Horizon Therapeutics plc Initiates PROTECT Trial Evaluating KRYSTEXXA® (pegloticase injection) to Improve Management of Uncontrolled Gout for Adults with a Kidney Transplant

-- Trial to evaluate the effectiveness of KRYSTEXXA to sustain reduced serum uric acid levels for post-kidney transplant adults; a vulnerable population --

DUBLIN, Ireland I October 17, 2019 I Horizon Therapeutics plc (Nasdaq: HZNP) today announced it has initiated an open-label clinical trial evaluating the use of KRYSTEXXA® (pegloticase injection) in adults with chronic gout refractory to conventional therapies – also known as uncontrolled gout – who have undergone a kidney transplant to demonstrate that KRYSTEXXA may provide effective disease control without burdening the kidneys.

PROspective sTudy of pEglotiCase in Transplant patients (PROTECT) is a multicenter, open-label study which will evaluate the efficacy and safety of KRYSTEXXA among 20 adults with uncontrolled gout who have received a kidney transplant at 15 centers across the U.S. The study and its protocol were thoughtfully designed in collaboration with the American Association of Kidney Patients to incorporate considerations for high-needs populations and their priorities in effectively managing uncontrolled gout.

“The prevalence of gout is more than ten-fold greater among patients who have undergone a kidney transplant than the general population,” said Abdul Abdellatif, M.D. F.A.S.N. primary investigator and assistant professor, Baylor College of Medicine. “Post-transplant medications to prevent organ rejection can also contribute to increased uric acid levels and lead to higher rates of uncontrolled gout. It is here where we have seen higher mortality rates compared to patients who have received a kidney transplant without uncontrolled gout. Strategies to effectively manage uncontrolled gout within the vulnerable post-transplant population are important to ensuring long-term protection of the kidney.”

During the study, participants will receive KRYSTEXXA 8 mg IV every two weeks over a six-month treatment period, with a 3-month post treatment follow-up. The study’s primary endpoint is response rate, as measured by sustained serum uric acid reduction to <6 mg/dL at Month 6 of treatment. It will also evaluate secondary outcomes such as complete resolution of at least one tophus, and scores related to pain and disability. This population was not originally studied in the KRYSTEXXA pivotal trials.

“The unique mechanism of action of KRYSTEXXA provides an opportunity for clinicians to address elevated uric acid levels irrespective of kidney function,” said Paul Peloso, M.D., M.Sc., vice president and therapeutic area head, rheumatology, Horizon. “Working together with our colleagues in the nephrology and transplant communities we designed the PROTECT trial to demonstrate that clinicians can safely and effectively manage uncontrolled gout for individuals who have undergone kidney transplantation.”

More information on the trial can be found on clinicaltrials.gov (NCT04087720).

About Uncontrolled Gout

Gout is a chronic, progressive inflammatory form of arthritis that is caused by excess uric acid in the body and needs to be managed aggressively.1 Over time uric acid can build up and form deposits, called tophi, inside the body and joints, which can have harmful effects including causing damage to the underlying bone.2 Patients with uncontrolled gout continue to have abnormally high levels of uric acid and continued symptoms of gout despite the use of conventional therapies. KRYSTEXXA is the only biologic approved by the U.S. Food and Drug Administration (FDA) for the treatment of uncontrolled gout in adult patients.



KRYSTEXXA® (pegloticase injection) is a PEGylated uric acid specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy.

Gout refractory to conventional therapy occurs in patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.

About Horizon

Horizon is focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, please visit www.horizontherapeutics.com, follow us @HorizonNews on Twitter, like us on Facebook or explore career opportunities on LinkedIn.

1 Keuhn B. Chronic Disease Approaches Needed to Curb Gout’s Growing Burden. Journal of the American Medical Association. 2018;319(13):1308-1309.

2 Zhu Y, Pandya BJ, Choi HK. Prevalence of gout and hyperuricemia in the US general population: the National Health and Nutrition Examination Survey 2007-2008. Arthritis Rheum. 2011;63(10):3136-3141.

SOURCE: Horizon Pharma

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