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Santarus and Pharming announce positive Top-Line Phase III results for RUCONEST in acute Hereditary Angioedema

November 7, 2012 - San Diego, Ca And Leiden, The Netherlands

Statisticallysignificant results for primary endpoint of time to beginning of symptomrelief

Santarus, Inc. (NASDAQ: SNTS) and Pharming Group NV (NYSE Euronext: PHARM)today announced that their pivotal Phase III clinical study to evaluate thesafety and efficacy of the investigational drug RUCONEST® (recombinanthuman C1 esterase inhibitor) 50 U/kg for the treatment of acute attacks ofangioedema in patients with Hereditary Angioedema (HAE) met the primaryendpoint of time to beginning ofsymptom relief.

A statistically significant difference in the time to beginning of symptomrelief was observed in the intent-to-treat population (n=75) betweenRUCONESTand placebo (p=0.031, log-rank test); the median time to beginning ofsymptomrelief was 90 minutes for RUCONEST patients (n=44) and 152 minutes forplacebopatients (n=31). The time to beginning of symptom relief was defined asthetime from the beginning of infusion of study medication (RUCONEST orplacebo)until the beginning of a persistent beneficial effect, based on thepatient'sresponses to a Treatment Effect Questionnaire for the primary attacklocation.

RUCONEST was generally well tolerated in this Phase III clinical study andthefrequency of patients experiencing at least one treatment emergent adverseeventin the RUCONEST treated group was less than in the placebo group. Within 72hours of the completion of infusion of study medication, four RUCONESTpatients(7%) experienced six adverse events: sneezing, procedural headache, backpain,skin burning sensation, an increase in fibrin D-dimer and lipoma. Withinthe 72hour period four placebo patients (22%) experienced four adverse events:sinuscongestion, vasomotor rhinitis, diarrhea and dyspepsia. Thromboembolicevents,anaphylaxis, or neutralizing antibodies to C1 inhibitor were not observedin anypatient. There was one patient in the RUCONEST group that experienced aseriousadverse event (abdominal hernia at Day 79) that was assessed by theinvestigatoras not related to the study drug.

"These positive results are consistent with the efficacy data previouslyreported from two smaller randomized, controlled clinical studies withRUCONESTin patients with HAE, and we believe the results provide strong support forourproposed dosing regimen of 50 U/kg in treating acute attacks of HAE," saidWendell Wierenga, Ph.D., executive vice president of research anddevelopment ofSantarus.

"We are very pleased with these pivotal study results and look forward toworking with our colleagues at Santarus to prepare and submit the BiologicsLicense Application (BLA) for RUCONEST to the FDA in the first half of2013,"said Bruno Giannetti, M.D., Ph.D., chief operations officer of Pharming."Weanticipate that additional data from this Phase III study will be presentedatan appropriate medical meeting in 2013."

Santarus licensed exclusive rights to commercialize RUCONEST in NorthAmericafor the treatment of acute attacks of HAE as well as other potential futureindications from Pharming. Under the terms of the license agreement, a $10million milestone is now payable to Pharming as a result of the successfulachievement of the primary endpoint of the Phase III clinical study. Anadditional $5 million milestone will be payable to Pharming upon U.S. FoodandDrug Administration (FDA) acceptance of the BLA for review.

RUCONEST Phase III Clinical Study (Study 1310)

Pharming conducted Study 1310 with RUCONEST under a Special ProtocolAssessment(SPA) agreement with the FDA, and the study is intended to support thesubmission of a BLA in the U.S.

* In Study 1310, RUCONEST was evaluated for the treatment of acuteattacks of angioedema in patients with HAE in an international, multicenter, randomized, placebo-controlled Phase III study comparing a single IV infusion of 50 U/kg of RUCONEST to a saline control with a primaryendpoint of time to beginning of symptom relief.

* The time to beginning of symptom relief was defined as the time lapsedfrom the beginning of the infusion of study medication to the beginning of a persistent beneficial effect based on the patient's responses to aTreatment Effect Questionnaire for the primary attack location.

* A 90 day follow-up period was required for each patient enrolled, oruntil such time that the patient required open-label treatment for asubsequent attack during the 90 day period. The data from the open-labelextension are being collected for additional efficacy and safety analyses.

* The study enrolled a total of 75 patients who were randomized 3:2 toreceive either RUCONEST or saline.

About RUCONEST and Hereditary Angioedema

RUCONEST (INN conestat alfa) is a recombinant version of the human proteinC1esterase inhibitor, and is produced with Pharming's proprietary technologywhichuses milk from transgenic rabbits. RUCONEST is approved in Europe for thetreatment of acute angioedema attacks in patients with HAE, a geneticdisorderin which the patient is deficient in or lacks a functional plasma proteinC1esterase inhibitor, resulting in unpredictable and debilitating episodes ofintense swelling. The swelling may occur in one or more anatomical areas,including the extremities, face, trunk, genitals, abdomen and upper airway.Thefrequency and severity of HAE attacks vary and are most serious when theyinvolve laryngeal edema, which can close the upper airway and cause deathbyasphyxiation. According to the U.S. Hereditary Angioedema Association,epidemiological estimates for HAE range from one in 10,000 to one in 50,000individuals. RUCONEST is an investigational drug in the U.S. and has beengranted orphan drug designation by the FDA both for the treatment of acuteattacks of HAE and for prophylactic treatment of HAE.

About Pharming Group NV

Pharming Group NV develops innovative products for the treatment of unmetmedical needs. RUCONEST® is a recombinant human C1 esterase inhibitorapprovedfor the treatment of angioedema attacks in patients with HAE in all 27 EUcountries plus Norway, Iceland and Liechtenstein, and is distributed in theEUby Swedish Orphan Biovitrum (OMX: SOBI). The product is also beingevaluated forvarious follow-on indications. Pharming's advanced technologies includeinnovative and validated platforms for the production of proteintherapeutics,technology and processes for the purification and formulation of theseproducts.A feasibility study, using the validated transgenic rabbit platform, aimedatthe development of recombinant Factor VIII for the treatment of HaemophiliaA isunderway with partner, Renova Life, Inc. Additional information isavailable onthe Pharming website,

About Santarus

Santarus, Inc. is a specialty biopharmaceutical company focused onacquiring,developing and commercializing proprietary products that address the needsofpatients treated by physician specialists. The company's currentcommercialefforts are focused on GLUMETZA® (metformin hydrochloride extendedreleasetablets) and CYCLOSET® (bromocriptine mesylate) tablets, which areindicatedas adjuncts to diet and exercise to improve glycemic control in adults withtype2 diabetes, and on FENOGLIDE® (fenofibrate) tablets, which isindicated as anadjunct to diet to reduce high cholesterol. Santarus also sellsZEGERID®(omeprazole/sodium bicarbonate), which is indicated for the treatment ofcertaingastrointestinal diseases and disorders.

Santarus has a diverse product development pipeline. A New DrugApplication forUCERIS™ (budesonide) for induction of remission of active mild tomoderateulcerative colitis is under review by the U.S. Food and Drug Administrationwitha response expected in January 2013. The pipeline also includes twolate-stageinvestigational drugs in Phase III clinical studies: RUCONEST®(recombinanthuman C1 esterase inhibitor) for treatment of acute attacks of hereditaryangioedema and rifamycin SV MMX® for treatment of travelers' diarrhea.Inaddition, the company's investigational monoclonal antibody, SAN-300, isbeingevaluated in a Phase I clinical program. More information about Santarusisavailable at

Santarus and Pharming caution you that statements included in this pressreleasethat are not a description of historical facts are forward-lookingstatements.The inclusion of forward-looking statements should not be regarded as arepresentation by Santarus or Pharming that any of its plans or objectiveswillbe achieved. Actual results may differ materially from those set forth inthisrelease due to the risks and uncertainties inherent in Santarus andPharming'sbusinesses, including, without limitation: risks related to the timing forsubmission of the BLA and whether the FDA will accept the BLA for reviewfollowing submission and ultimately approve it; whether the FDA will concurwiththe clinical interpretation of the Phase III study results or the conductof thestudy; whether the FDA ultimately will require additional clinical studiesorother development programs before accepting the RUCONEST BLA or approvingRUCONEST; risks related to Santarus' dependence on Pharming for manyfunctionsrelated to RUCONEST, and Pharming's ability to continue to perform thesefunctions based on its limited financial resources; risks related to thelicenseand supply arrangements between Santarus and Pharming, including thepotentialfor termination of the arrangements; other difficulties or delays indevelopment, testing, manufacturing and marketing of, and obtaining andmaintaining regulatory approvals for, Santarus' and Pharming's products;andother risks detailed in prior press releases as well as in public periodicfilings with the Securities and Exchange Commission.

You are cautioned not to place undue reliance on these forward-lookingstatements, which speak only as of the date hereof. All forward-lookingstatements are qualified in their entirety by this cautionary statement andneither Santarus nor Pharming undertakes any obligation to revise or updatethisnews release to reflect events or circumstances after the date hereof.Thiscaution is made under the safe harbor provisions of Section 21E of thePrivateSecurities Litigation Reform Act of 1995.

Santarus®, FENOGLIDE®, UCERIS™, and ZEGERID® aretrademarks ofSantarus, Inc. GLUMETZA® is a trademark of Biovail LaboratoriesInternationalS.r.l. licensed exclusively in the United States to Depomed, Inc.CYCLOSET®is a trademark of VeroScience LLC. MMX® is a trademark of CosmoTechnologiesLimited. RUCONEST® is a trademark of Pharming Group N.V.

Press release (PDF):

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Source: Pharming Group N.V. via Thomson Reuters ONE


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