Neuroblastoma - Pipeline Insight, 2024

This “Neuroblastoma - Pipeline Insight, 2024” 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.

Geography Covered

• Global coverage

Neuroblastoma: Understanding

Neuroblastoma: Overview

Neuroblastoma (NB) is the most frequently-occurring extracranial childhood tumor. It is classified as an embryonal neuroendocrine tumor, originating from neural crest progenitor cells. Hence, it can occur anywhere along the sympathetic nervous system, including the superior cervical, paraspinal, and celiac ganglia; the majority arise in the adrenal glands. Due to the high variability in its presentation, clinical signs and symptoms at presentation can range from a benign palpable mass with distension to major illness from substantial tumor spread. Although overall increases in five-year event-free survival have been reported, subgroup-specific analysis of mortality has revealed discordance between the high cure rates for the more benign low-risk forms and little improvement in the high-risk groups. Thus, the impetus for the development of targeted therapeutics in the intensive management of high-risk groups is strong. According to the National Cancer Institute, neuroblastoma is the first most common cancer in infants, and the third most common cancer in children. > 600 cases are diagnosed annually in the United States. It accounts for approximately 15% of all pediatric cancer fatalities. Incidence is higher in non-African-American children, and it is slightly more common in males than females.

Neuroblastoma sometimes forms before birth and may be discovered during a fetal ultrasound. Usually, by the time of diagnosis, the cancer has metastasized, most commonly to the lymph nodes, bones, bone marrow, and liver. In infants, metastasis also occurs to the skin. Neuroblastoma may be caused by a gene mutation, passed from a parent to a child. When this occurs, the child is usually at a younger age, and more than one tumor forms in the adrenal glands. The most common symptoms are abdominal pain, discomfort, and a sense of fullness caused by an abdominal mass. Although neuroblastoma may be encountered initially as a mass on abdominal radiographs or US, confirmation of the diagnosis and definition of the exact extent of disease is obtained with either CT or MRI. NB shows a broad spectrum of clinical behaviour, as in some cases it may spontaneously regress or mature, whereas in others it may progress despite intensive multimodality treatment. Clinical outcome appears to correlate with a series of well-known clinical, histological and biological features, which can be used for risk-group stratification and treatment assignment according to current treatment protocols.

Neuroblastoma is the most widespread solid tumor in childhood, presenting a plethora of symptoms and different responses to treatment. One of its features is the production of oxidative stress due to the accumulation of reactive species necessary for cancer metabolism, proliferation, and progression. Neuroblastoma treatment varies based on the risk group of the child. For low-risk neuroblastoma, observation or surgery may be sufficient. Intermediate-risk neuroblastoma typically involves surgery followed by chemotherapy. High-risk neuroblastoma is treated with a combination of therapies, including chemotherapy, surgery, and radiation, stem cell transplant, and immunotherapy. Newer treatments like monoclonal antibodies, CAR T-cell therapy, and anti-GD2 immunotherapy are also being explored through clinical trials. The International Neuroblastoma Risk Group Staging System (INRGSS) helps determine the appropriate treatment based on the stage and risk level of the cancer.

Neuroblastoma- Pipeline Insight, 2024 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 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

67-Cu-SARTATE: Clarity Pharmaceuticals67-Cu-SARTATE is a next generation, highly targeted theranostic radiopharmaceutical with increased specificity and in-vivo stability, being developed for diagnosing, staging and subsequently treating cancers that express somatostatin receptor 2 (SSTR2). 67Cu SARTATE™ Peptide Receptor Radionuclide Therapy (PRRT) is being administered to Pediatric Patients with High-Risk Neuroblastoma (CL04). 7Cu SARTATE and 64Cu SARTATE have been granted FDA Orphan Drug Designation (ODD) status for the treatment and clinical management of neuroblastoma. Currently the drug is in Phase II stage of its clinical trial for the treatment of neuroblastoma.

Alrizomadlin (APG-115): Ascentage PharmaAPG-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,Clarity Pharmaceuticals.

Phases

This 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

• Childrens Hospital of Philadelphia/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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