Caladrius Biosciences Announces Two-Year Study Results

Caladrius Biosciences, Inc. recently announced the publication of 2-year results from a Phase I clinical study of an autologous Regulatory T cell (Treg) immunotherapy for type 1 diabetes (T1D). The study investigated a product that is the forerunner of the company’s CLBS03 product candidate for recent-onset T1D.

The publication, titled Type 1 diabetes immunotherapy using polyclonal regulatory T cells, was published in Science Translational Medicine and authored by a team led by Jeffrey A. Bluestone, PhD, Professor at the Diabetes Center, University of California San Francisco, and a member of the company’s Type 1 Diabetes Scientific Advisory Board.

The Phase I open-label, uncontrolled dose-escalating study was conducted at the University of California, San Francisco and Yale University and provided evidence for safety and tolerability of autologous expanded polyclonal Treg cell therapy in 14 adults with recent-onset T1D. In the Phase I trial, subjects received ex vivo-expanded Tregs, ranging in dose from 5 million to 2.6 billion cells. The study found that autologous Tregs can be expanded and were well tolerated in patients with recent-onset T1D. Additionally, the Tregs retained their T cell receptor diversity and demonstrated enhanced functional activity.
The study demonstrated that:

• A subset of the adoptively transferred Tregs were long-lived, with up to 25% of the peak level remaining in circulation at 1-year post transfer, an indication that the therapy may be durable.
• No infusion reactions or cell-related high-grade adverse events were observed.
• C-peptide levels (a measure of beta-cell function preservation) persisted beyond 2 years post-transfer in several individuals.
• Expanded Tregs can be exported to clinical sites, increasing the feasibility of developing this cell therapy into a true therapeutic.

“These Phase I results are encouraging and, along with published results demonstrating safety and early signs of therapeutic effect of polyclonal Tregs in children with recent-onset type 1 diabetes, form the basis for our decision to embark upon a Phase II study (the Sanford Project: Trex study) of CLBS03 in recent-onset adolescent type I diabetics early next year,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “We expect that our Phase II trial will support the hypothesis of our Treg technology approach’s applicability in type 1 diabetes and across multiple autoimmune and inflammatory diseases, such as multiple sclerosis, COPD, and rheumatoid arthritis, to name a few.”

CLBS03 is a personalized medicine consisting of each patients’ own regulatory T cells that have been expanded in number and functionally enhanced by a proprietary method developed by the company’s PCT subsidiary, an external development and manufacturing partner for the growing cell therapy industry. Caladrius expects to commence patient enrollment in a Phase II study, in strategic collaboration with Sanford Research, as early as the first quarter of 2016. The Sanford Project: Trex Study has a planned enrollment of 111 subjects across approximately 12-15 US sites. Subjects will be randomized to placebo or one of two active arms receiving either 10 or 20 million cells/kg. Key endpoints in the study will include C-peptide measurement, insulin use, hypoglycemic episodes, and Hemoglobin A1 C levels in treated subjects in comparison to those receiving placebo. To learn more about the publication mentioned here, visit http://stm.sciencemag.org/content/7/315/315ra189

Caladrius Biosciences, Inc. is among the first of a new breed of immunotherapy companies with proven expertise and unique experience in cell process optimization, development, and manufacturing. Caladrius combines a leading cell therapy service provider with a development pipeline, including late-stage clinical programs based on a proprietary platform technology for immuno-oncology, as well as additional platform technologies for immunomodulation and ischemic repair. This integrated approach supports the industry in bringing significant life-improving medical treatments to market. For more information, visit www.caladrius.com.