Australian stem cell and regenerative medicine company Cynata Therapeutics (ASX:CYP) has announced positive safety and efficacy data from a 28 day analysis of patients in Cohort B of its Phase 1 clinical trial of CYP-001.
CYP-001 is the company’s lead Cymerus mesenchymal stem cell (MSC) product candidate in steroid-resistant acute graft-versus-host disease (GvHD).
According to the company, the overall response rate by Day 28 was 86 percent - six out of seven patients treated with CYP-001 showed an improvement in the severity of GvHD by at least one grade compared to baseline.
The complete response rate by day 28 was 57 percent - GvHD signs/symptoms completely resolved in four out of seven patients treated with CYP-001.
The company also said the higher dose of CYP-001 administered in Cohort B elicited a faster response than the lower dose in Cohort A. By Day 28, Cohort A had a complete response rate of 12.5 percent compared to 57 percent in Cohort B.
“The 28-day results for Cohort B are highly encouraging and, together with the excellent data from Cohort A, support the advancement of CYP-001 into a Phase 2 trial in GvHD," said Dr Ross Macdonald, CEO of Cynata.
"Importantly, CYP-001’s strong safety profile may enable us to advance the therapy directly into Phase 2 trials in other indications beyond GvHD where there is a high unmet medical need for a consistent and scalable source of high-quality MSCs. We are evaluating our options strategically and expect to provide more details at the appropriate time.”
Eight patients with steroid-resistant acute GvHD were enrolled in Cohort B, as originally planned, said the company.
"Seven out of eight patients enrolled in Cohort B received two infusions of CYP-001 at a dose level of 2 million cells per kilogram of body weight, up to a maximum of 200 million cells per infusion. All treated patients have now reached the pre-specified Day 28 endpoint," it said.
According to Dr Kilian Kelly, Cynata’s vice president of product development, “These results build on the very encouraging data from Cohort A of this trial and are particularly impressive given that all 15 patients enrolled in the Phase 1 trial had failed to respond to corticosteroid therapy, the only approved treatment for GvHD. The response to treatment is compelling, with an even higher Complete Response rate in Cohort B than in Cohort A and no safety concerns reported in either patient cohort. Moreover, the data suggest that the higher dose level has elicited a much quicker treatment response. This is important considering the severely debilitating nature of acute GvHD symptoms in many patients.”