Cynata Completes Phase 1 GvHD Clinical Trial Follow-up with Positive Results

4 August 2020

Release date 30 July 2020

Cynata Therapeutics Limited, a clinical-stage biotechnology company specialising in cell therapeutics, has today announced positive results from the two-year follow-up of patients enrolled in the Phase 1 clinical trial of CYP-001, Cynata’s lead induced pluripotent stem cell (iPSC)-derived CymerusTM mesenchymal stem cell (MSC) product. The Phase 1 trial, which was the world’s first clinical trial of an allogeneic iPSC-derived MSC product, evaluated the safety and efficacy of CYP-001 for the treatment of patients with steroid-resistant acute graft-versus-host disease (GvHD).


  • Overall survival after two years was 60% (9/15 patients), which compares favourably to previously published outcomes in this patient population
  • No treatment-related serious adverse events or safety concerns were identified
  • Cynata working with licensee FUJIFILM towards the planned Phase 2 GvHD clinical trial

The Phase 1 clinical trial enrolled 15 patients with steroid-resistant acute GvHD. The primary evaluation at Day 100 revealed highly promising safety and efficacy results, as announced on 18 December 2018. By Day 100, the Complete Response and Overall Response rates were 53% and 87% respectively, and overall survival was at least 87%.

Participants who completed the Primary Evaluation Period then continued to a follow-up period for up to two years after the initial dose, which has now been completed.

The overall survival rate after two years was 60% (9/15 patients), which compares favourably with previously published outcomes. An overall survival rate of just 17% after two years has been reported in patients with steroid-resistant acute GvHD who received standard of care treatment.1 Furthermore, a recently published review article summarised numerous studies with bone marrow and adipose tissue derived MSCs in patients with steroid-resistant acute GvHD. Six of the studies reviewed reported two year survival in MSC-treated patients, which ranged from 16.6% to 40%.2 Similarly, in another recently published Phase 3 trial of an investigational drug (ruxolitinib) in patients with steroid-resistant acute GvHD, overall survival after 12 months was 38%, while there were insufficient surviving patients remaining on study to calculate the survival rate at later timepoints.3 The results of Cynata’s trial illustrate the significant potential of CYP-001 as a new treatment option for GvHD.

Study close-out procedures are now underway, which will include the preparation of a formal addendum to the clinical study report. Cynata’s GvHD license partner, FUJIFILM, is responsible for the further development of CYP-001, with planning for a Phase 2 clinical trial advancing.

Dr. Kilian Kelly, Cynata’s Chief Operating Officer, said: “The prognosis in patients with this condition is extremely poor, not only due to GvHD itself, but also because these patients generally have an underlying cancer and a compromised immune system. These follow-up results suggest that the high initial treatment response rates observed with CYP-001 100 days after treatment may result in a longer-term benefit. Overall, our Phase 1 study results are very positive compared to published results with other methods of treatment. We continue to work with our partners at FUJIFILM toward the planned Phase 2 clinical trial in GvHD to build on these very encouraging results, and we look forward to that commencing in the near future”.

Read the full media release. 


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