CureFAKtor Pharmaceuticals, LLC, a privately-held biopharmaceutical company focused on the research and development of Focal Adhesion Kinase (FAK) inhibitors for cancer, announced that its lead compound, CFAK-C4, which is in development for the treatment of pancreatic cancer, has been granted Orphan Drug Designation by the US Food and Drug Administration (FDA).

Orphan status is given to drugs and biologics that are being developed to treat rare medical conditions, specifically those affecting fewer than 200,000 persons in the US. Orphan Drug Designation provides for a number of potential incentives related to CFAK-C4, including a seven-year period of marketing exclusivity, reduced regulatory fees, certain tax credits, and additional regulatory support for research and development initiatives.

“Receiving Orphan Drug Designation by the FDA is an important milestone for CureFAKtor, as well as for pancreatic cancer patients who have the lowest survival rate of any cancer and limited treatment options,” said H. Shep Wild, president and chief executive officer of CureFAKtor Pharmaceuticals. “CFAK-C4 is the first clinical candidate derived from our broad FAK technology platform and we look forward to progressing CFAK-C4 into human clinical trials.”

CureFAKtor Pharmaceuticals is planning a phase I clinical study of CFAK-C4 in combination with gemcitabine chemotherapy, the current standard of care for pancreatic cancer, in 2012 and based on fundamental discoveries of distinguished cancer researcher Dr William Cance, Chair, Surgical Oncology, Surgeon-in-Chief Roswell Park Cancer Institute.

The Company, to date, has been funded primarily by grants from the National Institutes of Health (NIH). CureFAKtor’s proprietary FAK technology platform may represent a significant breakthrough in the treatment of most solid tumours in that its unique mechanism of action disrupts the signalling of FAK in tumours by targeting specific protein to protein bindings. Since FAK is over-expressed in virtually all solid tumours, the technology has broad applicability in oncology.

More than 40 compounds have been identified that target FAK signalling sites. CFAK-C4, discovered by Dr Elena Kurenova, PhD associate professor, Roswell Park Cancer Institute, Senior Scientist and Co-Founder of CureFAKtor, is the lead product candidate in CureFAKtor’s pipeline. In pre-clinical studies, CFAK-C4 demonstrated efficacy in a number of cancers including pancreatic, breast, advanced melanoma and neuroblastoma.

The Company’s second product candidate, CFAK-Y15, discovered by Dr Vita Golubovskaya, PhD associate professor, Roswell Park Cancer Institute, Senior Scientist and Co-Founder of CureFAKtor, is being investigated for the treatment of breast, pancreatic, neuroblastoma and colon cancer.

CureFAKtor has attracted a renowned Board of Directors which includes David Keiser, former president and Co-Founder of Alexion Pharmaceuticals, Inc. and Dr Daniel Kleiner, Surgeon and Clinical Instructor at the University of Virginia. Scientific Advisory Board members include: Dr Murray Brennan, Former Chair of Surgery at Memorial Sloan-Kettering Cancer Centre; Dr Edison Tak-Bun Liu, executive director of Human Genome Institute of Singapore; and Dr Alex Adjei, senior vice president of Clinical Research and Chair of the Department of Medicine at Roswell Park Cancer Institute.

“Focal Adhesion Kinase protein binding inhibitors represent a promising area of research as FAK is expressed at extremely high levels in solid cancer tumours to serve as a survival mechanism by signalling tumour growth, invasion and metastasis. Dr William Cance’s groundbreaking work has shown that FAK protects tumours from chemotherapy, radiation and the body’s natural defences,” said Dr H. Shelton Earp III, MD, Professor and director of Lineberger Comprehensive Cancer Centre at the University of North Carolina at Chapel Hill School of Medicine. “While still early stage, I look forward to further research into the effect CFAK-C4 may have on solid tumours.”

Focal adhesion Kinase (FAK) is substantially over-expressed in many solid tumours. FAK operates by placing itself at the contact points between tumour cells and the extra cellular matrix that surrounds them. Studies by CureFAKtor and others found that in this role, FAK is an important facilitator for signals that cause tumour cells to survive, grow, and produce new blood vessels to sustain growth and travel to distant places within the body where they may establish new tumour sites. It also cocoons the tumour cells to protect them from the body's natural signalling mechanisms that would cause deviant tumour cells to be eliminated. In a similar fashion, FAK protects tumours from chemotherapeutic drugs and radiation, allowing the tumour cells to resist these therapies.

CureFAKtor Pharmaceuticals is a biopharmaceutical company focused on the research and development of Focal Adhesion Kinase therapies to prevent or treat cancer. CureFAKtor’s investigational and combination therapy products target pancreatic, breast, colon, melanoma, lung and brain oncology disorders. CureFAKtor research is conducted at the Roswell Park Cancer Institute.