and global insights. Contact us for accurate, timely data to maintain a competitive edge.

Contact Us

Kanishk

kkumar@delveinsight.com