Diffuse Intrinsic Pontine Glioma - Pipeline Insight, 2024

This “Diffuse Intrinsic Pontine Glioma- Pipeline Insight, 2024” report provides comprehensive insights about 25+ companies and 25+ pipeline drugs in Diffuse Intrinsic Pontine Glioma 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

Diffuse Intrinsic Pontine Glioma: Understanding

Diffuse Intrinsic Pontine Glioma: Overview

Diffuse intrinsic pontine gliomas (DIPGs) are highly-aggressive and difficult-to-treat brain tumors found at the base of the brain. They are glial tumors, meaning they arise from the brain's glial tissue - tissue made up of cells that help support and protect the brain's neurons. These tumors are found in an area of the brainstem called the pons, which controls many of the body's most vital functions such as breathing, blood pressure and heart rate. Approximately 300 children are diagnosed with DIPGs each year, usually between the ages of 5 and 9. Although the prognosis for DIPGs remains very poor but new research, with the help of clinical trials, may provide the key to improved treatment options. The symptoms of diffuse intrinsic pontine glioma (DIPG) usually develop very rapidly prior to diagnosis, reflecting the fast growth of these tumors. Most patients start experiencing symptoms less than three months - and often less than three weeks - before diagnosis. The most common symptoms include: rapidly developing problems controlling eye movements, facial expressions, speech, chewing, and swallowing, weakness in the arms and legs, problems with walking and coordination. The first step in treating the child is forming an accurate and complete diagnosis. Diffuse intrinsic pontine glioma (DIPG) is most commonly diagnosed from imaging studies. A number of treatments may be recommended. Some of them help to treat the tumor while others are intended to address complications of the disease or side effects of the treatment. Treatment for DIPG may include radiation therapy. DIPG falls into the Glioma staging system, so they can be classified according to the four stages below based on how the cells look under the microscope. The grades are from the least severe to the most severe. Low Grade: Grade I or II means that the tumor cells are the closest to normal. High Grade: Grade III or IV means that these are the most aggressive tumors. The main issue with DIPG is that most of these tumors are not classified by grade because surgery to obtain tissue by biopsy or to remove the tumor is not safe because of the location of the tumor. When these tumors are biopsied, they are usually grade III or grade IV tumors, which tend to behave very aggressively. Most of the tumors are diagnosed by their appearance on MRI. About 10% to 20% of all childhood brain tumors are DIPG or brainstem gliomas. They are more common in children between the ages of 5 and 10 years, but can occur at any age in childhood. Though more rare, they can also occur in adults.

Diffuse Intrinsic Pontine Glioma- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Diffuse Intrinsic Pontine Glioma pipeline landscape is provided which includes the disease overview and Diffuse Intrinsic Pontine Glioma treatment guidelines. The assessment part of the report embraces, in depth Diffuse Intrinsic Pontine Glioma 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, Diffuse Intrinsic Pontine Glioma 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 Diffuse Intrinsic Pontine Glioma R&D. The therapies under development are focused on novel approaches to treat/improve Diffuse Intrinsic Pontine Glioma.

Diffuse Intrinsic Pontine Glioma Emerging Drugs Chapters

This segment of the Diffuse Intrinsic Pontine Glioma 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.

Diffuse Intrinsic Pontine Glioma Emerging Drugs

MTX 110: Midatech MTX110 is a water-soluble form of panobinostat free base, achieved through complexation with hydroxypropyl-ß- 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). Based on favourable translational science data, MTX110 is being evaluated clinically as a treatment for DIPG (NCT03566199, NCT04264143) and recurrent medulloblastoma (NCT04315064), 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 minimizing 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 celllines.

Paxalisib: Kazia Therapeutics Paxalisib (originally GDC-0084) was invented by Genentech, Inc (South San Francisco, CA), the most successful developer of new cancer medicines in the world. Kazia entered into a worldwide exclusive license agreement with Genentech in October 2016. Genentech designed paxalisib to inhibit PI3K, a critical control mechanism in growth and cell division, which is activated in many forms of cancer. The distinguishing feature of paxalisib is its ability to cross the blood-brain barrier (BBB). Ordinarily, the BBB prevents many drugs from reaching brain tissue, and is a challenge in the treatment of any disease in the central nervous system. Paxalisib has been designed to cross the BBB, and a wealth of experimental data shows that it does so very successfully. This feature of paxalisib is almost unique in this class of medicines and differentiates it from the approved products in the PI3K inhibitor class.

Diffuse Intrinsic Pontine Glioma: Therapeutic Assessment

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

Major Players in Diffuse Intrinsic Pontine Glioma

There are approx. 25+ key companies which are developing the therapies for Diffuse Intrinsic Pontine Glioma. The companies which have their Diffuse Intrinsic Pontine Glioma drug candidates in the most advanced stage, i.e. phase I/II include, Midatech.

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

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

Diffuse Intrinsic Pontine Glioma: Pipeline Development Activities

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

Diffuse Intrinsic Pontine Glioma Report Insights

• Diffuse Intrinsic Pontine Glioma Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Midatech
• Kazia Therapeutics
• Kintara Therapeutics
• Oncotelic Therapeutics
• Antisense Pharma
• Y-mAbs Therapeutics
• PTC Therapeutics
• Celgene Corporation

Key Products

• MTX 110
• Paxalisib
• Trabedersen
• VAL-083
• Omburtamab I-124
• BXQ-350
• AloCELYVIR
• PTC-596
• Marizomib

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