Shanton Pharma has announced promising topline results from its Phase 2b clinical trial evaluating SAP-001, a novel oral treatment for refractory gout. The study demonstrated exceptional efficacy in lowering serum uric acid levels in patients who had previously failed to respond adequately to standard-of-care treatments.
By the end of the three-month treatment period, nearly 100% of patients receiving daily SAP-001 at 30mg and 60mg doses achieved the therapeutic target of serum uric acid levels below 6mg/dL. This stands in stark contrast to the approximately 10% success rate observed in patients receiving only conventional therapy. The treatment effects largely persisted through the full six-month study duration.
"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," said Dr. Bing Li, Shanton's CEO. "More than half of those patients confirmed exposed to a daily 60mg dose reached serum uric acid levels below 3mg/dL. Importantly, the study demonstrated an excellent safety profile in this population."
Novel Mechanism of Action Addresses Critical Unmet Need
SAP-001 represents a significant innovation in gout treatment as the first oral medication to successfully treat refractory gout patients, who typically require intravenous uricase therapy. The drug employs a first-in-class mechanism of action that selectively targets a distinct kidney transporter to enhance urate lowering.
Dr. Wenfeng Miao, Shanton's CMO, highlighted the significance of these results: "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 six months of treatment."
The medical community has long sought new mechanisms to address limitations in conventional gout treatments, particularly for refractory and tophaceous patients. SAP-001's unique approach could potentially transform the treatment landscape for these challenging cases.
Study Design and Patient Population
The Phase 2b trial was a six-month, multi-center, randomized, double-blind, placebo-controlled, dose-ranging study conducted across 21 US sites. It enrolled 87 patients with refractory gout, with or without tophi, evaluating the efficacy and safety of SAP-001 at 10mg, 30mg, or 60mg tablet doses.
The primary objective was to assess SAP-001's effectiveness in lowering serum urate levels in gout patients refractory to standard Xanthine Oxidase Inhibitor (XOI) therapy, such as allopurinol or febuxostat. Secondary endpoints included safety assessments and frequency of gout flares.
The Burden of Refractory Gout
Gout affects over 12 million adults in the US alone, making it the most common form of inflammatory arthritis and the second most prevalent metabolic disease. The condition results from elevated uric acid levels in the bloodstream, leading to the deposition of urate crystals in joints and tissues, causing painful inflammatory responses.
While approximately 29% of diagnosed gout patients in the US receive urate-lowering therapies, only half respond adequately or can tolerate current options. An estimated 20% of US gout patients on standard treatments like allopurinol or febuxostat are physiologically refractory to these medications, highlighting a significant unmet medical need.
Recurrent gout flares can be debilitating, and chronic gouty arthritis may eventually lead to joint destruction and disfigurement, significantly impacting patients' quality of life.
Path Forward for SAP-001
Building on these positive Phase 2b results and earlier successes in Phase 1 and 2a studies, Shanton plans to advance SAP-001 into pivotal trials later this year.
"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 mechanism of action," Dr. Miao stated. "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."
SAP-001 is designed as a once-daily oral urate-lowering therapy specifically targeting refractory gout. Its unique mechanism of action has demonstrated unparalleled efficacy and safety across multiple clinical studies, positioning it as a potential best-in-class treatment for patients with limited therapeutic options.
Shanton Pharma, founded in 2016 and headquartered in Singapore with research operations in the US, China, and Singapore, continues to focus on addressing unmet needs associated with hyperuricemia and gout through innovative approaches to treatment.
