SOURCE: Horizon Pharma plc

Horizon Pharma plc

January 12, 2015 08:00 ET

Horizon Pharma plc Provides Business Update

Expands Sales Organization, Accelerates Expansion of Prescriptions-Made-Easy™ (PME) Program and Launches PENNSAID®2% in United States; Secures Agreement With FDA on ACTIMMUNE® Phase 3 Clinical Trial Primary Endpoint

SAN FRANCISCO, CA--(Marketwired - Jan 12, 2015) - Horizon Pharma plc (NASDAQ: HZNP), a specialty biopharmaceutical company focused on improving patients' lives by identifying, developing, acquiring and commercializing differentiated products that address unmet medical needs, today announced a business update on its primary care and orphan businesses.

  • Horizon has begun execution of a comprehensive plan creating a new organization focused on the acceleration of its Prescriptions-Made-Easy™ (PME) program to ensure continued growth of its non-steroidal anti-inflammatory drug (NSAID) portfolio in 2015.

  • Horizon-labeled PENNSAID® (diclofenac sodium topical solution) 2% w/w is now available in the United States for the treatment of the pain of osteoarthritis (OA) of the knee(s). 

  • The Company has completed the hiring and training of an additional 75 sales representatives, with a total primary care sales force size of 325 representatives. All primary care representatives are now selling DUEXIS®, PENNSAID 2% and VIMOVO®.

  • The U.S. Food and Drug Administration (FDA) has agreed to the primary endpoint for the Company's Phase 3 study that will evaluate ACTIMMUNE® in the treatment of Friedreich's Ataxia (FA). The Company plans to submit an IND application in the first quarter of 2015 and anticipates the Phase 3 clinical study will begin enrolling patients in the second quarter of 2015.

"We have a unique commercial business model where focused representative promotion, PME-program execution and the maximization of the value of the product are all aligned to drive revenue growth," said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. "We are now executing a comprehensive plan to ensure patients maintain access to DUEXIS and VIMOVO when their physicians prescribe them. With the acceleration of our PME program, the creation of an innovative retail-PME program and recent pricing action, we are confident we will grow combined net revenues for DUEXIS and VIMOVO in the first quarter of 2015 versus the first quarter of 2014."

Primary Care Business Update

  • Dan Ulvila has been named group vice president, commercial operations, integration and general manager, Prescriptions-Made-Easy, or PME, leading a ten person organization focused on adding new physicians and driving prescriptions for the Company's products.

  • The Company has initiated a territory-by-territory PME-activation program, which began in December 2014 and will continue through February 2015. Early results are encouraging with PME-activated territories showing a 21 percent increase in DUEXIS and VIMOVO prescriptions versus prescription levels prior to initiation and each product achieving all-time Horizon high prescriptions in the week ending December 19, 2014.

  • The Company has initiated a retail-PME program, which enables patients going to most retail pharmacies to get similar benefits to Horizon's traditional PME program.

  • Horizon-labeled PENNSAID 2% is now available in the United States for the treatment of the pain of OA of the knee(s). PENNSAID 2% was approved by the FDA on January 16, 2014. It is the only topical NSAID used twice a day for treating the pain of OA of the knee(s) and is easy for patients to apply with a convenient measured dosage pump dispenser.

  • Horizon now has its full primary care sales force of 325 representatives selling DUEXIS, PENNSAID 2% and VIMOVO.

Orphan Business Update

  • ACTIMMUNE continues to see double digit quarter over quarter net sales growth in the current approved indications, Chronic Granulomatous Disease (CGD) and severe, malignant osteopetrosis (SMO).

  • Horizon has started to further study other dosage forms and additional orphan diseases such as the more common but still devastating forms of osteopetrosis (intermediate osteopetrosis and autosomal dominant osteopetrosis), severe atopic dermatitis and cutaneous T-cell lymphoma.

  • The U.S. Food and Drug Administration (FDA) has agreed to the primary endpoint for the Company's planned Phase 3 study that will evaluate ACTIMMUNE in the treatment of Friedreich's Ataxia (FA). 

  • The study will be conducted in collaboration with the Friedreich's Ataxia Research Alliance (FARA) and the investigators of FARA's Collaborative Clinical Research Network (CCRN) in Friedreich's Ataxia.

    • The primary endpoint is the change from baseline after 26 weeks in the Friedreich's Ataxia Rating Scale-modified neurological exam score (FARS-mNeuro) for patients treated with ACTIMMUNE compared to placebo.

    • The result of FARS-mNeuro score in the Phase 2 ACTIMMUNE FA study (p=0.0117) is consistent with the previously reported total FARS score in the study (p=0.0078). The FARS-mNeuro is a subscore of the total FARS score removing components of the total FARS score viewed by FDA to be more subjective and effort dependent.

    • The study is planned to be a randomized, double-blind, multicenter, placebo-controlled, 26-week study evaluating ACTIMMUNE in children and young adults (10-25 years of age) with FA functional stage > 1 (minimal disability) to < 5 (severe disability). 

    • It is anticipated that approximately 110 subjects will be screened at four U.S. centers for eligibility to randomize approximately 90 subjects 1:1 to receive either ACTIMMUNE or placebo. The Company anticipates the study will take 18 months to complete.

  • The Company plans to submit an IND application in the first quarter of 2015 and anticipates the Phase 3 clinical study will begin enrolling patients in the second quarter of 2015.

About PENNSAID® 2%
PENNSAID (diclofenac sodium topical solution) 2% w/w is a non-steroidal anti-inflammatory drug (NSAID) indicated for the treatment of pain of osteoarthritis (OA) of the knee(s). PENNSAID contains diclofenac sodium, an NSAID, and also includes dimethyl sulfoxide (DMSO), a powerful penetrating agent that helps ensure that diclofenac sodium is absorbed through the skin to the site of inflammation and pain. PENNSAID is an alternative to oral NSAID treatment, reducing systemic exposure to a fraction of that provided by the oral NSAID diclofenac. The only topical NSAID offered with the convenience of a metered-dose pump, PENNSAID is applied in two pumps, twice daily, delivering relief right to the site of OA knee pain. For more information, please see www.PENNSAID.com.

APPROVED USES FOR PENNSAID 2%
PENNSAID® (diclofenac sodium topical solution) 2% w/w is a non-steroidal anti-inflammatory drug (NSAID) used for treating the pain of osteoarthritis of the knee(s).

IMPORTANT SAFETY INFORMATION

WARNING: CARDIOVASCULAR AND GASTROINTESTINAL RISK

Heart Risk

  • Non-steroidal anti-inflammatory drugs (NSAIDs) may cause an increased risk of serious heart clotting events, heart attack and stroke, which can kill you. This risk may increase with longer use. Patients with heart disease or risk factors for heart disease may be at greater risk.

  • PENNSAID should not be used if you are in the hospital for certain heart surgeries.

Stomach and Intestine Risk

  • NSAIDs cause an increased risk of serious stomach and intestine events including bleeding, ulcers and holes in the stomach or intestines, which can kill you. These events can occur at any time during use and without warning symptoms. Elderly patients are at greater risk for serious stomach and intestine events.

CONTRAINDICATIONS

  • DO NOT USE PENNSAID if you:

    • are in the hospital for certain heart surgeries.

    • know you are allergic to diclofenac sodium or any other ingredient of PENNSAID.

    • have experienced asthma, hives, or allergic-type reactions after taking aspirin or other NSAIDs. Severe, allergic reactions, that will rarely kill you, to NSAIDs have been reported in such patients.

WARNINGS AND PRECAUTIONS

  • To minimize the potential for increased risk of serious heart events while being treated with an NSAID, use the lowest effective dose for the shortest duration possible.

  • Elevation of one or more liver tests may occur during therapy with NSAIDs. PENNSAID should be discontinued immediately if abnormal liver tests persist or worsen.

  • Use with caution in patients with fluid retention or heart failure.

  • Hypertension can occur with NSAID treatment. Monitor blood pressure closely with PENNSAID treatment.

  • Long-term use of NSAIDs can result in severe kidney injury. Use PENNSAID with caution in patients at greatest risk of this reaction, including the elderly, those with impaired kidney function, heart failure, liver dysfunction and those taking diuretics and ACE-inhibitors (certain blood pressure medicines).

  • Severe allergic reactions may occur without prior use of PENNSAID. NSAIDs can cause serious skin reactions such as exfoliative dermatitis, Stevens-Johnson Syndrome (SJS) and toxic epidermal necrolysis (TEN), which can kill you.

  • Wash and dry hands before and after use. Avoid contact of PENNSAID with the eyes, nose and mouth.

  • PENNSAID was not studied under the conditions of heat application, complete covering bandages or exercise; therefore, concurrent use of PENNSAID under these conditions is not recommended.

  • Do not:

    • Apply PENNSAID to open wounds.

    • Shower for at least 30 minutes after applying PENNSAID.

    • Wear clothing over the PENNSAID treated knee until the treated knee is dry.

  • Protect treated knee(s) from natural or artificial sunlight.

  • Protect treated knee(s) from sunlight (real and tanning booths).

  • Topicals, such as sunscreen and bug repellent, should not be used until after PENNSAID treated knee(s) are completely dry.

  • Do not use with oral NSAIDs unless your doctor says it is OK and you have lab tests to check your progress.

  • There is no consistent evidence that regular use of aspirin lessens the increased risk of serious heart events, such as heart clotting, heart attack and stroke associated with NSAID use. As with all NSAIDs, regular administration of PENNSAID and aspirin is not generally recommended because of the potential of increased risks.

ADVERSE REACTIONS

  • The most common adverse events in a phase 2 clinical trial of PENNSAID 2% were application site reactions, such as dryness (22%), peeling (7%), redness (4%), itching (2%), pain (2%), skin hardening (2%), rash (2%) and scabbing ( < 1%). Other adverse reactions occurring in > 1% of patients receiving PENNSAID 2% included bladder infection (3%), bruising (2%), sinus congestion (2%) and nausea (2%).

  • The most common treatment-related adverse events in patients receiving PENNSAID 1.5% were application site skin reactions including dry skin (32%), skin reaction characterized by redness and hardening (9%), skin reaction with blisters (2%) and itching (4%). In a long term safety study, skin reactions occurred in 13% and skin reactions with blisters in 10% of patients, generally within the first 6 months of exposure, leading to a withdrawal rate for an application site event of 14%. Other common adverse events greater than placebo include: stomach upset (9%), stomach pain (6%), gas (4%), diarrhea (4%) and nausea (4%).

USE IN SPECIFIC POPULATIONS

  • PENNSAID should not be used in pregnant woman or in women who are breastfeeding and is not approved for use in children.

For more information on PENNSAID 2%, please see the Medication Guide and Full Prescribing Information, available at www.PENNSAID.com.

About ACTIMMUNE®
ACTIMMUNE (interferon gamma-1b) 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). For more information, please see www.ACTIMMUNE.com.

APPROVED USES FOR ACTIMMUNE
ACTIMMUNE (Interferon gamma-1b) is indicated:

  • For reducing the frequency and severity of serious infections associated with Chronic Granulomatous Disease.

  • For delaying time to disease progression in patients with severe, malignant osteopetrosis.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • In patients who develop or have known hypersensitivity to interferon-gamma, E. coli-derived products, or any component of the product.

WARNINGS AND PRECAUTIONS

  • ACTIMMUNE should be used with caution in patients with preexisting cardiac conditions, including ischemia, congestive heart failure or arrhythmia.

  • Caution should be exercised when administering ACTIMMUNE to patients with seizure disorders or compromised central nervous system function.

  • Caution should be exercised when administering ACTIMMUNE to patients with Myelo suppression and in combination with other potentially myelo suppressive agents.

  • Patients begun on ACTIMMUNE before age one year should receive monthly assessments of liver function. If severe hepatic enzyme elevations develop, ACTIMMUNE dosage should be modified.

  • ACTIMMUNE should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus.

  • During nursing, ACTIMMUNE or nursing should be discontinued dependent on the importance of the drug to the mother.

ADVERSE REACTIONS

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

  • Isolated cases of acute serious hypersensitivity reactions have been observed in patients receiving ACTIMMUNE.

  • Reversible neutropenia, thrombocytopenia and elevations of AST and/or ALT have been observed during ACTIMMUNE therapy.

  • 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.

ACTIMMUNE is not indicated for the treatment of Friedreich's ataxia or any indications other than CGD and SMO. This data is not a substitute for FDA approval and should not be interpreted as such.

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

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. Horizon's U.S. marketed products are ACTIMMUNE® (interferon gamma-1b), DUEXIS® (ibuprofen/famotidine), PENNSAID® (diclofenac sodium topical solution) 2% w/w, RAYOS® (prednisone) delayed-release tablets and VIMOVO® (naproxen/esomeprazole magnesium). Horizon's global headquarters are in Dublin, Ireland. For more information, please visit www.horizonpharma.com.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding potential revenue growth, including growth of DUEXIS and VIMOVO net revenue in the first quarter of 2015, the design and timing of the Company's planned Phase 3 clinical trial of ACTIMMUNE in FA, the Company's plans to file an IND application, the potential for ACTIMMUNE as a treatment for FA patients, future collaborative efforts between the Company and FARA and the CCRN in FA, the planned expansion and expected benefits of the Company's PME program and the Company's growth strategy and prospects. 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 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, whether Horizon will be able to realize the anticipated benefits of recent product acquisitions and manage the integration of new products successfully, whether results of subsequent studies will be consistent with results of the Phase 2 clinical trial of ACTIMMINE in FA, the Company's ability to identify and enroll patients in the planned Phase 3 study, potential delays in initiating and completing the planned Phase 3 study, the Company's ability to file the planned IND application within the timeline it expects and whether and when the FDA allows the IND, risks related to regulatory review and approval of product candidates, Horizon's ability to successfully expand its PME program, whether the PME program continues to result in prescription growth 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

  • Contacts:

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

    Media:
    Geoff Curtis
    Email Contact
    +1 312-233-1253