Anacor Pharmaceuticals (NASDAQ:ANAC), the Drugs for Neglected Diseases initiative (DNDi), and SCYNEXIS Inc. today announced the successful completion of pre-clinical studies for the first new oral drug candidate discovered specifically to combat human African trypanosomiasis (HAT), also known as sleeping sickness. An article released today in the open-access journal PLoS Neglected Tropical Diseases, reveals the initial successful results of pre-clinical studies of the new compound, which will soon advance to Phase I human clinical trials.
“This innovative collaboration, expounding upon the expertise and engagement of two biotech companies and a not-for-profit R&D organization, has brought excellent results thus far, and this in an incredibly short timeframe”
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 are either 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.
Two-thirds of all reported sleeping sickness cases are found in the Democratic Republic of the Congo (DRC), where healthcare is often inaccessible to large parts of the population due to violent conflict, great distances patients must travel to health facilities, and extreme poverty. Dr. Miaka Mia Bilenge, special advisor to the National Control Program for Human African Trypanosomiasis in the DRC, explained that if the new drug candidate proves to be successful, it could become a major weapon to control sleeping sickness.
"People in the DRC and many countries in Africa have hoped for a safe, easy-to-use, and effective treatment against sleeping sickness for generations," Dr. Miaka Mia Bilenge said. "We very much look forward to the start of human trials, and we hope that at long last this will give us a tool for sustainable elimination of this dreaded disease."
In pre-clinical studies, the new drug candidate, SCYX-7158, also registered as AN5568, demonstrated safety and the ability to cross the blood-brain barrier making it efficacious against stage 1 and stage 2 of the disease. In addition, its oral formulation, short duration of therapy and excellent pre-clinical safety profile imply that SCYX-7158 (AN5568) could change the way sleeping sickness is treated, reduce its incidence in humans, and contribute to elimination of the disease.
The successful development of this new compound was the result of a unique collaboration between Anacor Pharmaceuticals, SCYNEXIS, a drug discovery and development company based in Research Triangle Park, North Carolina, and DNDi, a not-for-profit research and development (R&D) organization that develops new drugs to address the needs of the world's most neglected populations.
"This innovative collaboration, expounding upon the expertise and engagement of two biotech companies and a not-for-profit R&D organization, has brought excellent results thus far, and this in an incredibly short timeframe," said Dr. Bernard Pécoul, Executive Director of DNDi. "The message is clear that it is possible to stimulate patient needs-driven R&D for neglected tropical diseases, even if there is no commercial market," added Pécoul.
SCYX-7158 (AN5568) is a product of Anacor's novel boron chemistry which has produced a number of compounds with efficacy against a range of fungal, inflammatory and bacterial diseases. Realizing this technology could also be used for neglected diseases, Anacor, with the help of the Sandler Center for Drug Discovery of the University of California, San Francisco, screened its library of boron-based compounds for activity against the sleeping sickness parasites and identified an attractive lead series. In order to ensure further development of these compounds, Anacor approached DNDi, which was actively seeking compounds for its lead optimization program. DNDi, Anacor, SCYNEXIS, and a consortium including Pace University and the Swiss Tropical and Public Health Institute then worked on the series of molecules in pre-clinical studies that led to the development of the compound for which DNDi is filing a dossier for first in man (FIM) studies to enter Phase I clinical trials in Europe.
"Due to its safety and efficacy against both stages of the disease, and its ability to be administered orally, this compound could be a breakthrough in the treatment of sleeping sickness and could significantly improve the lives of those who suffer from this insidious disease," said David Perry, CEO of Anacor Pharmaceuticals. "We are committed to applying our boron chemistry platform to the discovery and development of new treatments for neglected diseases, and it's only with the support of organizations like DNDi and their donors that we are able to fulfill that commitment."
SCYNEXIS CEO, Dr. Yves Ribeill, said the program is part of SCYNEXIS' policy of contributing to neglected disease projects. "Success so far was in large part due to the work of several organizations working closely together. If we are going to find effective medicines against diseases that exact a terrible toll in developing countries, we need to find new approaches in the discovery process. This program is an example," said Ribeill.
Thanks to continuous support from public and private donors, DNDi secured USD$14.8 million in support of this lead optimization and pre-clinical program. Most of the funding was provided by the Bill & Melinda Gates Foundation. In addition, specific funding was obtained from the Ministry of Foreign and European Affairs (MAEE), France, and core funding from the Spanish Agency of International Cooperation for Development (AECID), Spain and Doctors Without Borders/Médecins Sans Frontières (MSF). Additional funding is being sought for the clinical phases of this program.
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