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Clinical Trial News

FDA Grants Breakthrough Therapy Designation to Avidity's Del-zota for Duchenne Muscular Dystrophy

Orphan DrugRare Disease
  • The FDA has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for treating Duchenne muscular dystrophy in patients with mutations amenable to exon 44 skipping.
  • Del-zota demonstrated statistically significant increases in exon skipping, substantial dystrophin production increases, and significant creatine kinase reduction to near-normal levels in Phase 1/2 trials.
  • Avidity Biosciences remains on track for a planned Biologics License Application submission at year-end 2025, with topline data from the ongoing Phase 2 extension trial expected in Q4 2025.
  • The therapy represents a novel Antibody Oligonucleotide Conjugate approach that combines monoclonal antibody specificity with oligonucleotide precision to deliver treatment directly to muscle tissue.

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

NCT06244082Active, Not RecruitingPhase 2
Avidity Biosciences, Inc.
Posted 1/22/2024

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730CompletedPhase 1
Avidity Biosciences, Inc.
Posted 11/9/2022

Global Clinical Trials Landscape for Alopecia Universalis Reveals Growing Research Activity Across Major Pharmaceutical Companies

  • A comprehensive 2025 clinical trials review reveals the current global research landscape for alopecia universalis, analyzing trial distribution across regions, phases, and sponsor types.
  • Major pharmaceutical companies including Pfizer, Johnson & Johnson, Roche, and Aclaris Therapeutics are actively participating in alopecia universalis therapeutic development programs.
  • The report provides critical insights into trial enrollment trends over the past five years and identifies prominent drugs currently in development for this severe autoimmune hair loss condition.
  • Clinical trial data is compiled from over 80 different registries worldwide, offering stakeholders comprehensive market intelligence for strategic decision-making in dermatology therapeutics.

Apollo Hospitals and Siemens Healthineers Launch AI-Driven Research Collaboration for Liver Disease Diagnostics in India

AI
  • Apollo Hospitals and Siemens Healthineers have established a research collaboration to develop AI-enabled diagnostic and imaging solutions for liver disease management, focusing on quantitative ultrasound imaging and clinical decision support tools.
  • The partnership specifically targets Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), which affects 9-32% of India's population and poses significant diagnostic challenges due to its underestimated prevalence.
  • The collaboration aims to enhance early detection of liver fibrosis through AI-driven disease progression models, enabling timely interventions and cost-effective screening for improved patient outcomes.
  • A Master Research Agreement was formalized in March 2025, with an addendum signed in July 2025 establishing the framework for collaborative research on diagnostic and interventional ultrasound imaging for liver care.

Eisai Launches Beova Tablets for Overactive Bladder Treatment in Thailand

Drug Approval
  • Eisai Thailand has launched Beova® Tablets (vibegron), a selective β3-adrenergic receptor agonist for overactive bladder treatment, marking the first market entry in Eisai's licensed ASEAN region.
  • The once-daily oral medication targets β3 receptors in the bladder to improve symptoms of urgency, frequent urination, and urge incontinence in patients with overactive bladder.
  • With overactive bladder affecting 15.8% of Thailand's population, the launch provides a new treatment option alongside Eisai's existing Uritos® tablets for addressing this condition that significantly impacts quality of life.
  • Approvals have also been obtained in the Philippines and Malaysia, with market preparations underway for these territories following the successful Thailand launch.

Crown Bioscience San Diego Receives CLIA Certification to Expand Clinical Testing Capabilities

Oncology
  • Crown Bioscience's San Diego laboratory has achieved Clinical Laboratory Improvement Amendments (CLIA) certification from the Centers for Medicare and Medicaid Services, enabling clinical-grade testing for drug development.
  • The certification allows the contract research organization to conduct clinical testing on human specimens, strengthening its position in oncology research and personalized medicine.
  • Crown Bioscience operates the world's largest commercially available patient-derived xenograft collection and approximately 1,000 tumor organoid models across 35 cancer indications.
  • The CLIA designation enhances the company's ability to support clients' clinical development and regulatory approval timelines with compliant biomarker data.

ILUVIEN Shows Mixed Results in NEW DAY Trial for Diabetic Macular Edema Treatment

  • ANI Pharmaceuticals' NEW DAY trial of ILUVIEN for diabetic macular edema failed to meet its primary endpoint of reducing supplemental aflibercept injections compared to aflibercept alone (2.4 vs. 2.5 injections, p=0.756).
  • A post-hoc analysis of patients without protocol deviations showed ILUVIEN significantly reduced supplemental injections (1.8 vs. 2.5, p=0.029), resulting in fewer total injections over 18 months.
  • ILUVIEN demonstrated a statistically significant longer time to first supplemental injection (185.4 vs. 132.8 days, p<0.001) and showed non-inferiority in visual acuity outcomes.
  • The safety profile was consistent with previous studies, with 41% of ILUVIEN patients experiencing treatment-related adverse events, primarily cataracts and increased intraocular pressure.

BD Libertas Wearable Injector Enters First Pharma-Sponsored Clinical Trial for Complex Biologics Delivery

  • BD announces the first pharma-sponsored combination product clinical trial using its BD Libertas™ Wearable Injector for subcutaneous delivery of complex biologics.
  • The selection follows successful outcomes from more than 50 BD-conducted pre-clinical and clinical studies, with 100% of participants stating they would likely use the device if prescribed.
  • The wearable injector supports high-viscosity biologics up to 50 centipoise and offers 2-10 mL configurations, potentially enabling conversion from hospital-based infusions to home-based self-injection.
  • The biologics market is projected to exceed $670 billion by 2030, positioning this drug delivery innovation as a significant advancement for patient-centric biologic therapy administration.

Bio Usawa and ServareGMP Partner to Establish Monoclonal Antibody Manufacturing in Africa

Monoclonal Antibody
  • Bio Usawa Biotechnology and ServareGMP announced a strategic partnership to build advanced monoclonal antibody manufacturing capabilities in Africa, addressing critical healthcare access gaps in low- and middle-income countries.
  • The collaboration focuses on four core pillars: local biomanufacturing infrastructure, workforce development, process innovation for cost reduction, and emergency response capabilities for pandemics and health crises.
  • The partnership aims to fundamentally restructure how biotherapeutics are developed, produced, and delivered in LMICs by investing in local talent, infrastructure, and innovation to ensure equitable access to life-saving therapies.

Dispatch Bio Emerges with $216M to Develop Universal Solid Tumor Immunotherapy

CAR-T
  • Dispatch Bio, a new biotech company formed through collaboration between Arch Venture Partners and the Parker Institute for Cancer Immunotherapy, has raised $216 million to develop a universal solid tumor treatment approach.
  • The company's lead therapy combines gene therapy, immunotherapy, and cell therapy by using engineered viruses to tag cancer cells with a synthetic "flare" antigen that CAR-T cells can then target and destroy.
  • Founded by renowned researchers including University of Pennsylvania's Carl June and Stanford's Chris Garcia, the approach aims to overcome current limitations of checkpoint inhibitors and CAR-T therapies in treating solid tumors.
  • The therapy is expected to enter clinical testing next year and could potentially address the challenge that many "cold" tumors pose to existing immunotherapies.

Ghana FDA and Company Deny COA-72 HIV Clinical Trial Claims Following Media Misinformation

  • Ghana's Food and Drugs Authority officially denied approving any clinical trials for COA-72 as an anti-HIV drug, calling media reports false and dangerous to public health.
  • COA Research and Manufacturing Limited Company clarified that COA-72 has not entered FDA-approved clinical trials, despite showing promising results in pre-clinical studies including reduced HIV viral loads.
  • The company applied for clinical trial approval in 2019 but has not yet fulfilled all FDA requirements, while estimating the product could contribute $76.8 billion annually to Ghana's economy if validated.

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