SAP-001 Demonstrates “Best in Class” Data for Gout With Hyperuricemia Refractory to SOC XOI Therapy

Shanton Pharma has announced positive phase 2b data on its novel urate-lowering therapy (ULT) SAP-001 in people with gout.1

"We are excited and very pleased with the outcome of our Phase 2b clinical study in this difficult-to-treat population of refractory gout patients" Bing Li, PhD, CEO of Shanton, said in a statement.1 "By the end of month 3 in our study, nearly 100% of patients that were confirmed exposed to daily SAP-001 30 and 60 mg dosages on top of conventional therapy reached the study's therapeutical target (serum uric acid levels below 6mg/dL), compared to only about 10% of patients on conventional treatment who reached that goal. The majority of treatment effects persisted until the end of treatment at month 6 of the study. More than half of those patients confirmed exposed to a daily 60 mg dose reached serum uric acid levels below 3mg/dL. Importantly, the study demonstrated an excellent safety profile in this population."

The phase 2b study is a 6-month, multi-center, randomized, double-blind, placebo-controlled, dose-ranging clinical study evaluating efficacy and safety of SAP-001 10, 30, or 60 mg in 87 participants with refractory gout, with or without tophi, from 21 US sites. Shanton previously announced that the phase study had completed enrollment in August 2024.2

The study’s primary outcome is lowering serum urate levels in patients with gout refractory to standard-of-care (SOC) Xanthine Oxidase Inhibitor (XOI) therapy.1 Secondary outcomes include frequency of gout flares and safety and tolerability assessments.

SAP-001 is an investigational compound for once-a-day oral urate-lowering therapy people with refractory gout by way of a unique mechanism-of-action (MOA) targeting a distinct kidney transporter. The therapy has previously shown efficacy and safety in Phase 1 and Phase 2a clinical studies. Shanton has called the phase 1 and 2a efficacy data “unparalleled” in gout patients with hyperuricemia, and the new phase 2b data “Best-in-Class” in gout patients with hyperuricemia refractory to SOC XOI therapy.

"This is the first successful clinical study with an oral gout drug to treat these refractory patients that normally need to be treated with an intravenous uricase. The efficacy of SAP-001 in this population is very impressive in all dosage groups at all assessed time points across the 6 months of treatment. We believe that the excellent efficacy and safety outcomes in this study show the exciting potential of our First-in-Class compound with its novel MOA that selectively targets a distinct kidney transporter for urate lowering treatment. The medical community has long been looking for this new mechanism in gout to address the shortcomings with conventional MOAs, especially where it comes to treating refractory and tophaceous patients. Our goal is to proceed into pivotal studies with our program later this year to eventually offer a paradigm-shifting gout treatment to our patients and make a big impact in their lives,” Wenfeng Miao, MD, PhD, Shanton's CMO added.1

REFERENCES

1. Shanton Reveals Topline Data from Phase 2b Study in Refractory Gout Patients. News release. Shanton Pharma. March 27, 2025. Accessed March 28, 2025. https://www.prnewswire.com/news-releases/shanton-reveals-topline-data-from-phase-2b-study-in-refractory-gout-patients-302413795.html

2. Shanton Pharma’s Phase IIb gout treatment trial reaches full enrolment. News release. Shanton Pharma. August 6, 2024. Accessed March 28, 2025. https://www.clinicaltrialsarena.com/news/shanton-gout-treatment-trial/?cf-view