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China Approves ORPATHYS-TAGRISSO Combination for EGFR-Mutated Lung Cancer with MET Amplification

Drug ApprovalTargeted Therapy
  • China's National Medical Products Administration approved the ORPATHYS-TAGRISSO combination for treating EGFR-mutated non-small cell lung cancer patients with MET amplification after disease progression on EGFR inhibitor therapy.
  • The Phase III SACHI trial demonstrated a 66% reduction in disease progression risk compared to chemotherapy, with median progression-free survival of 8.2 months versus 4.5 months.
  • This represents the first all-oral, chemotherapy-free treatment option for this patient population, addressing a critical resistance mechanism in lung cancer therapy.
  • The approval triggers an $11 million milestone payment from AstraZeneca to HUTCHMED and marks the third indication for ORPATHYS in China.

Savolitinib Plus Osimertinib Versus Platinum-based Doublet Chemotherapy in Participants With Non-Small Cell Lung Cancer Who Have Progressed on Osimertinib Treatment

NCT05261399RecruitingPhase 3
AstraZeneca
Posted 8/3/2022

A Phase II Study of HMPL-504 in Lung Sarcomatoid Carcinoma and Other Non-small Cell Lung Cancer

NCT02897479Unknown StatusPhase 2
Hutchison Medipharma Limited
Posted 12/1/2016

Savolitinib for Treating Gastric Cancer and Esophagogastric Junction Adenocarcinoma Patients

NCT04923932Active, Not RecruitingPhase 2
Hutchison Medipharma Limited
Posted 7/27/2021

MEDI4736 Combinations in Metastatic Renal Cell Carcinoma

NCT02819596CompletedPhase 2
Queen Mary University of London
Posted 5/3/2016

Clinical Study on Savolitinib + Osimertinib in Treatment of EGFRm+/MET+ Locally Advanced or Metastatic NSCLC

NCT05009836Active, Not RecruitingPhase 3
Hutchison Medipharma Limited
Posted 9/6/2021

Savolitinib for Treating Locally Advanced or Metastatic Non-small Cell Lung Cancer (NSCLC) Patients

NCT04923945CompletedPhase 3
Hutchison Medipharma Limited
Posted 8/19/2021

AZD9291 in Combination With Ascending Doses of Novel Therapeutics

NCT02143466Active, Not RecruitingPhase 1
AstraZeneca
Posted 8/5/2014

A trial to learn how well osimertinib with savolitinib works in people with non-small cell lung cancer (NSCLC) that has gotten worse after initial treatment with osimertinib.

2024-512581-34-00Active, Not RecruitingPhase 2
AstraZeneca AB

Study on Savolitinib Combined With Osimertinib in Treatment of Advanced NSCLC With MET Amplification

NCT05015608Active, Not RecruitingPhase 3
Hutchison Medipharma Limited
Posted 11/22/2021

Savolitinib vs. Sunitinib in MET-driven PRCC.

NCT03091192Active, Not RecruitingPhase 3
AstraZeneca
Posted 7/25/2017

FDA Approves Voranigo (Vorasidenib) as First Targeted Therapy for IDH-Mutant Glioma

Drug ApprovalTargeted Therapy
  • The FDA approved Voranigo (vorasidenib) on August 6, 2024, as the first targeted treatment for Grade 2 IDH-mutant astrocytoma or oligodendroglioma in patients 12 years and older.
  • Phase 3 INDIGO trial results showed Voranigo significantly extended progression-free survival to 27.7 months compared to 11.1 months with placebo.
  • The drug works by blocking mutant IDH1 and IDH2 enzymes, reducing tumor activity and crossing the blood-brain barrier to effectively treat brain tumors.
  • Common side effects include tiredness (37%), COVID-19 (33%), and muscle or joint pain (26%), with liver function monitoring required during treatment.

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

NCT04164901Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 1/5/2020

Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

NCT06478212RecruitingPhase 1
Institut de Recherches Internationales Servier
Posted 1/22/2025

AD HOC Trial: Artificial Intelligence-Based Drug Dosing In Hepatocellular Carcinoma

NCT05669339SuspendedPhase 2
University of Florida
Posted 12/2/2024

Study of Orally Administered AG-881 in Patients With Advanced Solid Tumors, Including Gliomas, With an IDH1 and/or IDH2 Mutation

NCT02481154CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 6/1/2015

Study of AG-120 and AG-881 in Subjects With Low Grade Glioma

NCT03343197CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 3/20/2018

ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

NCT05609994RecruitingPhase 1
Katy Peters, MD, PhD
Posted 7/15/2025

FDA Grants Breakthrough Therapy Designation for Daraxonrasib in KRAS-Mutated Pancreatic Cancer

Targeted TherapyPrecision MedicineOncology
  • FDA has granted Breakthrough Therapy Designation to daraxonrasib, a RAS(ON) multi-selective inhibitor, for previously treated metastatic pancreatic ductal adenocarcinoma (PDAC) in patients with KRAS G12 mutations.
  • The designation represents a significant regulatory milestone for targeting KRAS mutations in pancreatic cancer, one of the most challenging malignancies to treat.
  • Daraxonrasib's multi-selective RAS(ON) inhibition mechanism offers a novel therapeutic approach for patients with limited treatment options in the metastatic setting.

FDA-Approved Tovorafenib Offers New Treatment Option for Pediatric Low-Grade Glioma with BRAF V600 Mutations

Targeted TherapyDrug Approval
  • Tovorafenib (Ojemda) is approved for treating low-grade glioma that has returned after previous treatments failed, specifically targeting tumors with BRAF V600 mutations.
  • The oral medication is available for adults and children as young as 6 months old, administered once weekly as either tablets or oral suspension.
  • Treatment requires genetic testing to confirm BRAF V600 mutations before initiation and regular monitoring for potential side effects including bleeding, liver problems, and growth impacts in children.
  • Common side effects include fatigue, gastrointestinal symptoms, cold-like symptoms, and skin reactions, with serious adverse events requiring immediate medical attention.

Study for Patients With Hairy Cell Leukemia to Test the Targeted Agent Tovorafenib in Combination With Rituximab and Compare the Combination With Current Standard Treatment

NCT06965114Not Yet RecruitingPhase 1
National Cancer Institute (NCI)
Posted 9/30/2025

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

NCT05828069RecruitingPhase 2
National Cancer Institute (NCI)
Posted 3/28/2024

Zenocutuzumab Achieves 30% Response Rate in NRG1 Fusion-Positive Solid Tumors, Receives FDA Approval

Targeted TherapyDrug ApprovalPrecision Medicine
  • The Phase 2 eNRGy trial demonstrated zenocutuzumab's clinical efficacy with a 30% objective response rate and 11.1-month median duration of response in patients with NRG1 fusion-positive solid tumors.
  • Pancreatic cancer patients showed particularly promising results with a 42% response rate and 9.2-month median progression-free survival, offering hope for this historically difficult-to-treat malignancy.
  • Zenocutuzumab received accelerated FDA approval as the first targeted therapy for NRG1-positive non-small cell lung cancer and pancreatic adenocarcinoma.
  • The bispecific antibody demonstrated a favorable safety profile with treatment-related adverse events in 95% of participants but fewer than 10% experiencing grade 3 or higher toxicities.

Patritumab Deruxtecan in Patients with Solid Tumor Harboring an NRG1 Fusion

NCT06383884RecruitingPhase 2
Samsung Medical Center
Posted 8/13/2024

TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

NCT02693535RecruitingPhase 2
American Society of Clinical Oncology
Posted 3/14/2016

Study of Seribantumab in Adult Patients With NRG1 Gene Fusion Positive Advanced Solid Tumors

NCT04383210TerminatedPhase 2
Elevation Oncology
Posted 9/29/2020

A Study of Zenocutuzumab (MCLA-128) in Patients With Solid Tumors Harboring an NRG1 Fusion (eNRGy)

NCT02912949Active, Not RecruitingPhase 2
Partner Therapeutics, Inc.
Posted 1/1/2015

A Phase I/IIa Trial of HMBD-001 in Advanced HER3 Positive Solid Tumours

NCT05057013Active, Not RecruitingPhase 1
Cancer Research UK
Posted 11/10/2021

Dose Escalation Study to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of GSK2849330 in Subjects With Advanced Her3-Positive Solid Tumors

NCT01966445CompletedPhase 1
GlaxoSmithKline
Posted 11/26/2013

Phase 3 SYMPATICO Study Shows Promising First-Line Ibrutinib-Venetoclax Combination for Older MCL Patients

Targeted Therapy
  • The phase 3 SYMPATICO study demonstrated that first-line ibrutinib plus venetoclax achieved high complete response rates in older patients with mantle cell lymphoma, including those with challenging TP53 mutations.
  • Patients aged 65 and older with TP53-unmutated disease achieved a 76% complete response rate, while those with TP53-mutated disease reached a 44% complete response rate.
  • The combination showed durable responses with median progression-free survival of 40.2 months in TP53-unmutated patients and 22.0 months in TP53-mutated patients.
  • Safety profile was consistent with known individual agent profiles, with no new safety signals identified in the treatment-naive population.

Study of Ibrutinib Combined With Venetoclax in Subjects With Mantle Cell Lymphoma (MCL)

NCT03112174CompletedPhase 3
Pharmacyclics LLC.
Posted 6/19/2017

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Rare DiseaseTargeted TherapyDrug ApprovalOrphan Drug
  • Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder.
  • The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments.
  • APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available.
  • The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.

Budget Impact Analysis Shows Revumenib Cost-Neutral for Health Plans Despite High Specialty Drug Costs

Targeted TherapyRare Disease
  • A recent budget impact analysis demonstrates that revumenib (Revuforj) for relapsed/refractory acute leukemias with KMT2A translocation would be cost-neutral for health plans over three years.
  • The cost neutrality is primarily driven by revumenib's oral administration, which eliminates expensive infusion-related costs including chair time and administration fees.
  • The analysis highlights challenges in evaluating rare disease treatments affecting only 1.7 individuals per million patients, where traditional cost-effectiveness methods may not adequately capture therapeutic value.
  • Patient adherence to the oral therapy remains a critical variable that could influence long-term cost-effectiveness and clinical outcomes.

Advanced Mycosis Fungoides Treatment Requires Strategic Sequencing of HDAC Inhibitors and Targeted Therapies

Targeted Therapy
  • Advanced mycosis fungoides with nodal involvement requires systemic approaches adapted from peripheral T-cell lymphoma management, as standard skin-directed therapies provide limited benefit in this setting.
  • CTCL patients typically require 50% dose reductions compared to systemic T-cell lymphoma protocols due to enhanced treatment sensitivity and altered tumor cell metabolism.
  • HDAC inhibitors including romidapsin and vorinostat demonstrate efficacy in patients with both peripheral blood and nodal involvement, while mogamalizumab shows particular effectiveness in circulating disease.
  • Chronic use of single-agent chemotherapy introduces unique toxicity considerations, with gemcitabine carrying risks of hemolytic uremic syndrome after continuous use for over a year.

Eli Lilly's Olomorasib Receives FDA Orphan Drug Designation for KRAS G12C-Mutated NSCLC Treatment

Targeted Therapy
  • The FDA has granted orphan drug designation to Eli Lilly's Olomorasib for treating non-small cell lung cancer with KRAS G12C mutations, providing market exclusivity and development incentives.
  • Olomorasib is currently in Phase 3 clinical trials as a first-line treatment combined with Keytruda (pembrolizumab), with or without chemotherapy, for advanced NSCLC patients.
  • Previous ASCO data demonstrated that Olomorasib (50 or 100mg BID) combined with Keytruda showed promising safety and anti-tumor activity in KRAS G12C-mutated NSCLC patients.
  • The designation addresses an unmet medical need for patients with KRAS G12C mutations, a common driver mutation in NSCLC with limited treatment options.

A Study of First-Line Olomorasib (LY3537982) and Pembrolizumab With or Without Chemotherapy in Patients With Advanced KRAS G12C-Mutant Non-small Cell Lung Cancer

NCT06119581RecruitingPhase 3
Eli Lilly and Company
Posted 12/21/2023

Study of LY3537982 in Cancer Patients With a Specific Genetic Mutation (KRAS G12C)

NCT04956640RecruitingPhase 1
Eli Lilly and Company
Posted 7/19/2021

A Study of Multiple Olomorasib (LY3537982) Capsules in Healthy Participants

NCT07044271Not Yet RecruitingNot Applicable
Eli Lilly and Company
Posted 7/1/2025

Study of Olomorasib (LY3537982) in Combination With Standard of Care in Participants With Resected or Unresectable KRAS G12C-mutant Non-Small Cell Lung Cancer

NCT06890598RecruitingPhase 3
Eli Lilly and Company
Posted 3/27/2025

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