MedPath

Clinical Trial News

AstraZeneca's Anselamimab Fails Primary Endpoint in Phase III AL Amyloidosis Trial, Shows Promise in Patient Subgroup

Monoclonal AntibodyRare DiseaseOrphan Drug
  • AstraZeneca's CARES Phase III clinical programme evaluating anselamimab in patients with Mayo stages IIIa and IIIb light chain amyloidosis did not achieve statistical significance for the primary endpoint of time to all-cause mortality and cardiovascular hospitalizations.
  • Despite missing the primary endpoint in the overall population, anselamimab demonstrated highly clinically meaningful improvement in survival and cardiovascular hospitalization rates in a prespecified patient subgroup compared to placebo.
  • The investigational anti-fibril monoclonal antibody represents a potentially first-in-class approach to clearing amyloid deposits in AL amyloidosis, a rare and life-threatening disorder affecting an estimated 74,000 patients worldwide.
  • Anselamimab was well tolerated with balanced adverse events between treatment and placebo arms, and Alexion plans to share full results with global health authorities and present data at upcoming medical meetings.

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

NCT04512235Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 11/3/2020

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

NCT04504825Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 2/2/2021

A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis

NCT04304144CompletedPhase 2
Alexion Pharmaceuticals, Inc.
Posted 3/18/2020

Pragmatic Clinical Trials Bridge Gap Between Research and Real-World Healthcare Practice

Real World Evidence
  • Pragmatic clinical trials are designed to evaluate treatment effectiveness under routine clinical practice conditions, addressing the gap between traditional controlled trials and real-world care complexities.
  • Real-world evidence from electronic health records and registries enhances research by providing data from actual healthcare experiences outside controlled environments.
  • Recent developments challenge the conventional explanatory-pragmatic continuum, advocating for considering pragmatism and explanatorism as distinct but complementary dimensions in trial design.
  • Digital health innovations and alternative data collection techniques are facilitating the incorporation of real-world data into randomized designs to improve clinical applicability.

McMaster Study Identifies Most Effective Treatments for Chronic Hives Through Comprehensive Meta-Analysis

Monoclonal Antibody
  • A McMaster University-led international study analyzed 93 randomized controlled trials involving over 11,000 participants to identify the most effective treatments for chronic hives.
  • Omalizumab and remibrutinib emerged as the most effective treatments for reducing hives, itch, and swelling with favorable safety profiles.
  • Dupilumab showed particular promise for reducing hives, while cyclosporine demonstrated effectiveness but carried higher risks of side effects including kidney toxicity.
  • The comprehensive network meta-analysis examined more than 40 treatment options and will inform new international clinical guidelines for chronic urticaria management.

FDA Issues Warning Letters to Seven Companies Marketing Illegal Opioid Alternative Products Containing 7-Hydroxymitragynine

  • The FDA issued warning letters to seven companies illegally marketing products containing concentrated 7-hydroxymitragynine (7-OH), a compound found naturally in trace amounts in kratom.
  • These concentrated 7-OH products, including tablets, gummies, drink mixes, and shots, are being sold online and in retail outlets without FDA approval or proven safety.
  • The agency warns that 7-OH is not permitted in dietary supplements or conventional foods, and no FDA-approved drugs contain this compound.
  • Companies have 15 business days to respond and must cease marketing these products that pose potential safety risks to consumers.

WHO Includes ArkBio's Ziresovir in Pediatric RSV Drug Priority List, Marking First Chinese Anti-RSV Drug Recognition

  • The World Health Organization has included ziresovir (10mg) from Shanghai Ark Biopharmaceutical in its PADO-RSV priority list, making it the first and only RSV antiviral drug selected for the program.
  • RSV causes approximately 33 million lower respiratory tract infections, 3.6 million hospitalizations, and 101,400 deaths annually in infants and young children globally, with 97% of deaths occurring in low- and middle-income countries.
  • Ziresovir is a novel small-molecule RSV fusion protein inhibitor that has completed phase 3 pivotal clinical studies with positive results published in The New England Journal of Medicine and The Lancet Child & Adolescent Health.
  • ArkBio plans to expand ziresovir's clinical development globally and formulate accessibility plans for low- and middle-income countries to ensure reasonable pricing worldwide.

BTX A51 Shows Promise in Relapsed/Refractory AML with Notable Activity in RUNX1-Mutated Patients

  • BTX A51, an oral multi-kinase inhibitor targeting CK1α and CDK7/9, demonstrated tolerability and manageable safety in a phase I study of 31 patients with relapsed/refractory AML or MDS.
  • Three patients (10%) achieved complete remission with incomplete count recovery, with all responders harboring RUNX1 mutations and receiving doses of 11 mg or higher.
  • The study established 21 mg administered 3 days per week for 28 days as the recommended phase 2 dose, with encouraging activity particularly in RUNX1-mutated patients showing a 30% response rate.
  • Ex vivo studies demonstrated synergistic effects when BTX A51 was combined with venetoclax and azacitidine, supporting future combination therapy trials.

IO-202 as Monotherapy and IO-202 Plus Azacitidine ± Venetoclax in Patients in AML and CMML

NCT04372433CompletedPhase 1
Immune-Onc Therapeutics
Posted 9/14/2020

A Study of BTX-A51 in People With Advanced Solid Tumor and Breast Cancer

NCT04872166RecruitingPhase 1
Edgewood Oncology Inc.
Posted 6/7/2021

Expert Calls for Standardized Reflexive Biomarker Testing to Address Treatment Delays in Lung Cancer

Precision Medicine
  • Dr. Robert Kratzke from the University of Minnesota warns that delays in molecular testing for non-small cell lung cancer patients can lead to treatment lags that cost lives.
  • The expert advocates for making reflexive biomarker testing standard of care, requiring pathologists to automatically send lung cancer biopsy samples for genomic testing and immunohistochemistry.
  • Policy changes at the American College of Pathology level and insurer requirements are needed to ensure every NSCLC patient receives comprehensive biomarker testing regardless of geographic location.
  • Real-world data and AI integration into electronic health records could help reduce disparities in access to precision oncology treatments, particularly for underserved populations.

Aptar Pharma Acquires Mod3 Pharma's Clinical Trial Manufacturing Capabilities to Strengthen Early-Phase Drug Development Services

  • Aptar Pharma acquired clinical trial materials manufacturing capabilities from Mod3 Pharma to strengthen support for Phase 1 and 2 drug development programs.
  • The acquisition provides cGMP fill and finish services for orally inhaled nasal drug products, addressing a largely unmet market need.
  • Aptar will operate an FDA-inspected facility in Boonton, New Jersey, featuring cGMP cleanrooms, high-potency API suites, and biologics capabilities.
  • The strategic move accelerates adoption of new drug delivery technologies across multiple therapeutic areas including dermal, ophthalmic, and injectable solutions.

Repare Therapeutics Licenses PKMYT1 Inhibitor Lunresertib to Debiopharm in $267M Deal

  • Repare Therapeutics has entered into an exclusive worldwide licensing agreement with Debiopharm for lunresertib, a first-in-class PKMYT1 inhibitor targeting difficult-to-treat solid tumors.
  • The deal includes $10 million upfront payment and up to $257 million in potential milestones, with Debiopharm taking over development activities and the MYTHIC study.
  • Lunresertib has shown promising Phase 1/1b clinical data, particularly in combination with Debio 0123, a WEE1 inhibitor, demonstrating potential for rapid tumor regressions.
  • Repare will focus on advancing its remaining pipeline with Phase 1 trials POLAR (RP-3467 Polθ inhibitor) and LIONS (RP-1664 PLK4 inhibitor), with readouts expected in Q3 and Q4 2025.

Compremium Launches First-in-Human Study of Non-Invasive Thyroid Cancer Diagnostic Device

  • Swiss medtech company Compremium has initiated a first-in-human clinical study testing a novel non-invasive diagnostic device that measures tissue compressibility to distinguish between benign and malignant thyroid nodules.
  • The 18-month study will enroll up to 30 patients with Bethesda IV thyroid nodules at Bern University Hospital, targeting a patient population where only 25-40% of nodules are ultimately cancerous despite many undergoing precautionary surgery.
  • The CPMX2 system aims to reduce unnecessary thyroidectomies by providing real-time visualization of tissue stiffness, potentially addressing the current 75% false-positive rate in Bethesda IV nodule classification.
  • Compremium's FDA-cleared technology has been validated in over 40 clinical studies and shows promise across more than 30 medical indications beyond thyroid diagnostics.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.