BASKING RIDGE, N.J., July 28, 2016 — Caladrius Biosciences, Inc. (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 today that its product candidate CLBS03 (autologous expanded polyclonal regulatory T cells, or Tregs) was granted Fast Track designation by the US Food and Drug Administration (FDA) for the treatment of type 1 diabetes mellitus (T1D), making it the first known therapeutic candidate for treatment of T1D to receive the designation. CLBS03 has received Orphan Drug designation from the FDA as well as Advanced Therapeutic Medicinal Product (ATMP) classification from the European Medicines Agency. CLBS03 is currently being studied in a landmark Phase 2 clinical trial in collaboration with Sanford Health, The Sanford Project: T-Rex Study, which is expected to complete enrollment of the first defined cohort of 18 patients in the coming weeks.
Fast Track is a process designed to facilitate the development and expedite the review of drugs, biologics or treatments for serious conditions, thereby filling unmet medical needs. Through the Fast Track program, a product may be eligible for priority review at the time of a Biologic License Application (BLA) or New Drug Application (NDA) filing and may also be eligible to submit completed sections of the BLA/NDA on a rolling basis before the complete application is submitted. These expedited processes can significantly cut down the development time and cost associated with bringing a therapy to market. Furthermore, the therapy’s sponsors are eligible for more frequent written communication and meetings with the FDA, the benefit of which may be to foster a pivotal study design which more closely meets the FDA’s needs, thereby creating a more efficient and rapid pathway to approval.
The scientific basis for treating T1D with CLBS03 derives from the use of Tregs to treat autoimmune diseases caused by T-cell 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.
CLBS03 is a personalized, autologous medicine 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 published early clinical work conducted by respected leaders in the area of T regulatory cell science. 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 efficacy(1,2). 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 T1D(2). 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.
“Obtaining Fast Track designation is a key milestone in our regulatory and development strategy for CLBS03. It underscores the great need for innovative treatments, such as CLBS03, in the treatment of T1D and allows for the acceleration of its development,” said David J. Mazzo, PhD, Chief Executive Officer of Caladrius. “We are making excellent progress advancing the U.S.-based Phase 2 clinical program of CLBS03 to treat T1D and look to complete enrollment of the first cohort of 18 patients in the coming weeks. This, coupled with our Orphan Drug and Fast Track designations, should make CLBS03 an even more attractive opportunity for a potential partner.”
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 T1D with residual beta cell function in approximately 111 subjects age 12 to 17 in two cohorts (18 subjects followed by 93 subjects). The study 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. 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.
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. For more information, visit www.caladrius.com.
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 realization of the benefits of fast track designation for CLB03, the achievement of clinical milestones for CLB03 including the completion of enrollment for the initial cohort of 18 patients and the establishment of a partnership for CLBS03. 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.