Two double-blind, placebo-controlled Phase 3 trials (DISSOLVE I and II) evaluated nanoencapsulated sirolimus plus pegadricase (NASP) at high-dose (HD) and low-dose (LD) vs. placebo, given every 4 weeks for 6 doses (24 weeks), in 265 patients with uncontrolled gout (UG).
Serum urate (SU) <6 mg/dL for ≥80% of Weeks 21–24 was achieved by 51% (HD NASP), 43% (LD NASP), and 8% (placebo) — both comparisons p<0.0001. Gout flare rates were similar across groups (~42–44%) but declined after Week 12 only in NASP-treated patients. Stomatitis occurred in 9.2% (HD), 3.4% (LD), and 0% (placebo); early infusion reactions were infrequent at 4%.
- Total sample size of 265 across three arms limits subgroup precision. - Pooled reporting of two trials may obscure trial-specific differences. - The 24-week duration leaves longer-term efficacy and safety unknown.
For patients with uncontrolled gout who have failed standard urate-lowering therapy, NASP given every 4 weeks achieved meaningful SU control in roughly half of patients. Watch for stomatitis at the higher dose and counsel patients that flares are common in the first 12 weeks before urate levels stabilize.
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