Cynata Therapeutics (ASX:CYP) has reached a clinical milestone, completing patient enrolment in its Phase 2 trial of CYP-001, an investigational stem-cell therapy for acute graft-versus-host disease (aGvHD).
The condition is a life-threatening complication of bone marrow transplantation that currently has limited effective treatment options.
The study has now enrolled 65 adults across clinical centres in Australia, the United States and Europe. Participants were randomised to receive either standard steroid therapy plus CYP-001 or steroids plus placebo. The trial’s 100-day primary evaluation period is expected to conclude in March 2026, with topline results anticipated around June 2026. The primary endpoint is Overall Response Rate at Day 28, a key clinical measure in aGvHD treatment.
Cynata CEO and Managing Director Dr Kilian Kelly said the completion of enrolment represents a critical step forward for patients and for the company’s Cymerus technology. “We are delighted to complete patient enrolment in this trial, which has huge importance to Cynata as well as to patients afflicted with aGvHD,” Dr Kelly said. “There remains a significant unmet need for safer and more effective treatments, given that existing therapies often fail to prevent poor outcomes and may cause serious safety concerns. We are optimistic that the results will build on the very encouraging outcomes we saw in Phase 1.”
aGvHD affects up to 50 per cent of patients who receive donor-derived bone marrow transplants. In cases where patients do not respond to steroids, known as steroid-resistant aGvHD, two-year survival rates historically fall below 20 per cent, underscoring the severity of the condition.
CYP-001 is based on Cynata’s proprietary Cymerus induced pluripotent stem cell (iPSC) platform, designed to produce consistent, scalable mesenchymal stem cell (MSC) therapies without the donor-to-donor variability that affects conventional MSC manufacturing.
In a successful Phase 1 trial in steroid-resistant aGvHD, CYP-001 demonstrated an 87 per cent overall response rate, a 53 per cent complete response rate, and 60 per cent two-year survival, with no treatment-related serious adverse events—results that were subsequently published in Nature Medicine.
CYP-001 has been granted Orphan Drug Designation by the U.S. FDA for aGvHD, providing incentives including extended market exclusivity and fee waivers.