New Data from Teprotumumab Phase 3 OPTIC Study Shows Significantly Reduced Double Vision & Improved Quality of Life for Peopl...
October 11 2019 - 12:30PM
Business Wire
Horizon Therapeutics plc (Nasdaq: HZNP) today announced new data
from the Phase 3 OPTIC confirmatory clinical trial showing that
teprotumumab provided significant benefit on several devastating
effects of active thyroid eye disease (TED) compared with placebo,
including diplopia (double vision), quality of life (QoL) and
clinical activity score (CAS). These data were presented during the
American Society of Ophthalmic Plastic and Reconstructive Surgery
(ASOPRS) 50th Anniversary 2019 Fall Scientific Symposium, and build
upon data presented earlier this year that demonstrated the
significant benefit of teprotumumab on proptosis (bulging
eyes).
Teprotumumab is an investigational medicine for the treatment of
active TED and is currently under review by the U.S. Food and Drug
Administration (FDA). The teprotumumab Biologics License
Application (BLA) was recently granted Priority Review by the FDA
and if approved, teprotumumab would be the first FDA-approved
medicine for the treatment of active TED. The Prescription Drug
User Fee Act (PDUFA) goal date is March 8, 2020.
“Thyroid eye disease commonly causes a variety of vision
impairments, with double vision reported in about half of all
people living with the disease, and almost 70 percent of patients
enrolled in the OPTIC study,” said Raymond Douglas, M.D., Ph.D., of
Cedars Sinai Medical Center and lead investigator of the OPTIC
study. “People suffering from double vision often lose the ability
to perform daily tasks, like reading and driving, impacting their
ability to work and causing depression. The results of this study
are very encouraging, showing that 68 percent of patients had an
improvement of at least one grade in double vision, and also
improved on other measures including quality of life and CAS
score.”
Previously presented primary endpoint data from OPTIC (Treatment
of Graves’ Orbitopathy (Thyroid Eye Disease) to Reduce
Proptosis with Teprotumumab Infusions in a
Randomized, Placebo-Controlled, Clinical Study) showed that
significantly more patients treated with teprotumumab had a
meaningful improvement in proptosis, the primary study endpoint, as
compared with placebo (82.9% of teprotumumab patients compared to
9.5% of placebo patients; p˂0.001).
The following new data on three secondary endpoints were
presented at ASOPRS:
- Diplopia: At Week 24, 68% of
patients receiving teprotumumab had an improvement from baseline of
at least one grade in diplopia, compared to 29% of patients
receiving placebo (p=0.001). This endpoint measured the percentage
of patients who reported at least some diplopia at baseline in the
study eye and who had a reduction of ≥ 1 grade with no
corresponding deterioration (≥ 1 grade worsening) in the fellow eye
at Week 24.
- Quality of Life: Patients
receiving teprotumumab had a mean change of 13.79 on the Graves'
Ophthalmopathy Quality of Life (GO-QoL) scale compared with a
change of 4.43 for patients receiving placebo (p<0.001). These
scores indicate a statistical and clinically meaningful improvement
over placebo in these QoL measures. The GO-QoL scale consists of
two subscales to evaluate the quality of life of TED (Graves’
Ophthalmology) patients, including impacts on visual function and
self-assessment of appearance. A change of 6 points is considered
clinically significant.1
- CAS Score: At Week 24, more
patients achieved a CAS value of 0 or 1 with teprotumumab treatment
(59% vs 21% of placebo participants) (p<0.001). CAS is a scale
used to assess the disease activity of TED, and measures the degree
of inflammation, including pain, swelling and redness. The CAS
scale ranges from 0 to 7, with a score of 0 representing no
swelling or activity.2
- Significant improvement in other secondary endpoints, including
average change in proptosis and overall response rate over the
24-week treatment period, were presented during the 2019 American
Association of Clinical Endocrinologists (AACE) Annual Scientific
& Clinical Congress.
As previously reported, teprotumumab was generally well
tolerated; the majority of adverse events were mild or moderate,
manageable and resolved during or after treatment. The earlier
Phase 2 study results were published in The New England Journal of
Medicine in May 2017.
“The Phase 3 data further illustrate the potential for
teprotumumab to benefit the most prominent and challenging
characteristics of active thyroid eye disease, most notably the
vision impairment and subsequent detrimental effect on daily life,”
said Shao-Lee Lin, M.D., Ph.D., executive vice president, head of
research and development and chief scientific officer, Horizon.
“With the findings of our Phase 3 clinical trial demonstrating
benefit across all of the ranked endpoints studied, we are one step
closer to addressing the unmet need in the TED community for an
FDA-approved medicine.”
About Thyroid Eye Disease
Thyroid eye disease (TED) is a serious, progressive and
vision-threatening autoimmune disease with a limited window of
activity that can last up to three years.3,4,5 While TED often
occurs in people living with hyperthyroidism or Graves’ disease, it
is a distinct disease that is caused by autoantibodies activating
an IGF-1R-mediated signaling complex on cells within the orbit.6,7
This leads to a cascade of negative effects, which may cause
long-term, irreversible damage. Active TED lasts for up to three
years and is characterized by inflammation and tissue expansion
behind the eye.3,8 As TED progresses, it causes serious damage –
including proptosis (eye bulging), strabismus (misalignment of the
eyes), and diplopia (double vision) – and in some cases can lead to
blindness.4,9 TED has only been shown to respond to pharmacotherapy
while the disease is active and inflammation is ongoing.10
Currently, patients must live with active TED until the disease
becomes inactive – often left with permanent and vision-impairing
consequences.3,8
About Teprotumumab
Teprotumumab is a fully human monoclonal antibody (mAb) and a
targeted inhibitor of the insulin-like growth factor 1 receptor
(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 positive results from the Phase 3 OPTIC confirmatory
clinical trial as well as positive Phase 2 results, which were
published in The New England Journal of Medicine. The OPTIC trial
was 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 statements regarding the potential availability and
benefits of teprotumumab to patients. These forward-looking
statements are based on management 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 the actual timing
and process of review of the teprotumumab BLA and whether the BLA
is ultimately approved, as well as those 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 does not undertake any
obligation to update or revise these statements, except as may be
required by law.
References
- Terwee CB. Clinical Endocrinology 2001; 54: 391-398.
- Wiersinga WM, Perros P, Kahaly GJ, et al. Clinical assessment
of patients with Graves’ orbitopathy: the European Group on Graves’
Orbitopathy recommendations to generalists, specialists and
clinical researchers. Eur J Endocrinol 2006; 155: 387-9.
- Graves’ Ophthalmopathy: VISA versus EUGOGO Classification,
Assessment, and Management. Journal of Ophthalmology. 2015.
https://www.hindawi.com/journals/joph/2015/249125/cta/. Accessed
Feb 22, 2019.
- The 2016 European Thyroid Association/European Group on Graves'
Orbitopathy Guidelines for the Management of Graves' Orbitopathy.
European Thyroid Journal.2 March 2016.
https://www.ncbi.nlm.nih.gov/pubmed/27099835. Accessed Feb 22,
2019.
- Shan SJ, Douglas RS. The Pathophysiology of Thyroid Eye
Disease. Journal of Neuro-Ophthalmology. 2014; 34: 177-185.
- Graves' Ophthalmopathy. The New England Journal of Medicine. 25
February 2010. https://www.nejm.org/doi/full/10.1056/NEJMra0905750.
Accessed Feb 22, 2019.
- Igs from patients with Graves' disease induce the expression of
T cell chemoattractants in their fibroblasts. The Journal of
Immunology. 15 January 2002.
https://www.ncbi.nlm.nih.gov/pubmed/11777993. Accessed Feb 22,
2019.
- Update on thyroid eye disease and management. Clinical
Ophthalmology. 19 October 2009.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2770865/. Accessed Feb
22, 2019.
- Clinical features of dysthyroid optic neuropathy: a European
Group on Graves' Orbitopathy (EUGOGO) survey. British Journal of
Ophthalmology. 11 October 2006.
https://www.ncbi.nlm.nih.gov/pubmed/17035276. Accessed Feb 22,
2019.
- Mamoojee Y, Pearce SHS. Natural History. In: Wiersinga WM,
Kahaly GJ (eds): Graves’ Orbitopathy: A Multidisciplinary Approach
– Questions and Answers. Basel, Karger. 2017:93-104.
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Tina Ventura Senior Vice President, Investor Relations
Investor-relations@horizontherapeutics.com Ruth Venning
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Patient Advocacy media@horizontherapeutics.com Rachel Vann
Associate Director, Product Communications
media@horizontherapeutics.com Ireland Media Contact:
Gordon MRM Ray Gordon ray@gordonmrm.ie
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