UCSF Works with Anacor Pharmaceuticals and New York Blood Center to Discover Drugs 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.

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.

About the Sandler Center

Established in 2001, the Sandler Center for Basic Research in Parasitic Diseases is a research consortium at UCSF dedicated to the discovery of affordable, safe and effective drugs to treat the "neglected tropical diseases,” including Chagas’ disease, sleeping sickness, leishmaniasis and schistosomiasis. For further information, visit http://www.sandler.ucsf.edu.

About the New York Blood Center and the Lindsley F. Kimball Research Institute

New York Blood Center (NYBC) is one of the nation's largest non-profit, community-based blood centers. NYBC has been providing blood, transfusion products and services to hospitals since 1964 serving more than 20 million people in New York City, Long Island, the Hudson Valley, New Jersey, and parts of Connecticut and Pennsylvania. NYBC is also home to the Lindsley F. Kimball Research Institute (LFKRI) and the National Cord Blood Program at the Howard P. Milstein National Cord Blood Center, the world's largest public cord blood bank. NYBC provides medical services and programs (Clinical, Transfusion, and Hemophilia Services) through its medical professionals along with consultative services in transfusion medicine. Please visit us on Facebook at www.Facebook.com/newyorkbloodcenter. Follow us on Twitter: @NYBloodCenter.

Since 1964, LFKRI has led the way in blood research, breaking new ground in transfusion medicine and disease treatment and prevention. The institute is committed to furthering research efforts that support the discovery of new blood-related products, techniques, and therapies. LFKRI’s work has dramatically impacted global health, improved blood banking, nurtured a generation of scientists, and added significantly to the world’s store of biomedical knowledge. From the beginning, LFKRI has supported basic research to understand blood and disease at the molecular level as well as translational research that transforms the findings into major breakthroughs. With 14 state-of-the-art laboratories and more than 100 researchers, LFKRI brings world-class research to life every day. For further information, visit http://www.nybloodcenter.org/research-homepage.do?sid0=7.

About the Biocomputing and Media Research Group

The Biocomputing and Media Research group at San Francisco State University is an interdisciplinary research lab whose primary focus is on research problems at the intersection of computing, drug discovery, and Biology with special emphasis on the neglected diseases of Mankind. In addition to R&D activities, the mission of the group includes training students to tackle emerging scientific and technological challenges. The group is lead by Dr. Rahul Singh. For further information visit: http://tintin.sfsu.edu/WebHome.html.

About Anacor Pharmaceuticals

Anacor is a biopharmaceutical company focused on discovering, developing and commercializing novel small-molecule therapeutics derived from its boron chemistry platform. Anacor has discovered and is developing five clinical compounds, including its three lead programs: AN2690, a topical antifungal for the treatment of onychomycosis; AN2728, a topical anti-inflammatory PDE-4 inhibitor for the treatment of psoriasis; and GSK 2251052, or GSK ‘052 (formerly referred to as AN3365), a systemic antibiotic for the treatment of infections caused by Gram-negative bacteria, which has been licensed to GlaxoSmithKline under the companies' research and development agreement. In addition, Anacor is developing AN2718 as a topical antifungal product candidate for the treatment of onychomycosis and skin fungal infections, and AN2898 as a topical anti-inflammatory product candidate for the treatment of psoriasis and atopic dermatitis. For more information visit www.anacor.com.

About Anacor’s Neglected Disease Initiative

Neglected diseases are defined as diseases that disproportionately affect the world’s poorest people, including tuberculosis or TB, malaria, visceral leishmaniasis, Chagas disease, human African trypanosomiasis or African sleeping sickness, and filarial worm diseases such as onchocerciasis and lymphatic filariasis. Despite the fact that these diseases cause significant morbidity and mortality worldwide, and that the current standards of care are difficult to administer, have significant toxicities and are increasingly becoming less effective due to the development of resistance, there has been little investment in developing new therapies for these diseases due to the absence of a reasonable expectation of a financial return.

In recent years, a number of foundations and governments have created public-private partnerships to address this gap by funding promising technologies that may result in new drugs. In December 2007, Anacor established a partnership with the Drugs for Neglected Diseases initiative, or DNDi, to develop new therapeutics for African sleeping sickness, visceral leishmaniasis and Chagas disease. In May 2009, Anacor established a collaboration with the Global Alliance for TB Drug Development. In April 2010, Anacor entered into a research collaboration with the Medicines for Malaria Venture to identify lead compounds for the treatment and prophylaxis of malaria.

Forward-Looking Statements

This press release may contain forward-looking statements that relate to future events, including the potential discovery, development and commercialization of drug therapies for river blindness and other diseases. These forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual levels of activity, performance or achievement to differ materially from those expressed or implied by these forward-looking statements, including risks related to the difficulty in discovering new potential drug therapies, risks related to intellectual property position of any drug candidate we may identify, the difficulty and uncertainty of achieving regulatory approval for any drug candidate we may identify and, if we promote any drug candidate for river blindness to clinical development, the risks related to the commencement, enrollment and successful completion of clinical trials and risks related to unforeseen side effects. These statements reflect the views of Anacor as of the date of this press release with respect to future events and, except as required by law, it undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise after the date of this press release.