Earlier assessment of clinical safety permits acceleration of trial timeline
Sanford Research to Provide Operational Support at its Clinical Sites for Remainder of Trial
BASKING RIDGE, N.J., Oct. 31, 2016 — Caladrius Biosciences, Inc. (NASDAQ:CLBS) (“Caladrius” or the “Company”), a cell therapy company combining a unique, promising therapeutic development pipeline with an industry-leading development and manufacturing services provider through its subsidiary PCT, LLC a Caladrius Company™ (“PCT”), today announced that the independent Data Safety Monitoring Board (“DSMB”) for its Phase 2, The Sanford Project: T-Rex Study recommended that the trial proceed with the enrollment of the prescribed second cohort of subjects in the study to meet the target of 111 total participants. The Company’s decision to resume enrollment ahead of the original timeline represents an acceleration of the program. The decision was made in consultation with external clinical advisors based on a favorable safety profile for the first cohort of 19 subjects who reached their 28-day post-treatment follow-up. Sanford Research, the Company’s collaborator for the study, has agreed to extend operational support at its participating clinical sites for the remainder of the Study. The study is evaluating CLBS03 (the Company’s product candidate consisting of autologous expanded regulatory T cells, or Tregs) as a treatment for recent-onset type 1 diabetes (T1D).
Caladrius has modified the T-Rex study protocol to allow for an earlier than planned resumption of enrollment for the remaining patients based on the DSMB recommendation after the 28-day, rather than three-month, evaluation of the first cohort.
Based on the earlier than originally expected recommencement of Study enrollment and the high interest from subjects and investigators in the Study experienced from the first cohort, the Company now expects to reach the important milestone of treating 50% of subjects by mid-2017. The results from a pre-specified interim analysis of early therapeutic effect for the Study triggered after approximately 50% of subjects reach the 6-month post-treatment follow-up visit is expected to be announced late 2017/early 2018. The enrollment of the 70th subject in the Study, expected to occur in mid-2017, will trigger an expected $8.4 million infusion of capital pursuant to the terms of the recently announced private placements of Caladrius common stock.
CLBS03 has received Fast Track and Orphan Drug designations from the FDA as well as Advanced Therapeutic Medicinal Product (ATMP) classification from the European Medicines Agency.
“We are delighted to have identified multiple means by which we can accelerate our timeline for the T-Rex study and to have achieved this positive assessment of clinical safety earlier than originally planned. These results augment the existing body of evidence supporting the notion that CLBS03 has a favorable safety profile,” said David J. Mazzo, Ph.D., Chief Executive Officer of Caladrius. “We look forward to efficiently executing on the remainder of the T-Rex study, driving towards an understanding of CLBS03’s therapeutic effect in adolescents with recent-onset type 1 diabetes, starting with the planned interim analysis based upon a 6-month follow-up of ~50% of the targeted enrollment planned to occur late 2017 or early 2018.”
Sanford Research (“Sanford”), has extended the agreement with Caladrius to provide operational support for the T-Rex Study beyond the first cohort to the remainder of the study. Sanford will provide and cover the costs of its clinical performance sites. Sanford’s initial support resulted in the enrollment of most of the first cohort of subjects and its recent agreement to make a $5 million equity investment in Caladrius will contribute to the execution of the remainder of the Study. Sanford Research is a non-profit organization that is part of Sanford Health and supports an emerging translational research center focused on finding a cure for T1D, called the Sanford Project.
“Patients with type 1 diabetes benefit from having access to the newest and most innovative treatment options like cell therapy,” said David Pearce, Ph.D., executive vice president for Sanford Research. “Sanford understands how essential collaborations with organizations like Caladrius are to discovering those treatments and making them accessible to patients through clinical trials.”
About The Sanford Project: T-Rex Study
The landmark study, which is being conducted in collaboration with Sanford Research, a Sanford Health subsidiary, 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, in 111 subjects age 12 to 17 with recent-onset T1D. Subjects are randomized into one of three groups to receive, through a single administration, either a high dose of CLBS03, a low dose of CLBS03 or placebo. Enrollment of the first cohort of 19 subjects, designated for a preliminary safety evaluation, was completed in August 2016. 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 Sanford Project: T-Rex Study, please visit https://clinicaltrials.gov/ct2/show/NCT02691247.
About Sanford Health
Sanford Health is an integrated health system headquartered in the Dakotas. It is one of the largest health systems in the nation with 45 hospitals and nearly 300 clinics in nine states and four countries. Sanford Health’s 28,000 employees, including more than 1,300 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 sanfordhealth.org.
About Caladrius Biosciences, Inc.
Caladrius Biosciences, Inc. is advancing a proprietary platform technology for immunomodulation by pioneering the use of T regulatory cells as an innovative therapy for recent onset type 1 diabetes. The product candidate, CLBS03, is the subject of an ongoing Phase 2 clinical trial (The Sanford Project: T-Rex Study) in collaboration with Sanford Research, and has been granted Orphan Drug and Fast Track designation by the U.S. Food and Drug Administration and Advanced Therapeutic Medicinal Product classification by the European Medicines Agency. The Company’s PCT subsidiary 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. 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 enrollment of the second cohort of subjects in the T-Rex Study, the planned interim analysis based upon a 6-month follow-up of approximately 50% of the targeted enrollment, and the enrollment of the 70th subject in the 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. 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.