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Clinical Trial News

CirCode Biomed's HM2002 Becomes World's First Circular RNA Drug to Receive FDA IND Clearance for Ischemic Heart Disease

  • CirCode Biomed announced FDA clearance for HM2002, marking the first circular RNA drug to receive IND approval for ischemic heart disease treatment.
  • HM2002 achieved dual regulatory approval, having previously received IND clearance from China's NMPA in January 2025, making it the only circular RNA therapy approved in both countries.
  • The drug demonstrated significant cardiac function improvement without drug-related adverse events in its first-in-human trial at Ruijin Hospital.
  • HM2002 addresses critical unmet medical needs in ischemic heart disease by promoting angiogenesis through controlled VEGF expression in the myocardium.

Patient-Caregiver Preferences Study Reveals Key Treatment Decision Factors in ALK+ NSCLC

  • A discrete choice experiment with 205 ALK+ NSCLC patients and 125 caregivers found that both groups overwhelmingly prioritized 3-year progression-free survival when making treatment decisions.
  • Patients were willing to trade 3.9-8.7% of 3-year PFS to reduce risks like cognitive/mood effects, abnormal lab results, and lung complications, while caregivers showed similar but not identical risk tolerance patterns.
  • The study revealed significant differences in how patients and caregivers perceive adverse events, with caregivers ranking lab abnormalities as a major concern due to potential dose reductions, while patients prioritized quality-of-life impacts like muscle pain.
  • Researchers emphasized the importance of including both patients and caregivers in treatment discussions, as they observe different aspects of the treatment experience and have varying priorities regarding benefit-risk trade-offs.

Trump Administration Halts Major HIV Vaccine Research Programs Despite Scientific Progress

Drug Approval
  • The Trump administration has terminated funding for multiple HIV vaccine research programs, including major efforts at Duke Human Vaccine Institute and Scripps Research Institute that have been running since 2012.
  • Scientists warn the cuts represent a decade-long setback for HIV vaccine development, coming at a critical time when researchers report seeing "light at the end of the tunnel" with promising clinical trial results.
  • The decision affects 27 separate HIV/AIDS programs totaling $7.5 billion in funding, with HHS citing "duplication of efforts" as justification for prioritizing currently available HIV prevention approaches.
  • The timing coincides with the FDA's upcoming June 19 decision on lenacapavir, a twice-yearly injectable HIV prevention drug by Gilead Sciences that showed 100% effectiveness in preventing transmission.

Study of Lenacapavir for HIV Pre-Exposure Prophylaxis in People Who Are at Risk for HIV Infection

NCT04925752Active, Not RecruitingPhase 3
Gilead Sciences
Posted 6/28/2021

Machine Learning-Based Immune Risk Score Outperforms Traditional Methods in Predicting Colorectal Cancer Outcomes

  • Researchers developed an Immune Response-related Risk Score (IRRS) using machine learning and 13 immune-related genes that outperformed existing tools in predicting colorectal cancer prognosis and immunotherapy response.
  • The IRRS achieved the highest prognostic accuracy with an area under the curve of 0.861, successfully stratifying patients into high-risk and low-risk groups across multiple independent datasets.
  • Low-risk patients showed better overall survival, higher immune activity, increased immune cell infiltration, and elevated expression of immune checkpoint molecules including PDCD1, CD274, and CTLA4.
  • The multi-omics approach identified key genes like CTLA4, PDCD1, and CD274 that provide insights into tumor immunogenicity and represent potential therapeutic targets for colorectal cancer treatment.

Stage 4 Lung Cancer Patient Achieves Complete Response with Pembrolizumab Immunotherapy

  • A 76-year-old patient with stage 4 lung cancer achieved complete disease remission after two years of pembrolizumab treatment, defying typical survival statistics.
  • Pembrolizumab, a PD-1 checkpoint inhibitor, was initially deemed too expensive by the NHS at $84,000 per patient before cost negotiations with the manufacturer.
  • The case demonstrates the transformative potential of immunotherapy in advanced lung cancer, where average survival is typically less than one year.
  • The patient experienced no noticeable side effects during treatment and has maintained cancer-free status six years post-diagnosis.

Frontotemporal Dementia Therapeutics Enter New Era as Gene and Antibody Therapies Advance Toward Market

Monoclonal AntibodyOrphan DrugPrecision Medicine
  • Alector's Latozinemab (AL001) leads the frontotemporal dementia pipeline as a monoclonal antibody targeting progranulin deficiency, currently in pivotal Phase III trials with potential approval by 2026-2027.
  • Gene therapy companies AviadoBio and Passage Bio are developing AAV-based treatments AVB-101 and PBFT02 to restore progranulin expression through one-time administration, with readouts expected in late 2027 or early 2028.
  • The FTD therapeutic landscape is shifting from symptom management to disease modification, with multiple approaches including monoclonal antibodies, gene therapies, and oral small molecules targeting genetic biomarkers.
  • By 2028, the FTD market may include personalized therapies aligned with genetic profiles, potentially transforming clinical outcomes for patients aged 45-64 who currently lack disease-modifying treatment options.

HER3-DXd Demonstrates Promising Intracranial Activity in Phase II TUXEDO-3 Trial for Brain Metastases

Oncology
  • The phase II TUXEDO-3 trial showed patritumab deruxtecan (HER3-DXd) achieved intracranial response rates of 23.8% in metastatic breast cancer and 30% in advanced non-small cell lung cancer patients with active brain metastases.
  • HER3-DXd demonstrated activity across all breast cancer subtypes and showed potential to overcome resistance in patients previously treated with antibody-drug conjugates.
  • The study represents the first evaluation of intracranial and extracranial efficacy for this novel anti-cancer drug in patients with brain metastases and leptomeningeal disease from solid tumors.
  • Results from the leptomeningeal cohort showed 65% of patients remained alive after 3 months, with findings published simultaneously in Nature Medicine due to the high unmet medical need.

HER3-DXd in Breast Cancer and NSCLC Brain Metastases and Solid Tumor Leptomeningeal Disease

NCT05865990RecruitingPhase 2
MedSIR
Posted 11/24/2023

SLEIPNIR Multi-Arm Platform Receives £1 Million to Accelerate Parkinson's Drug Development in Norway

  • Cure Parkinson's has announced £1 million in funding for SLEIPNIR, a new multi-arm clinical trial platform in Norway that will test up to three potentially disease-modifying Parkinson's treatments simultaneously against one placebo group.
  • The platform, led by Professor Charalampos Tzoulis at Haukeland University Hospital and the University of Bergen, will assess drug brain penetrance and target interaction through blood and cerebrospinal fluid analysis.
  • SLEIPNIR aims to address the critical challenge of determining whether treatments reach the brain effectively, as many late-stage trials have failed due to insufficient brain penetrance.
  • The platform is designed to reduce the risk of larger clinical trial failures by evaluating multiple drugs in a smaller, shorter timeframe before advancing promising treatments to late-stage platforms like EJS ACT-PD.

Meitheal Pharmaceuticals Launches Generic Abraxane in US Market to Address Cancer Treatment Access

Oncology
  • Meitheal Pharmaceuticals has launched paclitaxel protein-bound particles for injectable suspension, a generic version of Abraxane, through an exclusive licensing agreement with parent company Hong Kong King-Friend Industry.
  • The 100mg/vial formulation is indicated for treating metastatic breast cancer, metastatic adenocarcinoma of the pancreas, and locally advanced or metastatic non-small cell lung cancer.
  • This launch expands Meitheal's generic injectable portfolio to 68 products and represents the company's 12th product launch in 2024, with 20 additional products planned for the year.
  • The generic paclitaxel aims to address treatment accessibility challenges for cancer patients facing expensive or unavailable complex therapies.

Immuron Advances Multi-Drug Pipeline with FDA Submissions and Clinical Milestones on Track for 2025

  • Immuron is on track to exceed A$7 million in sales this financial year, representing a significant increase from the previous year's A$4.9 million.
  • The company anticipates topline results from the Uniformed Services University Travelan® clinical study in October 2025, with plans to request an end-of-Phase 2 FDA meeting.
  • Immuron expects to submit an IND application to the FDA for IMM-529 targeting Clostridioides difficile infection in August 2025.
  • The company is developing IMM-986 for vancomycin-resistant enterococci with pre-clinical results anticipated in August 2025, addressing infections that cost the U.S. healthcare system over $4.6 billion annually.

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