Final Operational Milestone #5

Under CIRM grant CLIN2-09730, Caladrius conducted protocol CLBS03-P01 “A Prospective Randomized Placebo-Controlled Double Blind Clinical Trial to Evaluate the Safety and Efficacy of CLBS03 (Autologous Ex Vivo Expanded Polyclonal CD4+CD25+CD127lo/-FOXP3+ Regulatory T-cells [Tregs]) in Adolescents with Recent Onset Type 1 Diabetes Mellitus (T1DM)”. The study was initiated on February 29, 2016 and completed January 22, 2020. It successfully recruited, randomized, and treated the full cohort of 110 subjects across 3 treatment arms (placebo, low dose, high dose). Based on the evaluation of primary and secondary efficacy endpoints, there was no evidence that CLBS03 (either the low dose or the high dose) was effective at modifying the T1DM disease course, including preservation of β-cell function and improvements in measures of disease severity, as compared with placebo, in adolescents with recent onset T1DM. The safety results indicated that a single IV infusion of CLSB03 was safe and well tolerated in adolescent subjects with T1DM.