-- Increases KRYSTEXXA® (pegloticase
injection) Peak U.S. Annual Net Sales Expectations to More Than $1
Billion --
-- Increases Teprotumumab Peak U.S. Annual Net
Sales Expectations to More Than $1 Billion --
-- Announces 79 Percent of Patients Achieved a
Complete Response in MIRROR Open-Label Pilot Study, Supporting
KRYSTEXXA Immunomodulation Strategy to Optimize Treatment Outcomes
--
-- Announces Two New Pipeline Programs --
Horizon Therapeutics plc (Nasdaq: HZNP) announced today that it
is increasing the peak U.S. annual net sales expectations for its
key growth drivers KRYSTEXXA and teprotumumab, as well as providing
several pipeline updates.
“As we enter 2020, Horizon is in its strongest position ever,”
said Timothy Walbert, chairman, president and chief executive
officer, Horizon. “We believe KRYSTEXXA and teprotumumab each has
the potential for more than $1 billion in peak U.S. annual net
sales. In addition, our strong balance sheet allows us to augment
our pipeline to bolster long-term growth. Furthermore, we are
evaluating ways to help more patients benefit from our medicines,
including two new pipeline programs – one that explores more
convenient delivery for KRYSTEXXA by significantly reducing
infusion time, and the other for an additional indication for
teprotumumab, specifically for the rare disease, diffuse cutaneous
scleroderma.”
Continued Strong KRYSTEXXA Growth Drives Higher Peak Net
Sales Expectation; New Immunomodulation Study Data and
Shorter-Infusion Duration Development Program Offer Potential to
Benefit More Uncontrolled Gout Patients Based on current strong
performance, the Company is increasing its peak U.S. annual net
sales expectation for KRYSTEXXA to more than $1 billion, from the
previous expectation of more than $750 million. The Company
continues to project KRYSTEXXA full-year 2019 net sales growth of
more than 25 percent.
The Company also announced topline results from its MIRROR
open-label pilot study, which evaluated the use of the
immunomodulator methotrexate with KRYSTEXXA to increase the
response rate of KRYSTEXXA. The results of the study demonstrated
that 79 percent, or 11 of 14 patients, achieved a complete
response, defined as the proportion of serum uric acid (sUA)
responders (sUA < 6 mg/dL) at Month 6. The combination was also
well tolerated. Detailed results from the study will be presented
at a future medical meeting. The 79 percent response rate is
significantly higher than the 42 percent response rate in the
KRYSTEXXA Phase 3 clinical program, which evaluated KRYSTEXXA
alone.
KRYSTEXXA has demonstrated rapid reduction in sUA levels for
people with uncontrolled gout; however, treatment with biologic
medicines can, in some patients, trigger the body’s immune system
to develop anti-drug antibodies. These anti-drug antibodies can
reduce the effectiveness of the biologic therapy. Immunomodulators
such as methotrexate, which is commonly used by rheumatologists,
can help reduce this reaction.
The MIRROR open-label pilot study follows other studies that
showed an improved response rate when KRYSTEXXA is co-administered
with methotrexate. There is a growing body of data supporting the
potential of KRYSTEXXA plus immunomodulation to become the standard
therapy opposed to KRYSTEXXA therapy alone. This includes an
independent case series presented at the Annual European Congress
of Rheumatology meeting in June 2019 which demonstrated that the
administration of KRYSTEXXA with methotrexate resulted in a 100
percent response (10 of 10 patients) as defined by >80 percent
of sUA levels being maintained at goal (<6.0 mg/dL) during the
treatment period. Further, a case series presented at the American
College of Rheumatology meeting in November 2019 demonstrated an 80
percent response (8 of 10 patients) as defined by receiving ≥12
infusions without loss of sUA response.
The Company is currently conducting a separate,
placebo-controlled MIRROR trial evaluating the use of KRYSTEXXA and
methotrexate. The trial, with 135 randomized patients, is designed
to enable the potential for submission of results to the U.S. Food
and Drug Administration (FDA) for update to the label.
In addition, the Company is planning to evaluate the impact of
administering KRYSTEXXA over a significantly shorter infusion
duration. The initial proof-of-concept work will begin mid-2020.
Currently, KRYSTEXXA is infused over a two-hour or longer
timeframe. A shorter infusion duration could meaningfully improve
the experience and convenience for patients, physicians and sites
of care.
Increasing Teprotumumab Peak Net Sales Expectation;
Evaluating an Additional Indication for Diffuse Cutaneous
Scleroderma, a Rare Disease with No Treatment Options The FDA
is currently evaluating a Biologics License Application (BLA) for
teprotumumab for the treatment of Thyroid Eye Disease (TED) under
Priority Review, with a Prescription Drug User Fee Act (PDUFA)
action date of March 8, 2020. On Dec. 13, 2019, the Dermatologic
and Ophthalmic Drugs Advisory Committee of the FDA voted
unanimously (12-0) in favor of teprotumumab. Teprotumumab is a
fully human monoclonal antibody that targets and blocks the
insulin-like growth factor 1 receptor (IGF-1) with the potential to
be the first and only medicine approved for this rare eye
disease.
TED is a serious, progressive, vision-threatening rare
autoimmune disease in which the muscles and fatty tissue behind the
eye become inflamed and expand, which can lead to proptosis (eye
bulging) and diplopia (double vision) and seriously impact
activities of daily living and patients’ quality of life.
Based on an improved understanding of the TED landscape informed
by market research and launch preparation activities as well as the
dramatic results achieved in the teprotumumab Phase 3 OPTIC trial,
the Company is increasing its peak U.S. annual net sales
expectation to more than $1 billion, from the previous expectation
of more than $750 million.
The Company is evaluating additional indications for
teprotumumab and is initiating an exploratory study in diffuse
cutaneous scleroderma, a rare fibrotic disease with no treatment
options. Diffuse cutaneous scleroderma is a subtype of scleroderma
in which excess collagen production causes skin thickening and
hardening, or fibrosis, over large areas of the body, usually the
fingers, hands, arms, anterior trunk, legs and face. There can be
significant associated organ damage, including to the
gastrointestinal tract, kidneys, lungs and heart. Literature
suggests that the mechanism of action of teprotumumab, which is to
block the IGF-1 receptor, could have an impact on fibrotic
processes, such as those that are relevant to diffuse cutaneous
scleroderma. The Company expects to conduct an exploratory trial
beginning in the first half of 2020 to evaluate objective biomarker
and clinical endpoints to inform potential subsequent larger and
longer duration clinical trials.
“We are extremely pleased to be one step closer to bringing
teprotumumab to the thousands of patients with Thyroid Eye Disease
who have been waiting for a treatment for this debilitating,
painful and vision-threatening disease,” said Shao-Lee Lin, M.D.,
Ph.D., executive vice president, head of research and development
and chief scientific officer, Horizon. “Understanding the potential
of our medicines for additional diseases is also important. We
believe teprotumumab may impact underlying mechanisms of fibrosis
and could demonstrate utility beyond thyroid eye disease, including
diffuse cutaneous scleroderma, a rare autoimmune disease with
central characteristics of inflammation and fibrosis.”
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 patients 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 Teprotumumab
Teprotumumab is a fully human monoclonal antibody (mAb) and a
targeted inhibitor of the IGF-1R. Teprotumumab has received
Priority Review, Orphan Drug, Fast Track and Breakthrough Therapy
designations from the FDA. The clinical development program for
teprotumumab in the treatment of TED includes statistically
significant results from both the Phase 2 clinical study, which
were published in The New England Journal of Medicine, as well as
from the Phase 3 OPTIC confirmatory clinical trial. Both trials
were conducted at leading centers in the U.S., Germany and Italy,
with co-principal investigators Raymond Douglas, M.D., Ph.D.,
Cedars-Sinai Medical Center and George Kahaly, M.D., Ph.D.,
Johannes Gutenberg University Medical Center. Horizon is also
conducting the OPTIC-X extension trial to gather further insight
into the long-term efficacy and safety of teprotumumab.
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, but not limited to, statements related to expected
financial performance and operating results in future periods,
including potential growth in net sales of certain of Horizon’s
medicines; plans with respect to product development efforts;
potential market opportunity for, regulatory approval of and
benefits of Horizon’s medicines and medicine candidates; statements
regarding the timing of an FDA decision on the teprotumumab BLA;
expectations regarding the review of the BLA and the potential
benefits of teprotumumab to patients; and business and other
statements that are not historical facts. These forward-looking
statements are based on Horizon’s current expectations and
inherently involve significant risks and uncertainties. Actual
results and the timing of events could differ materially from those
anticipated in such forward-looking statements as a result of these
risks and uncertainties, which include, without limitation, risks
that Horizon’s actual future financial and operating results may
differ from its expectations or goals; Horizon’s ability to grow
net sales from existing medicines and successfully launch new
medicines; the availability of coverage and adequate reimbursement
and pricing from government and third-party payers; risks relating
to Horizon’s ability to successfully implement its business
strategies; risks inherent in developing novel medicine candidates
and existing medicines for new indications; risks associated with
regulatory approvals; risks in the ability to recruit, train and
retain qualified personnel; competition, including potential
generic competition; the ability to protect intellectual property
and defend patents; regulatory obligations and oversight, including
any changes in the legal and regulatory environment in which
Horizon operates and those risks detailed from time-to-time under
the caption "Risk Factors" and elsewhere in Horizon’s filings and
reports with the SEC. Horizon undertakes no duty or obligation to
update any forward-looking statements contained in this press
release as a result of new information.
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version on businesswire.com: https://www.businesswire.com/news/home/20200113005505/en/
Tina Ventura Senior Vice President, Investor Relations
Investor-relations@horizontherapeutics.com
Ruth Venning Executive Director, Investor Relations
Investor-relations@horizontherapeutics.com
U.S. Media Contact: Geoff Curtis Executive Vice
President, Corporate Affairs and Chief Communications Officer
media@horizontherapeutics.com
Ireland Media Contact: Gordon MRM Ray Gordon
ray@gordonmrm.ie
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