Pharmaceutical Business review

Horizon Therapeutics initiates PROTECT clinical trial of KRYSTEXXA

Image: KRYSTEXXA is a PEGylated uric acid specific enzyme indicated for the treatment of chronic gout in adult patients refractory to conventional therapy. Photo: Courtesy of PublicDomainPictures from Pixabay.

Horizon Therapeutics 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.”

Source: Company Press Release