Clinical Milestone for Immuno-Oncology Therapy That Works by Turning Cancer Against Itself
New York, June 22, 2015 — Caladrius Biosciences, Inc. (Nasdaq:CLBS), a cell therapy leader with a late-stage clinical program for immuno-oncology, announced today the dosing of the first patient in the Intus Phase 3 clinical trial. The Intus trial is investigating the efficacy of the Company’s patient-specific targeted cancer immunotherapy candidate CLBS20 (also known as NBS20). The trial is studying CLBS20 in patients with stage III recurrent or stage IV metastatic melanoma.
The patient was dosed (with either CLBS20 or control, in accordance with the randomized, double-blind design of the study) at the clinical trial site at Thomas Jefferson University Hospital in Philadelphia, Pennsylvania. The announcement comes on the heels of a $17.7 million grant from the California Institute for Regenerative Medicine awarded to help fund the trial.
“We are excited to be a part of the Intus study and to contribute to the advancement of this and any potentially life-prolonging investigational therapy,” said Dr. Takami Sato, principal investigator for this site and Professor of Medical Oncology at Thomas Jefferson University.
The Intus trial is based on consistent, compelling results from two Phase 2 trials in identical patient populations evaluating the therapeutic vaccine that has become CLBS20. The more recent of the two trials was a randomized trial comparing CLBS20 to injections of autologous irradiated (inactivated) tumor cells in 42 patients. At two years, survival was 72% compared to 31% for control patients, which was consistent with the previous Phase 2 trial’s findings in which CLBS20 demonstrated 73% two-year survival in 54 patients, with a median survival of five years. Treatments were well-tolerated; the most common side effects were mild to moderate local injections site reactions.
CLBS20 is an individualized therapy developed for each patient from that patient’s own tumor and immune cells. It works by combining certain irradiated tumor cells (those thought to be the cancer-initiating cells responsible for tumor proliferation) with the patient’s immune cells, called dendritic cells. The cell-based product is then admixed with an immune adjuvant that stimulates white blood cells and injected into the patient, essentially turning the cancer against itself.
Potential patients for the trial are pre-screened based on the trial’s eligibility criteria, and tumor tissue samples are collected and delivered to the Caladrius manufacturing facility. Once tumor cell lines have been successfully established, each patient is screened for enrollment and is randomized into either treatment or control. The second part of CLBS20 manufacture requires collection of specific immune cells from that patient using a standard blood collection technique, and manufacture of the final product from the immune cells and tumor cells, which takes one month. The product is shipped to the treating location, and at the time of treatment, the product is thawed for injection and admixed with an immune adjuvant. The patient then receives the therapy via subcutaneous injections once a week for three weeks and then once a month for five months.
In addition to metastatic melanoma, the technology platform on which CLBS20 is based is potentially applicable across lung, colon, renal and ovarian cancers, hepatocellular carcinoma and glioblastoma multiforme—indications that collectively lead to more than 200,000 deaths in the United States each year.
“The dosing of the first patient in this Phase 3 trial is an important milestone for our Company and the timing underscores our focus on this program and our commitment to impeccable trial execution,” said Dr. David J. Mazzo, Chief Executive Officer of Caladrius Biosciences. “We are delighted by the enthusiasm and productivity of the team at Jefferson University and other trial sites around the country and look forward to translating that into optimized patient enrollment and a rapid completion of the Phase 3 trial.”
About Metastatic Melanoma
Melanoma is the most lethal form of skin cancer. Patients who have progressed to stage IV melanoma have a cancer that has metastasized—or spread—to distant sites in the body such as the lymph nodes, lungs, liver or brain. As a result, advanced melanoma is exceedingly difficult to treat, with a five-year survival rate of approximately 15%. There are 20,000 estimated new cases of, and an estimated 10,000 deaths from, metastatic melanoma each year in the United States. Treatments for stage IV melanoma are typically directed at slowing the growth of the cancer and prolonging survival. Current treatment options include radiation, chemotherapy, surgical resection, immunotherapy, or a combination approach. Unfortunately, current therapies have had limited impact on long-term survival for most patients. Even with the newer targeted therapies (anti-checkpoint inhibitors and specific enzyme inhibitors), half of patients with metastatic melanoma still die within 13 to 17 months from the start of such treatment. Administering certain anti-melanoma drugs at higher doses or in combinations may be more effective, but often results in more severe side effects.
About Caladrius Biosciences, Inc. (formerly NeoStem, Inc.)
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 is a cell therapy leader with late-stage clinical programs based on a proprietary platform technology for immuno-oncology, as well as additional platform technologies for ischemic repair and immunomodulation, and capabilities that support other cell therapy developers. This integrated approach supports the industry in bringing significant life-improving medical treatments to market. 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, Ischemic Repair Program, Immune Modulation 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. The Company’s actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the Company in general, see the factors described under the heading, “Item 1A. Risk Factors” in the Company’s Annual Report on Form 10-K filed with the SEC on March 2, 2015 and those described in the Company’s other periodic filings with the SEC. The Company undertakes no obligation to update or revise any forward-looking statements.