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FDA-Approved FGFR Inhibitors Show Promise for Pediatric Brain Tumors in Preclinical Studies

Precision MedicineTargeted Therapy
  • Researchers from Dana-Farber/Boston Children's Cancer and Blood Disorders Center found that 8.9% of pediatric gliomas harbor FGFR alterations and may respond to existing FDA-approved FGFR inhibitors.
  • The study developed first-ever preclinical models of FGFR-altered gliomas using neural stem cells and demonstrated tumor sensitivity to FGFR inhibitors in laboratory testing.
  • Retrospective analysis of pediatric patients with FGFR-altered low-grade gliomas showed frequent stable disease after treatment with FGFR inhibitors.
  • The research opens potential pathways for targeted therapy in pediatric gliomas and rare brain tumors like ETMR, addressing an urgent unmet medical need.

Onco-Innovations Initiates Manufacturing of PNKP Inhibitor for Preclinical Testing

OncologyTargeted Therapy
  • Onco-Innovations has partnered with Dalton Pharma Services to begin manufacturing 50 grams of their proprietary PNKP inhibitor A83B4C63 for preclinical testing.
  • The nanoparticle-formulated inhibitor previously demonstrated significant survival benefits in colorectal cancer models, extending median survival from 23 to 60 days in PTEN-deficient mice.
  • This manufacturing milestone represents a critical transition from early research to production scale-up, supporting the company's planned IND submission to the FDA this year.

Eli Lilly Acquires Scorpion's Cancer Drug STX-478 in $2.5 Billion Deal

Targeted TherapyOncology
  • Eli Lilly has entered into a $2.5 billion agreement to acquire STX-478, an experimental oral PI3K alpha inhibitor from Scorpion Therapeutics for breast cancer and other solid tumors.
  • STX-478 is currently in phase 1/2 trials for hormone receptor-positive, HER2-negative breast cancer and could potentially address 30%-40% of people with HR-positive breast cancer.
  • The deal includes an upfront payment plus regulatory and commercial milestones, while Scorpion will spin off its remaining pipeline assets into a new independent company.
  • This acquisition expands Lilly's oncology portfolio following its $8 billion Loxo Oncology acquisition in 2019 and represents the company's renewed focus on PI3K inhibitors after abandoning LOXO-783 last year.

Iran Becomes First Asian Country to Develop Electrochemotherapy Cancer Treatment Technology

OncologyTargeted Therapy
  • Iran has successfully launched the first domestically-developed production line for electrochemotherapy (ECT) ablative devices, joining only Italy and the United Kingdom in producing this advanced cancer treatment technology.
  • The electrochemotherapy system uses electric pulses to increase cancer cell membrane permeability, significantly enhancing the effectiveness of anti-cancer drugs for targeted tumor treatment.
  • Over 1,000 patients have been treated with ECT technology, with more than 200 cases achieving successful outcomes that prevented the need for amputation procedures.
  • This achievement represents a major milestone for Iran's biotechnology sector and positions the country as a leader in advanced cancer treatment technologies within Asia.

Abbisko's FGFR4 Inhibitor Irpagratinib Receives China's Breakthrough Therapy Designation for Hepatocellular Carcinoma

Precision MedicineOncologyMonoclonal AntibodyTargeted Therapy
  • Abbisko Therapeutics' irpagratinib (ABSK011) has received Breakthrough Therapy Designation from China's NMPA for treating hepatocellular carcinoma, marking the first precision-targeted therapy using molecularly defined biomarkers for this indication.
  • The highly selective FGFR4 inhibitor targets FGF19 overexpression, which affects approximately 30% of HCC patients worldwide and is associated with significantly worse prognosis.
  • Clinical data from the 2024 ESMO GI Congress showed irpagratinib combined with atezolizumab achieved a 50% objective response rate in FGF19-positive HCC patients previously treated with immune checkpoint inhibitors.
  • The designation will expedite regulatory approval processes, potentially making irpagratinib the first globally approved FGFR4 inhibitor for HCC treatment.

A Study Evaluating Atezolizumab and Bevacizumab, With or Without Tiragolumab, in Participants With Untreated Locally Advanced or Metastatic Hepatocellular Carcinoma (IMbrave152)

NCT05904886Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 9/14/2023

A Study Evaluating the Efficacy and Safety of Multiple Immunotherapy-Based Treatment Combinations in Patients With Advanced Liver Cancers (Morpheus-Liver)

NCT04524871RecruitingPhase 1
Hoffmann-La Roche
Posted 11/1/2020

A Phase 2, Open-Label Study of ABSK-011 Combined Atezolizumab in HCC Patients

NCT05441475RecruitingPhase 2
Abbisko Therapeutics Co, Ltd
Posted 12/30/2021

Nuvalent to Present First Clinical Data for Next-Generation ALK Inhibitor NVL-655 at AACR-NCI-EORTC Symposium

OncologyTargeted Therapy
• Nuvalent will present preliminary dose-escalation data from the ALKOVE-1 Phase 1/2 trial of NVL-655, marking the first clinical disclosure for this next-generation ALK inhibitor. • NVL-655 is designed to overcome key limitations of existing ALK inhibitors, including resistance mutations, poor brain penetrance, and CNS adverse events. • The drug targets advanced ALK-positive non-small cell lung cancer and has been engineered to remain active against multiple resistance mutations including the challenging G1202R solvent front mutation. • The presentation will occur at the 35th AACR-NCI-EORTC Symposium in Boston on October 13, 2023, with a planned conference call the same day.

A Study of Neladalkib (NVL-655) in Patients With Advanced NSCLC and Other Solid Tumors Harboring ALK Rearrangement or Activating ALK Mutation (ALKOVE-1)

NCT05384626RecruitingPhase 1
Nuvalent Inc.
Posted 6/9/2022

Pfizer's BRAFTOVI Combination Therapy Halves Death Risk in BRAF-Mutant Metastatic Colorectal Cancer

Targeted TherapyPrecision MedicineOncology
  • Pfizer announced that its BRAFTOVI® combination regimen demonstrated a 50% reduction in death risk for patients with BRAF V600E-mutant metastatic colorectal cancer.
  • The targeted therapy represents a major advance in treating this specific genetic subtype of colorectal cancer.
  • This breakthrough could significantly improve survival outcomes for patients harboring the BRAF V600E mutation in metastatic disease.

Enhanced Premedication Protocol Reduces Infusion Reactions in NSCLC Patients Receiving Amivantamab

Monoclonal AntibodyTargeted Therapy
  • The SKIPPirr trial demonstrated that an enhanced premedication protocol significantly reduced infusion-related reactions (IRRs) for amivantamab from 67% to 22.5% in NSCLC patients.
  • The protocol includes dexamethasone 8 mg twice daily for two days prior to infusion plus standard premedications with diphenhydramine and acetaminophen.
  • Current NSCLC management faces critical gaps including optimal approaches for early-stage disease, tumor heterogeneity challenges, and understanding immunotherapy nonresponse mechanisms.
  • Institutional hypersensitivity guidelines are recommended for common NSCLC treatments including platinum agents, taxanes, monoclonal antibodies, and immunotherapy agents.

Mythic Therapeutics' MYTX-011 Shows Promising Efficacy in Phase 1 Trial for Advanced NSCLC

OncologyTargeted Therapy
  • Mythic Therapeutics will present updated dose escalation data from its Phase 1 KisMET-01 study of MYTX-011, a cMET-targeting antibody-drug conjugate, at the 2025 ASCO Annual Meeting.
  • The preliminary results demonstrate meaningful anti-tumor activity in cMET-positive non-small cell lung cancer patients across various cMET expression levels, histologies, and genetic alterations.
  • MYTX-011 leverages Mythic's proprietary FateControl™ technology, designed to enhance ADC uptake in tumor cells while minimizing impact on healthy tissues.

Clinical Study of Antibody-Drug Conjugate MYTX-011 in Subjects With Non-Small Cell Lung Cancer

NCT05652868RecruitingPhase 1
Mythic Therapeutics
Posted 3/23/2023

Dual-Targeting Radiopharmaceutical Shows 89% Response Rate in Advanced Cancer Trial

Targeted Therapy
  • A novel dual-targeting radiopharmaceutical therapy targeting two cancer cell markers simultaneously demonstrated safety and efficacy in a first-in-human trial with nine advanced adenocarcinoma patients.
  • The treatment achieved an 88.9% response rate, with patients experiencing either tumor shrinkage or disease stabilization after a single treatment cycle.
  • The therapy showed strong tumor uptake lasting for days, allowing prolonged radiation damage to cancer cells while causing no noticeable side effects.
  • Patients reported significant quality of life improvements including reduced pain, better appetite, and overall symptom relief compared to previous treatments.

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