BASKING RIDGE, N.J. (September 6, 2016) – Caladrius Biosciences (NASDAQ:CLBS), (“Caladrius” or the “Company”), a cell therapy company combining an industry-leading development and manufacturing services provider (PCT) with a select therapeutic development pipeline, announces that completion of enrollment of the first patient cohort in the Phase 2 Sanford Project: T-Rex Study occurred in August as planned. The T-Rex Study is investigating Caladrius’ product candidate, CLBS03 (autologous expanded regulatory T cells, or Tregs), for the treatment of recent-onset type 1 diabetes (T1D). The complete study targets inclusion of 111 patients, by design divided into an initial cohort of approximately 18 patients for an initial safety evaluation followed by a second cohort to meet patient enrollment goals. CLBS03 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) as well as Advanced Therapeutic Medicinal Product (ATMP) classification from the European Medicines Agency.
The Company expects to announce the results of the initial safety review of the data by the end of 2016. Pending review of the safety data from the first cohort and recommendation of the independent Data Safety Monitoring Board, enrollment of the second cohort will commence. An interim efficacy analysis is planned to occur after approximately 50% of all patients reach the six-month follow-up milestone.
Sanford Research provided and covered the clinical costs at two clinical trial sites that enrolled the majority of the first patient cohort.
“We expect that the review of safety data from the initial cohort of the T-Rex Study will provide evidence of a positive safety profile in adolescents, adding to the safety evidence provided by two published Phase 1 studies in adults and children, respectively,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “We have been encouraged by the rate of enrollment of patients in the first cohort both as an indicator of interest in the study and as it has allowed us to accelerate the expected timing of the initial safety review from early 2017 to late 2016.”
About The Sanford Project: T-Rex Study
The landmark study is a prospective, randomized, placebo-controlled, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of CLBS03 as a treatment for recent-onset T1D with residual beta cell function, in approximately 111 patients age 12 to 17. The study is being conducted in collaboration with Sanford Research, a subsidiary of Sanford Health. Patients are randomized into one of three groups to receive either a high dose of CLBS03, a low dose of CLBS03 or placebo. The key endpoints for the trial are the standard medical and regulatory endpoints for a T1D trial and include preservation of C-peptide, an accepted measure for pancreatic beta cell function; insulin use; severe hypoglycemic episodes; and glucose and hemoglobin A1c levels.
LEGEND OF ABBREVIATIONS
B = Brilacidin, the antibiotic
K = Kevetrin, the oncology drug
P = Prurisol, the psoriasis drug
SAE = Serious Adverse Event
IND = Investigational New Drug
