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Molecular Testing and Targeted Therapies Transform Treatment Landscape for Pediatric Low-Grade Gliomas

Targeted TherapyPrecision Medicine
  • Comprehensive molecular testing using NGS panels and methylation profiling has become foundational for accurately diagnosing pediatric low-grade gliomas and identifying actionable mutations like BRAF alterations.
  • Targeted therapies including BRAF and MEK inhibitors are emerging as compelling alternatives to traditional chemotherapy, offering oral administration and better tolerability for children.
  • The treatment approach has evolved from cytotoxic chemotherapy to precision medicine strategies that balance tumor control with quality of life over the chronic, multi-year treatment course.
  • Molecular diagnostics typically return within 2-4 weeks for full results, with initial pathology and specific mutations like BRAF V600E detectable within one week through targeted immunohistochemistry stains.

University of Houston Develops First Spleen-Targeted Nanoparticle System for Lupus Treatment

Targeted Therapy
  • University of Houston biomedical engineer Tianfu Wu has received a $1 million Department of Defense Impact Award to develop a novel spleen-specific drug delivery system for lupus treatment.
  • The innovative system uses mannose-modified lipid nanoparticles to directly target immune cells in the spleen, including B cells, plasmacytoid dendritic cells, and macrophages that drive lupus pathogenesis.
  • This approach represents the first instance of a spleen-specific targeting system designed for lupus models, potentially offering more precise treatment with reduced systemic side effects.
  • The technology aims to modulate immune responses rather than employing broad immunosuppression, addressing current therapeutic limitations in lupus management.

Bayer Partners with Kumquat Biosciences in $1.3 Billion Deal to Develop KRAS G12D Inhibitor for Hard-to-Treat Cancers

Targeted Therapy
  • Bayer and Kumquat Biosciences announced an exclusive global license and collaboration worth up to $1.3 billion to develop a KRAS G12D inhibitor targeting pancreatic, colorectal, and lung cancers.
  • The KRAS G12D mutation is found in 37% of pancreatic ductal adenocarcinoma cases, 13% of colorectal cancers, and 4% of non-small cell lung cancers, yet lacks effective treatment options despite occurring in nearly 25% of human cancers.
  • Kumquat received FDA clearance for its investigational new drug in July 2025 and will complete Phase Ia studies, while Bayer handles subsequent development and commercialization activities.
  • The collaboration addresses a critical unmet medical need in pancreatic cancer, which has a five-year survival rate of less than 10% and is projected to see a 95.4% increase in new cases by 2050.

Study of MRTX1133 in Patients With Advanced Solid Tumors Harboring a KRAS G12D Mutation

NCT05737706TerminatedPhase 1
Mirati Therapeutics Inc.
Posted 3/6/2023

A Study of ASP3082 in Adults With Advanced Solid Tumors

NCT05382559RecruitingPhase 1
Astellas Pharma Inc
Posted 6/8/2022

A Study to Investigate the Safety and Efficacy of KQB548 in Participants With Advanced Solid Malignancies

NCT07207707RecruitingNot Applicable
Kumquat Biosciences Inc.
Posted 9/16/2025

Day One Biopharmaceuticals Reports 310% Revenue Surge for Pediatric Cancer Drug OJEMDA

Targeted Therapy
  • Day One Biopharmaceuticals reported OJEMDA net product revenue of $33.6 million in Q2 2025, representing a 310% year-over-year increase driven by expanding adoption among pediatric oncologists.
  • The company wrote more than 1,000 OJEMDA prescriptions during the quarter, marking a 15% sequential increase and 346% rise compared to the prior year period.
  • Management reaffirmed annual OJEMDA revenue guidance of $140-150 million for 2025 and confirmed sufficient cash reserves to fund operations without external financing.
  • Two pivotal trials for OJEMDA, FIREFLY-1 and FIREFLY-2, remain on track with key clinical data expected in late 2025 and early 2026.

Vitiligo Treatment Pipeline Shows Promise with JAK Inhibitors and Novel Immunotherapies in Phase 3 Trials

Monoclonal AntibodyTargeted Therapy
  • Over 18 companies are actively developing 20+ pipeline therapies for vitiligo treatment, with JAK inhibitors leading the charge in late-stage clinical trials.
  • AbbVie's Upadacitinib and Pfizer's Ritlecitinib have both advanced to Phase 3 trials, demonstrating promising repigmentation results in vitiligo patients.
  • Novel approaches include Clinuvel's Afamelanotide in Phase 3 trials and Amgen's AMG 714 anti-IL-15 monoclonal antibody in Phase 2 development.
  • The pipeline reflects a paradigm shift towards targeted therapies and personalized medicine approaches, offering new hope for patients with this challenging autoimmune condition.

CDSCO Panel Recommends Approval for Vorasidenib with Phase-III Waiver for IDH-Mutant Gliomas in India

Drug ApprovalOrphan DrugTargeted Therapy
  • India's CDSCO panel has recommended approval for vorasidenib tablets (10 mg and 40 mg) with a waiver from local Phase-III clinical trials for treating IDH-mutant gliomas.
  • The dual IDH1/IDH2 inhibitor is indicated for adult and pediatric patients aged 12 years and older with Grade 2 astrocytoma or oligodendroglioma harboring IDH mutations.
  • The committee acknowledged the unmet medical need for these indications in India and noted the drug's orphan status in other countries.
  • A Phase-IV clinical trial in Indian patients will be mandatory, with the protocol to be submitted within 3 months of approval.

Novel Nanoparticle Formulations Enhance Paclitaxel Delivery to Cancer Tumors

Targeted Therapy
  • University of Arizona researchers developed Paclitaxome, a new nanovesicle formulation of paclitaxel that outperformed standard chemotherapy drugs Taxol and Abraxane in mouse models of triple-negative breast cancer and advanced pancreatic cancer.
  • The formulation chemically attaches paclitaxel to sphingomyelin to create nanovesicles that improve tumor delivery, extend circulation time, and reduce accumulation in healthy tissues like the liver and spleen.
  • Indian researchers created fluorescent nanoparticles coated with transferrin that selectively target cancer cells overexpressing transferrin receptors, releasing over 90% of paclitaxel over two days while sparing normal cells.
  • Both platforms demonstrate the potential for improved drug delivery systems that could reduce chemotherapy side effects while enhancing therapeutic efficacy across multiple cancer types.

Terbium-161 Radioimmunotherapy Shows Superior Efficacy Against Ovarian Cancer Stem Cells in Preclinical Study

Targeted Therapy
  • A novel radioimmunotherapy using Terbium-161 (¹⁶¹Tb) successfully eliminated ovarian cancer stem cells in preclinical models, outperforming the current gold standard Lutetium-177.
  • The ¹⁶¹Tb-DOTA-chCE7 conjugate completely eradicated all detected ovarian cancer stem cells and their differentiated progeny in live animal models, effectively preventing tumor growth.
  • The superior efficacy stems from ¹⁶¹Tb's emission of short-range conversion and Auger electrons alongside beta particles, enabling highly localized energy deposition and irreparable DNA damage within cancer stem cells.
  • This breakthrough offers renewed hope for targeting therapy-resistant cancer stem cells that drive tumor relapse, metastasis, and treatment resistance in ovarian cancer patients.

UNLV Researchers Achieve 99% Pancreas-Specific mRNA Delivery Using Novel ENDO Nanoparticle Platform

Targeted Therapy
  • UNLV researchers developed ENDO (Endogenous Targeting Lipid Nanoparticles) that achieve 99% selectivity for pancreatic delivery of mRNA therapeutics via intravenous injection.
  • The breakthrough technology exploits Vitamin D receptors on pancreatic cell surfaces to route nanoparticles specifically to the pancreas, marking the first material capable of such precision.
  • This targeted delivery system could revolutionize treatment for diabetes and pancreatic cancer by enabling direct mRNA therapy to pancreatic cells while minimizing systemic side effects.
  • The platform shows potential for expansion to other difficult-to-reach organs including the brain and heart, with commercialization efforts already underway through UNLV's Office of Economic Development.

Lung Cancer Experts Convene to Address Clinical Uncertainties in Rapidly Evolving Treatment Landscape

Targeted Therapy
  • The third annual Bridging the Gaps in Lung Cancer consensus meeting brings together approximately 30 global experts to identify and address unresolved clinical challenges in lung cancer management.
  • Key knowledge gaps persist in optimal duration of adjuvant targeted therapy, management of patients with complete pathologic response after neoadjuvant chemoimmunotherapy, and treatment guidelines for leptomeningeal disease.
  • The meeting's unique format focuses exclusively on clinical ambiguity rather than established standards, with consensus recommendations evolving annually as new trial data and biomarker validation studies emerge.
  • New features include livestreaming and social media integration to broaden reach and demonstrate variability in expert perspectives across different healthcare systems.

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