Horizon Pharma PLC (NASDAQ:HZNP) announced that a retrospective analysis of data from previous pivotal, randomized KRYSTEXXA® (pegloticase) clinical trials demonstrate that refractory chronic gout patients defined as “non-responders” based on serum uric acid levels in the trials still achieved significant clinical benefit despite a loss of response in uric acid levels.  These data (abstract 212) were presented during the American College of Rheumatology (ACR) and the Association of Rheumatology Health Professionals (ARHP) Annual Meeting in Washington D.C. on Sunday, November 13, 2016.

“In a recent survey of U.S. adults living with gout, nearly half said they could not imagine anything more painful than a gout flare, revealing a critical need for education and ongoing study of the available options for managing the condition,” said Jeffrey W. Sherman, M.D., FACP, executive vice president, research and development and chief medical officer, Horizon Pharma plc.  “Our clinical presence at the ACR meeting this year is the largest for KRYSTEXXA in three years and underscores our drive to expand awareness of this innovative treatment option for refractory chronic gout sufferers.”

KRYSTEXXA is the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of refractory chronic gout, which represents an orphan disease subset of the common form of gout.  In general, gout is a type of chronic inflammatory arthritis where uric acid builds up in the blood and can lead to severe pain and joint destruction, as well as the manifestation of tophi, which are hard, uric acid deposits under the skin that contribute to bone and cartilage destruction.  Patients with refractory chronic gout continue to have abnormally high levels of uric acid despite conventional therapies.  Refractory chronic gout impacts an estimated 50,000 people in the United States.

“The pivotal clinical trials evaluating KRYSTEXXA provide a wealth of data for health care providers treating people living with refractory chronic gout,” said Peter E. Lipsky, M.D., AMPEL BioSolutions, and one of the authors of the poster presentation.  “A retrospective look at this data uncovered that patients defined as “non-responders” in the trial actually experienced significant clinical benefits from KRYSTEXXA, with a quarter of non-responders achieving complete tophus resolution at six months.”

Summary of Results  

  • Gout symptom improvements were greatest for responders, but also significant for non-responders.
  • 25 percent of non-responders reported complete tophus resolution at six months.
  • Non-responders achieved a statistically significant decrease in gout flares at six months.
  • Non-responders reported improvements from baseline across several meaningful clinical endpoints including Patient Global Assessment, tender and swollen joints and pain.
  • No significant improvements were observed among patients who received placebo.
  • Across the pivotal clinical trials, the most common serious side effects in patients taking KRYSTEXXA were gout flare-ups or attacks, severe allergic reactions, and infusion reactions. (Original Source)

Shares of Horizon Pharma closed last Friday at $20.3, up $0.43 or 2.16%. HZNP has a 1-year high of $23.44 and a 1-year low of $13.05. The stock’s 50-day moving average is $18.40 and its 200-day moving average is $18.17.

On the ratings front, HZNP stock has been the subject of a number of recent research reports. In a report issued on November 8, Mizuho analyst Irina Rivkind Koffler reiterated a Buy rating on HZNP, with a price target of $30, which implies an upside of 48% from current levels. Separately, on November 7, BMO’s Gary Nachman reiterated a Buy rating on the stock and has a price target of $29.

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Irina Rivkind Koffler and Gary Nachman have a yearly average return of 18.4% and 14.1% respectively. Koffler has a success rate of 52% and is ranked #50 out of 4209 analysts, while Nachman has a success rate of 77% and is ranked #296.

Sentiment on the street is mostly bullish on HZNP stock. Out of 9 analysts who cover the stock, 8 suggest a Buy rating and one recommends to Hold the stock. The 12-month average price target assigned to the stock is $33.67, which implies an upside of 66% from current levels.

Horizon Pharma Plc engages in the research, development, and market of pharmaceutical products. Its medicines intend to treat arthritis, inflammation, and orphan diseases. It distributes under the following brands: Actimmune, Buphenyl, Duexis, Krystexxa, Migergot, Pennsaid, Ravicti, Rayos, and Vimovo.