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Horizon Pharma Presents Results of 48 Week Off-Therapy Follow-Up to the Phase 2 Trial of Teprotumumab.

M2 PHARMA-October 31, 2018-Horizon Pharma Presents Results of 48 Week Off-Therapy Follow-Up to the Phase 2 Trial of Teprotumumab

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- Irish drug developer Horizon Pharma plc (NASDAQ: HZNP) presented detailed results from the 48 week off-therapy follow-up to the Phase 2 clinical trial of teprotumumab for people living with moderate-to-severe active thyroid eye disease were presented at the American Academy of Ophthalmology annual meeting, Oct. 27 30, 2018, in Chicago, Ill, the company said.

The data was presented in an oral session, Diplopia Response in a Controlled Trial with Teprotumumab, an IGF-1 Receptor Antagonist Antibody for Thyroid Eye Disease, at 4: 57 p.m. CT TODAY.

During active TED, which can last up to three years, the insulin-like growth factor 1 receptor (IGF-1R) is overexpressed on orbital fibroblasts, resulting in local inflammation and tissue expansion.

This can lead to proptosis, or bulging of the eye, and depending on the degree of proptosis, pressure is created on the eyeball within a tight orbital space that can cause multiple eye symptoms.

Displacement of the eye muscles subsequently cause ocular misalignment, or strabismus; as a result of which, many people living with TED also endure challenges with double vision, known as diplopia.

The analysis of this Phase 2 exploratory endpoint shows that 69.2% (18/26 patients) of those with diplopia improvements of at least one grade at week 24 maintained these improvements at week 72 (48 weeks following treatment period).

The Phase 3 confirmatory trial and extension studies will evaluate diplopia in people with moderate-to-severe active TED. Teprotumumab is an investigational medicine and its safety and efficacy have not been established.

Thyroid eye disease is a rare autoimmune disease that can be 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.

Depending on the degree of proptosis, pressure is created on the eyeball within a tight orbital space that can cause multiple eye symptoms.

Lid retraction and inability to close the eyelids subsequently lead to dry eye symptoms, corneal ulcerations, infections and impaired vision.

Displacement of the eye muscles subsequently cause ocular misalignment, or strabismus; as a result of which, many people living with TED also endure challenges with double vision, known as diplopia.

In severe cases, pressure on the optic nerve that is responsible for vision, may compromise blood supply and potentially cause blindness.

Once TED is no longer active, the orbital muscles and tissues become fibrotic, and, for some, the resulting exophthalmos and diplopia can only be corrected with complicated surgical procedures which may not always successfully reverse patients' eye conditions.

Teprotumumab is a fully human monoclonal antibody 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 US Food and Drug Administration.

A Phase 3 confirmatory study began enrolling in October 2017 after results from the randomized double-blind, placebo controlled Phase 2 study and completed enrollment in September 2018.

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 clinical activity score of >= 2 points and reduction of proptosis of >= 2 mm at week 24.

In the intent-to-treat population, 29 of 42 patients receiving teprotumumab and 9 of 45 patients receiving placebo were responders at week 24 (p?0.001).

The most frequent adverse events (>= 5 %) reported with teprotumumab compared to placebo and not dose limiting 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 May 4, 2017, issue of The New England Journal of Medicine.

Horizon Pharma is focused on researching, developing and commercialising innovative medicines that address unmet treatment needs for rare and rheumatic diseases.

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