DelveInsight’s, “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.
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Key Takeaways from the Pediatric Brain Tumors Pipeline Report
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Pediatric Brain Tumors Emerging Drugs Profile
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 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.
The Pediatric Brain Tumors Pipeline Report Provides Insights into
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Pediatric Brain Tumors Companies
Biodexa Pharmaceuticals, Cellectar Biosciences, ZIOPHARM Oncology, Y-mAbs Therapeutics Inc., Neonc Technologiesd Inc. and others.
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
Pediatric Brain Tumors Products have been categorized under various Molecule types such as
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Scope of the Pediatric Brain Tumors Pipeline Report
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