Cynata Reports Positive Six-Month Data from Cohort A in Phase 1 Trial of CYP-001 in GvHD
Published: Jun 12, 2018
Australian stem cell and regenerative medicine company Cynata Therapeutics Limited (ASX: CYP) is pleased to announce positive safety and sustained overall survival rates from a six month follow-up assessment of patients enrolled in Cohort A of its Phase 1 clinical trial of CYP-001, the company’s lead Cymerus™ mesenchymal stem cell (MSC) product candidate, in steroid-resistant acute graft-versus-host disease (GvHD).
A follow up safety analysis was undertaken in all eligible patients enrolled in Cohort A, six months following their initial dose of CYP-001. The assessment found:
Overall survival in Cohort A remains at seven out of eight patients (87.5 percent)
No treatment-related serious adverse events (SAEs) or safety concerns have been identified
Dr Kilian Kelly, Cynata’s Vice President of Product Development, said, “There is a clear unmet need for improved treatment options in steroid-resistant GvHD, where the prognosis for patients remains poor and there are very high mortality rates. We are delighted to report that all patients evaluated in Cohort A continue to do well six months after treatment with CYP-001, and that no safety concerns have arisen. We look forward to reporting further follow-up data from these patients late this year, and to the first data readout from our higher-dose Cohort B in the coming weeks.”
In the Phase 1 trial, eight patients with steroid-resistant acute GvHD were enrolled in Cohort A, and received two infusions of CYP-001 administered one week apart. Each dose was 1 million cells per kilogram of body weight (cells/kg), up to a maximum dose of 100 million cells.
The primary evaluation period involved assessment of safety and efficacy (response to treatment) up to day 100. In accordance with the trial protocol, patients then entered the long term follow-up phase of the trial, primarily to monitor safety, with assessments at approximately six, 12, 18 and 24 months after the first dose of CYP-001.
As reported in February 2018, overall survival in Cohort A at day 100 was 87.5 percent, while the Overall and Complete Response rates by day 100 were 100 percent and 50 percent, respectively.1 As previously reported, one patient in Cohort A died after developing pneumonia, which is common in recipients of bone marrow transplants and similar procedures. This death was not considered to be treatment related.