Our Key Neglected Diseases Initiatives:

In recent years, a number of foundations and governments have created public-private partnerships to fund promising technologies that may result in new drugs for neglected diseases. Below is an overview of our key initiatives in this area:

DNDi

In December 2007, we established a partnership with DNDi to develop new therapeutics for HAT, visceral leishmaniasis and Chagas disease. In 2011, DNDi completed preclinical studies of AN5568 (also referred to as SCYX-7158) for HAT, a disease caused by parasites transmitted by the bite of a tsetse fly that threatens millions of people in sub-Saharan Africa and is fatal if left untreated. In March 2012, DNDi commenced Phase 1 human clinical studies of AN5568 in HAT, making AN5568 the first compound from our neglected diseases initiatives to enter human clinical studies. The AN5568 Phase 1 clinical study was completed in 2015 and a Phase 2/3 registration study is planned for 2016. If successful in clinical development, AN5568’s single oral dosage formulation, potential to treat all stages of the disease and short duration of therapy offer an opportunity to change the way HAT is treated and contribute to the elimination of the disease in conflict ridden areas such as the Democratic Republic of the Congo. DNDi is solely responsible for clinical development of AN5568 for this indication.

GSK

In September 2011, we and GSK entered into an amendment to our existing agreement to, among other things, add a new research program using our boron chemistry platform for TB. GSK selected a compound that resulted from the TB program in September 2013, subsequently selected a number of back-up compounds and is responsible for all further development and commercialization with respect thereto. The selected compound is currently in preclinical development. The research period under our agreement with GSK has expired. We may receive contingent payments if certain regulatory events occur and/or if certain sales levels are achieved and may also receive royalties on sales of resulting products.

Gates Foundation

In April 2013, we entered into a research agreement with the Gates Foundation to expand our boron chemistry library and to discover drug candidates intended to treat two filarial worm diseases (onchocerciasis, or river blindness, and lymphatic filariasis, commonly known as elephantiasis) and TB. Effective October 2014, we amended the research agreement to add an additional parasitic disease target, cryptosporidiosis. Under the amended research agreement, the Gates Foundation is obligated to pay us to conduct research activities directed at discovering potential neglected disease drug candidates in accordance with an agreed upon research plan. As part of the funded research activities, we created an expanded library of boron compounds to screen for additional potential drug candidates to treat neglected diseases. Now that the library is completed, we are responsible for storing the library compounds and making them accessible to the Gates Foundation and other parties to which the Gates Foundation grants access for an agreed period of time. Under the terms of the agreement, the Gates Foundation will have the exclusive right to commercialize selected drug candidates in specified neglected diseases in specified developing countries. We retain the exclusive right to commercialize any selected drug candidate outside of the specified neglected diseases, as well as with respect to the specified neglected diseases in specified developed countries. In addition, we would be obligated to pay the Gates Foundation royalties on specified license revenue received.