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Progress reported in global fight against diarrheal disease cryptosporidiosis

May 31, 2017

  • Novartis, the University of Georgia, Washington State University and collaborators advance therapeutic research for cryptosporidiosis

  • Research collaboration with novel drug discovery process counters slow progress in developing new cryptosporidiosis treatments

  • Scientists report discovery of Cryptosporidium PI(4)K inhibitor candidate KDU731 in new issue of Nature

Basel, May 31, 2017 - Infectious disease scientists from Novartis, the University of Georgia and Washington State University have reported the discovery and early validation of a drug candidate for treating cryptosporidiosis, a diarrheal disease which is a major cause of child mortality in lower-income countries. Currently there are no vaccines or effective treatments.

"There's a lot of uncertainty when embarking on drug discovery for a notoriously intractable parasite such as Cryptosporidium, the cause of cryptosporidiosis," said Thierry Diagana, Head of the Novartis Institute for Tropical Diseases (NITD). "Thanks to the commitment of our funding collaborators and urgent action of our academic colleagues, we've made an important step toward advancing a new treatment."

Diarrheal diseases cause more than 800,000 deaths annually[1]. Epidemiological studies have highlighted the vital need for new treatment options against the protozoan parasite Cryptosporidium, which often infects its victims from exposure to contaminated water supplies. Nitazoxanide, the only approved treatment for cryptosporidiosis, has shown poor results in vulnerable infants and immune-compromised patients[2,3].

Yet there are obstacles to finding new treatments. The parasite perishes relatively quickly in labs and scientists have lacked research tools to identify drug candidates. The team developed a novel drug discovery process using transgenic parasites and novel disease models, leading to the identification and validation of the Cryptosporidium PI(4)K inhibitor candidate KDU731. They reported the discovery and preclinical findings in a recent issue of Nature.

Novartis is advancing research of cryptosporidiosis through collaborations with the global health community and its scientists at NITD, which is ramping up its research and operations in Emeryville, CA.

Program funders include the Novartis Institutes for Biomedical Research (NIBR), the Wellcome Trust, and the National Institutes of Health. In addition to the Wellcome Trust, the Medicines for Malaria Venture (MMV) supported discovery of Plasmodium PI(4)K inhibitors screened in the development process.
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Disclaimer

This press release contains expressed or implied forward-looking statements, including statements that can be identified by terminology such as "commitment," "step toward," "ramping up," or similar expressions. Such forward-looking statements reflect the current views of the Group regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results expressed or implied by such statements. These expectations could be affected by, among other things, risks and factors referred to in the Risk Factors section of Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update it in the future.

About Novartis

Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 118,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.

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References

1. Liu, L. et al. Global, regional, and national causes of under-5 mortality in 2000-15: an updated systematic analysis with implications for the Sustainable Development Goals. The Lancet. 2016.
2. Amadi, B. et al. High dose prolonged treatment with nitazoxanide is not effective for cryptosporidiosis in HIV positive Zambian children: a randomised controlled trial. BMC infectious diseases. 2009.
3. Amadi, B. et al. Effect of nitazoxanide on morbidity and mortality in Zambian children with cryptosporidiosis: a randomised controlled trial. The Lancet. 2002.

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