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Clinical Trial News

MD Anderson Partners with TOPPAN Holdings to Develop Organoid-Based Personalized Cancer Treatment Platform

  • MD Anderson Cancer Center and TOPPAN Holdings announced a strategic alliance to advance invivoid™ 3D cell culture technology for personalized cancer treatments and drug screening.
  • The collaboration aims to obtain CAP/CLIA certification for an organoid-based assay that can evaluate cancer therapy effectiveness on patient-derived samples across multiple cancer types.
  • TOPPAN Holdings will provide approximately $10 million over five years to support joint research activities and regulatory certification efforts.
  • The technology enables rapid establishment of organoid models directly from patient biopsies to test various treatments and predict patient responses before therapy begins.

FDA Official Dr. Vinay Prasad Resigns After Three Months Amid Sarepta Gene Therapy Controversy

Drug Approval
  • Dr. Vinay Prasad resigned from his FDA leadership position after less than three months following criticism from right-wing activists and pressure from the White House.
  • His departure was linked to ongoing controversy over Sarepta Therapeutics' Duchenne muscular dystrophy drug Elevidys, which he had previously criticized for insufficient evidence.
  • The FDA recently requested Sarepta halt shipments of Elevidys after a reported death in Brazil, then reversed course to allow resumed shipments just before Prasad's resignation.
  • Prasad also faced backlash for overruling FDA scientists on COVID-19 vaccine approvals, limiting recommendations to older and immunocompromised patients only.

Cellipont Bioservices Expands Partnership with CellVax for Personalized GI Cancer Immunotherapy Manufacturing

  • Cellipont Bioservices and CellVax Therapeutics have expanded their collaboration to advance cGMP manufacturing of FK-GI101, a personalized autologous immunotherapy targeting gastrointestinal cancers including gastric, pancreatic, and colon cancer.
  • FK-GI101 utilizes patients' own tumor cells modified to express MHC Class II molecules, enhancing immune system recognition and creating a personalized immune response against residual or recurring tumor cells.
  • The partnership encompasses comprehensive manufacturing support including drug substance and drug product technology transfer, GMP facility preparation, and scaling capabilities to bring this innovative cell therapy to clinical trials.
  • This collaboration addresses urgent unmet medical needs in gastrointestinal oncology by combining Cellipont's manufacturing excellence with CellVax's proprietary tumor cell modification platform.

Kyowa Kirin to Present Phase 3 Rocatinlimab Results for Atopic Dermatitis at EADV 2025

Monoclonal Antibody
  • Kyowa Kirin will present results from the Phase 3 ROCKET IGNITE trial of rocatinlimab, an investigational anti-OX40 monoclonal antibody for moderate-to-severe atopic dermatitis, at the EADV 2025 Annual Meeting in Paris.
  • Rocatinlimab targets the OX40 receptor and has the potential to be the first T-cell rebalancing therapy that inhibits pathogenic T cells responsible for atopic dermatitis inflammation.
  • The ROCKET program comprises eight Phase 3 studies evaluating rocatinlimab's safety and efficacy in adults and adolescents with moderate to severe atopic dermatitis across multiple dosing regimens.
  • Atopic dermatitis affects 15-20% of children and up to 10% of adults, with more than half of moderate to severe patients experiencing severe itching that disrupts daily life.

Blood-Based Assay Detects Immunotherapy Response Up to 5 Months Earlier Than Standard Imaging

Precision Medicine
  • The RADIOHEAD study validated Guardant Reveal, a methylation-based liquid biopsy assay, for earlier detection of immunotherapy response in over 500 patients with advanced solid tumors across multiple cancer types.
  • Patients with an 80% or greater decrease in circulating tumor DNA showed a 75% lower risk of disease progression, while the assay identified treatment failure up to 5 months before standard imaging methods.
  • The tissue-free monitoring technology could enable oncologists to make faster treatment decisions, potentially sparing patients from ineffective therapies and improving clinical outcomes in immunotherapy management.

Diorasis Therapeutics Partners with Northway Biotech to Advance AAV Gene Therapy for Open Angle Glaucoma

  • Diorasis Therapeutics has partnered with Northway Biotech to establish scalable cGMP production for its lead AAV-based gene therapy candidate targeting open angle glaucoma.
  • The collaboration includes AAV process development, analytical method development, and manufacturing of pre-clinical and clinical materials for anticipated trials.
  • Diorasis' lead candidate DT003 delivers a modified tissue plasminogen activator gene to restore physiological aqueous humor outflow through a single intracameral injection.
  • In preclinical models including mice and sheep, DT003 demonstrated durable intraocular pressure reduction to normal levels with potential broad applicability.

Nota AI Receives Fast-Track KOSDAQ Approval for AI Optimization Technology IPO

AI
  • Nota AI secured preliminary KOSDAQ listing approval in just two months, positioning to become the first AI optimization company on the Korean exchange amid challenging market conditions.
  • The company's proprietary NetsPresso® platform has established partnerships with global semiconductor leaders including NVIDIA, Samsung Electronics, and Qualcomm for AI model optimization.
  • Nota AI achieved nearly tenfold revenue growth over five years and projects 72% year-over-year growth in 2025, with expansion into industrial safety, surveillance, and transportation systems.
  • The company became the first Korean firm to deliver generative AI solutions to a Middle East government agency through its Dubai RTA contract in April 2025.

FDA Grants Orphan Drug Designation to COAGADEX for Rare Bleeding Disorder Associated with AL Amyloidosis

Rare Disease
  • The FDA has granted Orphan Drug Designation to Kedrion Biopharma's COAGADEX for treating acquired Factor X deficiency, a rare blood disorder affecting fewer than one in one million people globally.
  • COAGADEX, already approved in 38 countries for hereditary Factor X deficiency, will be evaluated in a new clinical trial for patients with acquired deficiency caused by AL amyloidosis.
  • The condition occurs when amyloid fibrils bind to and reduce circulating Factor X levels, significantly increasing bleeding risk in affected patients.
  • Patient recruitment is underway for the trial, which will focus on moderate and severe forms of acquired Factor X deficiency with coagulant activity levels below 50% of normal.

BioNexus Gene Lab and Fidelion Diagnostics Form Strategic Alliance to Commercialize Revolutionary Liquid Biopsy Technology in Southeast Asia

  • BioNexus Gene Lab Corp. and Fidelion Diagnostics announced a strategic cross-equity partnership to bring VitaGuard™ minimal-residual-disease platform to Southeast Asia's 680 million population.
  • The VitaGuard™ technology dramatically reduces liquid biopsy costs from over $3,000 to under $300 while achieving 0.02% variant-allele-frequency sensitivity with tumor-naïve detection capabilities.
  • The alliance targets Southeast Asia's projected 2.4 million annual cancer cases by 2030, creating a multi-billion-dollar market opportunity for affordable precision oncology.
  • BGLC will secure exclusive commercial rights across ASEAN markets and lead regulatory submissions starting in Singapore and Malaysia.

NICE Recommends Vanzacaftor-Tezacaftor-Deutivacaftor for Cystic Fibrosis Treatment in England

Drug Approval
  • NICE has approved vanzacaftor-tezacaftor-deutivacaftor (Alyftrek) for NHS funding in England to treat cystic fibrosis in patients aged 6 years and older with at least one F508del mutation.
  • Clinical trial evidence demonstrates the triple combination therapy is as effective as the current standard treatment ivacaftor-tezacaftor-elexacaftor in improving lung function, growth, weight gain, and reducing lung infections.
  • The new therapy must be funded within 30 days of final guidance publication, with cost comparisons suggesting similar expenses to existing treatments.
  • NICE determined there is sufficient evidence showing the treatment provides benefits and value for money, allowing routine use across the NHS for this patient population.

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