Weekly Features


Biotech company based upon IU School of Medicine research earns double boost

Aug. 8, 2013

A biotechnology company based upon research conducted at the IU School of Medicine and technology licensed by IU Research and Technology Corp. recently received a boost from two prominent sources.

ApeX Therapeutics, a cancer-focused drug discovery and development company based upon research by Mark R. Kelley, Ph.D., received $240,332 July 18 through a Phase I, Small Business Innovation Research grant from the National Cancer Institute of the National Institutes of Health.


Mark R. Kelley, Ph.D.

In addition, BioCrossroads announced their investment in the company July 24 through the Indiana Seed Fund II, a $8.25 million early-stage fund focused on identifying, creating and developing the next generation of leading Indiana-based life sciences companies.

The funds will go towards ApeX’s mission to develop an oral or injectable medicine to more effectively treat leukemia and other cancerous tumors in children, including acute lymphoblastic leukemia, the most frequent cancer of childhood.

“It’s no secret that translating our excellent research into new therapies that help patients is an increasingly important goal for the IU School of Medicine — not to mention the National Institutes of Health and other funding organizations — ApeX is a great example of how this can happen,” said David Wilkes, M.D., executive associate dean for research affairs and August M. Watanabe Professor of Medical Research.

Dr. Kelley’s research has linked APE1 (or Ref-1), a protein that controls cell growth and cell death, with certain types of leukemia. Inhibiting this target causes leukemia cells in the blood and bone marrow to die. Ref-1 inhibitors do this by blocking formation of factors essential to leukemia cell growth and survival.

Although many children with leukemia now experience high cure rates and long-term survival, significant clinical challenges still remain, including leukemia recurrence in B-cell acute lymphoblastic leukemia; disease relapse, refractory disease and induction failure in T-cell acute lymphoblastic leukemia; and high relapse rates in adolescent and young adult patients.

Because these forms of leukemia are often associated with a grave prognosis, there is keen interest in research that translates into effective therapeutics benefiting this population of about 6,000 children per year. ALL remains the second cause of death for children.

“There are still those who do not respond to the standard treatments in this and other pediatric leukemias," said Dr. Kelley, associate director of the IU Herman B Wells Center for Pediatric Research and chief scientific founder of ApeX Therapeutics, noting investments from the NCI and BioCrossroads will help support new treatments for these vulnerable patients.

The ultimate goal of the project funded by these grants will be to develop an anticancer therapeutic agent in the form of oral or injectable medicine to more effectively treat leukemia and other tumors.

"ApeX is a great example of translational science - taking this important discovery from one of our leading research institutions to its first clinical trial with human patients," said David L. Johnson, president and CEO of BioCrossroads. “The companies executive team is comprised of leading experts and advisors from academia and industry that have come together to advance a platform that serves an unmet medical need in a very delicate population of patients – children."

Martin Haslanger, Ph.D., CEO of ApeX Therapeutics added that investments from the NCI and BioCrossroads will enable progress to the next milestone -- a pre-investigational new drug meeting with the U.S. Food and Drug Administration to establish a safe and efficient path to first-in-human trials in several different types of leukemia.”

"The results of the clinical trials are of particular translational interest since there is un-met medical need for effective therapy of relapse and refractory childhood leukemia,” he added.

In addition to Dr. Haslanger, ApeX also recently added industry veteran David Broecker to its board of directors. Broecker was President and CEO of Alkermes, a publically traded biotech company in Cambridge, Mass., and has more than 25 years leadership experience in the pharmaceutical, biotechnology and medical device industries, including establishing several start-up life sciences companies.

"We have a dynamic group of individuals leading ApeX to the next phase and we are excited for the promising future for the company, and more importantly for the impact our research will have on human lives," Haslanger added.

ApeX Therapeutics is also funded in part by the Innovate Indiana Fund and the Pearl Street Venture Fund.

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