Caladrius Biosciences secures FDA orphan drug designation for CLBS03 to treat type 1 diabetes

Orphan drug designation provides certain exclusivity benefits, tax credits for certain research, longer exclusivity and a waiver of the New Drug Application user fee.

The designation is made to promote safe and efficacious products for the treatment of rare diseases. T1D with residual beta cell function is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people (prevalence) nationwide.

The scientific basis for this therapeutic stems from the use of Tregs to treat autoimmune diseases caused by T cell imbalances in an individual’s immune system. This novel approach seeks to restore immune balance by enhancing Treg cell number and function.

Tregs are a natural part of the human immune system and regulate the activity of T effector cells, which are responsible for protecting the body from viruses and other foreign antigens.

When Tregs function properly, only harmful foreign materials are attacked by T effector cells. In autoimmune diseases, it is thought that deficient Treg activity permits the T effector cells to attack the body’s own beneficial cells, and in the case of T1D, insulin-producing pancreatic beta cells.

"Obtaining orphan drug designation is a key step in our regulatory and development strategy for CLBS03," said David J. Mazzo, PhD, Chief Executive Officer of Caladrius.

"Coupled with the progress we are making in advancing the product through to clinical milestones, we believe that this will make CLBS03 an even more attractive opportunity for a potential partner."

About The Sanford Project: T-Rex Study

The 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 type 1 diabetes mellitus 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 type 1 diabetes 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.