MedPath

Tagged News

£1.5M CONNECT TarGeT Trial Launches Precision Medicine Approach for Childhood Brain Cancer

Precision Medicine
  • The Brain Tumour Charity is contributing £1.5 million to CONNECT TarGeT, an adaptive umbrella clinical trial targeting paediatric high-grade gliomas in patients aged 12 months to 25 years.
  • The phase II trial uses genetic profiling to match patients with specific drug combinations, with the first UK arm testing everolimus and ribociclib for tumours with PI3K/mTOR pathway alterations.
  • The trial addresses a critical unmet need, as paediatric high-grade gliomas have less than 10% five-year survival rates and life expectancy of 9-12 months for conditions like DMG.
  • The international collaboration spans NHS children's cancer centres in the UK alongside institutions in the USA, Germany, Netherlands, Australia and New Zealand, with UK launch expected in Spring 2026.

Geneseeq's PanTRKare™ Receives NMPA Approval as China's First NGS-Based Pan-Solid Tumor Companion Diagnostic for NTRK Gene Fusions

Precision MedicineDrug ApprovalTargeted TherapyOncology
  • Geneseeq Technology Inc. has received NMPA approval for PanTRKare™, China's first next-generation sequencing-based pan-solid tumor companion diagnostic test for detecting NTRK1/2/3 gene fusions.
  • The assay was validated through a comprehensive multi-center clinical study involving seven leading hospitals, 33 tumor types, and more than 2,400 clinical samples, successfully detecting over 200 unique NTRK fusion variants.
  • PanTRKare™ is approved as a companion diagnostic test for Roche's ROZLYTREK® (entrectinib), addressing the clinical need to identify patients with rare NTRK gene fusions who can benefit from targeted TRK inhibitor therapy.
  • Clinical bridging studies demonstrated high concordance with ROZLYTREK® testing results and comparable objective response rates among NTRK fusion-positive patients, establishing the test's clinical utility for precision medicine.

Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options

NCT02650401Active, Not RecruitingPhase 1
Hoffmann-La Roche
Posted 5/3/2016

Study of Oral RXDX-101 in Adult Patients With Locally Advanced or Metastatic Cancer Targeting NTRK1, NTRK2, NTRK3, ROS1, or ALK Molecular Alterations.

NCT02097810CompletedPhase 1
Hoffmann-La Roche
Posted 7/28/2014

Basket Study of Entrectinib (RXDX-101) for the Treatment of Patients With Solid Tumors Harboring NTRK 1/2/3 (Trk A/B/C), ROS1, or ALK Gene Rearrangements (Fusions)

NCT02568267Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 11/19/2015

Novartis Opens New Radioligand Therapy Manufacturing Facility in California as Part of $23 Billion US Expansion

Precision MedicineOncology
  • Novartis has opened a new 10,000-square-foot radioligand therapy manufacturing facility in Carlsbad, California, marking the company's third US-based RLT production site.
  • The facility is designed to produce precision cancer treatments that deliver radioactive particles directly to tumors, ensuring continued on-time delivery rates of >99.9% to patients across the western US, Alaska, and Hawaii.
  • This opening represents a key milestone in Novartis' previously announced $23 billion investment in US infrastructure over the next five years, with additional RLT facilities planned for Florida and Texas.
  • Novartis maintains its position as the only pharmaceutical company with a dedicated commercial RLT portfolio and global leadership in radioligand therapies for more than seven years.

Targeted Vitamin D3 Supplementation Cuts Heart Attack Recurrence Risk by 50% in Clinical Trial

Precision Medicine
  • Intermountain Health researchers found that a "target-to-treat" approach for vitamin D3 supplementation reduced the risk of second heart attacks by 50% in patients who previously suffered cardiac events.
  • The TARGET-D clinical trial enrolled 630 heart attack patients and monitored vitamin D blood levels, adjusting dosages to achieve optimal levels above 40 ng/mL rather than using standard supplementation doses.
  • More than half of patients receiving targeted therapy required initial doses of 5,000 IU of vitamin D3, significantly higher than typical supplement recommendations of 600-800 IU.
  • The findings are particularly significant given that 85% of enrolled heart attack patients had insufficient vitamin D levels below 40 ng/mL at study entry.

Inka Health Publishes Framework to Enable Cross-Border Use of Real-World Evidence in Oncology

Real World EvidenceAIPrecision MedicineRare DiseaseOncology
  • Inka Health, a subsidiary of Onco-Innovations, published a technical primer in the Journal of Clinical Evaluative Research addressing how to adapt medical evidence from one country for use in another through advanced statistical transportability methods.
  • The framework outlines three foundational principles for reliable cross-border evidence sharing: consistency, positivity, and conditional exchangeability, which will be integrated into Inka Health's AI-powered SynoGraph platform.
  • These transportability methods are particularly important for rare disease drug approvals where patient populations are small and local evidence is often lacking, potentially accelerating regulatory approvals and improving patient access to innovative therapies.
  • The primer was co-authored with experts at AstraZeneca Canada and details proven statistical techniques including matching, weighting, standardization, and bias analysis to address differences in patient populations across regions.

CareDx Launches HistoMap Kidney Tissue-Based Test to Enhance Transplant Rejection Diagnosis

AIPrecision Medicine
  • CareDx announced the launch of HistoMap Kidney, a tissue-based molecular test that uses gene expression profiling to objectively characterize rejection type and enhance biopsy results for kidney transplant patients.
  • New clinical data presented at ASN Kidney Week 2025 demonstrated that AlloSure Plus, an AI-powered rejection risk assessment tool, showed performance for predicting antibody-mediated rejection in kidney transplants.
  • Studies revealed that AlloSure levels remained consistent regardless of donor kidney size across 54 living donor recipients, supporting its use as a robust biomarker for allograft injury in both adult and pediatric patients.
  • Research showed that AlloSure and blood gene expression profiling were not affected by early graft function status, enabling rejection screening as early as week 1 post-transplant.

Jaya Biosciences Secures U.S. Patent for Gene Therapy Approach to Alzheimer's Disease Treatment

Precision Medicine
  • Jaya Biosciences received U.S. Patent No. 12,460,262 for methods of detecting, preventing, reversing, and treating neurological diseases linked to lysosomal enzyme gene mutations.
  • The patent covers a novel approach targeting heterozygous mutations in lysosomal enzyme genes that increase susceptibility to adult-onset neurodegeneration, including Alzheimer's disease.
  • The company's lead therapy JB111, an AAV9-mediated PPT1 gene therapy, demonstrated improved survival, cognition, and reduced amyloid pathology in preclinical models with a single intracerebroventricular injection.
  • This patent expands JayaBio's global intellectual property portfolio following prior patents in Japan, China, and Singapore, strengthening its position in the gene therapy market for neurodegenerative diseases.

PacBio's Sequel II CNDx Becomes World's First Clinically Approved Long-Read Sequencer in China

Precision Medicine
  • PacBio's Sequel II CNDx system received Class III Medical Device Registration approval from China's NMPA, marking the world's first regulatory clearance of a clinical-grade long-read sequencer.
  • Berry Genomics becomes the first company worldwide authorized to deploy long-read sequencing in clinical settings, enabling comprehensive genomic testing for complex genetic disorders like thalassemia.
  • The approval delivers the first end-to-end long-read sequencing workflow for China's hospitals, optimized for carrier, prenatal, newborn, and rare disease testing with reduced turnaround times.
  • The technology captures single nucleotide variants, insertions, deletions, copy number variants, structural variants, and repeat expansions in a single test with exceptional accuracy.

Neok Bio Emerges with $75M to Develop Dual-Targeting ADCs for Cancer Treatment

OncologyPrecision Medicine
  • Neok Bio launched with $75 million in Series A funding to develop bispecific antibody-drug conjugates that simultaneously target two distinct cancer proteins.
  • The company's lead programs Neok001 and Neok002 target B7-H3/ROR1 and EGFR/MUC1 protein pairs respectively, aiming to enhance efficacy and safety over conventional ADCs.
  • Both programs are expected to enter human testing in 2026 with results anticipated in 2027, backed by Korean antibody specialist ABL Bio.
  • The dual-targeting approach aims to overcome limitations of traditional single-target ADCs, including limited therapeutic windows and off-target effects.

OncoHost Advances Plasma Proteomics for NSCLC Immunotherapy Monitoring and Prediction

Precision Medicine
  • OncoHost presented two research posters at the 2025 ISLB Annual Congress demonstrating plasma proteomics' potential to improve clinical decision-making for NSCLC patients.
  • The first study showed plasma proteomic signatures can detect immunotherapy non-responders up to 6.6 months before standard imaging, outperforming ctDNA analysis.
  • The second study validated PROphetNSCLC® test in 308 patients, showing it predicts immunotherapy benefit independent of genomic alterations with significant survival advantage (HR=0.39, p<0.0001).
  • The proteomics approach captures comprehensive tumor-immune interactions beyond genomic testing, establishing it as a universal biomarker for treatment selection.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.