Chronic graft-versus-host disease is a complication of allogeneic stem cell or bone marrow transplantation in which donor immune cells attack the recipient's body. It can affect the skin, mouth, eyes, liver, lungs, joints, and other organs.
The FDA approved Tregzi (Orca-T), a first-of-its-kind regulatory T cell-based immunotherapy, for adults with blood cancers undergoing matched donor stem cell transplants. Approval was based on the PRECISION-T trial, in which 78% of treated patients achieved chronic GVHD-free survival at one year versus 38.4% with standard transplant.
The FDA has approved Tregzi, a donor-derived T-cell immunotherapy, to reduce the risk of chronic graft-versus-host disease following stem cell transplant in adults with high-risk blood cancers. In a pivotal trial, 78% of recipients were alive and free of severe chronic GVHD at one year versus 38.4% with standard transplant.
Sanofi announces leadership transition with CEO departure and interim appointment, while reporting key regulatory milestones including FDA Breakthrough Therapy designation for venglustat and expanded Dupixent approvals. The company advances an observational RSV study in Australia and outlines strategic initiatives in AI drug discovery and potential consumer healthcare spin-off.
MARGO, BIOPREVENT, and FOCaL are new machine-learning frameworks for adaptive clinical trial randomization, post-transplant risk prediction, and personalized treatment effects. Each showed improved performance over conventional methods.
Syndax Pharmaceuticals announced a collaboration to expand global access to Revuforj while reporting strong commercial performance for its two marketed products, with Revuforj generating $125 million and Niktimvo $152 million in the first 11 months of sales.
Researchers developed BIOPREVENT, an AI-based tool that predicts chronic graft-versus-host disease and transplant-related mortality in stem cell and bone marrow transplant patients by analyzing blood biomarkers and clinical data collected 90-100 days post-transplant.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07771439 |
A Study to Investigate the Efficacy and Safety of Belumosudil Compared With Best Available Therapy in Participants Aged 12 Years or Older With Chronic Graft-versus-host Disease |
NOT_YET_RECRUITING | PHASE3 |
| NCT07733141 |
Mindfulness Intervention to Reduce Patient Reported Symptoms in Patients With Chronic Graft-Versus-Host Disease |
NOT_YET_RECRUITING | NA |
| NCT07726914 |
Study to Compare the Plasma Concentration of Belumosudil Given as an Oral Suspension and as a Tablet to Healthy Adult Male Participants |
NOT_YET_RECRUITING | PHASE1 |
| NCT07535008 |
Dental Cleaning to Prevent Chronic Graft-Versus-Host Disease |
RECRUITING | NA |
| NCT07135973 |
A Study to Investigate Safety of Belumosudil in Participants Aged 12 Years and Above, With Chronic Graft-versus-host Disease (cGVHD) |
NOT_YET_RECRUITING | PHASE4 |
| NCT07127926 |
Evaluation of Belumosudil Whole Tablets vs. Crushed Tablets Pharmacokinetics in Patients Suffering From Chronic GvHD |
RECRUITING | |
| NCT07116031 |
A Study of Belumosudil in Children With Chronic Graft Versus Host Disease (schoolROCK) |
RECRUITING | PHASE2 |
| NCT07012304 |
Gecacitinib for cGVHD: Safety and Efficacy in Patients After ≥2 Lines of Prior Therapy |
RECRUITING | PHASE1/PHASE2 |
| NCT07011810 |
Axatilimab for Sclerotic Chronic Graft-versus-Host Disease |
RECRUITING | PHASE2 |
| NCT06695507 |
Upfront Ruxolitinib for Chronic Graft-vs-host Disease |
TERMINATED | PHASE1 |
