Horizon Pharma PLC (NASDAQ: HZNP) has announced results from its late-stage confirmatory trial evaluating teprotumumab for the treatment of active thyroid eye disease (TED), and that sent shares soaring on Thursday.
The study met its primary endpoint, showing more patients treated with teprotumumab, compared with placebo, had a meaningful improvement in proptosis, or bulging of the eye. Some 82.9% of teprotumumab patients compared to 9.5% of placebo patients achieved the primary endpoint.
Note that proptosis is the main cause of morbidity in TED. All secondary endpoints were also met, and the safety profile was consistent with the Phase 2 study of teprotumumab in TED.
Looking ahead, Horizon expects to submit a Biologics License Application to the U.S. Food and Drug Administration (FDA) in mid-2019. Teprotumumab previously received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA.
Timothy P. Walbert, board chair, president and CEO, commented:
The dramatic results of the teprotumumab Phase 3 confirmatory trial, in addition to positive Phase 2 data, form a highly convincing body of clinical evidence supporting teprotumumab for the treatment of active thyroid eye disease. This is a key milestone as we evolve into a research-focused company developing innovative new medicines to address challenging diseases with very few effective options. We are one step closer to delivering the first FDA-approved treatment to people living with active thyroid eye disease, and we are grateful to the patients, physicians and investigational sites who have partnered with Horizon to make the teprotumumab research and development program possible.
Shares of Horizon Pharma were last seen up about 25% to $27.30, in a 52-week range of $12.55 to $28.20. The consensus price target is $25.27.