Caladrius Biosciences, Inc., a cell therapy company combining an industry-leading development and manufacturing services provider, PCT, with a select therapeutic development pipeline, announces that its product candidate CLBS03 (autologous expanded polyclonal regulatory T cells, or Tregs) was granted Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of type 1 diabetes mellitus (TID), making it the first known therapeutic candidate for treatment of T1D to receive the designation.
CLBS03 has received Orphan Drug designation from the FDA as well as Advanced Therapeutic Medicinal Product (ATMP) classification from the European Medicines Agency. CLBS03 is currently being studied in a landmark phase 2 clinical trial in collaboration with Sanford Health, The Sanford Project: T-Rex Study, which is expected to complete enrollment of the first defined cohort of 18 patients in the coming weeks.
CLBS03 is a personalized, autologous medicine consisting of each patient’s own Tregs, which have been expanded in number and functionally enhanced by a proprietary method developed by a collaboration between PCT and the University of California, San Francisco. The program is supported by promising published early clinical work conducted by respected leaders in the area of T regulatory cell science. Two phase 1 clinical trials of this technology in T1D patients demonstrated safety and tolerance, feasibility of manufacturing, infused Treg persistence and an early indication of efficacy. In particular, one of those trials provided supportive evidence of the utility of Tregs for T1D in pediatric patients 5 to 18 years of age with new onset T1D2. In that open label, randomized study, the authors reported that treatment with expanded autologous Tregs preserved function of pancreatic beta cells and reduced the need for exogenous insulin in the majority of patients treated.
“Obtaining Fast Track designation is a key milestone in our regulatory and development strategy for CLBS03. It underscores the great need for innovative treatments, such as CLBS03, in the treatment of T1D and allows for the acceleration of its development,” said David J. Mazzo, CEO of Caladrius. “We are making excellent progress advancing the US-based phase 2 clinical program of CLBS03 to treat T1D and look to complete enrollment of the first cohort of 18 patients in the coming weeks. This, coupled with our Orphan Drug and Fast Track designations, should make CLBS03 an even more attractive opportunity for a potential partner.”
The Sanford Project: T-Rex 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 T1D with residual beta cell function in approximately 111 subjects age 12 to 17 in two cohorts (18 subjects followed by 93 subjects). The study is being conducted in collaboration with Sanford Research, a subsidiary of Sanford Health. Subjects will be randomized into one of three groups and will 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.
