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    Neglected Diseases

Funds from public-private partnerships like the TB Alliance and DNDi make it possible to meet both our responsibilities to our shareholders and help reduce the burden of neglected diseases. Learn more about Kerydin
Learn more about AN2728



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, (HAT or sleeping sickness), and filarial worms. 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 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.

Our boron chemistry platform appears to be particularly well suited for the treatment of these types of infectious diseases, and we feel a responsibility to apply our technology to the development of new treatments. Until such time as we are profitable, however, we are committed to doing that research only when we can use grants and other non-dilutive sources of funding in a cash-neutral manner.

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, we established a partnership with the Drugs for Neglected Diseases initiative, or DNDi, to develop new therapeutics for sleeping sickness, visceral leishmaniasis and Chagas disease. In May 2009, we established a collaboration with the Global Alliance for TB Drug Development. In April 2010, we entered into a research collaboration with the Medicines for Malaria Venture to identify lead compounds for the treatment of prophylaxis of malaria.

Our work in this area also allows us the potential benefits of expanding the chemical diversity of our boron compounds, understanding new properties of our boron compounds, receiving future incentives, such as the potential grant of a priority review voucher by the FDA, and, ultimately if a drug is approved, potential revenue in some regions.

Sleeping Sickness

Sleeping sickness, which threatens millions in 36 countries in sub-Saharan Africa, is fatal if left untreated. The disease is caused by parasites transmitted by the bite of a tsetse fly and is often asymptomatic for years until the infection reaches "stage 2" where it crosses into the central nervous system and brain. Without effective treatment, sleeping sickness is fatal. Currently available treatments are limited to drugs developed decades ago that can be highly toxic, difficult to administer in resource-limited settings, or are only effective in one stage of the disease. In addition, prior to being treated, the stage of the disease must be determined using a diagnostic spinal tap to extract cerebrospinal fluid from the patient.

Our Contribution to a Potential Solution
In March 2012 DNDi announced the initiation of Phase 1 clinical trials of AN5568 (also referred to as SCYX-7158), the first new investigational oral drug candidate discovered specifically to combat sleeping sickness.

Visceral Leishmaniasis

VL mainly affects populations in 70 countries across Asia, East Africa and South America. Worldwide around 350 million people are at risk. The parasite that leads to infection is called Leishmania and transmitted by sandflies. Existing treatments are difficult to administer, toxic, and costly. Drug resistance is also an increasing problem.

Chagas Disease

Chagas disease is endemic in 21 countries across Latin America and kills more people in the region than any other parasite-borne disease, including malaria. In total, 100 million people are at risk worldwide and patient numbers are growing in non-endemic countries such as the United States, Australia, and Europe. The disease is transmitted by an insect known as the "kissing bug" and without treatment, is potentially fatal. Existing treatments may present limitations related to efficacy and toxicity.

Our Contribution to Potential Solutions
In May 2009, Anacor announced that it had extended an existing license and drug development agreement with not-for-profit, Drugs for Neglected Diseases initiative (DNDi) to develop new therapeutics for Human African Trypanosomiasis (HAT or African Sleeping Sickness), Visceral Leishmaniasis (kala azar) and Chagas disease. Under the terms of the agreement, Anacor will provide DNDi with a non-exclusive, royalty-free license for any boron-based therapeutics for neglected diseases identified through the collaboration for developing world countries.


About Malaria
Malaria is a preventable and treatable infectious disease transmitted by mosquitoes that kills almost one million people each year, most of them in sub-Saharan Africa, where malaria is the leading cause of death for children under five.

Malaria is not only a major killer in Africa but a primary cause of death and poverty - undermining development in some of the poorest countries in the world. Though the majority of the cases and the malaria deaths are found in Sub-Saharan Africa, the disease is also present in Asia and Latin America.

Our Contribution to a Potential Solution
In April 2010, we entered into a research agreement with not-for-profit Medicines for Malaria Venture (MMV) to explore Anacor's boron chemistry platform for developing new therapeutics for the treatment of malaria. Under the agreement, Anacor is responsible for leading the research effort and MMV contributes its extensive malaria drug research expertise as well as financial support. In March 2011, we entered into a development agreement with MMV to develop AN3661, the first investigational candidate arising out of the research agreement to move into preclinical development. Under the new development agreement, Anacor and MMV will work together on the development of AN3661 through human proof-of-concept studies.

River Blindness

About River Blindness
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 an investigational macrofilaricidal drug candidate for the treatment of onchocerciasis, it is expected that parallel screening of the closely related filarid, Brugia malayi may also yield investigational 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.

Our Contribution to a Potential Solution
In November 2010, we signed 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 combines 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 Lindsley F. Kimball Research Institute 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 wormscwould simplify ongoing onchocerciasis treatment programs and provide improved outcomes to patients and communities affected by this devastating disease.

Funding for the project is being provided by the Bill and Melinda Gates Foundation through a grant to UCSF over two years for a total of $3.61 million, of which Anacor will receive $2.24 million.


About Tuberculosis
Every year, more than 1.6 million people worldwide die from TB. Today's TB drugs are more than 40 years old, and a cocktail of up to four drugs must be taken for six to nine months to treat drug-sensitive disease and up to 24 months to treat drug-resistant disease. Long, demanding treatment schedules prove too much for many patients and the resulting erratic or incomplete treatment can result in drug resistance, treatment failure or death.

Our Contribution to a Potential Solution
In May of 2009, we entered into a license and research agreement with not-for-profit Global Alliance for TB Drug Development (TB Alliance) to explore a novel investigational anti-bacterial drug target for use in tuberculosis (TB) therapy. Under the agreement, Anacor will provide the TB Alliance with a non-exclusive, royalty-free worldwide license for TB. Anacor will receive support from the TB Alliance for its prominent role in the joint research efforts.


About Shigellosis (Bloody diarrhea)
Shigellosis, or bloody diarrhea, is an infectious disease caused by the gram-negative bacterium Shigella. An estimated 80 to 165 million people worldwide are impacted by this disease annually, which is responsible for an estimated 600,000 deaths each year, mostly among children in the poorest countries. Current therapies are losing their effectiveness due to bacterial resistance, which demands the development of new medicines to ensure effective treatment against this deadly disease.

Our Contribution to a Potential Solution
In March 2011, we announced we had established a joint research agreement with the Institute for OneWorld Health (iOWH) to discover investigational antibacterial compounds for treating shigellosis. iOWH is a non-profit that develops safe, effective and affordable new medicines for people with infectious diseases in the developing world with emphasis on diseases that disproportionately affect children. iOWH is currently focused on developing a treatment for children with cholera and other life threatening diarrheal diseases. iOWH received significant funding for this project from the Department for International Development (DfID) in the United Kingdom for research and development of medicines to treat diarrheal diseases. iOWH has also received funding from the Bill & Melinda Gates Foundation to develop medicines to treat diarrheal diseases, and has research collaborations with Roche and Novartis.


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