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A first for Fast Track designation
September 2016
by Rachel Flehinger  |  Email the author
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BASKING RIDGE, N.J.—Caladrius Biosciences recently announced that its product candidate CLBS03 was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of type 1 diabetes mellitus (TID), making it the first known therapeutic candidate for T1D to receive the designation.
 
CLBS03 is a personalized, autologous medicine consisting of each patient’s own regulatory T cells (Tregs), which have been expanded in number and functionally enhanced by a proprietary method developed by a collaboration between Caladrius’ subsidiary PCT and the University of California, San Francisco.
 
The scientific basis for treating type 1 diabetes with CLBS03 derives from using Tregs to treat autoimmune diseases caused by imbalances in an individual’s immune system. Tregs are a natural part of the human immune system and regulate the activity of T 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 cells. In autoimmune diseases, deficient Treg activity permits the T cells to attack the body’s own beneficial cells, such as insulin-producing pancreatic beta cells in the case of T1D.
 
CLBS03 demonstrates the possibility for long, durable effect that may prove to be potentially curative. The product is unique in that it targets the root cause of the disease, rather than treating or masking symptoms. The therapy aims to reeducate the patient’s immune system, helping it return to a more normal state consistent with that of a person without the disease. Caladrius Biosciences’ potential therapy for T1D also stands out in that it employs polyclonal Treg cells.
 
CLBS03 targets the fundamental imbalance in the immune system that is believed to exist in patients with most autoimmune diseases. According to Caladrius CEO Dr. David J. Mazzo, their strategy is to “demonstrate proof of [the Treg] concept with type 1 diabetes, and then potentially expand this program to other autoimmune indications that should respond similarly, such as lupus, graft versus host disease, chronic obstructive pulmonary disease (COPD), multiple sclerosis, inflammatory bowel disease and steroid-resistant asthma.”
 
CLBS03 is currently being studied in a landmark Phase 2 clinical trial, The Sanford Project: T-Rex Study in collaboration with Sanford Research. This prospective, randomized, placebo-controlled, double-blind landmark study is being conducted with subjects age 12 to 17 years old. It is currently completing enrollment of the first defined cohort of 18 patients, with preliminary safety evaluation expected by the end of 2016.
 
From there, they will initiate cohort two, with the goal to complete that cohort over the course of 2017. There will be an interim efficacy analysis when 50 percent of the total number of patients (111 patients total from cohorts 1 and 2 combined) reach six-month follow-up, which Caladrius expects to hit by the end of 2017.
 
CLBS03 has also been granted Orphan Drug status. This designation is focused almost exclusively on prevalence, on those diseases that affect less than 200,000 patients per year. Approximately 18,000 people under the age of 20 are newly diagnosed with type 1 diabetes mellitus with residual cell beta cell function in the United States every year, and there are currently no curative treatments, only lifelong insulin therapy, often with serious co-morbidities.
 
In the case of Fast Track, the criteria for designation go beyond prevalence—it is intended to facilitate the development of novel new therapeutics, those that are considered disruptive to traditional care and that could provide a major step forward in a particular disease area. “We believe that Fast Track designation shows that there is a medical and/or regulatory community that believes that a particular product represents a significant advancement, and we are pleased to count CLBS03 in that category,” states Mazzo.
 
Code: E091627

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