SOURCE: Horizon Pharma plc

October 09, 2014 13:37 ET

Horizon Pharma plc Announces Presentation of ACTIMMUNE® Phase 2 Data in Friedreich's Ataxia

Positive Clinical Efficacy Signal Seen in Study of Twelve Patients; Company Planning Phase 3 Program

DUBLIN, IRELAND--(Marketwired - Oct 9, 2014) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company with a portfolio of products in arthritis, inflammation and orphan diseases, announced today the presentation of data from a Phase 2 clinical study of ACTIMMUNE® (interferon gamma-1b) treatment in children with Friedreich's ataxia (FA). An abstract of the data has been published in a supplement to the Annals of Neurology and is being presented as a poster during the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13.

This single center, Phase 2 open-label study of 12 children ages five to 17 years with genetically confirmed FA with treatment of up to 12 weeks, evaluated the safety, tolerability and efficacy of ACTIMMUNE. The study measured frataxin levels, a biomarker of disease and clinical measures (Friedreich's Ataxia Rating Scale [FARS] score and other neurological evaluations) as efficacy markers. Additional details on the study design may be found at

The results, as noted in the published abstract, showed ACTIMMUNE was well tolerated with no serious adverse events and two subjects reporting severe events and subsequent dose reductions. The safety findings generally reflected the labeled safety profile for ACTIMMUNE. Changes in frataxin protein levels, the primary study endpoint, were statistically significant in red blood cells, white blood cells and platelets. The magnitude of change observed was small and varied between tissues. Mean improvement in the FARS score, a clinically validated measurement of patient performance and secondary endpoint, was statistically significant (p=0.008) and was equivalent to two years of extension of disease progression. No other changes were observed.

"The highly significant improvement in the FARS score, a key measure of improvement in disease progression, supports further study of ACTIMMUNE in Friedreich's ataxia," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We plan to work with the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) and the Friedreich's Ataxia Research Alliance (FARA) to rapidly move into a registration program for ACTIMMUNE in FA. We plan to meet with the FDA this quarter regarding the clinical development and regulatory pathway for ACTIMMUNE in FA."

The study was sponsored by FARA and conducted by David Lynch, M.D., Ph.D., professor of neurology at Children's Hospital of Philadelphia (CHOP) and principal investigator of the CCRN in FA. Investigational product was provided by Vidara Therapeutics Research Limited, now part of Horizon Pharma plc.

"The FA community has been working toward a viable treatment option and we were proud to be able to sponsor this initial pilot study. We are encouraged that ACTIMMUNE was well tolerated and there was a significant improvement observed in a clinical outcome measure," said Ronald Bartek, president, director and co-founder of FARA. "We are very pleased to partner with Horizon Pharma and look forward to working together to explore the potential for ACTIMMUNE in FA."

About Friedreich's Ataxia
FA is a debilitating, life-shortening and degenerative neuro-muscular disorder that affects about one in 50,000 people in the United States. Onset of symptoms can vary from five years old to adulthood, with the childhood onset tending to be associated with a more rapid progression. A progressive loss of coordination and muscle strength leads to motor incapacitation and often the full-time use of a wheelchair. Most young people diagnosed with FA require mobility aids such as a cane, walker or wheelchair by their teens or early 20's. There are currently no approved treatments for FA. For more information about FA, visit the Friedreich's Ataxia Research Alliance (FARA) website at

About ACTIMMUNE® (interferon gamma-1b)
ACTIMMUNE is currently approved for two rare diseases in the United States. It is approved by the FDA to reduce the frequency and severity of serious infections associated with Chronic Granulomatous Disease (CGD), a genetic disorder that affects the functioning of a type of white blood cell of the immune system, neutrophils or phagocytes, leading to recurrent severe bacterial and fungal infections and chronic inflammatory conditions. In addition, ACTIMMUNE is approved by the FDA to slow the worsening of severe, malignant osteopetrosis (SMO), another genetic disorder that affects normal bone formation causing the abnormal accumulation of bone material which tends to narrow the space inside bones where bone marrow is formed. This can cause failure of the bone marrow, leading to a decrease in various blood cells such as red blood cells (anemia) and white blood cells (decreased ability to fight infection).

Important Safety Information
ACTIMMUNE is contraindicated in patients who develop or have known hypersensitivity to interferon-gamma, E. coli-derived products or any component of the product.

The most common adverse experiences occurring with ACTIMMUNE therapy are "flu like" or constitutional symptoms such as fever, headache, chills, myalgia, or fatigue, which may decrease in severity as treatment continues. Some of the "flu-like" symptoms may be minimized by bedtime administration of ACTIMMUNE. Acetaminophen may be used to prevent or partially alleviate the fever and headache.

Reversible neutropenia and thrombocytopenia have been observed during ACTIMMUNE therapy. Caution should be exercised when administering ACTIMMUNE in patients with myelosuppression or in combination with other potentially myelosuppressive agents. ACTIMMUNE may also depress hepatic metabolism of certain drugs that are metabolized by the hepatic cytochrome P-450 system. ACTIMMUNE has not been tested for carcinogenic potential. ACTIMMUNE is pregnancy Category C and it is unknown if ACTIMMUNE is excreted in human milk.

Reversible elevations of AST and/or ALT have been observed during ACTIMMUNE therapy. Patients initiated on ACTIMMUNE therapy before one year of age should receive monthly assessments of liver function. If severe hepatic enzyme elevations develop, ACTIMMUNE dosage should be modified.

At doses 10 times greater than the weekly recommended dose, ACTIMMUNE may exacerbate pre-existing cardiac conditions or may cause reversible neurological effects such as decreased mental status, gait disturbance and dizziness. Therefore, caution is advised when ACTIMMUNE is administered to patients with seizure disorders or compromised CNS function or when administered to patients with cardiac conditions such as ischemia, heart failure or arrhythmia.

For more information on ACTIMMUNE, please see the Full Prescribing Information at

About Horizon Pharma plc
Horizon Pharma plc is a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated products that address unmet medical needs. The company markets a portfolio of products in arthritis, inflammation and orphan diseases. The company's U.S. marketed products are ACTIMMUNE® (interferon gamma-1b), DUEXIS® (ibuprofen/famotidine), RAYOS® (prednisone) delayed-release tablets and VIMOVO® (naproxen/esomeprazole). Horizon's global headquarters are in Dublin, Ireland. For more information, please visit

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding results of the Phase 2 clinical trial of ACTIMMUNE and management expectations and plans regarding further study of ACTIMMUNE in FA and related discussions with the FDA and the potential for ACTIMMUNE as a treatment for FA patients. Forward-looking statements speak only as of the date of this press release and Horizon does not undertake any obligation to update or revise these statements, except as may be required by law. These forward-looking statements are based on management's 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, but are not limited to, risks regarding whether results of subsequent studies will be consistent with results of the Phase 2 clinical trial, regulatory review and approval of product candidates, Horizon's ability to commercialize products successfully, including risks relating to availability of coverage and adequate reimbursement and pricing from government and third party payers, Horizon's ability to enforce its intellectual property rights to its products, and Horizon's ability to execute on its growth strategy. For a further description of these and other risks facing the Company, please see the risk factors described in the Company'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 the Company undertakes no obligation to update or revise these statements, except as may be required by law.

Contact Information

  • Investor and Media Contact:
    Elizabeth M. Higashi, CFA
    Vice President, Investor Relations
    Email Contact
    +1 224 383-3285