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Clinical Trial News

NextCell Pharma Completes Patient Dosing in Phase II ProTrans-Young Trial for Pediatric Type 1 Diabetes

  • NextCell Pharma has successfully completed treatment of all patients in the ProTrans-Young Phase II clinical trial, marking the completion of patient dosing in the company's largest clinical study to date.
  • The trial evaluates ProTrans, an allogeneic mesenchymal stromal cell therapy, in 66 pediatric and adolescent patients with recently diagnosed type 1 diabetes across three age cohorts.
  • One-year follow-up results from the 12-21 years age group showed a positive trend in preserved insulin production, with full study population data expected after summer 2026.
  • ProTrans is being developed as an off-the-shelf, single-dose infusion therapy aimed at modulating the immune system and preserving residual beta cell function in autoimmune diabetes.

Vial Doses First Participants in Phase 1 Trial of Half-Life Extended Anti-TL1A Antibody for IBD Treatment

Monoclonal Antibody
  • Vial has successfully dosed the first healthy volunteers in a Phase 1 trial of VIAL-TL1A-HLE, a novel subcutaneous monoclonal antibody targeting TL1A for inflammatory bowel disease treatment.
  • The first-in-human, single-ascending dose trial will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of the half-life extended antibody with interim data expected in H2 2025.
  • Preclinical studies demonstrate the antibody has potential for best-in-class dosing intervals compared to first and second-generation anti-TL1A candidates, offering improved patient convenience.
  • Vial plans to initiate a multi-region Phase 2 trial in IBD later in 2025 following the interim Phase 1 results.

Panavance Therapeutics Reports Promising Phase 1 Results for Misetionamide in Pancreatic Cancer

Oncology
  • Panavance Therapeutics announced interim Phase 1 results for misetionamide (GP-2250) combined with gemcitabine in advanced pancreatic cancer patients.
  • The combination demonstrated a 42% response rate with partial response or stable disease in 55 enrolled patients across seven US sites.
  • Misetionamide showed excellent tolerability with no new toxicities compared to gemcitabine alone, targeting multiple oncogenic transcription factors including c-MYC and NFκB.
  • The company plans additional trials in platinum-resistant ovarian cancer and first-line maintenance therapy for non-BRCA mutated pancreatic cancer patients.

UCB Announces $5 Billion U.S. Biologics Manufacturing Investment to Strengthen Supply Chain

FDA Approval
  • UCB plans to invest $5 billion in a new state-of-the-art biologics manufacturing facility in the United States, creating 300 permanent high-skilled jobs and over 500 construction positions.
  • The investment reflects UCB's commitment to serving its growing U.S. patient base and building a more resilient supply chain for immunology and central nervous system disease treatments.
  • Since 2017, UCB has expanded its U.S. workforce by 73% to around 2,000 employees and achieved 15 FDA approvals, with eight coming in the past two years alone.

FDA Places Clinical Hold on Rocket Pharmaceuticals' Gene Therapy Trial After Patient Death

Rare Disease
  • The FDA placed a clinical hold on Rocket Pharmaceuticals' Phase 2 pivotal trial for RP-A501, a gene therapy for Danon disease, following a patient death from a serious adverse event.
  • Rocket Pharmaceuticals' stock price crashed over 60% on May 27, 2025, after the company disclosed the FDA's clinical hold and revealed undisclosed protocol amendments.
  • A securities class action lawsuit has been filed alleging the company failed to properly disclose safety risks and protocol changes, including the addition of a novel immunomodulatory agent.
  • The lawsuit claims Rocket Pharmaceuticals made materially false statements about RP-A501's safety profile and trial protocol while knowing serious adverse events were at risk.

Citius Pharmaceuticals Closes $15.8M Direct Offering to Fund LYMPHIR Commercial Launch

Drug ApprovalOncology
  • Citius Pharmaceuticals completed a $15.8 million registered direct offering with $6 million raised upfront and potential additional $9.8 million from warrant exercises.
  • The company plans to use proceeds to support the commercial launch of LYMPHIR, an FDA-approved targeted immunotherapy for cutaneous T-cell lymphoma.
  • The offering included 4.92 million shares at $1.22 per share with accompanying warrants exercisable at $1.00 per share over 24 months.
  • Citius maintains a late-stage pipeline including Mino-Lok for catheter-related infections and CITI-002 for hemorrhoid treatment, both having completed pivotal trials.

Quest Diagnostics Partners with MD Anderson to Develop Multi-Cancer Risk Blood Test for 2026 Launch

  • Quest Diagnostics has partnered with The University of Texas MD Anderson Cancer Center to develop a blood test that identifies elevated cancer risk across nine cancer types using circulating protein biomarkers.
  • The Multi-Cancer Stratification Test (MCaST) technology is based on extensive clinical research involving tens of thousands of individuals and aims to supplement conventional screening methods.
  • The test is designed to be more affordable and accessible than existing multi-cancer early detection liquid biopsies, with potential commercialization in North America by 2026.
  • Only 51% of U.S. adults have had routine cancer screening in the past year, highlighting the need for more convenient and cost-effective screening approaches.

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.

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