Buffalo-Made Anti-Cancer Drug Begins Human Trials

BUFFALO, N.Y. -- An anti-cancer drug developed by Kinex Pharmaceuticals of Buffalo and a University at Buffalo faculty researcher has begun clinical testing with patients.

As part of FDA-mandated phase 1 testing, KX2-391 is being administered to a group of patients with advanced cancer who have not responded to other therapies. In non-human testing, the drug has been shown to be active against all cancers, according to Kinex Pharmaceuticals CEO Allen Barnett.

KX2-391 may be the first small-molecule drug discovered and developed in Buffalo that has progressed to the human trial stage. The phase 1 trial is a first step toward FDA approval of the drug and is intended to test the safety and dosage tolerability of the drug.

"We're very excited about the drug's potential," Barnett says. "As we go further in the drug's development, and do broader testing, we get better and better data. If the drug works half or a third as well as it's worked in pre-clinical trials, it will have blockbuster, billion-dollar potential."

During non-human testing by Kinex over the past two years, KX2-391 has reduced tumors in several types of cancer. Though the drug must complete two additional phases of testing after phase 1, Kinex has attracted significant interest from venture capital firms, private investors and several major pharmaceutical companies, Barnett says.

Barnett is negotiating with several pharmaceutical companies considering funding Kinex's development of the drug through phase 1 and other clinical phases needed to test the drug's effectiveness with larger patient populations. A deal, which would include an upfront payment, milestones and royalties, could be made by early next year.

An alternate source of funding would be via venture capital investment, which would provide necessary funds to progress KX2-391 further in clinical trials before partnering with a large pharmaceutical company in a bigger deal. Those discussions are in progress as well.

The drug was created from the work of David Hangauer, Ph.D., UB associate professor of chemistry, who developed a compound that targets Src ("sark") kinase, a protein that is linked to the survival of cancer cells. Hangauer's drug compounds, known as protein kinase inhibitors, are designed to shrink tumors and prevent metastases.

Kinases are considered one of the most lucrative classes of drug targets in the pharmaceutical industry, and Hangauer is the first to develop a kinase drug that targets a unique site on the kinase target. KX2-391 is the first in this class of drugs to progress to the clinical trial stage.

"We have the first success to come from this approach," explains Hangauer, who also serves as Kinex senior vice president of research and development. "Our drug compound has been shown to be active against all cancers.

"Cancer is a very tough disease to treat with drugs," he adds, "but we think this will be better than any kinase inhibitor currently available."

Hangauer also sees great potential for using related drug compounds, under development at Kinex, to treat autoimmune diseases like lupus, ulcerative colitis and rheumatoid arthritis.

Roswell Park Cancer Institute and M.D. Anderson Cancer Center in Houston are performing the phase 1 trials, which are expected to enroll a total of 50 patients and last about a year. Alex Adjei, Ph.D., M.D., senior vice president of clinical research at Roswell, is principal investigator for the phase 1 study at Roswell. Adjei is a national leader in translational research, drug development and thoracic oncology.

The drug was synthesized by Albany Molecular Research Institute, a contract research organization, and the final dosage form for human trials was prepared in UB's New York State Center of Excellence in Bioinformatics and Life Sciences, where Kinex Pharmaceuticals is headquartered. Kinex's progress has been bolstered by significant cost savings from their use of Center of Excellence facilities and its funding programs, including funding from the UB Center for Advanced Biomedical and Bioengineering Technology (UB CAT).

Kinex originally licensed three patent filings from UB that describe the drug's makeup, as well as a methodology for the design and synthesis of this and other kinase inhibitors. Kinex now has four issued patents and six other filings under review.

Marnie LaVagine, Ph.D., director of business development at UB's Center of Excellence, says the drug's progression to human trials "is a critical milestone" in the development of a local life-sciences economy.

"We're banking on our ability to create private-sector jobs in drug discovery and development, rather than licensing university-developed technologies to out-of-state firms, which had been the more common path for moving locally grown inventions from the lab to the marketplace."

Success for KX2-391 and Kinex would be a huge win for Buffalo's emerging biotechnology industry, agrees Lyn Dyster, Ph.D., Kinex vice president for operations, who, like Barnett and Hangauer, earned a doctoral degree at UB.

"Big pharma is thriving by licensing innovations from small-drug discovery and development companies like ours," Dyster says. "The success of Kinex and other Buffalo companies like Smart Pill will put Buffalo on the biotech map and help other local companies grow."

Collaboration between scientists at UB's Center of Excellence, Roswell and Hauptman Woodward Medical Research Institute -- as well as the attraction of new companies like Cleveland Biolabs to Buffalo -- should continue to fuel groundbreaking life-sciences research in Buffalo, according to Barnett.

"You're starting to see the right kinds of scientific and entrepreneurial activity and partnerships you need to build a biotech industry in Buffalo," Barnett says. "If Kinex is successful, you'll start to see more local investment in local companies and also more investors from the outside starting to take a closer look at Buffalo opportunities."

The University at Buffalo is a premier research-intensive public university, the largest and most comprehensive campus in the State University of New York. UB's more than 28,000 students pursue their academic interests through more than 300 undergraduate, graduate and professional degree programs. Founded in 1846, the University at Buffalo is a member of the Association of American Universities.