Cynata Completes Phase 2 Enrolment for Promising aGvHD Therapy
Cynata Therapeutics has finalised patient enrolment in its pivotal Phase 2 trial of CYP-001 for acute graft versus host disease, setting the stage for critical data due mid-2026.
- 65 patients enrolled across US, Europe, and Australia
- Trial compares steroids plus CYP-001 against steroids plus placebo
- Primary endpoint, Overall Response Rate at Day 28
- Results expected by June 2026
- CYP-001 holds US FDA Orphan Drug Designation
Trial Milestone Achieved
Cynata Therapeutics Limited (ASX – CYP) has announced the completion of patient enrolment in its Phase 2 clinical trial evaluating CYP-001 for acute graft versus host disease (aGvHD). This milestone marks a significant step forward for the Australian biotech, with 65 participants recruited across multiple sites in the United States, Europe, and Australia.
The trial is designed to assess the efficacy and safety of CYP-001 when added to standard steroid treatment, compared to steroids plus placebo. Patients enrolled are adults newly diagnosed with high-risk aGvHD, a serious complication following bone marrow transplantation where donor immune cells attack the recipient’s tissues.
Addressing a Critical Unmet Need
aGvHD remains a life-threatening condition with limited effective treatment options. Standard steroid therapy fails in approximately half of cases, leading to poor survival outcomes. Cynata’s CYP-001, derived from their proprietary Cymerus™ stem cell platform, aims to modulate the immune response and improve patient prognosis.
Previous Phase 1 data were encouraging, showing high overall response rates and improved survival without serious safety concerns. The FDA has granted CYP-001 Orphan Drug Designation, underscoring its potential to fill a significant therapeutic gap.
Looking Ahead to Results
The trial’s primary evaluation period spans 100 days post-treatment, expected to conclude in March 2026, with topline results anticipated by June. The primary endpoint is the Overall Response Rate at Day 28, a key measure of early treatment efficacy.
CEO Dr Kilian Kelly expressed optimism about the trial’s potential impact, highlighting the urgent need for safer, more effective therapies in aGvHD. Investors and stakeholders will be watching closely as Cynata moves toward this critical data readout.
Beyond aGvHD, Cynata’s Cymerus™ platform is advancing multiple clinical programs, reflecting the company’s broader ambition in regenerative medicine and cell therapy.
Bottom Line?
Cynata’s next six months will be pivotal as Phase 2 data could validate CYP-001’s promise and reshape aGvHD treatment.
Questions in the middle?
- Will CYP-001 demonstrate statistically significant improvement over steroids alone?
- How might the Phase 2 results influence Cynata’s commercial and partnership strategies?
- What are the prospects for regulatory approval timelines following the trial?
