Caladrius Biosciences Announces First Patient Dosed in Phase 3 Immunotherapy Trial for Melanoma

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.”

For more information on the Intus Study please visit www.theintusstudy.com or www.clinicaltrials.gov/ct2/show/NCT01875653.