BASKING RIDGE, N.J., September 6, 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 that completion of enrollment of the first patient cohort in the Phase 2 Sanford Project: T-Rex Study occurred in August as planned. The T-Rex Study is investigating Caladrius’ product candidate, CLBS03 (autologous expanded regulatory T cells, or Tregs), for the treatment of recent-onset type 1 diabetes (T1D). The complete study targets inclusion of 111 patients, by design divided into an initial cohort of approximately 18 patients for an initial safety evaluation followed by a second cohort to meet patient enrollment goals. CLBS03 has received Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) as well as Advanced Therapeutic Medicinal Product (ATMP) classification from the European Medicines Agency.
The Company expects to announce the results of the initial safety review of the data by the end of 2016. Pending review of the safety data from the first cohort and recommendation of the independent Data Safety Monitoring Board, enrollment of the second cohort will commence. An interim efficacy analysis is planned to occur after approximately 50% of all patients reach the six-month follow-up milestone.
Sanford Research provided and covered the clinical costs at two clinical trial sites that enrolled the majority of the first patient cohort.
“We expect that the review of safety data from the initial cohort of the T-Rex Study will provide evidence of a positive safety profile in adolescents, adding to the safety evidence provided by two published Phase 1 studies in adults and children, respectively,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “We have been encouraged by the rate of enrollment of patients in the first cohort both as an indicator of interest in the study and as it has allowed us to accelerate the expected timing of the initial safety review from early 2017 to late 2016.”
About The Sanford Project: T-Rex Study
The landmark study is a prospective, randomized, placebo-controlled, double-blind Phase 2 clinical trial to evaluate the safety and efficacy of CLBS03 as a treatment for recent-onset T1D with residual beta cell function, in approximately 111 patients age 12 to 17. The study is being conducted in collaboration with Sanford Research, a subsidiary of Sanford Health. Patients are randomized into one of three groups to receive either a high dose of CLBS03, a low dose of CLBS03 or placebo. The key endpoints for the trial are the standard medical and regulatory endpoints for a T1D trial and include preservation of C-peptide, an accepted measure for pancreatic beta cell function; insulin use; severe hypoglycemic episodes; and glucose and hemoglobin A1c levels.
For more information on the T-Rex Study, please visit https://clinicaltrials.gov/ct2/show/NCT02691247.
CLBS03 is a personalized, autologous therapy consisting of each patient’s own Tregs, which have been expanded in number and functionally enhanced by a proprietary method developed by a collaboration between PCT and the University of California, San Francisco. The program is supported by promising early clinical work conducted by respected leaders in the area of T regulatory cell science and published in peer reviewed journals. The scientific basis for treating T1D with CLBS03 derives from the use of Tregs to treat autoimmune diseases caused by imbalances in an individual’s immune system. This innovative 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, deficient Treg activity permits the T effector cells to attack the body’s own beneficial cells, for example, insulin-producing pancreatic beta cells in the case of T1D.
Two Phase 1 clinical trials of this technology in T1D patients demonstrated safety and tolerance, feasibility of manufacturing, infused Treg persistence and an early indication of efficacy1,2. In particular, one of those trials provided supportive evidence of the utility of Tregs for T1D in pediatric patients 8 to 16 years of age with new onset T1D2. In that open label study, the authors reported that treatment with expanded autologous Tregs preserved function of pancreatic beta cells and reduced the need for exogenous insulin in the majority of patients treated.
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., Ltd. 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.
Forward Looking Statements
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 review of data from the initial 18-patient cohort in the T-Rex Study, the announcement of the results of that review and the enrollment of the remaining 93 patients. 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.
- Bluestone, J., et al. Type 1 diabetes immunotherapy using polyclonal regulatory T cells. Science Translational Medicine, 2015 Nov;7(315): pp. 315ra189.
- Marek-Trzonkowsa, N., et al. Therapy of type 1 diabetes with CD4(+)CD25(high)CD127-regulatory T cells prolongs survival of pancreatic islets – results of one year follow-up. Clinical Immunology. 2014 Jul;153(1): 23-30.