Caladrius Announces Publication of Two-Year Results from Phase 1 Trial of Type 1 Diabetes Product Candidate in Science Translational Medicine
Study demonstrated persistence of Tregs, safety and feasibility; supports upcoming Phase 2 study to test efficacy of the therapy
Basking Ridge, NJ / CRWEPRESSRELEASE / December 1, 2015– Caladrius Biosciences, Inc. (“Caladrius” or the “Company”, NASDAQ:CLBS), a cell therapy company combining an industry-leading external development and manufacturing provider with a development pipeline in immunotherapy, announces today the publication of two-year results from a Phase 1 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, entitled “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 1 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 fourteen adults with recent-onset T1D. In the Phase 1 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 one-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 two 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 1 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 diabetes1, form the basis for our decision to embark upon a Phase 2 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 2 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 which 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 2 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.
Marek-Trzonkowska N, Mysliwiecb M, Dobyszukc A, et al. Therapy of type 1 diabetes with CD4+CD25highCD127-regulatory T cells prolongs survival of pancreatic islets — results of one year follow-up. Clin Immunol. 2014;153(1):23–30.
About Caladrius Biosciences
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
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s current expectations, as of the date of this press release, and involve certain risks and uncertainties. Forward-looking statements include statements herein with respect to the successful execution of the Company’s business strategy, the Company’s ability to develop and grow its business, the successful development of cellular therapies with respect to the Company’s research and development and clinical evaluation efforts in connection with the Company’s Immuno-oncology Program, Immune Modulation Program, Ischemic Repair Program and other cell therapies, the future of the regenerative medicine industry and the role of stem cells and cellular therapy in that industry, and the performance and planned expansion of the Company’s wholly-owned subsidiary and its center of excellence for cell therapy process development, engineering and manufacturing, PCT, as well as its efforts to expand its capabilities into the cell therapy tools market. The Company’s actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. Factors that could cause future results to materially differ from the recent results or those projected in forward-looking statements include the “Risk Factors” described in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 2, 2015, and in the Company’s other periodic filings with the SEC. The Company’s further development is highly dependent on future medical and research developments and market acceptance, which is outside of its control.
Anne Marie Fields
Senior Vice President
Caladrius Biosciences, Inc.
Manager, Communications and Marketing
Source: Caladrius Biosciences, Inc.