La Jolla Pharmaceutical Company Acquires Rights to Next-Generation Gentamicin Derivatives

La Jolla Pharmaceutical Company has entered into an exclusive option agreement to acquire the Indiana University Research and Technology Center’s (IURTC) intellectual property rights covering next-generation gentamicin derivatives. Gentamicin has become one of the most commonly prescribed hospital antibiotics, despite causing kidney toxicity. Gentamicin consists of a mixture of distinct but closely related chemical entities that may contribute differentially to the product’s toxicity profile. IURTC’s technology covers the use of next-generation, parenteral gentamicin derivatives as antimicrobial agents with the potential for reduced toxicity.

La Jolla also entered into a second option agreement with IURTC and the University of Alabama at Birmingham (UAB) for the use of these next-generation compounds for the treatment of certain rare genetic diseases, such as cystic fibrosis and Duchenne muscular dystrophy. Gentamicin’s ability to induce a lack of fidelity in gene transcription, intrinsic to its antimicrobial mechanism of action, can be leveraged in the correction of certain human genetic mutations that lead to rare genetic disorders. In spite of favorable short-term clinical proof-of-efficacy data in cystic fibrosis, development of gentamicin as a chronic treatment for these genetic diseases has been limited by its toxicity profile.

La Jolla has initially selected two lead development candidates from the technology, LJPC-30Sa and LJPC-30Sb, which are purified components of the currently marketed gentamicin product. LJPC-30Sa and LJPC-30Sb retain the biologic activity of gentamicin, yet appear to lack the traditional kidney toxicity associated with it. La Jolla plans to pursue a dual development strategy in serious bacterial infections and rare genetic disorders characterized by stop codon mutations, such as cystic fibrosis and Duchenne muscular dystrophy. Following a pre-Investigational New Drug application (IND) meeting with the FDA, La Jolla has received guidance that it may proceed with its proposed Phase I clinical trial following the submission of an IND.

“We are very pleased to gain access to this intellectual property covering next-generation gentamicin derivatives,” said George F. Tidmarsh, President and CEO of La Jolla. “The use of aminoglycoside antibiotics has been limited primarily by treatment-related toxicity. We believe that our next-generation gentamicin derivatives may retain the activity of gentamicin, but improve the therapeutic window, thereby improving the outcome for patients requiring antimicrobial agents and potentially creating new opportunities for the treatment of rare genetic disorders.”