Gout: novel compound as a future option for refractory patients?

Treatment with SAP-001, a first-in-class urate-lowering agent targeting a novel renal urate transport mechanism, resulted in successful reduction of serum urate (sUA) levels to ≤6 mg/dL in 43.8%–70% of patients, across different dosing regimens. A dose-response effect could not be established.

Prof. Kenneth G. Saag (University of Alabama at Birmingham, AL, USA) presented phase 2 data on SAP-001 (NCT05690204) [1]. Eligible participants were adults with refractory gout and sUA levels ≥7 mg/dl. After a placebo run-in, patients were treated either with placebo, or SAP-001 at doses of 10 mg, 30 mg, or 60 mg, over 85 days, at which point the primary endpoint of proportion of participants with sUA ≤6 mg/dL was evaluated. Treatment then continued until day 169.

Across the study arms, participants were predominantly male (87.5%–96.9%), the mean age ranged between 50.3 and 55.9 years, and all had a BMI above 30 kg/m² and preserved kidney function. Mean baseline sUA levels were 8.37 mg/dL.

“At all study time points, a significant reduction in sUA levels and a higher proportion of participants meeting the primary endpoint was observed in the active arms compared with placebo,” Prof. Saag mentioned. The proportion of patients achieving target sUA levels was 56.3% in the 10 mg arm, 70.0% in the 30 mg arm, and 43.8% in the 60 mg arm, compared with just 10.5% in the placebo group. A statistically significant dose-response relationship was not established (P=0.148).

Prof. Saag commented that the safety profile was favourable. All adverse events of special interest, which included transient creatine elevations or decreased glomerular filtration rate, were few (15 events in 11 participants), with a numerically slightly higher incidence in the SAP-001 arms. All were mild or moderate and non-serious. Gout flares occurred in 26.3% of participants on placebo and in 25%–30% of those receiving SAP-001.

“SAP-001 demonstrated sustained urate-lowering effects in patients with refractory gout, with or without tophi,” Prof. Saag summarised. He concluded that the drug may offer a novel, efficacious, and safe treatment option for patients with difficult-to-treat gout.

1. SAP-001, A first-in-class compound targeting a renal urate transporter, shows potent serum urate-lowering effects and favourable safety profile in a US phase 2b study in patients with refractory gout with or without palpable tophi. LB0008, EULAR 2025, 11–14 June, Barcelona, Spain.

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Posted on 18 June 20258 August 2025