Medulloblastoma- Pipeline Insight, 2025

This “Medulloblastoma- Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Medulloblastoma 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.

Medulloblastoma: Understanding

Medulloblastoma: Overview

Medulloblastoma is a type of malignant brain tumor that primarily affects children. It arises in the cerebellum, which is the part of the brain responsible for coordination and balance. This tumor is relatively rare, accounting for around 20% of all childhood brain tumors. Despite its rarity, it is the most common malignant brain tumor in children.

Signs and symptoms of medulloblastoma often include headaches, nausea, vomiting, and problems with balance and coordination. Children may also experience changes in their vision or have difficulty walking. As the tumor grows, it can compress the surrounding brain tissue, leading to increased pressure within the skull and potentially causing symptoms such as irritability, lethargy, and seizures.

The exact cause of medulloblastoma is unknown, but researchers believe that it may result from genetic mutations that occur during fetal development. Certain genetic syndromes, such as Gorlin syndrome and Turcot syndrome, are associated with an increased risk of developing medulloblastoma. Exposure to radiation, particularly at a young age, is also considered a risk factor for this type of brain tumor.

In terms of pathophysiology, medulloblastoma arises from primitive neuroectodermal cells, which are precursor cells that give rise to various types of nervous tissue. These cells proliferate uncontrollably, forming a mass within the cerebellum. The tumor may spread to other parts of the brain and spinal cord via cerebrospinal fluid, leading to metastasis.

Diagnosis of medulloblastoma typically involves imaging studies such as magnetic resonance imaging (MRI) or computed tomography (CT) scans to visualize the tumor and assess its size and location. A biopsy may be performed to confirm the diagnosis by examining the tumor tissue under a microscope.

Treatment for medulloblastoma usually involves a combination of surgery, radiation therapy, and chemotherapy. The primary goal of surgery is to remove as much of the tumor as possible without causing damage to surrounding brain tissue. Radiation therapy is used to kill any remaining cancer cells and reduce the risk of recurrence, while chemotherapy may be administered to further destroy cancer cells and prevent metastasis. The specific treatment approach may vary depending on factors such as the patient's age, the size and location of the tumor, and whether the cancer has spread to other parts of the body. With advances in treatment, the prognosis for children with medulloblastoma has improved significantly in recent years, with many patients achieving long-term remission.

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

Medulloblastoma Emerging Drugs Chapters

This segment of the Medulloblastoma 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.

Medulloblastoma Emerging Drugs

Pomalidomide: Bristol-Myers Squibb

Pomalidomide is a novel investigative drug candidate being developed by Bristol-Myers Squibb. The drug candidate was also under development for the treatment of metastatic adenocarcinoma of the pancreas, soft tissue sarcoma, metastatic hormone-refractory prostate cancer, systemic sclerosis (scleroderma), chronic cough and small-cell lung cancer. It was under development for acute myelocytic leukemia, primary myelofibrosis (chronic idiopathic myelofibrosis) and post-essential thrombocythaemia myelofibrosis. Currently, the drug is in Phase II stage of its development for the treatment of medulloblastoma.

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 tumour. 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 favourable translational science data, MTX110 is being evaluated clinically as a treatment for DIPG and recurrent medulloblastoma and preclinically for treatment of glioblastoma. MTX110 is delivered directly into and around the patient’s tumour via a catheter system (e.g. CED or fourth ventricle infusions) to bypass the blood-brain barrier. This technique exposes the tumour to very high drug concentrations while simultaneously minimising systemic drug levels and the potential for toxicity and other side effects. Panobinostat has demonstrated high potency against DIPG tumour 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 stage of development for the treatment of medulloblstoma.

Medulloblastoma: Therapeutic Assessment

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

Major Players in Medulloblastoma

• There are approx. 10+ key companies which are developing the therapies for Medulloblastoma. The companies which have their Medulloblastoma drug candidates in the most advanced stage, i.e. phase II include, Bristol-Myers Squibb.

Phases

The report covers around 12+ 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

Medulloblastoma 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.

Medulloblastoma: Pipeline Development Activities

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

Medulloblastoma Report Insights

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

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

Key Players

• Bristol-Myers Squibb
• Biodexa Pharmaceuticals
• Valent Technologies, LLC
• Novartis Oncology
• Sun Pharmaceutical Industries
• Chimerix
• Curis

Key Products

• Pomalidomide
• MTX110
• VAL-413
• Ribociclib
• Sonidegib
• ONC206
• Fimepinostat

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