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Belzutifan Demonstrates Superior Quality-Adjusted Survival Time Compared to Everolimus in Advanced Renal Cell Carcinoma

OncologyTargeted Therapy
  • Belzutifan showed a clinically meaningful 2.66-month improvement in quality-adjusted time without symptoms or toxicity (Q-TWiST) compared to everolimus in advanced renal cell carcinoma patients.
  • The phase 3 LITESPARK-005 trial demonstrated an 11.32% relative gain in Q-TWiST with belzutifan, despite numerically longer time with grade 3/4 toxicity.
  • Belzutifan patients experienced significantly longer time without symptoms and progression (10.73 vs 6.07 months) driven by prolonged periods without disease progression.
  • The analysis supports belzutifan's clinical benefit profile, with lead investigator Thomas Powles noting superior response rates, progression-free survival, and quality of life outcomes.

A Study of Belzutifan (MK-6482) Versus Everolimus in Participants With Advanced Renal Cell Carcinoma (MK-6482-005)

NCT04195750Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 2/27/2020

Yale Researchers Engineer Novel Antibody-RNA Therapy Platform for Treatment-Resistant Cancers

Targeted Therapy
  • Yale researchers developed TMAB3, a specially engineered antibody that successfully delivers RNA therapies to treatment-resistant "cold" tumors, significantly reducing tumor size and extending survival in animal models of pancreatic, brain, and skin cancers.
  • The therapy demonstrated ability to penetrate the blood-brain barrier and target cancerous cells while avoiding healthy tissue, showing pronounced tumor suppression without severe side effects across multiple cancer types.
  • Computer modeling enabled antibody modification for RNA binding and humanization, laying groundwork for personalized immuno-RNA therapies and potential first-in-human clinical trials.

Korean Researchers Develop Precision Nanobody Therapy for Lung Adenocarcinoma with 90% Tumor Reduction

Targeted TherapyPrecision Medicine
  • Researchers at Korea Research Institute of Bioscience and Biotechnology developed the A5 nanobody that specifically targets CD155 protein overexpressed in lung adenocarcinoma cells.
  • The A5-LNP-DOX delivery system achieved 2-3 fold higher drug uptake in cancer cells and demonstrated 70-90% tumor burden reduction in animal models.
  • The nanobody therapy suppressed cancer cell migration and invasion by over 50% while showing no major organ toxicity in preclinical studies.
  • Clinical analysis revealed that patients with high CD155 and PXN expression levels had significantly worse survival outcomes, validating the therapeutic target.

UCLA Researchers Receive $950,000 Grant to Launch Multi-Institutional Trial of Zipalertinib for EGFR-Mutated Cancers

Targeted TherapyOncology
  • UCLA's Arjan Gower received a $950,000 grant from the National Comprehensive Cancer Network and Taiho Oncology Inc. to launch a multi-institutional clinical trial testing zipalertinib.
  • The investigational drug targets specific EGFR mutations, including Exon 20 insertions, which drive cancer growth and resist standard treatments.
  • This funding represents a significant investment in developing targeted therapies for previously difficult-to-treat EGFR-mutated cancers.
  • The multi-institutional approach suggests broad clinical collaboration to evaluate this novel targeted therapy's potential.

Real-World Study Reveals Persistent Gaps in Molecular Testing and Targeted Therapy Implementation for AML and MDS

Targeted TherapyReal World Evidence
  • A five-year retrospective analysis of over 16,000 patients with AML and MDS found significant gaps in molecular testing implementation, with mean turnaround times of 14-16 days exceeding recommended guidelines.
  • Despite mutations being detected in 68% of AML patients and 72% of MDS patients, transition rates to targeted therapies remained suboptimal, particularly in older AML patients and across all MDS patient groups.
  • The study highlighted the need for streamlined approaches, including ultra-rapid NGS platforms, to improve guideline adherence and enhance patient outcomes in hematological malignancies.

Targeted Therapies Transform Biliary Tract Cancer Treatment Landscape with FGFR2 Inhibitors Leading Clinical Advances

Targeted TherapyPrecision MedicineOncology
  • Biliary tract cancer treatment has evolved from traditional cytotoxic chemotherapy to include targeted therapies based on specific molecular alterations, with FGFR2 inhibitors showing particularly promising results.
  • Pemigatinib and futibatinib, selective FGFR inhibitors, have demonstrated significant clinical efficacy with response rates of 35% and 42% respectively in patients with FGFR2 fusions.
  • Comprehensive next-generation sequencing has become the standard approach for identifying actionable molecular alterations, with IDH1/2 mutations, FGFR2 fusions, and ERBB2 alterations being the most clinically significant biomarkers.
  • The rarity of biliary tract cancers presents unique challenges in treatment access, making clinical trials essential for patients with molecular alterations that have FDA-approved therapies in other disease states.

Real-World Data Confirm Ivosidenib Efficacy in IDH1-Mutated Cholangiocarcinoma

Targeted TherapyReal World EvidenceOncology
  • The phase 3b ProvIDHe study demonstrated that ivosidenib achieved a median progression-free survival of 4.7 months and overall survival of 15.5 months in patients with IDH1-mutated cholangiocarcinoma.
  • Real-world data from 262 patients across 80 sites in 15 countries confirmed the efficacy profile established in the pivotal ClarIDHy trial that led to FDA approval.
  • The study enrolled heavily pretreated patients with a median of 2 prior lines of therapy, showing a disease control rate of 51.5% and objective response rate of 5.7%.

An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma

NCT05876754RecruitingPhase 3
Servier Affaires Médicales
Posted 5/3/2023

Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy)

NCT02989857CompletedPhase 3
Institut de Recherches Internationales Servier
Posted 2/20/2017

Australian-First "Seek-and-Destroy" Cancer Therapy Begins Human Trials Targeting CDCP1 Protein

Targeted Therapy
  • A groundbreaking radioactive therapy targeting the CDCP1 protein in cancer cells has begun its first human trial in Australia, with a 69-year-old Brisbane woman becoming the first patient to receive the treatment.
  • The therapy, developed over 24 years by Professor John Hooper, uses an innovative antibody that binds to CDCP1 found in ovarian, bladder, and other common cancers to deliver targeted radiotherapy.
  • Up to 45 patients with ovarian and bladder cancer will participate in the trial, which aims to provide exceptionally accurate imaging of metastasizing cancer and potentially become the gold standard treatment for multiple cancer types.
  • The research represents a collaboration between Mater Research, The University of Queensland, CSIRO, and other institutions, with ovarian cancer having a five-year survival rate of just 49 percent.

Arvinas CEO John Houston Announces Retirement Following PROTAC Breakthrough Success

Leadership ChangeTargeted Therapy
  • John Houston, Ph.D., CEO and President of Arvinas, announces plans to retire from executive roles after eight years of leadership, while remaining as Board Chairperson.
  • Under Houston's leadership, Arvinas achieved multiple industry firsts for PROTAC protein degraders, including the first positive pivotal Phase 3 trial and first new drug application.
  • The company has advanced six PROTAC programs into clinical trials and demonstrated for the first time that orally administered PROTACs can achieve pharmacodynamic activity in the central nervous system.
  • Arvinas Board of Directors has initiated a search for Houston's successor to continue advancing the company's targeted protein degradation platform across multiple therapeutic areas.

Comprehensive Genomic Profiling Emerges as Essential Standard for Personalized Cancer Treatment

Precision MedicineTargeted Therapy
  • Comprehensive genomic profiling has become standard practice for non-small cell lung cancer treatment, enabling identification of actionable genomic alterations that guide personalized therapeutic decisions.
  • The integration of both tissue-based testing and plasma-based next-generation sequencing provides complementary molecular profiling capabilities throughout the patient care continuum.
  • Incomplete genomic testing can leave patients without access to potentially beneficial targeted therapies, emphasizing the critical importance of comprehensive molecular profiling for optimal outcomes.
  • Future applications include expanded serial testing for resistance monitoring and potential expansion into earlier cancer stages as precision medicine approaches continue evolving.

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