Today, there is important progress to report at all stages of DNDi’s drug R&D pipeline for sleeping sickness, with projects moving forward to address the treatment needs of some of the most neglected patients.

Commonly known as sleeping sickness, human African trypanosomiasis (HAT) is a fatal illness which threatens 60 million people in 36 countries, primarily in sub-Saharan Africa.

The two current treatment options for stage 2 HAT are inadequate:
• Melarsoprol is toxic (associated with 3-6% fatality) and increasingly ineffective
• Eflornithine: inconvenient to use (56 slow intravenous infusions administered every 6 hours over 14 days) and only used in approximately 30% of stage 2 HAT patients for that reason
NECT, a simplified co-administration of oral nifurtimox and intravenous eflornithine, can make an immediate, practical improvement for patients and health care staff today. If NECT is effectively implemented as a replacement for melarsoprol, it could save 4 to 5 lives for every 100 patients treated as there is much less toxicity with NECT. This is critical as there are no new drugs for stage 2 HAT expected in the next five years. Although NECT radically improves upon current treatment options by being safer to use than melarsoprol and far easier to use than eflornithine, the treatment is still far from ideal.
Having a truly simplified treatment which can be orally administered, implemented at the primary health care level, and effective against both stages of the disease, is still the ultimate goal. Drugs for Neglected Diseases initiative is pleased to announce that fexinidazole is entering clinical development for HAT and that we have signed an agreement with sanofi-aventis for its further development. Additionally, the pipeline is being strengthened by renewed commitments of the United Kingdom’s Department for International Development and research partners like Anacor towards the discovery of new drugs against HAT.

NOW AVAILABLE FOR USE: AN IMMEDIATE IMPROVEMENT FOR PATIENTS WITH STAGE 2 HAT
• NECT, the nifurtimox-eflornithine combination therapy: the first improved treatment in 25 years is now included on the WHO’s Essential Medicines List and is now available for use in treating stage 2 HAT
• May 15: MSF, Epicentre and DNDi announce that NECT is on WHO Essential Medicines List
• NECT is an improved option because it’s much easier to use and as efficacious / safe as eflornithine
• Clinical evidence from a 5-year, multicentre, randomized controlled trial in central Africa, represents some of the strongest evidence in the field to date
• According to WHO, NECT “provides a new opportunity to improve the management of sleeping sickness cases” by reducing treatment duration and making it easier to administer…
• Key donors for NECT-related DNDi activities: the Department for International Development (DFID) of the United Kingdom, Médecins Sans Frontières (MSF) International, the Medicor Foundation, the Ministry of Foreign and European Affairs (MAEE) of France, and the Spanish Agency of International Cooperation for Development (AECID).

COLLABORATION TO DEVELOP A PROMISING NEW DRUG CANDIDATE
• FEXINIDAZOLE: has potential to be an oral treatment effective against both stages of sleeping sickness; a collaboration agreement with sanofi-aventis announced yesterday
• “Rediscovered” by DNDi after extensive compound mining efforts with industry and academia
• Entering clinical development in 2009 and the only new clinical candidate currently in the drug pipeline for sleeping sickness
• Offers the potential of an oral treatment that can treat both stages of disease (no painful lumbar punctures and no more infusions over 2 weeks)
• May 18: Sanofi-aventis and DNDi sign agreement to develop and make available a new drug for sleeping sickness, fexinidazole
• The following donors have financially supported DNDi’s fexinidazole-related activities: DFID of the United Kingdom, GTZ of Germany, MSF, MAEE of France, the Spanish AECID, and a Swiss foundation.

DNDI’S PARTNERS STRENGTHEN COMMITMENTS
• Private biopharma: This week, the biopharmaceutical company, Anacor, and DNDi will announce their continuing partnership on boron-based therapeutics for sleeping sickness and others neglected diseases
• Public donor: The UK’s Department for International Development (DFID) renews multi-year support of DNDi’s efforts to improve medicines for the most neglected, with a 5-year, GBP 18 million grant