Horizon’s strategy is to transform into a balanced, diversified, sustainable-growth biopharmaceutical company, predominantly focused on rare disease medicines.
The company has been focused on expanding its orphan drug business, as evidenced by its long-range plan, wherein the orphan business is expected to constitute approximately 60% of net sales in 2020. Meanwhile, the company has been quite active on the acquisition front. In May 2015, Horizon acquired Hyperion in a deal valued at about $1.1 billion and added a couple of UCD drugs like Ravicti and Buphenyl/Ammonaps to its portfolio.
The FDA approved a supplemental new drug application (sNDA) for Ravicti to expand the age range, for chronic management of urea cycle disorders (UCD) in patients, to two months and older from two years and older, in April 2017. In December 2018, the FDA approved an sNDA to expand the age range for Ravicti Oral Liquid to include infants younger than two months, living with a UCD.
Also, in January 2016, Horizon acquired Crealta for approximately $540 million in cash. The acquisition added Krystexxa and Migergot to its portfolio. Krystexxa is the first and only FDA-approved medicine for chronic refractory gout. Sales of Krystexxa increased 56% year over year in the first nine months of 2018. In addition, Krystexxa has a long patent life running through 2027.
Meanwhile, to evaluate the safety and efficacy related to the immunogenicity profile of Krystexxa, two investigator-initiated studies — TRIPLE and RECIPE — are currently enrolling patients. Horizon initiated a new company-sponsored immunomodulation MIRROR study with Krystexxa in September 2018 to continue exploring a broader clinical profile of the drug.
The study will evaluate the impact of adding methotrexate to Krystexxa to enhance the patient response rate. The company expects to begin enrolling patients into the adapted protocol in the second quarter of 2019.
Earlier in 2017, the company acquired River Vision, thereby adding the latter’s biologic candidate teprotumumab in late-stage development for Thyroid Eye Disease (TED). The company completed enrollment in phase III confirmatory clinical study, OPTIC, evaluating teprotumumab for the treatment of TED in September 2018.Top-line data from the study is expected in the second quarter of 2019.
Horizon Pharma is pursuing two development programs — HZN-003 and PASylated uricase technology — for next-generation biologics for uncontrolled gout. These programs are expected to support and sustain the company’s market leadership in the said indication.
The company is also working to expand the label of Actimmune, which is approved for the treatment of chronic granulomatous disease (CGD) and severe malignant osteopetrosis (SMO). The company has collaborated with the Fox Chase Cancer Center to evaluate Actimmune in combination with Bristol-Myers Squibb Company’s BMY Opdivo in a phase I dosing study for the treatment of kidney and bladder cancer.
The National Cancer Institute is evaluating Actimmune in combination with Merck & Co’s MRK Keytruda in phase II study to treat cutaneous t-cell lymphoma patients. A dose ranging study is evaluating Actimmune in combination with Bristol-Myers’ Taxol, and Roche’s RHHBY Herceptin and Perjeta in a certain type of advanced breast cancer.
Horizon Pharma currently carries a Zacks Rank#3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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