-- Trial to evaluate the effectiveness of
KRYSTEXXA to sustain reduced serum uric acid levels for post-kidney
transplant adults; a vulnerable population --
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.
About KRYSTEXXA
INDICATIONS AND USAGE
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.
Important Limitations of Use: KRYSTEXXA is not recommended
for the treatment of asymptomatic hyperuricemia.
IMPORTANT SAFETY INFORMATION WARNING: ANAPHYLAXIS AND
INFUSION REACTIONS
Anaphylaxis and infusion reactions have been reported to
occur during and after administration of KRYSTEXXA. Anaphylaxis may
occur with any infusion, including a first infusion, and generally
manifests within 2 hours of the infusion. However, delayed-type
hypersensitivity reactions have also been reported. KRYSTEXXA
should be administered in healthcare settings and by healthcare
providers prepared to manage anaphylaxis and infusion reactions.
Patients should be premedicated with antihistamines and
corticosteroids. Patients should be closely monitored for an
appropriate period of time for anaphylaxis after administration of
KRYSTEXXA. Serum uric acid levels should be monitored prior to
infusions, and healthcare providers should consider discontinuing
treatment if levels increase to above 6 mg/dL, particularly when 2
consecutive levels above 6 mg/dL are observed.
The risk of anaphylaxis and infusion reactions is higher in
patients who have lost therapeutic response.
Concomitant use of KRYSTEXXA and oral urate-lowering agents may
blunt the rise of sUA levels. Patients should discontinue oral
urate-lowering agents and not institute therapy with oral
urate-lowering agents while taking KRYSTEXXA.
In the event of anaphylaxis or infusion reaction, the infusion
should be slowed, or stopped and restarted at a slower rate.
Patients should be informed of the symptoms and signs of
anaphylaxis and instructed to seek immediate medical care should
anaphylaxis occur after discharge from the healthcare setting.
CONTRAINDICATIONS: G6PD DEFICIENCY ASSOCIATED HEMOLYSIS AND
METHEMOGLOBINEMIA
Patients should be screened for G6PD deficiency prior to
starting KRYSTEXXA. Hemolysis and methemoglobinemia have been
reported with KRYSTEXXA in patients with G6PD deficiency. KRYSTEXXA
should not be administered to these patients.
GOUT FLARES
An increase in gout flares is frequently observed upon
initiation of anti-hyperuricemic therapy, including treatment with
KRYSTEXXA. If a gout flare occurs during treatment, KRYSTEXXA need
not be discontinued. Gout flare prophylaxis with a non-steroidal
anti-inflammatory drug (NSAID) or colchicine is recommended
starting at least 1 week before initiation of KRYSTEXXA therapy and
lasting at least 6 months, unless medically contraindicated or not
tolerated.
CONGESTIVE HEART FAILURE
KRYSTEXXA has not been studied in patients with congestive heart
failure, but some patients in the clinical trials experienced
exacerbation. Caution should be exercised when using KRYSTEXXA in
patients who have congestive heart failure, and patients should be
monitored closely following infusion.
ADVERSE REACTIONS
The most commonly reported adverse reactions in clinical trials
with KRYSTEXXA were gout flares, infusion reactions, nausea,
contusion or ecchymosis, nasopharyngitis, constipation, chest pain,
anaphylaxis and vomiting.
Please see Full Prescribing Information and Medication Guide
for more information.
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.
Forward-Looking Statements
This press release contains forward-looking statements,
including statements regarding the potential benefits of KRYSTEXXA
in treating uncontrolled gout in kidney transplant patients and
expectations regarding the PROTECT clinical trial. These
forward-looking statements are based on management's expectations
and assumptions as of the date of this press release and actual
results may differ materially from those in these forward-looking
statements as a result of various factors. These factors include,
but are not limited to, risks regarding whether results of the
PROTECT trial will be consistent with results of prior trials or
Horizon’s expectations, and the risks associated with clinical
development of drug candidates. For a further description of these
and other risks facing Horizon, please see the risk factors
described in Horizon’s filings with the United States Securities
and Exchange Commission, including those factors discussed under
the caption “Risk Factors” in those filings. Forward-looking
statements speak only as of the date of this press release and
Horizon undertakes no obligation to update or revise these
statements, except as may be required by law.
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.
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