MedPath

Clinical Trial News

Daewoong Pharmaceutical Launches NABOTA Botulinum Toxin in Qatar, Completing Gulf Market Expansion

  • Daewoong Pharmaceutical officially launched NABOTA, its high-purity botulinum toxin product, in Qatar through a symposium attended by approximately 200 local healthcare professionals.
  • The launch completes NABOTA's availability across all three key Gulf Cooperation Council markets—Qatar, Saudi Arabia, and the UAE—targeting high-income populations with growing demand for aesthetic procedures.
  • NABOTA features over 98% purity of the 900kDa complex and is positioned to compete with existing products like AbbVie's Botox in the premium Qatar market.
  • The company plans to expand local engagement through strategic partnerships and awareness campaigns featuring its proprietary NABOlift microinjection technique for wrinkle reduction and facial contouring.

Medera Showcases Human Mini-Heart Technology Breakthroughs at ISSCR 2025, Advancing Gene Therapy Clinical Trials

FDA Approval
  • Medera and Novoheart presented seven scientific abstracts at ISSCR 2025, featuring their proprietary human mini-Heart technology that has informed FDA approvals for first-in-human gene therapy trials.
  • The company's human-based cardiac tissue platform received FDA recognition as an animal-free alternative under the FDA Modernization Act 2.0, supporting IND and Fast Track designations for heart failure treatments.
  • Breakthrough technologies demonstrated include high-throughput screening automation that reduces cell use by over 90% and AI-driven drug classification algorithms that outperform conventional approaches.
  • The platform has directly supported three ongoing clinical trials targeting different forms of heart failure, including HFpEF and HFrEF, with gene therapies SRD-001 and SRD-002.

Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Preserved Ejection Fraction

NCT06061549RecruitingPhase 1
Sardocor Corp.
Posted 8/24/2023

Modulation of SERCA2a of Intra-myocytic Calcium Trafficking in Heart Failure With Reduced Ejection Fraction

NCT04703842RecruitingPhase 1
Sardocor Corp.
Posted 9/23/2021

WHO's mRNA Technology Transfer Programme Enters Phase 2.0, Targeting Commercial-Scale Production in Low- and Middle-Income Countries

  • The WHO and Medicines Patent Pool's mRNA Technology Transfer Programme is transitioning to Phase 2.0 (2026-2030), moving from proof of concept to commercially sustainable manufacturing in 15 partner countries across Latin America, Africa, Eastern Europe and Asia.
  • The initiative aims to empower regional manufacturers to scale up GMP-grade production of mRNA vaccines for pandemic and priority diseases including influenza, TB, HIV, malaria, dengue, and leishmaniasis, as well as mRNA therapeutics for oncology applications.
  • Several manufacturers including Bio-Manguinhos, Sinergium, BioFarma, and Biovac are already piloting investment roadmaps with detailed market and regulatory modeling, while Afrigen Biologics is approaching GMP accreditation.
  • The programme faces structural challenges including misinformation, vaccine hesitancy, shifting donor funding priorities, high clinical trial costs, and the need for supportive procurement policies to ensure sustainability.

MRD Testing Emerges as Key Decision Tool for Post-Transplant Multiple Myeloma Management

Precision Medicine
  • MRD testing is gaining clinical importance for treatment decisions following autologous stem cell transplant in multiple myeloma patients, with FDA approval as an endpoint.
  • High-risk patients who fail to achieve MRD negativity after transplant require treatment intensification, while those with sustained MRD negativity may be candidates for treatment discontinuation.
  • Sustained MRD negativity over 2-4 years offers potential for treatment-free intervals, providing both clinical benefits for patients experiencing adverse effects and economic advantages for healthcare systems.

BCMA-Targeted Bispecific Antibody CM336 Shows Promise for Refractory Autoimmune Hemolytic Anemia in NEJM Study

  • Keymed Biosciences' CM336, a BCMA x CD3 bispecific antibody, achieved rapid partial remission in two patients with refractory autoimmune hemolytic anemia within 13-19 days of treatment.
  • Both patients maintained sustained remission for over six months without requiring immunosuppressive therapies or transfusions, marking the first global report of BCMA-targeted therapy for this indication.
  • The treatment demonstrated excellent safety with no cytokine release syndrome, neurotoxicity, or infections during the entire treatment and follow-up period.
  • CM336 represents a potential breakthrough for patients with relapsed/refractory AIHA who have exhausted multiple conventional therapies including CAR-T cell treatments.

Dry Eye Disease Affects 16.4 Million Americans as New Targeted Therapies Enter Development Pipeline

  • Dry eye disease affects approximately 16.4 million diagnosed Americans with an additional 6 million experiencing undiagnosed symptoms, creating substantial economic burden exceeding $55 billion annually in indirect costs alone.
  • Current treatment approaches follow a staged management strategy from artificial tears to prescription anti-inflammatory agents like cyclosporine and lifitegrast, though significant gaps remain in symptom relief and patient adherence.
  • Multiple investigational therapies are in development targeting various pathophysiologic mechanisms including tear production enhancement, inflammation reduction, and evaporation prevention to address current treatment limitations.

RadioMedix Launches RAHA-100 Bench Top Generator to Expand Lead-212 Supply for Targeted Alpha Therapy

Oncology
  • RadioMedix has launched the RAHA-100, a proprietary bench top generator designed to provide on-demand supply of Lead-212 isotope for targeted alpha therapy research and clinical development.
  • The generator features a fully automated process with multiple high-activity modular columns and proprietary purification steps, making Lead-212 more accessible compared to earlier-generation systems requiring specialized facilities.
  • Lead-212 offers potential advantages over conventional beta-emitting isotopes, including a shorter half-life and alpha-emitting profile that may enable more selective tumor cell destruction while limiting exposure to healthy tissue.
  • The technology aims to democratize Lead-212 availability and accelerate development of targeted alpha therapies for hard-to-treat cancers, addressing historical supply limitations that have restricted clinical adoption.

Novel Therapies Show Promise for Idiopathic Pulmonary Fibrosis and Progressive Pulmonary Fibrosis Treatment

  • An LPA1 receptor antagonist demonstrated preservation of lung function in phase 2 trials, showing 1.4% to 3.2% less decline in forced vital capacity compared to placebo over 26 weeks.
  • A selective oral integrin inhibitor targeting α_v_β_6 and α_v_β_1 showed initial improvements in lung function but was halted due to adverse event imbalances.
  • Inhaled prostaglandins, originally developed for pulmonary arterial hypertension, are being evaluated in phase 3 trials as add-on therapy for progressive pulmonary fibrosis.
  • Current IPF and PPF management emphasizes early intervention, multidisciplinary collaboration, and proactive management of antifibrotic therapy side effects to improve patient adherence.

FDA Approves First-in-Human Trial for Novel CAR-T Therapy Targeting Neuroblastoma

CAR-TDrug Approval
  • The FDA has approved Myrio Therapeutics' IND application for PHOX2B PC-CAR T, marking the first human trial for a therapy targeting the PHOX2B protein in neuroblastoma cells.
  • The novel CAR-T therapy breaks HLA restriction by recognizing peptides across multiple HLA allotypes, potentially expanding treatment to a broader patient population.
  • Neuroblastoma affects approximately 800 children annually in the US and accounts for 15% of pediatric cancer deaths, with current high-risk treatments showing low response rates.
  • The Phase 1 trial will be led by Prof. John Maris at a leading Philadelphia children's hospital, with first patient enrollment anticipated for mid-2025.

Large Real-World Study Finds No Increased Cancer or Cardiovascular Risk with JAK Inhibitors in Rheumatoid Arthritis

Real World Evidence
  • A comprehensive analysis of 53,169 treatment initiations across 13 international registries found no significantly higher cancer risk in rheumatoid arthritis patients treated with JAK inhibitors compared to biologic DMARDs.
  • Separate cardiovascular safety data from 51,233 patients showed JAK inhibitors did not increase major adverse cardiovascular events compared to TNF inhibitors, with rates of 6.97 versus 7.57 per 1,000 person-years respectively.
  • The findings provide reassuring real-world evidence addressing previous safety concerns raised by the ORAL Surveillance trial, though increased keratinocyte cancer risk remains a consideration for JAK inhibitor therapy.
  • GLP-1 receptor agonists showed potential cardioprotective effects in RA patients on JAK inhibitors, with significantly lower rates of acute coronary syndromes and deep venous thrombosis.

MedPath

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

© 2025 MedPath, Inc. All rights reserved.