Positive Top-Line Results from BCX4208 Phase 2B Gout Study
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Patients had failed to reach the clinically important serum uric acid (sUA) goal of <6 mg/dL on allopurinol alone. Detailed study findings will be shared at an upcoming medical meeting.
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 | ||||||||
|
Once-daily treatment: |
(N=55) |
5 mg
(N=56) |
10 mg
(N=55) |
20 mg
(N=56) |
40 mg
(N=53) |
|||||
| Primary endpoint: Proportion of patients with uric acid levels <6mg/dL at day 85 | ||||||||||
|
Response Rate
(mITT Analysis) |
18% | 45% | 33% | 39% | 49% | |||||
| P value vs. placebo | p=0.004* | p=0.125 | p=0.021* | p<0.001* | ||||||
*statistically significant
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. William P. Sheridan, Senior Vice President & Chief Medical Officer of BioCryst Pharmaceuticals. “We expect to report 6-month results from the ongoing extension study in early 2012, which will provide additional insight into BCX4208’s safety and efficacy profile. We are on track to conclude this Phase 2 program during the first half of next year and we look forward to discussing the results in more detail with regulatory authorities and potential partners.”
