• Patient enrolment completed in Cynata's Phase 2 clinical trial of CYP-001 in acute graft versus host disease
• 65 participants enrolled across the US, Europe and Australia
• Primary evaluation period expected to complete in March 2026, with results in June 2026

Cynata Therapeutics Limited has announced that patient enrolment has been completed in its Phase 2 clinical trial of CYP-001 in adults with newly diagnosed, high risk acute graft versus host disease (aGvHD). A total of 65 participants have been enrolled in the trial across numerous clinical centres in Australia, the USA, and Europe. Each participant was randomised to receive either steroids plus CYP-001, or steroids plus placebo. The trial involves a 100-day primary evaluation period, which is expected to conclude in March 2026, with results anticipated around June 2026. The primary endpoint is Overall Response Rate at Day 28. aGvHD is a serious and often life-threatening complication of bone marrow transplantation and similar procedures, where the donor's immune cells (the graft) attack the recipient's tissues (the host). Standard first-line treatment with steroids fails in around half of all aGvHD cases, which are known as 'steroid-resistant' or SR-aGvHD cases. Historical two-year survival rates in patients with SR-aGvHD are less than 20%. Cynata's Cymerus™ iPSC-derived MSC product for intravenous use, CYP-001, is designed to modulate the immune system and improve both response rates and survival outcomes in aGvHD. In a successful Phase 1 trial in patients with SR-aGvHD, 87% of patients showed an Overall Response, 53% showed a Complete Response, and 60% survived for at least two years. CYP-001 has been granted Orphan Drug Designation by the US FDA for the treatment of aGvHD.