USC Keck School of Medicine's Dr. Alan Epstein Receives Prestigious National Cancer Institute RAID Award For Breakthrough IL-2 Molecule Therapeutic

July 7, 2009

$3.5 Million Award Will Advance Clinical "Proof of Principle"
for Expanded Clinical Evaluation

Second RAID Award for Dr. Epstein

LOS ANGELES (July 7, 2009) – The University of Southern California today announced that Alan Epstein, M.D., Ph.D., Professor, Department of Pathology at the USC Keck School of Medicine of USC, has received approval for a $3.5 million drug development project through the Rapid Access to Intervention Development (RAID) program of the National Cancer Institute (NCI) for his breakthrough Interleukin-2 (IL-2) cytokine immunotherapy analog.   This is the second RAID award Dr. Epstein has received from the National Cancer Institute.

The NCI RAID program is a peer-reviewed competitive award program designed to assist translation of novel anticancer therapeutics to the clinic.  The goal of the program is to show proof of principle that a new molecule is a viable candidate for expanded clinical evaluation. As a RAID award recipient, Dr. Epstein will receive access to drug development resources of the Developmental Therapeutics Program. These resources will be used to further the preclinical development of his IL-2 analog therapy for the treatment of certain cancers that appears to retain the benefits of existing IL-2 therapies without the major side effects that currently limit their application.

IL-2 cytokine immunotherapy is used to treat metastatic melanoma and metastatic renal cell carcinoma, diseases that afflict over 60,000 and 50,000 people in the United States each year, respectively.  An increase in melanoma incidence has elevated the disease to the sixth most common cancer in the United States.   Approximately 60,000 Americans developed invasive cutaneous melanoma in 2007, with an estimated additional 48,000 or more cases of melanoma in situ. The current lifetime risk for developing invasive melanoma is 1 case per 60 Americans.  Currently, IL-2 is the only FDA approved immunotherapeutic agent.

But while the current therapy provides unparalleled recovery for certain cancer patients, the application of IL-2 is severely limited by its significant toxicity and resulting side effects, especially that of capillary leak syndrome where leakage of fluid from the blood results in dangerously low blood pressure (hypotension), edema and multiple organ failure. Capillary leak syndrome affects as many as 65% of patients and can lead to the cessation of therapy.

Dr. Epstein has developed an IL-2 analog that eliminates the side effect of capillary leak syndrome while retaining the cytokine and therapeutic activity of IL-2.  His extensive pre-clinical data indicates that this IL-2 analog will allow more clinically effective doses to be administered, resulting in significantly improved disease control with fewer complications. 

“We believe the full potential of IL-2 has yet to be realized because of its limiting toxicity. This discovery could mean an exponentially improved success rate for cancer therapies,” said Dr. Epstein.  “I am thrilled NCI saw the priority value of this therapeutic and awarded RAID assistance to help propel the manufacturing of this novel molecule toward Phase 1 clinical trials.”

“We are in the midst of a biomedical revolution. New technologies promise to reshape medicine – with novel applications in new therapies, drug delivery, and patient care,” said Dr. Richard Hull, Senior Director of Innovation Advancement and Business Development for the USC Stevens Institute for Innovation. “The work and success of Dr. Epstein is a great example of how USC is focused on translating top-notch laboratory research into breakthrough clinical applications that save lives and improve healthcare.”

Pivotal BioSciences, Inc., a privately-held Los Angeles-based biotechnology company developing innovative low toxicity therapies for cancer, has licensed the molecule from USC.  The technology is covered under U.S. Patent #7371371 issued on May 13, 2008 titled, “Interleukin-2 Mutants with Reduced Toxicity.” 

Dimitri Villard, CEO of Pivotal BioSciences, Inc. added, “We are delighted that the National Cancer Institute’s RAID Program will undertake the critical next steps of process development, manufacturing and toxicology studies that will enable us to apply to the FDA for a Phase I clinical trial. This fortuitous award enables us to bridge the funding gap that so many pre-clinical stage biotechnology companies have experienced lately; we are looking forward to working with Dr. Epstein and USC Stevens to see this important project through to fruition.”

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About the Keck School of Medicine of USC
The Keck School of Medicine of the University of Southern California trains tomorrow’s leaders in patient care and biomedical research and provides advanced health care to the people of Southern California. Increasingly, the Keck School of Medicine of USC is expanding its biomedical research enterprise and, in so doing, raising its profile as a premier medical school. At present, the Keck School of Medicine of USC ranks among the nation’s top 25 percent of medical schools in federally sponsored research (Association of American Medical Colleges, 2003). Research programs in genetics, cancer, neurogenetics, transplantation medicine, and other key areas have attracted national recognition. The School’s dual commitments to collaborative interdisciplinary research and rapidly translating scientific findings from the lab bench to the patient’s bedside have proved pivotal in its continuing success in research.

About the USC Stevens Institute for Innovation
The USC Stevens Institute for Innovation (http://stevens.usc.edu) is a university-wide institute in the office of the Provost designed to harness the creative thinking and innovative work at all of USC’s 17 professional schools, the USC College, and research centers across campus to build a multidisciplinary approach to innovation. To empower USC innovators to make maximum societal impact with their ideas, the USC Stevens Institute identifies, nurtures, protects, and transfers to the market the most exciting innovations from USC, and in turn, provides a central connection for industry seeking cutting edge innovations in which to invest. Furthermore, the USC Stevens Institute develops the innovator as well as innovations, through educational programs, community-building events, and showcase opportunities.

About Pivotal BioSciences, Inc.
Pivotal BioSciences, Inc. is an angel-backed spin-out of the University of Southern California Keck School of Medicine that has been developing several promising discoveries in the field of oncology licensed from USC. For more information, visit www.pivotalbiosciences.com.

Media Inquiries:

Elisa Wiefel Schreiber
USC Stevens Institute for Innovation
University of Southern California
p: 213-821-6063  f: 213-821-5001
wiefel@usc.edu

Dimitri Villard
Pivotal BioSciences, Inc.
310-289-7111
villard@pivotalbiosciences.com

Leslie Ridgeway
Keck School of Medicine of USC
323-442-2823
213-200-8015
leslie.ridgeway@usc.edu