Pediatric Brain Tumors - Pipeline Insight, 2025

This “Pediatric Brain Tumors- Pipeline Insight, 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Pediatric Brain Tumors 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.

Pediatric Brain Tumors: Understanding

Pediatric Brain Tumors: Overview

Pediatric brain tumors are a diverse group of neoplasms that occur in the brain and spinal cord of children. These tumors are the second most common type of childhood cancer after leukemia. They can be benign or malignant, with varying degrees of aggressiveness. The most common types include gliomas, medulloblastomas, and ependymomas. The location of the tumor in the brain significantly affects the clinical presentation and prognosis. Advances in imaging techniques and molecular biology have improved the understanding and management of these tumors, although they still pose significant challenges due to the delicate nature of the developing brain.

The signs and symptoms of pediatric brain tumors depend on the tumor's location, size, and growth rate. Common symptoms include headaches, which may be more severe in the morning, nausea, vomiting, and balance problems. Other signs can include vision changes, seizures, behavioral changes, and cognitive impairments. In infants and very young children, increased head circumference, irritability, and developmental delays may be observed. These symptoms often overlap with other less severe conditions, which can delay diagnosis.

The causes of pediatric brain tumors are not fully understood. Most are thought to arise sporadically without a clear genetic predisposition. However, some genetic syndromes, such as neurofibromatosis type 1 and 2, Li-Fraumeni syndrome, and tuberous sclerosis, increase the risk of developing brain tumors. Environmental factors, including exposure to ionizing radiation, have also been implicated in the development of brain tumors, but these cases are rare.

Pathophysiologically, pediatric brain tumors result from uncontrolled cell growth in the central nervous system due to genetic mutations and aberrant cellular signaling pathways. These mutations can lead to the activation of oncogenes or the inactivation of tumor suppressor genes, promoting tumorigenesis. The brain’s unique environment and the presence of the blood-brain barrier pose challenges in understanding and treating these tumors, as therapeutic agents must be able to penetrate this barrier to be effective.

Diagnosis of pediatric brain tumors typically involves a combination of clinical evaluation, neuroimaging, and sometimes biopsy. Magnetic resonance imaging (MRI) is the gold standard for detecting and characterizing brain tumors. Advanced imaging techniques, such as functional MRI and diffusion tensor imaging, can provide additional information about the tumor's impact on surrounding brain structures. Histopathological examination of biopsy samples, when feasible, helps in definitive diagnosis and guides treatment planning. Molecular and genetic testing of tumor tissue is increasingly used to tailor therapies to the tumor's specific characteristics.

Treatment of pediatric brain tumors depends on the type, location, and extent of the tumor, as well as the child’s age and overall health. It often involves a combination of surgery, radiation therapy, and chemotherapy. Surgery aims to remove as much of the tumor as safely possible. Radiation therapy, including advanced techniques like proton beam therapy, is used to target residual tumor cells while minimizing damage to healthy tissue. Chemotherapy may be used in conjunction with surgery and radiation or as a primary treatment in certain cases. Newer treatment modalities, such as targeted therapies and immunotherapy, are being explored and hold promise for improving outcomes. Multidisciplinary care, including support from neuro-oncologists, neurosurgeons, radiation oncologists, and rehabilitation specialists, is essential for optimizing treatment and quality of life for affected children.

'Pediatric Brain Tumors- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Pediatric Brain Tumors pipeline landscape is provided which includes the disease overview and Pediatric Brain Tumors treatment guidelines. The assessment part of the report embraces, in depth Pediatric Brain Tumors 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, Pediatric Brain Tumors 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 Pediatric Brain Tumors R&D. The therapies under development are focused on novel approaches to treat/improve Pediatric Brain Tumors.

Pediatric Brain Tumors Emerging Drugs Chapters

This segment of the Pediatric Brain Tumors 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.

Pediatric Brain Tumors Emerging Drugs

MTX110: Biodexa Pharmaceuticals

MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-β-cyclodextrin (HPBCD) that enables convection-enhanced delivery (CED) at potentially chemotherapeutic doses directly to the site of the tumor. Panobinostat is a hydroxamic acid and acts as a non-selective histone deacetylase inhibitor (pan-HDAC inhibitor). The currently available oral formulation of panobinostat lactate (Farydak®) is not suitable for treatment of brain cancers owing to poor blood-brain barrier penetration and inadequate brain drug concentrations. Based on favorable translational science data, MTX110 is being evaluated clinically as a treatment for DIPG and recurrent medulloblastoma and preclinical for treatment of glioblastoma. MTX110 is delivered directly into and around the patient’s tumor via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumor to very high drug concentrations while simultaneously minimizing systemic drug levels and the potential for toxicity and other side effects. Panobinostat has demonstrated high potency against DIPG tumor cells in in vitro and in vivo models, and in a key study it was the most promising of 83 anticancer agents tested in 14 patient-derived DIPG cell lines. Currently, the drug is in Phase I/II stage of its development for the treatment of diffuse intrinsic pontine glioma.

Iopofosine: Cellectar Biosciences

Iopofosine is a small-molecule Phospholipid Drug Conjugate™ designed to provide targeted delivery of iodine-131 (radioisotope) directly to cancer cells, while limiting exposure to healthy cells. Iopofosine is currently being evaluated in the CLOVER-WaM Phase II pivotal study in patients with relapsed/refractory (r/r) Waldenstrom’s macroglobulinemia (WM), a Phase IIb study in r/r multiple myeloma (MM) patients and the CLOVER-2 Phase I study for a variety of pediatric cancers. The U.S. Food and Drug Administration granted iopofosine Fast Track Designation for WM patients having received two or more prior treatment regimens, as well as r/r MM and r/r diffuse large B-cell lymphoma (DLBCL). Orphan Drug Designations (ODDs) have been granted for WM, MM, neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. Iopofosine was also granted Rare Pediatric Disease Designation (RPDD) for the treatment of neuroblastoma, rhabdomyosarcoma, Ewing’s sarcoma and osteosarcoma. The European Commission granted an ODDs for r/r MM and WM. Currently, the drug is in Phase I stage of its development for the treatment of pediatric brain tumor.

Pediatric Brain Tumors: Therapeutic Assessment

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

Major Players in Pediatric Brain Tumors

• There are approx. 5+ key companies which are developing the therapies for Pediatric Brain Tumors. The companies which have their Pediatric Brain Tumors drug candidates in the most advanced stage, i.e. phase I/II include, Biodexa Pharmaceuticals.

Phases

The report covers around 5+ 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

Pediatric Brain Tumors 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.

Pediatric Brain Tumors: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Pediatric Brain Tumors 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 Pediatric Brain Tumors drugs.

Pediatric Brain Tumors Report Insights

• Pediatric Brain Tumors Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Pediatric Brain Tumors 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 Pediatric Brain Tumors drugs?
• How many Pediatric Brain Tumors drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pediatric Brain Tumors?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pediatric Brain Tumors 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 Pediatric Brain Tumors and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Biodexa Pharmaceuticals
• Cellectar Biosciences
• ZIOPHARM Oncology
• Y-mAbs Therapeutics, Inc.

Neonc Technologies, Inc.

Key Products

• MTX110
• Iopofosine
• INXN 2001
• BCC018
• NEO100-03

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