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Ulodesine

Ulodesine (BCX4208): PNP inhibitor

With its unique mechanism of action, clinical activity and safety in clinical studies to date, ulodesine is a Phase 3-ready-asset for development as an add-on therapy to xanthine inhibitors to address unmet medical needs in patients with gout.

Ulodesine is a purine nucleoside phosphorylase (PNP) inhibitor in development as a once-daily oral, chronic treatment for gout. It acts upstream of xanthine oxidase in the purine metabolism pathway to reduce the production of serum uric acid (sUA). Xanthine oxidase inhibitors, such as allopurinol and febuxostat, reduce uric acid production. In a Phase 2 clinical trial, the combination of low doses of ulodesine and allopurinol resulted in a synergistic effect in reducing sUA.

BioCryst initiated the ulodesine Phase 2 clinical program in September 2009 and since then has announced positive results from a monotherapy trial, evaluating the efficacy and safety of orally administered ulodesine in patients with gout, as well as a combination trial, evaluating ulodesine alone and in combination with allopurinol in gout patients. Furthermore, ulodesine was found to be generally safe and well-tolerated through 24 weeks of treatment, when evaluated as an add-on therapy to allopurinol in gout patients who have not adequately responded to allopurinol alone. Connect to slides for further information.

Following the successful outcome of the Phase 2b 24-week ulodesine clinical trial reported in January 2012, BioCryst completed its end-of-Phase 2 meeting with the FDA as well as initiated the Scientific Advice Process with the EMA. BioCryst remains focused on securing the right partner to fund the Phase 3 development and commercialization of ulodesine.

About Gout

Gout is a chronic inflammatory arthritis caused by monosodium urate crystal deposits in joints and the kidneys resulting from elevated sUA levels in the blood, a condition known as hyperuricemia. The consequences of gout may include intense, painful flares affecting one or more joints, impaired kidney function and joint destruction. Gout continues to grow in prevalence and severity, affecting over 17 million people in major markets, including 8.3 million in the U.S. A majority of gout patients are also treated to manage other chronic conditions, including hypertension, diabetes and/or high cholesterol. Decreasing sUA to the recommended level (less than 6 mg/dL) can reduce the risk of gout attacks over the long-term. A minority of patients treated with the current standard of care, allopurinol, achieve this therapeutic goal. There is a need for new therapies that effectively and safely get a larger portion of gout suffers to goal without the risk of drug-drug interactions. More information regarding gout and hyperuricemia is available on the CDC website at www.cdc.gov/arthritis/basics/gout.htm.

Resources

Ulodesine Scientific Publications

For more information on gout visit the following websites: