TGen, Scottsdale Healthcare, Mayo Clinic study new drug to stimulate immune system of cancer victims

December 19, 2008

SCOTTSDALE, Ariz. - Dec. 19, 2008 - The Translational Genomics Research Institute (TGen), Scottsdale Healthcare and Mayo Clinic are testing a new drug that could help cancer patients by stimulating the immune system.

Clinical trials of the drug VTX-2337 are being conducted at TGen Clinical Research Services at Scottsdale Healthcare, a partnership of Phoenix-based TGen and Scottsdale-based Scottsdale Healthcare Corp., and at Mayo Clinic in Arizona.

Dr. Ramesh Ramanathan, Medical Director of TGen Clincal Research Services at Scottsdale Healthcare, said the new drug appears promising.

"VTX-2337 is a new, novel, small molecule aimed at stimulating the immune cells in the blood, lymph nodes, and in and around the tumor. It represents an exciting new class of agents for cancer therapy with good preclinical evidence of activity,'' Dr. Ramanathan said.

The Phase I trial, a yearlong first-in-humans test, will study the drug's safety. If successful, a Phase II trial will test the drug's effectiveness on tumors.

A weakened immune system is often the result of advanced cancer. The hope is that this new drug will actually help enable the immune system to slow down the growth of tumors, and perhaps even shrink them, Dr. Ramanathan said.

VTX-2337 is the first drug of its kind developed by San Diego-based VentiRx Pharmaceuticals Inc. The biopharmaceutical company is focused on the development of new Toll-Like Receptor 8 (TLR8) agonists, which are small molecules that prompt a response in the body's immune system. The drugs are intended to treat cancer, respiratory and autoimmune diseases.

"VentiRx is very excited to be working with TGen, Scottsdale Healthcare and Mayo Clinic on this important and novel program,'' said Michael Kamdar, Executive Vice President and Chief Business Officer at VentiRx. "Entering Phase I clinical trials represents a significant milestone for VentiRx and our TLR efforts in that we have rapidly advanced into a clinical development company with a novel molecule that may play an important role and have broad application in the treatment of cancer."

VTX-2337 is a small molecule TLR8 agonist that is expected to be used in combination with standard of care for the treatment of patients with cancer. Preclinical evaluation of VTX-2337 suggests that it may play a key role in augmenting the innate arm of the immune system.

There are two broad components of the immune system, the innate arm, and the adaptive arm. Both generally aim to eliminate viruses and bacteria.

-- The innate arm senses infectious agents as they infect the body by recognizing structures they have in common, such as lipids, proteins, sugars, and nucleic acids (DNA and RNA). This is an initial rapid response, which is not precise but potent.

-- The adaptive arm of the immune system is instructed by the innate arm to devise more specific responses to unique components of the invading pathogens. This is a more precise response and takes longer, especially when an infectious agent is encountered for the first time.

The first clinical trial at TCRS at Scottsdale Healthcare will investigate the safety and pharmacology of multiple doses of VTX-2337 in patients with late-stage cancer. For more information about this clinical trial, please call Joyce Ingold, R.N., research patient care coordinator for Scottsdale Healthcare, at 480-323-1339.

The clinical trial coordinator for Mayo Clinic is Dianna Boughter, who can be reached at 480-301-9875.

"VTX-2337 is the first selective TLR8 compound to reach the clinic, and we are hopeful that modulation of the innate immune response will provide a benefit to patients in a number of oncology indications," said Dr. Robert Hershberg, Executive Vice President and Chief Medical Officer at VentiRx.
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About TGen

The Translational Genomics Research Institute (TGen) is a non-profit organization dedicated to conducting groundbreaking research with life changing results. Research at TGen is focused on helping patients with diseases such as cancer, neurological disorders and diabetes. TGen is on the cutting edge of translational research where investigators are able to unravel the genetic components of common and complex diseases. Working with collaborators in the scientific and medical communities, TGen believes it can make a substantial contribution to the efficiency and effectiveness of the translational process. For more information, visit: www.tgen.org.

Press Contact:
Steve Yozwiak
TGen Senior Science Writer
602-343-8704
syozwiak@tgen.org

About Scottsdale Healthcare

Scottsdale Healthcare is a primary clinical research site for TGen. TGen Clinical Research Services (TCRS) at Scottsdale Healthcare is housed in the Virginia G. Piper Cancer Center at Scottsdale Healthcare, located on the Scottsdale Healthcare Shea medical campus. Scottsdale Healthcare is the not-for-profit parent organization of the Scottsdale Healthcare Shea, Scottsdale Healthcare Osborn and Scottsdale Healthcare Thompson Peak hospitals, Virginia G. Piper Cancer Center, Scottsdale Clinical Research Institute, TGen Clinical Research Services at Scottsdale Healthcare, Scottsdale Healthcare Home Health Services, Scottsdale Healthcare Community Health Services, and Scottsdale Healthcare Foundation. For additional information, visit www.shc.org.

Contact:
Alice Sluga
Public Relations Coordinator
Scottsdale Healthcare
480-882-4915
asluga@shc.org

About Mayo Clinic

Mayo Clinic Cancer Center is one of only 39 U.S. medical centers that have been named as a National Cancer Institute (NCI) Comprehensive Cancer Center. To receive this designation, an institution must meet rigorous standards demonstrating scientific excellence and the ability to integrate diverse research approaches to address the problem of cancer. Mayo Clinic Cancer Center is the only national, multi-site center with the NCI's Comprehensive Cancer Center designation. In Arizona, Mayo's clinical and research experts work together to address the complex needs of cancer patients, with a dedication to understanding the biology of cancer; discovering new ways to predict, prevent, diagnose and treat cancer; and transforming the quality of life for cancer patients today and in the future.

About VentiRx Pharmaceuticals

VentiRx Pharmaceuticals Inc. is a biopharmaceutical company committed to the development and commercialization of novel medicines for the treatment of cancer, respiratory and autoimmune diseases. The company's initial focus is on developing small molecule TLR-based product candidates for oncology and allergy. VentiRx is a privately held organization headquartered in San Diego with operations in Seattle. For additional information, visit www.ventirx.com.

Contact:
Michael Kamdar
VentiRx Pharmaceuticals, Inc.
(858) 436-1525
mkamdar@ventirx.com

The Translational Genomics Research Institute

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