Lisata Therapeutics Inc (LSTA)

[F1ash]

Caladrius Biosciences Announces


First Subject Treated in CLBS03 Type 1 Diabetes Phase 2 Trial




BASKING RIDGE, N.J. (March 29, 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 treatment of the first subject in The Sanford Project: T-Rex Study. The T-Rex Study is a Phase 2 trial of Caladrius’ product candidate CLBS03 (autologous expanded regulatory T cells, or Tregs) for the treatment of recent-onset type 1 diabetes (T1D) in adolescents.

The prospective, randomized, placebo-controlled, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of CLBS03 will enroll 111 subjects age 12 to 17 in two cohorts (18 subjects followed by 93 subjects) and 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. Investigational product will be administered as a single infusion and the subjects will be evaluated over the course of 2 years. 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 hemoglobin A1c level in comparison to placebo.

An initial safety analysis of the data and early analysis of immunological biomarkers will occur after the first cohort of 18 subjects has completed the three-month post-treatment visit. Pending review of the data from the first cohort and recommendation of the independent Data Safety Monitoring Board, a second cohort of 93 subjects will be enrolled. An interim efficacy analysis will occur after approximately 50% of all subjects reach the six-month follow-up milestone. Sanford Research will provide and cover the costs of two initial clinical trial sites which are expected to enroll the first cohort of subjects. Additional sites could be added to the study to facilitate timely completion of enrollment of the second cohort.

“The initiation of enrollment of this trial marks an important milestone for our investigational type 1 diabetes program and moves this program into the clinical development stage,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “Achieving this milestone supports our goal to create shareholder value by developing select, early-stage, highly-promising cell therapy candidates to proof-of-concept as a complement to our robust, revenue-generating cell therapy development and manufacturing services business. We look forward to presenting the initial safety analysis on the first 18 subjects by early 2017.”

The scientific basis for this program stems from the use of Tregs to treat diseases caused by imbalances in an individual's immune system, such as T1D. 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.

“We were encouraged by the findings of our Phase 1 study indicating safety and tolerability in adult patients and are now excited to begin this Phase 2 program to further assess safety and to evaluate the efficacy of this novel therapy,” said Stephen Gitelman, MD, Professor of Clinical Pediatrics at the University of California, San Francisco and Chair of the T-Rex Study Executive Steering Committee.

“If proven effective, such a therapy could have a profound impact on the lives of tens of thousands of young people suffering from diabetes, for whom there are currently no curative treatments but only lifelong insulin therapy,” said Douglas W. Losordo, MD, Senior Vice President, Clinical, Medical and Regulatory Affairs and Chief Medical Officer at Caladrius.

“Sanford Health has long been a leader of innovation and research,” stated David Pearce, PhD, president of Sanford Research. “Sanford is driven to help advance research to cure type 1 diabetes. That is why we were committed to being at the forefront of bringing The Sanford Project: T-Rex Study to patients.”

For more information on the T-Rex Study, please visit https://clinicaltrials.gov/ct2/show/