BASKING RIDGE, N.J., March 29, 2016 (GLOBE NEWSWIRE) -- 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/NCT02691247.
About Sanford Heath
Sanford Health is an integrated health system headquartered in the Dakotas. It is one of the largest health systems in the nation with 43 hospitals and nearly 250 clinics in nine states and three countries. Sanford Health’s 27,000 employees, including 1,400 physicians, make it the largest employer in the Dakotas. Nearly $1 billion in gifts from philanthropist Denny Sanford have allowed for several initiatives, including global children's clinics, genomic medicine and specialized centers researching cures for type 1 diabetes, breast cancer and other diseases. For more information, visit www.sanfordhealth.org.
About Caladrius Biosciences
Caladrius Biosciences, Inc., through its subsidiary, PCT, is a leading development and manufacturing partner to the cell therapy industry. PCT works with its clients to overcome the fundamental challenges of cell therapy manufacturing by providing a wide range of innovative services including product and process development, GMP manufacturing, engineering and automation, cell and tissue processing, logistics, storage and distribution, as well as expert consulting and regulatory support. PCT and Hitachi Chemical Co. have entered into a strategic global collaboration to accelerate the creation of a global commercial cell therapy development and manufacturing enterprise with deep engineering expertise. Around the core expertise of PCT, Caladrius strategically develops select product candidates, which currently includes an innovative therapy for type 1 diabetes based on a proprietary platform technology for immunomodulation. 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. All statements other than statements of historical fact contained in this press release are forward-looking statements, including statements regarding the timing and results of the initial safety analysis, efficacy results and the expansion to include further clinical sites for the T-Rex Study. 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 15, 2016, and in the Company’s other periodic filings with the SEC, including: risks related to: (i) our expected continued losses and negative cash flows; (ii) our anticipated need for substantial additional financing; (iii) the significant costs and management resources required to comply with the requirements of being a public company; (iv) the possibility that a significant market for cell therapy may not emerge; (v) the potential variability in PCT’s revenues; (vi) PCT’s limited manufacturing capacity; (vii) the need to improve manufacturing efficiency at PCT; (viii) the limited marketing staff and budget at PCT; (ix) the logistics associated with the distribution of materials produced by PCT; (x) government regulation; (xi) our intellectual property; (xii) cybersecurity; (xiii) the development, approval and commercialization of our products; (xiv) enrolling patients in and completing, clinical trials; (xv) the variability of autologous cell therapy; (xvi) our access to reagents we use in the clinical development of our cell therapy product candidates; (xvii) the validation and establishment of manufacturing controls; (xviii) the failure to obtain regulatory approvals outside the United States; (xix) our failure to realize benefits relating to “fast track” and “orphan drug” designations; (xx) the failure of our clinical trials to demonstrate the safety and efficacy of our product candidates; (xxi) our current lack of sufficient manufacturing capabilities to produce our product candidates at commercial scale; (xxii) our lack of revenue from product sales; (xxiii) the commercial potential and profitability of our products; (xxiv) our failure to realize benefits from collaborations, strategic alliances or licensing arrangements; (xxv) the novelty and expense of the technology used in our cell therapy business; (xxvi) the possibility that our competitors will develop and market more effective, safer or less expensive products than our product candidates; (xxvii) product liability claims and litigation, including exposure from the use of our products; (xxviii) our potential inability to retain or hire key employees; and (xxix) risks related to our capital stock. The Company’s further development is highly dependent on, among other things, future medical and research developments and market acceptance, which are outside of its control.
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