Horizon Pharma PLC (NASDAQ:HZNP) shareholders are having a rough morning after the drug maker announced that its Phase III STEADFAST trial of Actimmune in Friedreich’s Ataxia did not meet its primary endpoint of improvement on the modified Friedreich’s Ataxia Rating Scale (FARSmNEURO) at 26 weeks. The study also failed to demonstrate efficacy on any of the secondary endpoints. Based on these data, management is discontinuing its FA program, including the 26-week extension and longterm safety study, which we had previously viewed as a possible “second chance” for the program.
In reaction, Horizon Pharma shares are falling nearly 19% to $15.79 in pre-market trading Thursday.
FARS‐mNeuro is an exam-based rating scale that measures disease progression based on functional parameters such as speech, ability to swallow, upper and lower limb coordination, gait and posture.
In addition, the secondary endpoints did not meet statistical significance. No new safety findings were identified on initial review of data other than those already noted in the ACTIMMUNE prescribing information for approved indications. The Company, in conjunction with the independent Data Safety Monitoring Board, the principal investigator and the Friedreich’s Ataxia Research Alliance (FARA) Collaborative Clinical Research Network (CCRN) in FA, has determined that, based on the trial results, the FA development program will be discontinued, including the 26-week extension study and the long-term safety study.
Patients participating in the ongoing extension studies should contact their study site coordinator for further information and next steps.
The Company will continue to work with FARA and the principal investigator to further analyze the data to help inform future research efforts as well as future data presentation or publication.
“A well-designed, rigorous study like STEADFAST would not have been possible without the extraordinary drive of the FA community, particularly the people who enrolled in the study, the clinical trial investigators and the Friedreich’s Ataxia Research Alliance,” said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. “While the results were not what we hoped for, this is the very reason why research and development is important – to find answers that may help inform future research.”
“FARA’s mission is to drive research to develop therapies that will treat and cure Friedreich’s ataxia and we remain passionately committed to that mission so that one day soon patients and caregivers impacted by this devastating disease will have effective treatment options,” said Ronald J. Bartek, co-founder and founding president, FARA. “We want to extend our sincere appreciation to all of the patients, patient families and investigators who were a part of this study as well as Horizon for collaborating with us so impressively on this important research.”
The announcement today does not impact Horizon Pharma’s full-year 2016 adjusted net sales or adjusted EBITDA guidance, and the Company believes it is well-positioned for growth in 2017 and beyond based on its existing portfolio of medicines. Horizon’s management will host a live conference call and webcast at 8 a.m. ET to discuss the results of the STEADFAST study. (Original Source)
On the ratings front, Horizon Pharma has been the subject of a number of recent research reports. In a report released yesterday, JMP analyst Donald Ellis reiterated a Buy rating on HZNP, with a price target of $33, which represents a potential upside of 70% from where the stock is currently trading. Separately, on November 28, Citigroup’s Liav Abraham upgraded the stock to Buy .
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Donald Ellis and Liav Abraham have a yearly average return of 14.7% and 10.8% respectively. Ellis has a success rate of 56% and is ranked #859 out of 4271 analysts, while Abraham has a success rate of 53% and is ranked #671.
Overall, one research analyst has assigned a Hold rating and 8 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $35.00 which is 80.5% above where the stock closed yesterday.
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.