Anacor, UCSF and NYBC collaborate to discover drug therapies for river blindness

Anacor Pharmaceuticals (NASDAQ:ANAC), the University of California San Francisco (UCSF) Sandler Center and the Lindsley F. Kimball Research Institute (LFKRI) of the New York Blood Center (NYBC) today announced the establishment of a research and development collaboration to discover new drug therapies for the treatment of river blindness (onchocerciasis), a parasitic disease that is the second leading cause of infectious blindness worldwide, and is most prevalent in Africa. The collaboration will combine Anacor's novel boron-based chemistry platform and drug discovery and development capabilities with the Sandler Center's expertise in neglected disease biology and drug discovery and the LFKRI's expertise in onchocerciasis. The collaboration's goal is to identify a novel, potent macro-filaricidal drug candidate that is capable of killing adult worms. Current medications kill only microfilaria, resulting in the need to treat the same infected individual repeatedly over several years to outlast the lifecycle of the adult worms and to stop transmission of the disease. A drug that kills adult worms would simplify ongoing onchocerciasis elimination programs and provide improved outcomes to patients and communities affected by this devastating disease.

“Today, LFKRI houses one of the world's largest collections of Onchocerca larvae and is the global leader in understanding their basic biology and host-parasite interactions. Our shared goal is to discover new ways to intervene in key pathways essential for parasite development, survival and/or propagation.”

The collaboration will bring together drug discovery experts from Anacor's neglected diseases program, James H. McKerrow, PhD, Robert E. Smith professor of experimental pathology and the leader of the Sandler Center for Drug Discovery at UCSF, and Sara Lustigman, PhD, head of the Laboratory of Molecular Parasitology, Lindsley F. Kimball Research Institute, New York Blood Center. The collaboration also includes the BioComputing and Media Research Group led by Rahul Singh, PhD, an associate professor of computer science at San Francisco State University, which will develop algorithms and software to automate the screening process for lead identification. Funding for the project is being provided by the Bill and Melinda Gates Foundation (BMGF) through a grant to UCSF over two years for a total of $3.61 million, of which Anacor will receive $2.24 million.

"The Sandler Center has been a research pioneer in the development and application of state-of-the-art drug development techniques to identify new therapeutic strategies to combat deadly parasitic diseases that prey upon the world's poorest populations, and in working with collaborators to transform biological insights into innovative medicines," said Dr. McKerrow. "We have a long-standing history of collaboration with Anacor on applying their novel boron-based chemistry platform to neglected parasitic diseases. We believe this approach holds significant promise to discover novel and potent compounds that can potentially meet this significant unmet need and help prevent unnecessary blindness."

"Our guiding principle is that academic/non-profit/ private collaborations can best bring together the needed technological expertise to address neglected diseases, and we appreciate the opportunity to join forces with UCSF and Anacor's discovery and development experts to eradicate river blindness," said Dr. Lustigman. "Today, LFKRI houses one of the world's largest collections of Onchocerca larvae and is the global leader in understanding their basic biology and host-parasite interactions. Our shared goal is to discover new ways to intervene in key pathways essential for parasite development, survival and/or propagation."

River blindness afflicts over 37 million people, primarily in Africa, and is the second most common cause of infectious blindness. It is spread by a biting black fly that hosts the parasite, Onchocerca volvulus, in an early phase of its five-stage life cycle. Adult worms live in humans in subcutaneous tissues for up to 15 years and produce millions of minute worms called microfilaria. These burrow under the skin and cause debilitating, severe itching, and when they invade the eyes, they produce lesions that can lead to blindness. While the primary focus is development of a macrofilaricidal drug candidate for the treatment of onchocerciasis, it is expected that parallel screening of the closely related filarid, Brugia malayi will also yield drug candidates for the treatment of lymphatic filariasis, a disfiguring and disabling neglected parasitic disease. The filarial nematodes causing elephantiasis are transmitted by mosquitos and infect 120 million people in developing countries. These parasites can live for 5 years or more and reside in the lymphatic system.