CARLSBAD, CA -- (Marketwired) -- 02/18/14 -- International Stem Cell Corporation (OTCQB: ISCO) (www.internationalstemcell.com), a California-based biotechnology company developing novel stem cell-based therapies and biomedical products, today announced the completion of discussions with the U.S. Food and Drug Administration (FDA) regarding its Parkinson's disease (PD) cell therapy program through a pre-Investigational New Drug (pre-IND) meeting. The meeting provided formal feedback and guidance from the FDA on the proposed pre-clinical plan, manufacturing approach, and clinical study design necessary to file the IND.
"We're pleased with the outcomes of the meeting with the FDA," said Yale School of Medicine Professor D. Eugene Redmond Jr. MD, who is leading ISCO's pharmacology/toxicology study. "The FDA's feedback has provided clear direction for the remaining studies required for the IND."
Following this meeting, ISCO will be working to complete the Good Laboratory Practice (GLP) pharmacology and safety studies, including tumorigenicity, toxicology and tolerability, using the human parthenogenetic neural stem cell (hPNSC) product, a type of self-renewing multipotent cell that is a precursor for the major cells of the central nervous system. To date pre-clinical pilot studies in rodents and primates have shown the hPNSC to be safe, well tolerated, and provide therapeutic benefit by differentiating into dopaminergic neurons and expressing specific neurotrophic factors to protect the nigrostriatal system of the brain. We anticipate being able to report interim primate data before the end of March 2014 and top line final results from these GLP studies later in 2014 and expect to file the IND soon thereafter.
"As this will be the first IND for the Company, we're pleased to have completed this critical milestone," said Dr. Ruslan Semechkin, Chief Scientific Officer of ISCO. "Based on data from our pilot studies, along with the guidance from the FDA, we believe that we're well positioned to successfully transition this program to the clinical stage."
According to the Parkinson's Disease Foundation, an estimated seven to 10 million people worldwide live with PD, with as many as one million of those in the United States alone, more than the combined total of people diagnosed with multiple sclerosis, muscular distrophy, and Lou Gehrig's disease. The total direct and indirect cost of Parkinson's disease is estimated to be nearly $25 billion per year in the United States alone.
A more complete description of the hPNSC product and the GMP (21CFR211) manufacturing method was published together with Prof. Evan Y. Snyder, Director of Stem Cell Research Center at Sanford-Burnham Medical Research Institute in early 2013. Importantly, unlike many other stem cell therapy products, ISCO's proprietary hPNSC are cryopreservable, meaning they can be delivered in a frozen state to hospitals and clinics.
About International Stem Cell Corporation
International Stem Cell Corporation is focused on the therapeutic applications of human parthenogenetic stem cells (hpSCs) and the development and commercialization of cell-based research and cosmetic products. ISCO's core technology, parthenogenesis, results in the creation of pluripotent human stem cells from unfertilized oocytes (eggs) hence avoiding ethical issues associated with the use or destruction of viable human embryos. ISCO scientists have created the first parthenogenetic, homozygous stem cell line that can be a source of therapeutic cells for hundreds of millions of individuals of differing genders, ages and racial background with minimal immune rejection after transplantation. hpSCs offer the potential to create the first true stem cell bank, UniStemCellTM. ISCO also produces and markets specialized cells and growth media for therapeutic research worldwide through its subsidiary Lifeline Cell Technology (www.lifelinecelltech.com), and stem cell-based skin care products through its subsidiary Lifeline Skin Care (www.lifelineskincare.com). More information is available at www.internationalstemcell.com.
ISCO's Parkinson's disease program uses human parthenogenetic neural stem cells (hPNSC), a novel therapeutic cellular product derived from the company's proprietary histocompatible human pluripotent stem cells. hPNSC are self-renewing multipotent cells that are precursors for the major cells of the central nervous system. The ability of hPNSC to (i) differentiate into dopaminergic (DA) neurons and (ii) express neurotrophic factors such as glial derived neurotrophic factor (GDNF) and brain derived neurotrophic factor (BDNF) to protect the nigrostriatal system, offers a new opportunity for the treatment of Parkinson's disease, especially in cases where current small molecule approaches fail to adequately control the symptoms.
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Safe harbor statement
Statements pertaining to anticipated developments, expected progress of our Parkinson's disease research and development program, and other opportunities for the company and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates,") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in clinical trials and the development and/or commercialization of potential products, the management of collaborations, regulatory approvals, need and ability to obtain future capital, application of capital resources among competing uses, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the company's business, particularly those mentioned in the cautionary statements found in the company's Securities and Exchange Commission filings. The company disclaims any intent or obligation to update forward-looking statements.
International Stem Cell Corporation
Dr. Simon Craw
Executive Vice President
Dr. Ruslan Semechkin
Chief Scientific Officer