Atypical Teratoid Rhabdoid Tumors (ATRT) - Pipeline Insight, 2024

This “Atypical Teratoid Rhabdoid Tumors (ATRT)- Pipeline Insight, 2024” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Atypical Teratoid Rhabdoid Tumors (ATRT) 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

Atypical Teratoid Rhabdoid Tumors (ATRT): Understanding

Atypical Teratoid Rhabdoid Tumors (ATRT): Overview

ATRT is a primary central nervous system (CNS) tumor. This means it begins in the brain or spinal cord. Genes may be mutated (changed) in many types of cancer, which can increase the growth and spread of cancer cells. Most ATRTs are caused by changes in a gene known as SMARCB1 (also called INI1). This gene normally signals proteins to stop tumor growth. But in ATRTs, SMARCB1 doesn’t function properly and tumor growth is uncontrolled. SMARCB1 can sometimes be found in a person’s DNA, which means they are born with it.

ATRTs occur in both children and adults and are very rare in both age groups. There have been only 50 reported cases in adults. An estimated 596 people are living with this tumor in the United States. ATRTs occur slightly more often in males than females. Additionally, they can form anywhere in the CNS. They often occur in the brain and often spread to the spinal cord. But ATRTs rarely begin in the spinal cord. ATRTs can form outside the CNS, such as in the kidney, but this is rare. ATRTs develop from several different very young cells called embryonal cells. These cells include rhabdoid, neuroepithelial, epithelial and mesenchymal.

Symptoms
Symptoms related to an ATRT depend on the tumor’s location and the person’s age. Since ATRTs are fast-growing, symptoms usually get worse quickly. The possible symptoms that can occur include:

• ATRT Symptoms
• Morning headaches
• Vomiting
• Changes in activity levels
• Loss of balance
• Increase in head size (in infants)

Diagnosis
Diagnosis can be done through several tests performed on the individual which consists of the following:

Neurological exam: A series of questions and tests to check the brain, spinal cord, and nerve function. The exam checks a person’s mental status, coordination, and ability to walk normally, and how well the muscles, senses, and reflexes work. This may also be called a neuro exam or a neurologic exam.- MRI (magnetic resonance imaging): A procedure that uses a magnet, radio waves, and a computer to make a series of detailed pictures of areas inside the brain and spinal cord. This procedure is also called nuclear magnetic resonance imaging (NMRI).

Lumbar puncture: A procedure used to collect cerebrospinal fluid (CSF) from the spinal column. This is done by placing a needle between two bones in the spine and into the lining around the spinal cord to remove a sample of the CSF. The sample of CSF is checked under a microscope for signs of tumor cells. The sample may also be checked for the amounts of protein and glucose. This procedure is also called an LP or spinal tap.- SMARCB1 and SMARCA4 gene testing: A laboratory test in which a sample of blood or tissue is tested for certain changes in the SMARCB1 and SMARCA4 genes.

Treatment
The first treatment for an ATRT is surgery, if possible. The goal of surgery is to obtain tissue to determine the tumor type and to remove as much tumor as possible without causing more symptoms for the person.

People with ATRTs usually receive further treatments after surgery, which may include radiation, chemotherapy, High-dose chemotherapy with stem cell transplant, or clinical trials. Clinical trials, with new chemotherapy, targeted therapy, or immunotherapy drugs, may also be available and can be a possible treatment option. Treatments are decided by the based on the patient’s age, remaining tumor after surgery, tumor type, and tumor location.

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

Atypical Teratoid Rhabdoid Tumors (ATRT) Emerging Drugs Chapters

This segment of the Atypical Teratoid Rhabdoid Tumors (ATRT) 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.

Atypical Teratoid Rhabdoid Tumors (ATRT) Emerging Drugs

Alisertib: Takeda Pharmaceuticals
Alisertib (MLN8237) is an oral, selective, inhibitor of Aurora A kinase being investigated by Takeda in Phase II trials for the treatment of Atypical Teratoid Rhabdoid Tumors. Aurora A kinase is required for cells to divide properly and has been shown to be over-expressed in a variety of cancers, and inhibition of Aurora A kinase represents a novel approach in cancer research.

ONC206: Chimerix
ONC206 is a DRD2 antagonist and ClpP agonist with nanomolar potency that demonstrates enhanced non-competitive DRD2 antagonism relative to ONC201 and disruption of DRD2 homodimers. ONC206 exhibits a distinct gene expression profile as well as single agent and combinatorial efficacy with ONC201 in cells with acquired resistance to ONC201. ONC206 is effective in preclinical models of difficult-to-treat neuroendocrine tumors and high-grade gliomas. It affects some of the same downstream pathways as ONC201, including activation of the integrated stress response and inhibition of Ras signaling, leading to selective killing of tumor cells.

ONC206 is a currently being evaluated in Phase I clinical trials for adult and pediatric patients with brain tumors.

Atypical Teratoid Rhabdoid Tumors (ATRT): Therapeutic Assessment

This segment of the report provides insights about the different Atypical Teratoid Rhabdoid Tumors (ATRT) drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Atypical Teratoid Rhabdoid Tumors (ATRT)

There are approx. 6+ key companies which are developing the therapies for Atypical Teratoid Rhabdoid Tumors (ATRT). The companies which have their Atypical Teratoid Rhabdoid Tumors (ATRT) drug candidates in the advanced stage, i.e. phase II include, Takeda Pharmaceuticals.

Phases

This report covers around 6+ 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

Atypical Teratoid Rhabdoid Tumors (ATRT) 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.

Atypical Teratoid Rhabdoid Tumors (ATRT): Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Atypical Teratoid Rhabdoid Tumors (ATRT) 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 Atypical Teratoid Rhabdoid Tumors (ATRT) drugs.

Atypical Teratoid Rhabdoid Tumors (ATRT) Report Insights

• Atypical Teratoid Rhabdoid Tumors (ATRT) Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Novartis Pharmaceuticals
• Ipsen Pharma
• Chimerix
• Exelixis
• Takeda Pharmaceuticals

Key Products

• Ribociclib
• Tazemetostat
• ONC206
• Cabozantinib
• Alisertib

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