Celgene Cellular Therapeutics Receives FDA Clearance for First Placenta-Derived Stem Cell Clinical Study
-- Trial is First Application of Company's Proprietary Cell Therapy Product--
WARREN, N.J.--(BUSINESS WIRE)--Celgene Cellular Therapeutics (CCT), a wholly owned subsidiary of Celgene Corporation (NASDAQ: CELG), today announced the U.S. Food and Drug Administration (FDA) has accepted the company's investigational new drug application to initiate a clinical trial using PDA001, an immunomodulatory therapy utilizing human placenta-derived stem cells obtained via CCT's proprietary processes. Clinical development will begin by the end of the year with the initiation of this Phase I, multi-center clinical trial in the U.S. for patients with moderate-to-severe Crohn's disease, who are refractory to oral corticosteroids, such as prednisone, and immune suppressants.
PDA001 is a novel culture expanded stem cell population that has broad therapeutic potential in areas such as immunology, inflammation, hematology and oncology. Pre-clinical evaluation of PDA001 has demonstrated its safety and potent immune-suppressive properties. A placenta-derived stem cell therapy, PDA001 has several potential significant benefits in that the cells are derived from a safe and almost unlimited source and are scalable to a traditional pharmaceutical level.
CCT owns an array of proprietary technologies directed to novel placental cell types and cell populations, as well as methods for collecting, processing and storing many types of stem cells from the placenta. PDA001 is the first product to be developed as a result of this expanding portfolio.
"This first placental-derived stem cell clinical trial is of vital importance given the significant number of people suffering from this debilitating disease," said lead investigator Dr. Lloyd F. Mayer, Professor and Director of the Immunology Institute, Professor of Medicine and Chief of the Divisions of Clinical Immunology and Gastroenterology at Mount Sinai in New York City. "With the positive results from in vivo biodistribution and safety studies, our hope is that PDA001 will suppress the atypical immune and inflammatory reactions involved in the pathogenesis of Crohn's disease, thereby resulting in a decrease in symptoms and improved quality of life for patients."
Crohn's disease, a chronic inflammatory condition of the gastrointestinal tract, impacts almost one million people in the United States alone. Traditional treatment for Crohn's disease has focused on non-specific anti-inflammatory or immunosuppressive agents. Unfortunately, a considerable proportion of patients develops intolerable side-effects, requires surgery or becomes unresponsive to therapy.
"This is the first step in our program to initiate clinical evaluations in a range of indications including not only Crohn's disease, but other serious inflammatory and autoimmune diseases, such as multiple sclerosis and rheumatological disorders as well," said Robert Hariri, M.D., Ph.D., CEO of Celgene Cellular Therapeutics. "By creating a novel cell therapy from a readily available source that does not require human leukocyte antigen-matching, we are hopeful that we can treat a large number of patients with a variety of devastating diseases."
About Celgene Cellular Therapeutics
At Celgene Cellular Therapeutics (CCT) we discover and develop therapeutics from stem cells derived from the human placenta as well as from the umbilical cord. CCT is a state-of-the-art research and development organization dedicated to fulfilling the promise of cellular therapeutics by developing cutting-edge products and therapies that will significantly benefit patients. Our goal is to develop proprietary cell therapy products for the treatment of unmet medical needs. Stem cell based therapies offer the potential to provide disease-modifying or even curative outcomes for serious diseases, which today lack adequate therapy.
About Placenta-Derived Stem Cells
Human Placenta-Derived Cells (PDA001) is a cellular immune modulatory agent with significant therapeutic potential. PDA001 is a novel cell population derived from normal, full-term human placental tissue. PDA001 is a culture-expanded plastic adherent cell, undifferentiated in vitro cell population that expresses the nominal phenotype CD34-, CD10+, CD105+ and CD200+. PDA001 cells constitutively express moderate levels of HLA Class I and undetectable levels of HLA Class II, and they do not express the co-stimulatory molecules CD80 and CD86. PDA001 is genetically stable, displaying a normal diploid chromosome count, normal karyotype and exhibit normal senescence after prolonged in vitro culture. PDA001 is capable of immunomodulation and suppresses T-cell proliferation. PDA001 also exhibits immunomodulatory effects on other cell types involved in an immune response such as T-cell subsets, macrophages and dendritic cells.
This release contains forward-looking statements which are subject to known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations expressed or implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and other factors described in the Company's filings with the Securities and Exchange Commission such as our 10K, 10Q and 8K reports.