Horizon Pharma plc Announces Presentation of Data From 48 Week Off-Therapy Follow-Up to the Phase 2 Trial of Teprotumumab
Horizon Pharma plc Announces Presentation of Data From 48 Week Off-Therapy Follow-Up to the Phase 2 Trial of Teprotumumab
-- Data presented during an oral session at the
TED is a rare autoimmune disease that is active for up to three years. During active TED the insulin-like growth factor 1 receptor (IGF-1R) is overexpressed on orbital fibroblasts, resulting in local inflammation and tissue expansion, which can in turn lead to proptosis, or bulging of the eye. This can result in stiffness of the muscles, displacement of the eyes so they are no longer in line with each other, and/or the eyelids are unable to close. The inability for people living with active TED to close their eyelids can lead to corneal ulcerations and potential blindness, and many also endure challenges with double vision, known as diplopia. Once TED is no longer active, the orbital muscles and tissues become fibrotic and, for some, changes due to local inflammation and tissue expansion can only be corrected with surgery.
“Proptosis creates numerous challenges for people living with thyroid
eye disease and is the most important measure from a clinical
perspective,” said
The analysis of the Phase 2 data, 48-Week Follow-Up Of A Multicenter, Randomized, Double-Masked, Placebo Controlled Treatment Trial Of Teprotumumab In Thyroid-Associated Ophthalmopathy, shows that at four weeks following the initial 24 week treatment period, proptosis response was 73.8 percent for patients receiving teprotumumab versus 13.3 percent for patients receiving placebo (p<0.001). In addition, 53 percent of week 24 teprotumumab proptosis responders maintained at least a 2 mm improvement relative to baseline at week 72 (48 weeks following treatment period). Teprotumumab is an investigational medicine and its safety and efficacy have not been established.
“Teprotumumab has the potential to be an impactful treatment for people
living TED and the first therapy to demonstrate reduction in proptosis,”
said
Additional data from the 48 week off-therapy follow-up will be presented
during an oral session on
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 Breakthrough Therapy, Orphan Drug and Fast
Track designations from the
The Phase 2 study was designed to evaluate the efficacy and safety of
teprotumumab in patients with recent onset, moderate-to-severe active
TED. The primary endpoint was response in the study eye defined as a
reduction in CAS of ≥ 2 points and reduction of proptosis of ≥ 2 mm at
week 24. In the intent-to-treat population, 29 of 42 (69%) patients
receiving teprotumumab and 9 of 45 (20%) patients receiving placebo were
responders at week 24 (p˂0.001). The most frequent adverse events
reported (≥ 5 percent) were nausea, muscle spasms, diarrhea, alopecia,
hyperglycemia, dry skin, dysgeusia, headache, paresthesia, hearing
impairment and weight loss. Results from this study were published
in the
Forward-Looking Statements
This press release contains forward-looking statements, including
statements regarding the potential for teprotumumab as a treatment for
TED. Forward-looking statements speak only as of the date of this press
release and
Horizon Pharma plc is focused on researching, developing and
commercializing innovative medicines that address unmet treatment needs
for rare and rheumatic diseases. By fostering a growing pipeline of
medicines in development and exploring all potential uses for currently
marketed medicines, we strive to make a powerful difference for
patients, their caregivers and physicians. For us, it’s personal: by
living up to our own potential, we are helping others live up to theirs.
For more information, please visit www.horizonpharma.com,
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Source:
Horizon Pharma plc
Tina Ventura
Senior Vice President,
Investor Relations
Investor-relations@horizonpharma.com
or
Ruth
Venning
Executive Director, Investor Relations
Investor-relations@horizonpharma.com
or
U.S.
Media Contact:
Amanda Phraner
Senior Manager, Public
Relations and Social Media
media@horizonpharma.com
or
Ireland
Media Contact:
Ray Gordon
Gordon MRM
ray@gordonmrm.ie