Cynata gains EU endorsement for Graft-vs-Host iPSC Study Plans
The Australian biotech company is hoping to tackle aGvHD in Europe, which appears to have twice as many cases as the USA.
Key Points
- Cynata has received a positive opinion from the EMA’s Pediatric Committee on its study plan for CYP-001 in acute graft-versus-host disease.
- The plan covers a combined Phase 2/3 trial in about 72 pediatric patients, while the company plans to begin with an adult Phase 3 study.
- Cynata expects Phase 2 data in adults with high-risk acute graft-versus-host disease in late June or early July 2026.
The Australian reports that Cynata, an Australian stem cell company, has received a positive opinion from the EMA’s Pediatric Committee on its investigation plan for CYP-001, its induced pluripotent stem cell (iPSC) therapy, for acute graft-versus-host disease (aGvHD).
For context, in the EU, sponsors generally must agree a Pediatric Investigation Plan (PIP) with the EMA before initiating Phase 3 studies, even for drugs initially being developed only in adults, unless a waiver is granted. Pediatric studies themselves are frequently deferred until later in development.
The opinion will now be sent to the European Commission for formal adoption.
Nuket Desem, director of regulatory and scientific affairs at Cynata, said that “aGvHD is more common in Europe than in any other region, with approximately twice as many cases as in the USA, and there are currently no EMA-approved cell-based therapies for the treatment of aGvHD in any age group.”
Trial plan in adults and children
The pediatric investigation plan outlines a single randomized controlled Phase 2/3 trial of CYP-001 in about 72 patients aged 28 days to 18 years with aGvHD.
Cynata plans to start with a Phase 3 trial in adults, then move to the pediatric study after the primary analysis from the adult trial is complete. Subject to positive Phase 2 data in adults, the development plan would include:
- One Phase 3 trial in adults
- A separate Phase 2/3 pediatric trial
The Pediatric Committee also agreed with the proposed timing. This would allow Cynata to submit a marketing authorization application for adults while the pediatric trial is still ongoing.
Background and upcoming Phase 2 readout
Acute graft-versus-host disease is a serious complication of bone marrow transplantation and related procedures. Standard first-line steroid treatment fails in about half of the cases. In steroid-resistant aGvHD, the source material says two-year survival rates are less than 20%.
Cynata said an earlier Phase 1 trial in adults with steroid-resistant aGvHD showed:
- 87% overall response rate
- 53% complete response rate
- 60% two-year overall survival
- No serious adverse events or safety concerns related to CYP-001 treatment
The company has also completed the primary evaluation period in its Phase 2 trial of CYP-001 in adults with high-risk aGvHD. That study enrolled 65 participants across sites in Australia, the US, and Europe, with patients randomized to receive steroids plus CYP-001 or steroids plus placebo.
Cynata expects results from the Phase 2 trial in late June or early July 2026.
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