The study randomized 279 patients to five study arms: BCX4208 at doses of 5 mg, 10 mg, 20 mg, 40 mg and placebo, administered once-daily for 12-weeks. Allopurinol 300 mg once-daily was administered in all study arms. The primary endpoint of the study was the proportion of patients with sUA <6 mg/dL at day 85.
The primary endpoint of the study was successfully achieved. When added to allopurinol 300 mg, BCX4208 was superior to allopurinol plus placebo (p=0.009 overall). BCX4208 doses evaluated in the study showed response rates ranging from 33% to 49%, compared to 18% for placebo (table below).
|Top-Line Efficacy Results: BCX4208 Phase 2b Study in Patients Not Responding to Allopurinol Alone|
|Placebo||BCX4208 Treatment Groups|
|Primary endpoint: Proportion of patients with uric acid levels <6mg/dL at day 85|
|P value vs. placebo||p=0.004*||p=0.125||p=0.021*||p<0.001*|
Adding BCX4208 to allopurinol was generally safe and well-tolerated at all doses studied. Both the frequency and types of adverse events, including infections, were similar between the groups treated with BCX4208 and placebo. No opportunistic or unusual infections were reported in either the BCX4208 treated groups or placebo. As expected, a dose-dependent effect on lymphocyte counts was observed and this effect appeared to plateau within 12 weeks of treatment. No patients from the placebo, 5 mg or 10 mg cohorts discontinued study drug due to confirmed lymphocyte or CD4+ cell counts below certain pre-specified thresholds. Two patients were discontinued from the 20 mg group and eight patients from the 40 mg group due to pre-specified stopping rules based on CD4+ cell counts.
"These positive results in 2nd line treatment are consistent
with findings from our prior 1st line combination study, and
reinforce BCX4208's potential to safely address the unmet medical need
for new treatment options that help gout patients reach their
therapeutic goal," said Dr.
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Gout is a chronic inflammatory arthritis caused by monosodium urate crystal deposits in joints and the kidneys resulting from elevated serum uric acid (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.
BCX4208 is a novel enzyme inhibitor with the potential for once-a-day oral dosing suitable for chronic administration to treat gout. It acts upstream of xanthine oxidase in the purine metabolism pathway to reduce sUA in patients with gout and has a mechanism of action that complements xanthine oxidase inhibitors, such as allopurinol and febuxostat, in reducing uric acid production. With its unique mechanism of action, clinical activity and safety in clinical studies to date, BCX4208 is nearing the end of Phase 2 development as an add-on therapy to xanthine oxidase inhibitors to address unmet medical needs in patients with gout. To date, BCX4208 has been studied in over 500 subjects in clinical trials.
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Catherine Kyroulis, 212-301-7174
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