LOS ANGELES--(BUSINESS WIRE)-- ImmunoCellular Therapeutics (OTCBB: IMUC) announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for ICT-107, the company’s dendritic cell-based cancer vaccine candidate which targets glioblastoma multiforme (GBM). As a result of the orphan drug status, ImmunoCellular will be eligible to receive a number of benefits, including access to grant funding for clinical trials, tax credits, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of up to seven years. US orphan drug designation is granted to companies with products aimed at treatment of a rare disease or condition that affects fewer than 200,000 Americans.
A recent Phase I clinical study of ICT-107 in GBM revealed that newly diagnosed patients who received the vaccine demonstrated a median progression-free survival (PFS) of 17.7 months after surgery, compared with the historical median PFS of 6.9 months observed with standard treatment with surgery, radiation and chemotherapy. Seven of the 16 patients (44%) who participated in the study went on to live with no disease progression with an average time over 2 years, significantly better than historical data of less than 15% disease free survival. The drug has exceeded its competitors in the overall survival rate in newly diagnosed GBM patients by demonstrating an 80% survival, which is about three times larges than the historical control.
"We are pleased that FDA has recognized the potential that ICT-107 has to treat GBM and has granted us orphan drug status”, said ImmunoCellular Therapeutics’ president and CEO said Manish Singh, Ph.D. "The designation should facilitate a shorter time period to approval and reflects the FDA’s views on the importance of developing a treatment which will improve survival outcomes for those afflicted with this terrible disease."
The 12-month disease-free survival from the time of surgery was 75% with ICT-107, compared with a historical control survival rate of 26.9%, and the 18-month disease-free survival with ICT-107 was 49.2%, compared with 18.4% historically. Safety data for ICT-107 also compared favorably to current treatments: no serious adverse events were reported and minor side effects were limited to fatigue, skin rash and pruritis.
About ImmunoCellular Therapeutics, Ltd.
IMUC is a Los Angeles-based clinical-stage company that is developing immune-based therapies for the treatment of brain and other cancers. The Company recently completed a Phase I trial of its lead product candidate, ICT-107, a dendritic cell-based vaccine targeting multiple tumor associated antigens for glioblastoma. The Company is planning to initiate a multicenter phase II study of this vaccine in the second half of 2010. The Company’s “off the shelf” therapeutic vaccine product candidate (ICT-121) targeting cancer stem cells for multiple cancer indications is targeted by IMUC to enter clinical trials for glioblastoma during the second half of 2010. IMUC has entered into a research and license option deal with the Roche Group for one of the Company’s monoclonal antibody product candidates for the diagnosis and treatment of ovarian cancer and multiple myeloma, which provides for potential licensing and milestone payments of $32MM and royalties if the Roche Group exercises its option and commercializes this antibody technology for multiple indications. IMUC is in pre-clinical development of another monoclonal antibody product candidate for the treatment of small cell lung cancer and pancreatic cancer, and is also evaluating its platform technology for monoclonal antibody discovery to target cancer stem cells. To learn more about IMUC, please visit www.imuc.com.
This press release contains certain forward-looking statements that are subject to a number of risks and uncertainties, including without limitation, the risks associated with the potential inability to obtain FDA marketing approval for ICT-107 if another orphan drug version of this vaccine product receives FDA marketing approval first; potential inability to obtain licenses from third parties that will be needed to commercialize ICT-107 in many major commercial territories; the potential inability to secure a partner for ICT-107; the risk that future trials of ICT-107, if any, do not confirm the safety and efficacy data generated in the Phase I trial; the need to satisfy performance milestones to maintain the vaccine technology licenses with Cedars-Sinai; the risks associated with obtaining a patent that provides commercially significant protection for ICT-107; and the need for substantial additional capital to fund development of product candidates beyond their initial clinical or pre-clinical stages and to continue IMUC’s operations. Additional risks and uncertainties are described in IMUC’s most recently filed SEC documents, such as its most recent annual report on Form 10-K, all quarterly reports on Form 10-Q and any current reports on Form 8-K. IMUC undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
CEOcast, Inc. for ImmunoCellular Therapeutics
Dan Schustack, 212-732-4300
KEYWORDS: United States North America California
INDUSTRY KEYWORDS: Health Biotechnology Clinical Trials Oncology Pharmaceutical FDA