La Jolla Pharmaceutical Acquires Rights To Gentamicin Derivatives For Cystic Fibrosis Treatment

In a recent agreement with the Indiana University Research and Technology Corp. (IURTC), La Jolla Pharmaceutical Co. acquired intellectual property rights covering next-generation derivatives of gentamicin, an antibiotic used in the treatment of several types of bacterial infections, namely heart, urinary tract, and pregnancy-related infections.

Gentamicin treatment is effective but can only be performed for a limited time period as prolonged exposure to this antibiotic leads to kidney toxicity. Researchers at Indiana University have identified distinct components in gentamicin that are potentially less toxic. The goal of La Jolla Pharmaceuticals is to adapt these gentamicin derivatives in order to improve treatment for bacterial infections. Based on another agreement between La Jolla, IURTC and the University of Alabama at Birmingham (UAB), La Jolla is eligible to use gentamicin derivatives also for the treatment of certain genetic disorders like cystic fibrosis (CF).

CF is a life-threatening genetic disease in which a defective gene (CFTR) causes the body to form unusually thick, sticky mucus that can obstruct the airways and promote dangerous lung infections resulting in serious respiratory and also gastrointestinal manifestations. The majority of the CF patients die due to respiratory failure. There is no cure for the disease and the therapies available aim at the management of disease symptoms. It is estimated that almost 70,000 individuals worldwide suffer from CF, including 30,000 in the United States.

The development of antibiotics like gentamicin is crucial to the CF community, since being able to effectively treat bacterial infections in the lung is key to increasing the survival rate of those with the disease.

Short-term clinical tests with gentamicin revealed a favorable proof-of-efficacy against CF, although its application as a chronic treatment is also impaired by toxicity issues. La Jolla has now two next-generation gentamicin derivatives – LJPC-30Sa and LJPC-30Sb, and the company has reported that both antimicrobial compounds maintain gentamicin’s biological properties while apparently lacking the toxicity effects associated with it.

The company’s plan is to use these gentamicin derivative compounds as “lead candidates” in the development of therapeutic strategies against serious bacterial infections and rare genetic disorders, including CF.

“We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives,” said La Jolla’s President and CEO, Dr. George Tidmarsh in a news release. “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.”