Benzinga | Nov 4, 2021 01:02PM EDT

Horizon Therapeutics Highlights Presentation Of Results From Completed PROTECT Trial On KRYSTEXXA For Gout At American Society Of Nephrology Kidney Week

-- Complete data from PROTECT trial shows 89% of patients achieved the primary endpoint --

-- Additional presentations call attention to the high prevalence of gout and its associated comorbidities among individuals undergoing dialysis --

DUBLIN--(BUSINESS WIRE)-- Horizon Therapeutics plc (NASDAQ:HZNP) today announced the presentation of results from the completed PROTECT trial evaluating KRYSTEXXA(r) (pegloticase injection) in people with chronic gout refractory to conventional therapies, also known as uncontrolled gout, who had received a kidney transplant. In additional presentations, the company shared new insights on the challenges of gout among individuals with chronic kidney disease who undergo dialysis. These data are presented as part of the American Society of Nephrology (ASN) Kidney Week, Nov. 4-7, 2021.

Key KRYSTEXXA presentations include:

Pegloticase for Uncontrolled Gout in Kidney Transplant Recipients: Provisional Data Report of a Multicenter, Open-Label, Efficacy and Safety Study. The results from the completed PROTECT trial (NCT04087720) demonstrated that KRYSTEXXA provided a substantial and sustained decrease in serum uric acid (sUA) for patients with uncontrolled gout who had received a kidney transplant and were treated with two to three immunosuppressive agents to prevent organ rejection. Twenty patients were enrolled in the PROTECT trial. Analysis was conducted based on 18 of 20 patients who did not have a lapse or cessation in treatment due to COVID-19 prior to the Month 6 analysis time-point. Sixteen of 18 patients (88.9%) achieved the primary endpoint, defined as sUA <6 mg/dL for at least 80% of the time during Month 6. In total, six patients discontinued the treatment early, two based on meeting monitoring rules (pre-dose sUA >6 mg/dL at two consecutive visits), three based on COVID-19 pandemic concerns (one included in primary analysis, and two excluded from the primary analysis), and one based on personal reasons not related to adverse events.

At Week 24, health assessment scores for pain and disability improved by 35.5 (baseline: 42.2) and 0.3 (baseline: 1.0), respectively, among those who completed 24 weeks of treatment. Seven serious adverse events reported in five patients were deemed unrelated to treatment, and no anaphylaxis or infusion reaction events occurred.

"Evidence indicates gout is more common, and often more severe, among those who have undergone kidney transplantation, with some data showing a gout prevalence more than 10-fold higher than among non-transplant patients," said Theresa Podrebarac, M.D., MSc., senior vice president, clinical development, Horizon. "These results indicate KRYSTEXXA can help address uncontrolled gout for one of the most vulnerable populations."

Please see below for Important Safety Information.

Key gout presentations include:

Risk Factors and Outcomes of Gout in Dialysis Patients: A Cohort Study of the United States Renal Data System (USRDS). A recent review of the 2017 U.S. Renal Data System (USRDS) highlighted new insights on risk factors and outcomes associated with gout among dialysis patients. The study identified more than 41,000 dialysis patients with gout following initiation of chronic outpatient dialysis and compared baseline characteristics, comorbid conditions and clinical outcomes to those without gout. The review found a high prevalence of gout among Medicare patients on dialysis (15%). In all, gout patients were more likely to have a higher prevalence of diabetes, hypertension and cardiovascular conditions (such as heart failure, acute myocardial infarction, ischemic heart disease and stroke) than those without gout. Importantly, the risks of hospitalization and mortality were 11% in the year after index claim.

Characterization of Gout in U.S. Patients Treated with Hemodialysis (HD) and Peritoneal Dialysis (PD). A separate evaluation sought to understand the impact of gout for people undergoing hemodialysis (HD) and peritoneal dialysis (PD) by reviewing patterns of 70,297 HD patients and 5,117 PD patients from the Dialysis and Peritoneal Dialysis Outcomes and Practice Patterns Studies (DOPPS and PDOPPS) for outcomes such as erythropoietin resistance index (ERI), mortality and hospitalization, and patient-reported outcomes (PROs). Gout was found to be common in HD and PD patients (13% prevalence in HD patients and 21% in PD patients). Both HD and PD patients with gout, compared to those without, had a higher prevalence of cardiovascular comorbidities. These comorbidities are a leading cause of mortality in the dialysis patient population.1,2

"One-in-four adults with moderate-to-severe CKD are living with gout, creating additional complexities in managing both diseases and contributing additive burden that impacts functional abilities," said Richard J. Johnson, M.D., professor, Division of Renal Diseases and Hypertension, University of Colorado Denver - Anschutz Medical Campus. "Better understanding the relationship between gout, kidney disease and dialysis offers research opportunities to improve care for patients with gout, particularly to mitigate potentially fatal comorbid conditions such as cardiovascular disease."

Additional presentations supported by Horizon during ASN Kidney Week include:

* Claims-Based Evaluation of Pegloticase Use in Gout Patients with a History of Kidney Transplant [PUB317]

* Safety of Pegloticase with Immunomodulation Co-Therapy: Literature Review [PUB318]

* Baseline Renal Characteristics and Trial Design for MIRROR RCT, Randomized Trial of Pegloticase with or without Methotrexate for Uncontrolled Gout [PUB314]

* Gout in Advanced CKD Patients: Prevalence and Impact on Patient Health [PUB316]