Press Release Source: Pharming Group N.V. on Tuesday December 28, 2010, 8:13 am EST
LEIDEN, NETHERLANDS–(Marketwire – 12/28/10) – Biotech companyPharming Group NV (NYSE Euronext: PHARM) and specialty biopharmaceuticalcompanySantarus, inc (NASDAQ:SNTS – News) today announced the submission of aBiologicsLicense Application (BLA) to the US Food and Drug Administration (FDA) toobtainmarketing approval for Rhucin(®) (recombinant human C1inhibitor) for thetreatment of acute angioedema attacks in patients with HereditaryAngioedema(HAE).
The safety and efficacy of Rhucin for the treatment of HAE attackswereevaluated in two randomized placebo-controlled studies and are supported byfouropen label treatment studies. Both placebo-controlled clinical studiesshowedstatistically significant and clinically relevant improvement in theprimaryendpoint of time to beginning of relief of symptoms at Rhucin dosagestrengthsof 50 U/kg and 100 U/kg compared to placebo. In October 2010, PharmingreceivedMarketing Authorization for Ruconest(™ )(Rhucin in non-Europeancountries)for the treatment of acute HAE in the European Union. Pharming hasupdated theclinical dataset reviewed and approved by the European Medicines Agencywithadditional patient data and analyses. In total, the BLA dossier includesnineclinical studies covering 714 administrations in 190 subjects.
Santarus has licensed certain exclusive rights from Pharming tocommercializeRhucin in North America for the treatment of acute attacks of HAE andotherfuture indications. under the terms of the license agreement, a $5millionmilestone is payable to Pharming upon FDA acceptance for review of theBLA forRhucin.
About Rhucin (Ruconest in European countries) and Hereditary Angioedema
Rhucin (INN conestat alfa) is a recombinant version of the human protein C1inhibitor (C1INH). Rhucin is produced through Pharming’s proprietarytechnologyin milk of transgenic rabbits and in Europe is approved under the nameRuconestfor treatment of acute angioedema attacks in patients with HAE. The FDA hasgranted Orphan Drug and Fast Track Status to Rhucin for the treatment ofacuteattacks of HAE, a genetic disorder in which the patient is deficient in orlacksa functional plasma protein C1 inhibitor, resulting in unpredictable anddebilitating episodes of intense swelling of the extremities, face, trunk,genitals, abdomen and upper airway. The frequency and severity of HAEattacksvary and are most serious when they involve laryngeal edema, which canclose theupper airway and cause death by asphyxiation. According to the USHereditaryAngioedema Association, epidemiological estimates for HAE range from one in10,000 to one in 50,000 individuals. Based on prior discussions with theFDA,Pharming is planning to initiate an additional randomizedplacebo-controlled, clinical study with Rhucin in approximately 50 patientsto provide additionaldata in support of the 50 U/kg dose. Data from the placebo-controlledstudywill also be used to provide additional validation of the visual analogscaleused in measuring the clinical effects of Rhucin.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment ofunmetmedical needs. Ruconest™ (Rhucin(®) in non-European territories) isarecombinant human C1 inhibitor approved for the treatment of angioedemaattacksin patients with HAE in all 27 EU countries plus Norway, Iceland andLiechtenstein. The product is also under development for follow-onindications,i.e. antibody-mediated rejection (AMR) and delayed graft function (DGF)following kidney transplantation. Pharming’s advanced technologies includeinnovative platforms for the production of protein therapeutics, technologyandprocesses for the purification and formulation of these products.Additionalinformation is available on the Pharming website, pharming.com.
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. CYCLOSET was commercially launched in November 2010.
Santarus also has a diverse development pipeline with three late-stageproductcandidates in Phase III clinical programs: ULTESA™ (budesonideMMX(®)) forinduction of remission of active ulcerative colitis, rifamycin SVMMX(®) fortreatment of travelers’ diarrhea and RHUCIN(®) (recombinant human C1inhibitor)for treatment of acute attacks of hereditary angioedema. In addition,Santarusplans to initiate a Phase I clinical study in the first half of 2011 withSAN-300, its anti-VLA-1 antibody, which the company expects toinvestigate for thetreatment of rheumatoid arthritis. more information about Santarus isavailableon the company’s website at santarus.com.
Pharming and Santarus caution you that statements included in this pressreleasethat are not a description of historical facts are forward-lookingstatements.These forward-looking statements include statements regarding thepotentialattributes of the RHUCIN (recombinant human C1 inhibitor) developmentproductand its potential to treat HAE or other indications. The inclusion offorward-looking statements should not be regarded as a representationby Pharming orSantarus that any of its plans or objectives will be achieved. Actualresultsmay differ materially from those set forth in this release due to therisks anduncertainties inherent in Pharming’s and Santarus’ businesses,including,without limitation: whether the FDA accepts the BLA submission andapprovesRHUCIN in a timely manner or at all; risks related to the timing andsuccess ofplanned development programs for RHUCIN, including the planned clinicalstudydesigned to provide additional data in support of the 50 U/kg dose;Santarus’ability to generate market demand and sales of RHUCIN, if approved;competitionfrom other products, unexpected adverse side effects or inadequatetherapeuticefficacy of RHUCIN; the ability to ensure continued supply of RHUCIN; thescopeand validity of patent protection or other regulatory exclusivity forRHUCIN;risks related to the license and supply arrangements betweenPharming andSantarus, including the potential for termination of the arrangements;otherdifficulties or delays in development, testing, manufacturing andmarketing of,and obtaining and maintaining regulatory approvals for, Pharming’s andSantarus’products; and other risks detailed in prior press releases as well as inpublicperiodic filings with the Securities and Exchange Commission.
You are cautioned not to place undue reliance on theseforward-looking statements, which speak only as of the date hereof.All forward-looking statements are qualified in their entirety by thiscautionary statement andneither Pharming nor Santarus 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(®) and ULTESA(™) are trademarks of Santarus, inc.GLUMETZA(®) is aregistered trademark of Biovail Laboratories International S.r.l.licensedexclusively in the United States to Depomed, inc. CYCLOSET(®) is aregisteredtrademark of VeroScience LLC. MMX(®) is a registeredtrademark of CosmoTechnologies Limited. RHUCIN(®) and RUCONEST™ are trademarksof PharmingGroup NV.
Press release (PDF):hugin.info/132866/R/1475839/411934.pdf
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Source: Pharming Group N.V. via Thomson Reuters ONE