Horizon Pharma Announces Collaboration With Alliance For Lupus Research To Study RAYOS (Prednisone) Delayed-Release Tablets In Systemic Lupus Erythematosus

DUBLIN, IRELAND--(Marketwired - November 11, 2015) - Horizon Pharma plc (NASDAQ: HZNP), a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs, today announced a collaboration with the Alliance for Lupus Research (ALR) to study RAYOS® (prednisone) delayed-release tablets in systemic lupus erythematosus (SLE), a chronic autoimmune disease that causes inflammation and pain in the joints and muscles as well as overall fatigue.

“Despite advances in SLE therapies, sustained control of disease activity is limited to a small fraction of patients,” said Jeffrey D. Kent, M.D., FACG, senior vice president, medical affairs and outcomes research, Horizon Pharma plc. “Prednisone is the steroid most commonly prescribed for lupus and given the unique delayed-release mechanism of RAYOS, we’re excited to partner with the Alliance for Lupus Research to further explore its potential in this disease.”

The first study planned as part of the collaboration, RAYOS Inhibits Fatigue in Lupus Erythematosus (RIFLE), will investigate the effect of RAYOS on the fatigue experienced by lupus erythematosus patients, a common symptom of the disease. RIFLE is an investigator-initiated, randomized, double-blind, active comparator, cross-over study in which patients will be randomized to receive either prednisone for three months or RAYOS at 10 p.m. for three months, and then switched to the alternative medication for an additional three months. Approximately 62 patients across 25 sites will be enrolled in the United States. The primary endpoint will assess fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), a 13-question survey to be completed by study participants that focuses on the daily fatigue experienced in patients with chronic illnesses.

“This study presents an opportunity to explore the debilitating fatigue associated with SLE -- one of the most common symptoms that can decrease quality of life and increase risk of work disability and associated healthcare costs,” said Kenneth M. Farber, president of the Alliance for Lupus Research “Through this study we hope to uncover another treatment option that may improve outcomes for SLE patients.”

The secondary and exploratory endpoints of the study will include measurement of fatigue according to the Fatigue Severity Scale, a 9-item questionnaire related to how fatigue interferes with certain activities; the SF-36 short-form health survey, which determines overall physical and mental health; assessments of overall quality of life and morning stiffness; an evaluation of the activity of lupus using the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI), British Isles Lupus Activity Group (BILAG), Systemic Lupus Activity Questionnaire (SLAQ), British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) and the SF 14.

This trial will be carried out by the ALR-founded and supported Lupus Clinical Investigators Network (LuCIN) that was formed to specifically carry out this kind of trial to explore new treatments that might make life better for patients with lupus. It is part of the new ALR program to identify and test drugs and treatments that address the concerns of lupus patients. More than three fourths of lupus patients experience fatigue, which is often debilitating. This is one of the first trials to address that problem in a scientifically rational way.

About RAYOS
RAYOS, known as LODOTRA® in Europe, is a proprietary delayed-release formulation of low-dose prednisone. The pharmacokinetic profile of RAYOS is different with an approximately four-hour lag time from that of immediate-release prednisone formulations. In clinical trials studying use of RAYOS in rheumatoid arthritis (RA), patients were administered RAYOS at 10 p.m. with food. The delayed-release profile of RAYOS helps to achieve therapeutic prednisone blood levels at a time point when cytokine levels start rising during the middle of the night. While the pharmacokinetic profile of RAYOS differs in terms of lag time from immediate-release prednisone, its absorption, distribution and elimination processes are comparable. For more information, please visit www.RAYOSrx.com.

RAYOS utilizes Skyepharma’s proprietary Geoclock™ technology.

Outside the United States, LODOTRA is approved for the treatment of moderate to severe active RA when accompanied by morning stiffness in over thirty-five countries. Horizon has granted commercialization rights for LODOTRA in Europe, Asia (excluding Japan) and Latin America to its distribution partner Mundipharma International Corporation Limited. Horizon has an exclusive license from Skyepharma for RAYOS/LODOTRA.

Approved Uses for RAYOS
RAYOS, a delayed-release form of prednisone, prevents the release of substances in the body that cause inflammation. RAYOS is approved to treat a broad range of diseases including RA, polymyalgia rheumatica (PMR), psoriatic arthritis (PsA), ankylosing spondylitis (AS), lupus asthma and chronic obstructive pulmonary disease (COPD). For a full list of RAYOS indications, please see full prescribing information at www.RAYOSrx.com. RAYOS is contraindicated in patients who have known hypersensitivity to prednisone or to any of the excipients. Rare instances of anaphylaxis have occurred in patients receiving corticosteroids.

Important Safety Information about RAYOS
Do not use RAYOS if you are allergic to prednisone. Long-term use of RAYOS can affect how your body responds to stress. Symptoms can include weight gain, severe fatigue, weak muscles and high blood sugar. RAYOS can weaken your immune system, making it easier for you to get an infection or worsening an infection you already have or have recently had. RAYOS can cause high blood pressure, salt and water retention and low blood potassium. There is an increased risk of developing holes in the stomach or intestines if you have certain stomach and intestinal disorders. Behavior and mood changes can occur, including intense excitement or happiness, sleeplessness, mood swings, personality changes or severe depression.

Long-term use of RAYOS can cause decreases in bone density. RAYOS can cause cataracts, eye infections and glaucoma. Do not receive a “live” vaccine while taking RAYOS. The vaccine may not work as well during this time and may not fully protect you from disease. Taking RAYOS during the first trimester of pregnancy can harm an unborn baby. Long-term use of RAYOS can slow growth and development in children.

The most common side effects with RAYOS are water retention, high blood sugar, high blood pressure, unusual behavior and mood changes, increased appetite and weight gain. Please see full prescribing information for RAYOS at www.RAYOSrx.com.

About Horizon Pharma plc
Horizon Pharma plc is a biopharmaceutical company focused on improving patients’ lives by identifying, developing, acquiring and commercializing differentiated and accessible medicines that address unmet medical needs. The Company markets seven medicines through its orphan, primary care and specialty business units. Horizon’s global headquarters are in Dublin, Ireland. For more information, please visit www.horizonpharma.com. Follow @HZNPplc on Twitter or view careers on our LinkedIn page.

About the ALR
The Alliance for Lupus Research (ALR) is a national voluntary health organization dedicated to finding better treatments and ultimately preventing and curing systemic lupus erythematosus (SLE, or lupus). The organization is based in New York City and chaired by Robert Wood Johnson IV. Since its founding in 1999, the ALR has given more money to lupus research than any non-governmental agency in the world. To date the ALR has committed nearly $100 million to fund the most innovative and aggressive research into the cause, prevention and cure of lupus. The board of directors funds all administrative and fundraising costs, allowing one hundred percent of all donations from the public and the proceeds of our signature grassroots fundraising program, Walk with Us to Cure Lupus, to go directly to support research programs. More information can be found at www.alr.org.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding planned clinical trials of RAYOS in collaboration with the Alliance for Lupus Research and the potential of RAYOS to treat lupus symptoms. 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 risks regarding whether planned clinical trials will be initiated or completed, whether RAYOS will be shown to be safe and effective in any indications studied in planned clinical trials, whether RAYOS is ultimately approved for any additional indications, and other factors described in Horizon Pharma’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 Pharma does not undertake any obligation to update or revise these statements, except as may be required by law.