ImmunoCellular Therapeutics seeks FDA orphan drug status for ICT-107 dendritic-cell based vaccine for GBM

ImmunoCellular Therapeutics (OTCBB: IMUC) announced today it has filed an orphan drug application with the U.S. Food and Drug Administration (FDA) for its cancer treatment candidate ICT-107. ICT-107 is a dendritic-cell based cancer vaccine product which targets glioblastoma multiforme (GBM), the most prevalent, aggressive and malignant form of primary brain tumors.

“By harnessing the body's own immune system, ICT-107 has been shown to keep glioblastoma patients free of disease for significantly longer than has been seen with current standard treatments”

"By harnessing the body's own immune system, ICT-107 has been shown to keep glioblastoma patients free of disease for significantly longer than has been seen with current standard treatments," said Manish Singh, Ph.D., ImmunoCellular Therapeutics' president and CEO. "We are applying for orphan drug status with the goal of advancing this promising cancer vaccine candidate to market as efficiently as possible."

U.S. 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. If the status is granted by the FDA, the company will be eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA review and allowance for marketing exclusivity after drug approval for a period of as long as seven years.

In a recent Phase I study of ICT-107 in GBM, newly diagnosed patients who received the vaccine demonstrated a median progression-free survival (PFS) of 17.7 months after surgery. This compared favorably 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 continue to live with no disease progression with an average time of over two years, which is significantly better than historical data of less than 15% PFS at two years. Safety data for ICT-107 also compared favorably to currently available treatments: no serious adverse events were reported and minor side effects were limited to mild (grade 1) fatigue, skin rash and pruritis.