Phase I follow-up results for CYP-001, a mesenchymal stem cell product for steroid-refractory acute GvHD

Positive results were announced today for the phase I trial (NCT02923375) of CYP-001, the first commercially developed mesenchymal stem cell product available.¹ The primary endpoints of this trial were the safety and efficacy of CYP-001 in adults with steroid-refractory acute graft-versus-host disease (SRaGvHD).  The secondary endpoints were complete response, partial response, and overall survival (OS) at Days 28 and 100. The initial evaluation of this multicenter, non-randomized study showed favorable results, with a CR rate of 53% and an OS of 87% at Day 100.

The follow-up results, obtained from 15 patients included in this trial, showed that the OS rate was 60% after 2 years, which compared favorably to historic controls: 1- or 2-year OS ranged from 17%−40% in previous studies. No safety concerns or serious treatment-related adverse events were recorded.

Allogeneic mesenchymoangioblast-derived mesenchymal stem cells make up the active agent of CYP-001. These cells are derived from induced pluripotent stem cells. CYP-001 will be advanced into a phase II trial for SR-GvHD, COVID-19, and critical limb ischemia, and a phase III trial for osteoarthritis.