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Compounds identified in IU-led research are ‘lead candidates’ for improved cystic fibrosis treatment


INDIANAPOLIS — A San Diego-based drug maker plans to adapt antibiotic derivatives identified through Indiana University-led research toward improved treatment of bacterial infections and genetic disorders such as cystic fibrosis.

Under an agreement with the Indiana University Research and Technology Corp., La Jolla Pharmaceutical Co. obtained an exclusive option to acquire intellectual property rights to “next-generation” offshoots of gentamicin. Gentamicin is part of a class of hospital-grade antibiotics known as aminoglycosides that are commonly used to treat staph, urinary tract, heart and pregnancy-related infections.

Despite its effectiveness, gentamicin can only be used for a limited time because prolonged exposure leads to kidney toxicity. However, research led by Dr. Bruce Molitoris of IU’s Department of Medicine identified purified components of gentamicin that potentially are less toxic.

Under another agreement with IURTC and the University of Alabama at Birmingham, La Jolla also obtained the option to apply gentamicin derivatives toward treatment of certain genetic diseases that include cystic fibrosis.                       

In short-term clinical tests, gentamicin’s antimicrobial qualities showed favorable proof-of-efficacy against cystic fibrosis, yet its development as a chronic treatment also is limited by its toxicity. However, two IURTC compounds selected by La Jolla — LJPC-30Sa and LJPC-30Sb — retain gentamicin’s biologic activity while appearing to lack such toxicity.

La Jolla plans to use the compounds as “lead candidates” to develop therapies for serious bacterial infections as well as cystic fibrosis. The pharmaceutical company also may evaluate their potential to treat other genetic disorders such as Duchenne muscular dystrophy.

 “We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives,” said Dr. George Tidmarsh, La Jolla’s president and CEO. “We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin but improve the therapeutic window, thereby potentially increasing the market opportunity as antimicrobial agents and potentially creating new opportunities for aminoglycosides in rare genetic disorders.”

Cystic fibrosis is a life-threatening genetic disease found in about 70,000 people worldwide, including 30,000 in the United States, according to the Cystic Fibrosis Foundation. The disease is caused by an inherited, defective gene and mainly affects the lungs and digestive system. Although cystic fibrosis has yet to be cured, specialized care, drug treatments and therapies have significantly lengthened and improved the quality of life for patients.

About the Indiana University Research and Technology Corp.

IURTC is a not-for-profit agency that helps IU faculty and researchers realize the commercial potential of their discoveries. Since 1997, IURTC’s university clients have accounted for more than 2,800 inventions, nearly 1,900 patent applications and 77 startup companies. IURTC is part of the Innovate Indiana initiative, which engages strategic partners to leverage and advance IU’s intellectual resources and expertise, enhance Indiana’s economic growth and contribute to the overall quality of life for Hoosiers.

About La Jolla Pharmaceutical Co.

La Jolla Pharmaceutical Co. is a biopharmaceutical company focused on the discovery, development and commercialization of innovative therapies intended to significantly improve outcomes in patients suffering from life-threatening diseases. The company has several product candidates in development. LJPC-501 is La Jolla’s proprietary formulation of angiotensin II for the potential treatment of catecholamine-resistant hypotension and hepatorenal syndrome. LJPC-401 is La Jolla’s novel formulation of hepcidin for the potential treatment of conditions characterized by iron overload, such as hemochromatosis and beta thalassemia. LJPC-30Sa and LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for the potential treatment of serious bacterial infections and rare genetic disorders, such as cystic fibrosis and Duchenne muscular dystrophy.