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Clinical Trial News

Ascend Advanced Therapies Achieves GMP Certification for AAV Gene Therapy Quality Control in Munich

  • Ascend Advanced Therapies received GMP certification for Quality Control testing at its Munich facility following a joint inspection by the government of Upper Bavaria and the Paul-Ehrlich-Institute in September 2024.
  • The certification enables streamlined stability testing and release of higher quality AAV-based gene therapy products using advanced techniques including droplet digital PCR and automated ELISA.
  • The Munich facility can now perform commercial assays for DNA impurities, vector genome titer, and capsid titer, providing much-needed European capacity for AAV characterization and release.
  • Additional methods such as potency assays are planned to be added to the Munich GMP license through 2025, while GMP expansions continue in Alachua, Florida.

Cogent Biosciences Secures $400M Debt Financing to Advance Bezuclastinib Through Pivotal Trials

Precision MedicineRare Disease
  • Cogent Biosciences secured a $400 million non-dilutive debt financing facility with SLR Capital Partners, with an initial $50 million already drawn to support the company's growth strategy.
  • The company is on track to report results from three pivotal bezuclastinib trials in 2025, starting with SUMMIT results in July for NonAdvSM patients.
  • Additional funding tranches are tied to clinical milestones, with $100 million available upon successful SUMMIT and PEAK trial readouts and commercial success.
  • The financing enhances Cogent's financial flexibility as it prepares for the expected bezuclastinib launch in 2026 for genetically defined diseases.

Triplet Therapy Shows Superior Efficacy in BRAF-Mutant Melanoma Brain Metastases

Targeted Therapy
  • The triplet combination of encorafenib, binimetinib, and nivolumab achieved a median progression-free survival of 6.2 months compared to 1.5 months with nivolumab plus ipilimumab in BRAF V600-mutant melanoma brain metastases.
  • Intracranial response rates were significantly higher with the triplet regimen at 75% versus 13% with the standard immunotherapy doublet.
  • The phase 2 SWOG S2000 trial represents the first randomized study comparing treatment approaches in this challenging patient population with historically poor outcomes.

Anti-PD 1 Brain Collaboration for Patients With Melanoma Brain Metastases

NCT02374242Active, Not RecruitingPhase 2
Melanoma Institute Australia
Posted 11/4/2014

A Study to Compare the Administration of Encorafenib + Binimetinib + Nivolumab Versus Ipilimumab + Nivolumab in BRAF-V600 Mutant Melanoma With Brain Metastases

NCT04511013RecruitingPhase 2
SWOG Cancer Research Network
Posted 1/6/2021

Capstan Therapeutics Initiates First-in-Human Trial of Novel In Vivo CAR-T Therapy for Autoimmune Diseases

CAR-T
  • Capstan Therapeutics has successfully dosed the first participants in a Phase 1 trial of CPTX2309, an innovative in vivo CAR-T therapy targeting B cell-mediated autoimmune disorders.
  • CPTX2309 uses targeted lipid nanoparticles to deliver anti-CD19 CAR mRNA directly to CD8+ T cells, eliminating the need for chemotherapy, cell manipulation, or viral vectors.
  • The therapy demonstrated rapid, deep, and transient B cell depletion in preclinical studies, with B cell repopulation occurring predominantly with naïve B cells.
  • The Phase 1 trial in healthy volunteers will evaluate safety, tolerability, and pharmacodynamic activity to determine optimal dosing for future autoimmune disease studies.

Oncolytics Biotech Appoints $2B Deal Architect as CEO to Advance Pelareorep Immunotherapy

Oncology
  • Oncolytics Biotech has appointed Jared Kelly as CEO, leveraging his expertise from orchestrating the $2 billion sale of Ambrx Biopharma to Johnson & Johnson.
  • The company's lead immunotherapy pelareorep has achieved FDA Fast Track designation for metastatic pancreatic and breast cancers, with >60% objective response rates in pancreatic cancer trials.
  • Pelareorep demonstrated survival benefits 4-6 times higher than historical controls in pancreatic cancer and meaningful survival improvements in over 100 metastatic breast cancer patients.
  • The appointment aims to accelerate pelareorep's development toward registrational studies and potential strategic partnerships in the immunotherapy space.

Novo Nordisk Launches New Phase 3 Trial for CagriSema Following Mixed REDEFINE 1 Results

  • Novo Nordisk has initiated a new Phase 3 trial for CagriSema, its next-generation weight loss drug, after the previous REDEFINE 1 trial showed disappointing results with up to 23% weight loss over 68 weeks.
  • The new study will test CagriSema's efficacy in weight loss and long-term weight maintenance in approximately 600 adults with obesity over a three-year period.
  • The trial officially began on June 3 and is structured in two parts, with the primary endpoint measuring percentage change in body weight over 80 weeks.
  • Novo Nordisk stock rallied following the announcement as investors view this as another opportunity to demonstrate CagriSema's potential in the competitive obesity treatment market.

Cantargia's Nadunolimab Receives FDA Fast Track Designation for Metastatic Pancreatic Cancer

Monoclonal AntibodyOncology
  • The FDA has granted Fast Track Designation to nadunolimab, Cantargia's anti-IL1RAP antibody, for treating previously untreated metastatic pancreatic ductal adenocarcinoma with high IL1RAP expression levels.
  • The designation follows strong clinical data from the CANFOUR study showing 35% two-year survival, 14.2 months overall survival, and 48% overall response rate in patients with high IL1RAP expression.
  • Fast Track Designation provides benefits including more frequent FDA meetings, eligibility for Accelerated Approval and Priority Review, and rolling review opportunities for the Biologic License Application.
  • The recognition addresses the high unmet medical need in metastatic pancreatic cancer, where current treatment options are limited and overall survival after first-line treatment is typically less than 12 months.

Nadunolimab in Combination with Gemcitabine Plus Carboplatin in Patients with Advanced Triple Negative Breast Cancer.

NCT05181462Active, Not RecruitingPhase 1
Cantargia AB
Posted 1/11/2022

A First-in-Human Study of CAN04 in Patients With Solid Malignant Tumors

NCT03267316CompletedPhase 1
Cantargia AB
Posted 9/19/2017

IQVIA and NVIDIA Launch AI Orchestrator Agents to Accelerate Clinical Research and Drug Development

AI
  • IQVIA announced the launch of multiple AI orchestrator agents in collaboration with NVIDIA at GTC Paris, designed to manage and accelerate complex pharmaceutical development workflows for thousands of global customers.
  • The AI agents can reduce clinical trial start-up processes from 200 days and clinical data review from seven weeks to as little as two weeks through automated workflows and specialized sub-agents.
  • The orchestrator agents utilize NVIDIA NIM microservices and IQVIA's petabytes of healthcare data to streamline target identification, clinical site start-up, and drug commercialization processes.
  • The technology enables pharmaceutical companies to focus on decision-making rather than time-consuming administrative tasks while providing comprehensive market analysis in days instead of weeks.

Memo Therapeutics Demonstrates Therapeutic Antibodies Can Cross Kidney Barrier, Supporting BK Virus Treatment Approach

Monoclonal Antibody
  • Memo Therapeutics published research in Frontiers in Pharmacology showing that intravenously administered therapeutic IgG antibodies can cross the kidney endothelial barrier and be detected in urine.
  • The study found that 0.015% (median) of serum rituximab concentration appears in urine, with levels reaching up to 4.2%, challenging previous assumptions about size-exclusion limitations.
  • These findings provide scientific support for the dosing strategy of potravitug, MTx's investigational anti-BK polyomavirus antibody currently in Phase II trials for kidney transplant recipients.
  • BKV nephropathy affects up to 70% of kidney transplant recipients with established BK viremia and is associated with compromised graft function and reduced long-term survival.

Israeli Researchers Develop Bioengineered Skin Graft That Accelerates Burn Healing by 50%

  • Researchers from Tel Aviv University and Sheba Medical Center have developed a bioengineered skin graft using patients' own cells that heals burns in half the time of standard treatments.
  • The innovative graft achieved wound closure in just four days compared to eight days with conventional methods in animal models, while also promoting hair follicle growth.
  • The technology uses FDA-approved nanofiber scaffolds that allow cells to self-organize into natural skin structure without requiring animal-derived materials or causing tissue shrinkage.
  • The breakthrough was driven by urgent clinical needs arising from treating numerous burn victims, including soldiers injured in recent conflicts.

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