Glioblastoma - Pipeline Insight, 2025

This “Glioblastoma - Pipeline Insight, 2025” report provides comprehensive insights about 180+ companies and 200+ pipeline drugs in Glioblastoma 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.

Glioblastoma: Understanding

Glioblastoma: Overview

Glioblastoma Multiforme (GBM) is the most frequently occurring type of primary tumors of the central nervous system (CNS) mostly in adults, and its poor prognosis has not been significantly improved despite the fact that the innovative diagnostic strategies and new therapies have been developed. Somatic evolution promotes the progression of cancer in which the genome of the cancer cell is being deviated from that of the healthy cell due the accumulation of mutations. There is a remarkable development in GBM because it occurs via a complex network of various different molecular and genetic aberrations, which leads to significant changes in major signaling pathways. GBMs, as they extensively disperse throughout the parenchyma, making maximal surgical resection unattainable and having high level of vascularization, are lethal in nature. Glioblastoma, a highly aggressive brain cancer, is characterized by nonspecific symptoms such as headaches, personality changes, nausea, and stroke-like symptoms. Neurological symptoms like headaches, vomiting, seizures, and cognitive dysfunction, and major clinical signs like focal neurological deficits and cranial nerve dysfunction. In rare cases, patients can present with unusual symptoms like vertigo or psychiatric symptoms. GBM is the most common and aggressive primary brain tumor, accounting for 52% of all primary brain tumor cases and 20% of all intracranial tumors.

The glioblastoma multiforme preferentially invades along myelinated axons, vascular basement membranes, and the subependyma, mostly occurring in the subcortical matter of the cerebrum (65%). Within the cerebrum, the tumors proliferate through the temporal (31%), parietal (24%), frontal (23%), and occipital (16%) lobes. However, tumor formation is rarely isolated exclusively to one of these lobes, instead presenting in a combination of these lobes within the cerebrum. GBM also forms in the hypothalamus-thalamus area (20%) and the cerebellum and posterior fossa (15%). In addition, GBM can also potentially develop in the brain stem and the spinal cord, though these cases are extremely rare.

A patient with any neurological symptoms will first be given a physical exam that includes neurologic function tests (reflexes, muscle strength, eye and mouth movement, coordination and alertness). If a tumor is suspected, the patient will have imaging tests so that doctors can look into the brain for any abnormality. A neurological exam alone is not sufficient to make a glioblastoma diagnosis, but the results will indicate whether additional testing is needed. Treatment for glioblastoma multiforme usually includes a combination of surgery, chemotherapy, radiation, or stereotactic radiosurgery. Surgery is usually one of the most important aspects of treatment, although rarely used alone. Since glioblastomas develop very rapidly, they are often difficult to remove in their entirety. Therefore, surgery is performed to achieve a maximum safe resection - removing as much of the tumor as possible while preserving the patient’s brain function and sparing healthy tissues. Radiation therapy and chemotherapy usually follow surgery once the diagnosis or name of the tumor is determined.

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

Glioblastoma Emerging Drugs Chapters

This segment of the Glioblastoma report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, 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.

Glioblastoma Emerging Drugs

Enzastaurin: Denovo BioPharma

DB102 (enzastaurin) is an orally available investigational first-in-class small molecule, serine/threonine kinase inhibitor of the PKC beta, PI3K, and AKT pathways that has been studied in more than 3,000 patients across a range of solid and hematological tumor types. DB102 was originally developed by Eli Lilly and for which Denovo has acquired worldwide rights. DB102 received Orphan Drug Designation in DLBCL and glioblastoma multiforme (GBM) from the FDA and EMA and Fast Track Designation from the FDA. DB102 is the world's first oral small-molecule kinase inhibitor targeting PKC. A retrospective analysis found that it has significant curative effects in high-risk DLBCL patients who are DGM1 positive. The company has initiated a biomarker guided Phase III clinical study evaluating the DB102 (enzastaurin) in combination with temozolomide and radiation as first line therapy to treat newly-diagnosed glioblastoma multiforme (GBM).

Asunercept: Apogenix

CAN008 (asunercept) is a CD95-Fc fusion protein that binds to the CD95 ligand and blocks the interaction between the ligand and the endogenous CD95 receptor. CAN008 has a unique dual mechanism of action, inhibiting both the invasive growth and migration of tumor cells, as well as T-cell apoptosis, which enhances immune recognition of the cancer. Earlier asunercept glioblastoma multiforme (GBM) clinical trial data showed favorable safety and tolerability, prolonged survival and improved quality-of-life. Asunercept has been granted US FDA Orphan Drug Designation and Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for GBM. It has also been accepted into the EMA’s PRIME (Priority Medicines) program, which provides support to medicines that could address unmet medical needs. In China, CAN008 has been classified as a Class 1 New Drug by the National Medical Products Administration. CANbridge holds the rights to develop and commercialize CAN008 for any indication in Greater China and is currently conducting a CAN008 Phase II trial in GBM in China.

Berubicin: CNS Pharmaceuticals

Berubicin is an anthracycline, a class of anticancer agents that are among the most powerful chemotherapy drugs and effective against more types of cancer than any other class of chemotherapeutic agents. Anthracyclines are designed to utilize natural processes to induce deoxyribonucleic acid (DNA) damage in targeted cancer cells by interfering with the action of topoisomerase II, a critical enzyme enabling cell proliferation. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Glioblastoma.

Azeliragon: Cantex

Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer's disease by vTv Therapeutics from which Cantex licensed worldwide rights to azeliragon. Currently, the drug is in Phase II stage of its clinical trial for the treatment of glioblastoma, brain metastasis, pancreatic cancer, breast cancer.

VXM01: VAXIMM

VXM01 is an oral T-cell immunotherapy that is designed to activate T-cells to attack the tumor vasculature and, in several tumor types, attack cancer cells directly. VXM01 carries the vascular endothelial growth factor receptor-2 (VEGFR2), which is highly overexpressed on the tumor vasculature and on certain cancer cells as the target gene. The active, T-cell-mediated destruction of tumor vasculature cells leads to an increased infiltration of various immune cells into tumor tissue (inflammation). In preclinical studies, a murine analog VXM01 vaccine showed broad anti-tumor activity in different tumor types. This activity was linked to a VEGFR2-specific T-cell response and was accompanied by the destruction of the tumor vasculature and increased immune cell infiltration. Currently, the drug is in Phase I/II stage of its clinical trial for the treatment of Glioblastoma.

MB-101: Mustang Bio

MB-101 is a CAR-T cell therapy developed by Mustang Bio that targets IL13Rα2, a protein expressed on the surface of certain glioma cells. The therapy involves the infusion of CAR-T cells that are engineered to recognize and bind to IL13Rα2, leading to the activation and proliferation of these cells. This results in the killing of glioma cells and the potential for long-term remission. MB-101 has shown promising results in clinical trials, including achieving complete responses in two patients with high-grade glioma, and has been well-tolerated with manageable side effects. Currently, the drug is in Phase I stage of its clinical trial for the treatment of Glioblastoma.

Glioblastoma: Therapeutic Assessment

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

Major Players in Glioblastoma

• There are approx. 180+ key companies which are developing the therapies for Glioblastoma. The companies which have their Glioblastoma drug candidates in the most advanced stage, i.e. Phase III include, Denovo

Phases

The report covers around 200+ 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

Glioblastoma 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.

Glioblastoma: Pipeline Development Activities

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

Glioblastoma Report Insights

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

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

Key Players

• Denovo Biopharma
• Cantex Pharmaceuticals
• CNS Pharmaceuticals
• CANbridge Pharmaceuticals
• Vaximm
• Inovio Pharmaceuticals
• Mustang Bio
• Bullfrog AI Holdings
• Cantex
• Chimeric Therapeutics
• Philogen
• Boehringer Ingelheim
• Photonamic GmbH
• Berg Pharma
• Beyond Bio
• Genenta Science
• Polaris Pharmaceuticals
• Telix Pharmaceuticals
• Shanghai Simnova Biotechnology
• NEONC Technologies

Key Products

• Enzastaurin
• Azeliragon
• Berubicin
• CAN008
• VXM01
• INO-5401
• MB-101
• BF-222
• Azeliragon
• CHM 1101
• Fibromun
• BI 907828
• 5-aminolevulinic acid
• BPM31510
• BEY1107
• Temferon
• ADI-PEG20
• 131I-IPA
• SNC-109
• Perillyl alcohol

This product will be updated with the latest data at the time of order. Consequently, dispatch time for this product will be 7-10 business days.