MedPath

Clinical Trial News

Monalizumab Shows Promise in NeoCOAST-2 Trial for Early-Stage NSCLC

  • Interim results from the NeoCOAST-2 Phase 2 trial show monalizumab, combined with durvalumab and chemotherapy, demonstrates encouraging clinical outcomes in early-stage NSCLC.
  • The monalizumab arm achieved a pathological complete response rate of 26.7% and a major pathological response rate of 53.3% in resectable NSCLC patients.
  • Monalizumab, targeting the NKG2A receptor, aims to enhance anti-tumor responses mediated by NK and T cells, potentially improving outcomes in NSCLC treatment.
  • The manageable safety profile and maintained surgical rate in the monalizumab arm support its further investigation in combination strategies for NSCLC.

Neoadjuvant and Adjuvant Treatment in Resectable Non-small Cell Lung Cancer

NCT05061550RecruitingPhase 2
AstraZeneca
Posted 4/14/2022

PolTREG's PTG-007 Shows Long-Term Remission in Type 1 Diabetes Patients

  • PolTREG's PTG-007 cell therapy induced clinical remission in some patients with type 1 diabetes for up to 12 years, showcasing its long-term potential.
  • A subset of patients treated with PTG-007 remained insulin-independent for a period of 18 to 24 months, suggesting a significant therapeutic effect.
  • The company is preparing to initiate a pivotal Phase II/III trial for PTG-007 in type 1 diabetes and is actively seeking partnership funding to support this endeavor.
  • Data from the study, which monitored 54 patients over 7-12 years, were presented at the INNODIA EASD conference, highlighting the therapy's safety and efficacy.

Alzheimer's Disease: Late-Stage Pipeline Explores Novel Therapeutic Targets Beyond Amyloid and Tau

• The Alzheimer's disease therapeutic pipeline is expanding beyond amyloid and tau targets, with 32 therapeutics in 48 Phase III trials exploring neuroprotection, neurotransmitters, and inflammation. • Recent approvals of anti-amyloid antibodies like Leqembi and Kisunla have shown modest clinical benefits but also carry risks, prompting the development of alternative approaches. • Several Phase III candidates, such as Athira's fosgonimeton, Anavex's ANAVEX 2-73, and Novo Nordisk's semaglutide, target different mechanisms like neurotrophic factors, neuronal homeostasis, and neuroinflammation. • Experts suggest that combination therapies and brain-healthy lifestyles may be essential for effectively managing the heterogeneous nature of Alzheimer's disease.

A Research Study Investigating Semaglutide in People With Early Alzheimer's Disease (EVOKE)

NCT04777396Active, Not RecruitingPhase 3
Novo Nordisk A/S
Posted 5/18/2021

MDA Launches Kickstart Program for Ultra-Rare Neuromuscular Disease Gene Therapy Development

• The Muscular Dystrophy Association (MDA) has initiated the MDA Kickstart program to advance gene therapy development for ultra-rare neuromuscular diseases. • The first project focuses on gene therapy for Congenital Myasthenic Syndrome (CMS) caused by mutations in the CHAT gene, affecting around 200 individuals in the US. • MDA partners with UC Davis and Forge Biologics to accelerate preclinical development and manufacturing of AAV-based gene therapies for ultra-rare NMDs. • The program seeks FDA Orphan Drug and Rare Pediatric Disease designations, emphasizing the need for Rare Pediatric Disease Priority Review Voucher reauthorization.

Healios KK's MultiStem Therapy Advances to Global Phase 3 Trial for ARDS

  • Healios KK has secured FDA agreement to proceed with a pivotal global Phase 3 trial, REVIVE-ARDS, evaluating MultiStem therapy for Acute Respiratory Distress Syndrome (ARDS).
  • The REVIVE-ARDS trial will enroll up to 550 patients and use Ventilator Free Days as the primary endpoint, with interim analyses planned at 300 and 400 patient milestones.
  • Healios intends to pursue conditional and time-limited approval in Japan, leveraging prior Phase 2 results and the forthcoming confirmatory Phase 3 study.
  • MultiStem's efficacy and safety in treating ARDS will be rigorously assessed in this global trial, potentially addressing a critical unmet need in respiratory medicine.

Boryung Pharmaceutical's BR-6002 Advances in Cardiovascular Disease Treatment

  • Boryung Pharmaceutical is developing BR-6002, an orally administered drug, for cardiovascular diseases, as well as gastric and duodenal ulcers.
  • BR-6002's development leverages GlobalData's predictive analytics for assessing the likelihood of approval based on historical drug development data.
  • Boryung Pharmaceutical focuses on chronic diseases, including hypertension, dyslipidemia, diabetes, CNS disorders, and cancers, with BR-6002 potentially expanding their cardiovascular portfolio.

Pidnarulex Shows Promise in Advanced Solid Tumors with BRCA/PALB2 Mutations

  • Pidnarulex (CX-5461) demonstrates acceptable tolerability and preliminary efficacy in end-stage oncology patients with BRCA1/2 or PALB2 mutations, even after PARP inhibitor failure.
  • In a Phase Ib study, 40% of evaluable patients achieved clinical benefit with stable disease as the best response, including ovarian cancer patients with BRCA1/HRD mutations.
  • The trial aims to determine the Phase II dose and evaluate the safety and antitumor activity of Pidnarulex in patients with BRCA2/PALB2 deficiencies across various tumor types.
  • Senhwa Biosciences' Pidnarulex, a novel DDR drug, shows potential as a rescue medication for PARP inhibitor-resistant patients and may enhance immunotherapy efficacy.

HUTCHMED Highlights Clinical Data on Fruquintinib, Surufatinib, and Savolitinib at Upcoming Conferences

• HUTCHMED will present Phase II trial results of osimertinib plus savolitinib as first-line treatment for EGFRm, MET-aberrant advanced NSCLC, showing a significantly higher objective response rate compared to osimertinib alone. • Subgroup analyses from the FRESCO-2 trial will be presented, demonstrating the efficacy and safety of fruquintinib in refractory metastatic colorectal cancer patients with and without liver metastasis, and by age. • Data on surufatinib, both as a monotherapy and in combination with other treatments, will be presented for advanced hepatocellular carcinoma, neuroendocrine tumors, and soft tissue sarcoma.

Antennova to Present Phase I/Ib Data on ATN-037 at ESMO Congress 2024

  • Antennova will present data from the STAMINA-01 trial of ATN-037, a CD73 inhibitor, at the ESMO Congress 2024 on September 16.
  • The study evaluates ATN-037 as a monotherapy and in combination with pembrolizumab in patients with advanced solid tumors.
  • Early data shows a 43.8% disease control rate with ATN-037 monotherapy and a 20% overall response rate with the combination therapy.
  • The Phase II dose optimization and expansion of ATN-037 is ongoing in Australia and planned for China in late October 2024.

Natera's Signatera Shows 10-Fold Overall Survival Advantage in Colorectal Cancer

  • Natera will present new data on Signatera, its MRD test, at the ESMO Congress, including findings from the CIRCULATE-Japan GALAXY trial.
  • Signatera-positive patients in the GALAXY trial had a significantly shorter overall survival compared to Signatera-negative patients, with a hazard ratio of approximately 10.
  • Updated analysis from the GALAXY arm of CIRCULATE-Japan includes the first prospective read-out of overall survival based on MRD in over 2,100 patients.
  • Additional presentations at ESMO will highlight new Signatera data in breast cancer and squamous cell carcinoma of the head and neck.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.