DNage initiates observational study in children suffering from
Cockayne Syndrome
(Thomson Reuters ONE) - Leiden, The Netherlands, September 16, 2009. Biotech companyPharming Group NV ("Pharming" or "the Company") (NYSE Euronext:PHARM) announced today that its wholly-owned subsidiary DNage hasinitiated an observational study in children suffering from CockayneSyndrome (CS).This study is designed to assess the natural (untreated) progressionof the disease during a period of 6-12 months and will includedetailed data collected from existing medical records as wellprospective measurements and assessments. Data collected in theobservational study will enable the Company to conduct one or moreclinical studies to assess the efficacy of Prodarsan®, a product indevelopment for the treatment of CS. Although the final design ofthese planned international efficacy studies is not yet completed andalso dependent on the outcome of discussions with regulatoryauthorities, it is likely that in these studies Prodarsan® will betested, amongst other parameters, for its effect on growth ofpaediatric patients compared to their growth when untreated.DNage is developing Prodarsan® as a potential therapy for CS. Theproduct is a combination of small molecules formulated formulated asan oral liquid and is believed to reduce the accumulation ofDNA-damage, the underlying biochemical cause of Cockayne Syndrome.Prodarsan® has been successfully tested in animal models of CS andappeared to be safe and well-tolerated in a Phase I study in healthyhuman volunteers. Prodarsan® has an Orphan Drug Designation from theUS Food and Drug Administration (FDA) who recently also approved anInvestigational New Drug (IND) application allowing the Company toconduct a clinical trial in children suffering from CS.The Principal Investigator of the observational study, Dr. Edward G.Neilan, Staff Physician at the Children's Hospital Boston/HarvardMedical School, commented: "The initiation of this observationalstudy is an important milestone in the development of new treatmentoptions for CS-patients and in particular for the development ofProdarsan®. For the first time, we will be collecting detailedmeasurements of the natural progression of this disease. This studywill hopefully provide us with data needed to complete the design ofa pivotal clinical study necessary to achieve registration ofProdarsan® as a therapeutic. While our center in Boston will serve asa central site in the current study, I look forward to collaboratewith many colleagues in the US and abroad to make this study asuccess."About Cockayne SyndromeCS is a premature ageing disease. Premature ageing diseases are agroup of rare diseases caused by a genetic defect leading todeficient repair of DNA-damage. Patients suffering from thesediseases develop multiple 'ageing-pathologies', normally associatedwith old age, early on in their lives. Generally, these patients havea strongly reduced quality of life and reduced life expectancy. CS ischaracterized (amongst other symptoms) by growth failure, mentalretardation, hearing loss, a prematurely aged appearance (progeria)and premature death. The average lifespan of CS patients is 12.5years and quality of life for these patients is seriously impaired.At present, there is neither a cure nor an effective therapyavailable for CS patients. Disease management consists of treatingthe symptoms as they arise and providing assistive devices.About DNage BVDNage is a wholly owned subsidiary of Pharming Group NV since itsacquisition in 2006. DNage is developing products that interfere withthe development of ageing-related pathologies. Its technologyplatform is based on the scientific work by the group of Dr.Hoeijmakers, at the Erasmus Medical Centre in Rotterdam, whoestablished a link between insufficient DNA-repair (accumulation ofDNA-damage) and the development of ageing-related diseases. DNage hasactive programs in the field of neurodegeneration, bone disease andother ageing-related pathologies. Additional information is availableon the Pharming website, http://www.pharming.com and onhttp://www.dnage.nlAbout Pharming Group NVPharming Group NV is developing innovative products for the treatmentof genetic disorders, ageing diseases, specialty products forsurgical indications, intermediates for various applications andnutritional products. Pharming has two products in late stagedevelopment - Rhucin® for Hereditary Angioedema and human Lactoferrinfor use in food products and one product in early stage clinicaldevelopment - Prodarsan® for Cockayne Syndrome. The advancedtechnologies of the Company include innovative platforms for theproduction of protein therapeutics, technology and processes for thepurification and formulation of these products, as well as technologyin the field of DNA repair (via DNage). Additional information isavailable on the Pharming website, http://www.pharming.com.This press release contains forward looking statements that involveknown and unknown risks, uncertainties and other factors, which maycause the actual results, performance or achievements of the Companyto be materially different from the results, performance orachievements expressed or implied by these forward lookingstatements.Contact:Ms. Marjolein van Helmond, Pharming Group NV, T: +31 (0)71 52 47 431or +31 (0)6 109 299 54http://hugin.info/132866/R/1341507/320895.pdfThis announcement was originally distributed by Hugin. The issuer is solely responsible for the content of this announcement.
Datum: 16.09.2009 - 08:00 Uhr
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