Rare Daily Staff

Cynata Therapeutics said that CYP-001, its experimental off-the-shelf stem cell therapy, met all clinical endpoints in an early-stage trial for the treatment of steroid-resistant acute graft-versus-host disease (GvHD).

GvHD is a complication that can occur after a bone marrow transplant or similar procedure when the donor’s immune cells attack the recipient of the transplant. The only approved treatment for GvHD is corticosteroid therapy, which is typically only effective in about half of patients. When GvHD fails to improve or worsens despite steroid treatment, patients are described as having steroid-resistant GvHD. The prognosis for these patients is poor, with mortality rates in excess of 90 percent.

CYP-001 is based on the company’s stem cell platform, which leverages iPSCs taken from one banked blood donation from one adult donor to enable the economical manufacture of an unlimited number of consistent, commercial-scale mesenchymal stem cells for therapeutic use. Mesenchymal stem cells are present in bone marrow and give rise to a variety of cells including skeletal tissue, bone, and fat.

The study showed a 93 percent showed improvement in their GvHD symptoms with 53 percent of patients showing an absence of symptoms. There was at least an 87 percent overall survival at day 100. There were no treatment-related serious adverse events or safety concerns.

The company previously reported that one patient in the study died after developing pneumonia, which is a common finding in recipients of bone marrow transplants and similar procedures. This death was not considered to be treatment related. One patient in the study withdrew from the trial to commence palliative care, which meant it was not possible to collect any further data regarding that patient as part of this trial. All other patients remained alive at Day 100.

The company is preparing a formal clinical study report to support further clinical development and commercialization of CYP-001 in GvHD. The report will be provided to Fujifilm under the existing agreement with Cynata. Upon receipt of the report, Fujifilm will have 90 days to exercise its license option to the indication and to trigger a $3 million upfront payment plus the potential for $43.6 million in milestones and double-digit royalties for Cynata.

Based on the phase 1 safety data in GvHD, Cynata is exploring additional indications, and beginning to plan for a phase 2 trial in critical limb ischemia.

“Steroid-resistant GvHD is a devastating disease with a high rate of mortality,” said Kilian Kelly, vice president of product development for Cynata. “The clinical results from patients in cohorts A and B are highly encouraging, as all of the patients had failed to respond to corticosteroid therapy, the only approved treatment for GvHD.

August 30, 2018
Photo: Kilian Kelly, vice president of product development for Cynata