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FDA Grants Breakthrough Therapy Designation to Novel Triplet Combination for BRAF-Mutant Metastatic Colorectal Cancer

Targeted Therapy
  • The FDA has granted breakthrough therapy designation to the triplet combination of encorafenib, binimetinib, and cetuximab for treating BRAF V600E-mutant metastatic colorectal cancer after 1-2 prior treatments.
  • The designation is based on promising data from the BEACON CRC trial showing a 48% overall response rate and 62% one-year overall survival rate.
  • This represents a significant advancement for patients with BRAF-mutant mCRC, who currently have no approved regimens specifically targeting their mutation.
  • The breakthrough designation will expedite development and regulatory review of this combination therapy for patients with limited treatment options.

Study of Encorafenib + Cetuximab Plus or Minus Binimetinib vs. Irinotecan/Cetuximab or Infusional 5-Fluorouracil (5-FU)/Folinic Acid (FA)/Irinotecan (FOLFIRI)/Cetuximab With a Safety Lead-in of Encorafenib + Binimetinib + Cetuximab in Patients With BRAF V600E-mutant Metastatic Colorectal Cancer

NCT02928224CompletedPhase 3
Pfizer
Posted 10/13/2016

Boehringer Ingelheim Partners with UK Consortium to Develop Novel Gene Therapy for Cystic Fibrosis

  • Boehringer Ingelheim has formed a groundbreaking partnership with the UK Cystic Fibrosis Gene Therapy Consortium and Oxford BioMedica to develop a first-in-class gene therapy for cystic fibrosis.
  • The collaboration focuses on developing an inhaled lentiviral vector therapy that can deliver healthy CFTR genes to lung cells, potentially offering a universal treatment for all CF mutations.
  • The partnership combines the consortium's gene therapy expertise, Oxford BioMedica's manufacturing capabilities, and Boehringer Ingelheim's drug development experience, with BI securing exclusive global rights.

Oncolytic Virus Therapy Emerges as Promising Cancer Treatment Breakthrough

  • Oncolytic virus therapy shows remarkable potential in cancer treatment by selectively targeting and destroying cancer cells while activating immune responses, marking a significant advancement in immunotherapy.
  • Recent clinical trials demonstrate that combining oncolytic viruses with checkpoint inhibitors, such as Amgen's Imlygic with Keytruda, achieved a 62% response rate in melanoma patients.
  • Major pharmaceutical companies are heavily investing in oncolytic virus platforms, with Janssen's $1 billion acquisition of BeneVir BioPharm and Merck's $400 million purchase of Viralytics highlighting industry confidence.

Advancing Precision Medicine with Synthetic Nearest Neighbors in Clinical Trials

A novel approach using Synthetic Nearest Neighbors (SNN) estimator to predict patient-level outcomes from population-level randomized control trials (RCTs) has shown promising results, particularly in Alzheimer’s Disease research. This method addresses the challenges of missing data and patient heterogeneity, offering a pathway towards personalized medicine.

CHMP Backs Novel Therapies: Onpattro for hATTR and Symkevi for Cystic Fibrosis

• The European Medicines Agency's CHMP has recommended approval for Alnylam's Onpattro (patisiran), a groundbreaking siRNA therapy for hereditary transthyretin-mediated amyloidosis with polyneuropathy.
• Vertex's Symkevi (tezacaftor/ivacaftor), in combination with Kalydeco, received positive CHMP opinion for treating cystic fibrosis patients with specific F508del mutations.
• The committee also endorsed several other medications, including Pierre Fabre's melanoma drugs and AstraZeneca's Imfinzi for lung cancer, while rejecting BMS's Opdivo-Yervoy combination for first-line kidney cancer.

Sanofi Establishes €66 Million Global R&D Hub in Chengdu, Expanding China Research Footprint

  • Sanofi is investing €66 million to establish a new global research hub in Chengdu, China, focusing on data analysis and management of multi-center clinical trials.
  • The facility aims to recruit 300 R&D staff by 2020 and will support research across multiple therapeutic areas including diabetes, cardiovascular diseases, oncology, and rare diseases.
  • This expansion establishes China as Sanofi's third major research pillar alongside France and the US, building upon their existing R&D presence in Shanghai.

Genetic Variants and Their Impact on Clinical Responses to Treatments

Recent studies have explored the relationship between genetic variants and clinical responses to various treatments, including mesalamine for ulcerative colitis, ALK inhibitors for non-small cell lung cancer, and metformin for type 2 diabetes. Findings suggest that while some genetic variants do not significantly affect treatment outcomes, others may influence drug efficacy and patient survival.

Vedolizumab: A New Therapeutic Option for Inflammatory Bowel Disease

Vedolizumab, a fully humanised monoclonal antibody, has emerged as a promising treatment for patients with moderate-to-severe ulcerative colitis and Crohn’s disease who are refractory or intolerant to conventional treatments or anti-TNFα agents. This article reviews the efficacy, safety, and tolerability of vedolizumab, highlighting its role in the treatment algorithms for inflammatory bowel disease based on clinical trials and real-world studies.

Pfizer, Amgen, and Sanofi Partner to Leverage Blockchain Technology in Clinical Trials

  • Three pharmaceutical giants - Pfizer, Amgen, and Sanofi - have joined forces to explore blockchain technology's potential in enhancing data management and reducing drug development costs in clinical trials.
  • Blockchain technology offers a secure, immutable platform for storing clinical trial data, addressing critical challenges in patient privacy, data integrity, and efficient information sharing across multiple databases.
  • Research indicates only 16% of patients are aware of relevant clinical trials, highlighting the potential for blockchain to revolutionize patient recruitment and trial participation through anonymous data sharing and smart contracts.

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