SAP-001 Promising for Refractory Gout

Released on March 29, positive findings from a phase 2b clinical trial highlight the potential of SAP-001 as a treatment for patients with refractory gout (NCT05690204).^1,2 SAP-001 is an oral, once daily, urate-lowering treatment that targets refractory gout being researched by Shanton Pharmaceuticals, which developed the agent. SAP-001’s urate-lowering properties are based on a mechanism of action that targets a distinct kidney transporter (no further information reported).²

The study’s primary outcome assessed the effectiveness of SAP-001 in lowering serum urate levels in patients with gout refractory to standard treatment with xanthine oxidase inhibitors. Secondary outcomes investigated include its safety and tolerability, as well as the frequency of gout flares and changes in the number and size of tophi.³

In previously completed phase 1 and 2 studies, SAP-001 was well tolerated in patients with gout and hyperuricemia at single doses up to 120 mg, as well as doses of up to 60 mg given daily for 28 days. Patients in these studies had statistically significant reductions in serum uric acid levels compared with those who received placebo (data not reported).²

Study Methods & Results

The new, randomized, double-blind, placebo-controlled study examined three different, orally administered doses of SAP-001 compared with placebo in adults with gout, with or without tophi, and hyperuricemia refractory to xanthine oxidase inhibitor therapy (n=87). The study assessed doses of 10 mg, 30 mg and 60 mg of SAP-001 given once daily.¹

By the end of month 3, almost 100% of patients treated with SAP-001 at the 30 mg and 60 mg daily doses, along with conventional therapy, achieved a serum uric acid level of <6 mg/dL.³ Approximately 10% of patients receiving only conventional treatment achieved a serum uric acid level of <6 mg/dL.

Most effects lasted through month 6, the end of the study. Of the patients who received 60 mg of SAP-001 daily, more than half achieved a serum uric acid level of less than 3 mg/dL. The study also demonstrated a positive safety profile (data not reported).

The company plans to run phase 3 trials to further assess SAP-001’s potential for patients with difficult-to-treat gout.

Michele B. Kaufman, PharmD, BCGP, is a freelance medical writer based in New York City and a pharmacist at New York Presbyterian Lower Manhattan Hospital.

References

1. Shanton reveals topline data from phase 2b study in refractory gout [news release]. Shanton Pharma. 2025 Mar 29.
2. Dose finding study to evaluate safety and efficacy of 3 dosages of SAP 001 [NCT05690204]. Shanton Pharma. ClinicalTrials.gov. 2024 Nov 14.
3. Shanton Pharma’s phase IIb gout treatment reaches full enrolment [Sic] [news release]. Clinical Trials Arena. 2024 Aug 26.