Orphan Drug Designation Granted by the U.S. FDA for Ceflatonin(R)
Posted by rob on March 20, 2006 under Uncategorized | 
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MELBOURNE, Australia and MENLO PARK, Calif., March 19 /PRNewswire-FirstCall/ -- ChemGenex Pharmaceuticals Limited (Nasdaq: CXSP; ASX: CXS)announced today that the United States Food and Drug Administration (FDA) hasgranted Orphan Drug status for the company's most advanced drug, Ceflatonin,for the treatment of chronic myeloid leukemia (CML). Ceflatonin is the firstof a new class of novel drugs that induces apoptosis (programmed cell death)and inhibits angiogenesis (new blood vessel formation). Ongoing or soon to beinitiated clinical trials are and will be evaluating Ceflatonin in a broadrange of conditions, including: chronic myeloid leukemia (CML),myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Orphan drug status is granted by the FDA to promote the development ofdrugs for diseases affecting less than 200,000 people in the United States.Orphan drug status entitles ChemGenex to seven years of market exclusivity forthe use of Ceflatonin in the treatment of CML; protocol assistance by the FDAto optimize drug development in the preparation of a dossier that will meetregulatory requirements; and reduced fees associated with applying for marketapproval. A similar designation for Ceflatonin in the European market, with similaradvantages, was granted by the European Union on September 9, 2004, based on apositive recommendation by the Committee for Orphan Medicinal Products (COMP)of the European Medicines Agency (EMEA). The European Union granted Ceflatoninorphan status on October 20, 2004 for the treatment of acute myeloid leukemia(AML). "The decision by the U.S. FDA to grant Ceflatonin orphan drug status forCML is further incentive to move Ceflatonin as quickly as possible through theclinical development and regulatory approval process worldwide," said GregCollier, ChemGenex chief executive officer and managing director. "We are veryencouraged by the growing body of clinical data on Ceflatonin in resistantCML, MDS and AML, and by the ongoing support of key opinion leaders in thefield. Now with the granting of Orphan Drug status by the FDA, we are fullycommitted to accelerate our development efforts to help address the unmetmedical needs of CML patients worldwide." About Ceflatonin(R) Ceflatonin (HHT) is a potent inducer of apoptosis (programmed cell death)in myeloid cells and inhibits angiogenesis (blood vessel formation).Ceflatonin has confirmed Phase 2 clinical activity in resistant CML, both as asingle agent and in combination with other approved drugs. ChemGenex isdeveloping Ceflatonin for the treatment of CML, myelodysplastic syndrome (MDS)and acute myeloid leukemia (AML). Recent studies have demonstrated that Ceflatonin has potential in CMLpatients who are developing resistance to tyrosine kinase inhibitor (TKI)therapy, including Gleevec(R), and in-combination with TKI's in early stageCML to increase the cytogenetic and molecular response rate. A summary of recent clinical data on Ceflatonin includes the followingstudies: * In a nine patient single-agent Phase 1/2 study in accelerated-phase CML who had become resistant to Gleevec, seven (80%) patients returned to chronic-phase and 6 (67%) achieved a complete hematologic response. * In a separate five patient single-agent Phase 2 study in late chronic-phase CML, five (100%) achieved a complete hematologic response and two (40%) achieved a cytogenetic response. Two patients with detectable resistance mutations achieved a complete hematologic response and their mutations (p-loop) were not longer detectable after Ceflatonin treatment. * In another recent 10 patient Phase 1/2 study, Ceflatonin was added to Gleevec in patients who had a partial or complete cytogenetic response, in order to reduce bcr-abl transcript counts further. Seven (70%) patients had a significant decrease in bcr-abl counts. Two patients with partial cytogenetic responses achieved complete cytogenetic responses. Two patients achieved a complete molecular response, or not detectable evidence of residual disease. * Pre-clinical studies on Ceflatonin have shown both additive and synergistic activity when combined with Gleevec, as measured by a reduction in the marker (bcr-abl protein expression) that is a hallmark of resistance in CML. A multinational Phase 2/3 study in CML patients harboring the T315I pointmutation, known to confer complete resistance to tyrosine kinase (TKI)inhibitors like Gleevec, is scheduled to begin in Q2, 2006. A secondmultinational Phase 2/3 study in CML patients resistant to TKI inhibitors isscheduled to begin soon thereafter. Each study will include centers in theUnited States, Germany, France and Italy. Ceflatonin is not approved by the FDA as a treatment in any indication andis currently being evaluated in clinical trials for efficacy and safety forfuture regulatory applications. Gleevec(R) is a registered trademark of the Novartis PharmaceuticalsCorporation. About Chronic Myeloid Leukemia Chronic Myeloid Leukemia (CML) is a cancer of the blood cells caused by anacquired genetic defect called the bcr-abl mutation. This defect occurs whengenetic material from two chromosomes (9 and 22) swaps places, creating theso-called Philadelphia chromosome. The bcr-abl mutation interferes with normalcell replication processes, leading to an abnormal proliferation of whiteblood cells. CML usually occurs in adults and typically progresses through threephases. Patients generally are diagnosed in 'chronic phase', progress throughan 'accelerated phase' and then may die if the disease progresses to 'blastphase'. CML incidence is relatively consistent occurring at about 1 to 2 per100,000 people and the global CML market for therapeutics is estimated at inexcess of US$2 billion. Patients with chronic phase CML have been effectively treated in recentyears by the drug Gleevec. However, over time many patients become resistantto the therapeutic effects of the drug and the disease progresses. About ChemGenex Pharmaceuticals Limited (http://www.chemgenex.com) ChemGenex Pharmaceuticals is a pharmaceutical development companydedicated to improving the lives of patients by developing therapeutics in theareas of oncology, diabetes, obesity, and depression. ChemGenex harnesses thepower of genomics for target discovery and validation, and in clinical trialsto develop more individualized therapeutic outcomes. ChemGenex's leadcompound, Ceflatonin(R), is currently in phase 2/3 clinical trials forleukemia and Quinamed(R) is in phase 2 clinical trials for prostate, breastand ovarian cancers. The company has a significant portfolio of anti-cancer,diabetes, obesity and depression programs, several of which have beenpartnered with international pharmaceutical companies. ChemGenex currentlytrades on the Australian Stock Exchange under the symbol "CXS" and on NASDAQunder the symbol "CXSP". Safe Harbor Statement Certain statements made herein that use the words "estimate," "project,""intend," "expect," "believe," and similar expressions are intended toidentify forward-looking statements within the meaning of the US PrivateSecurities Litigation Reform Act of 1995. These forward-looking statementsinvolve known and unknown risks and uncertainties which could cause the actualresults, performance or achievements of the company to be materially differentfrom those which may be expressed or implied by such statements, including,among others, risks or uncertainties associated with the development of thecompany's technology, the ability to successfully market products in theclinical pipeline, the ability to advance promising therapeutics throughclinical trials, the ability to establish our fully integrated technologies,the ability to enter into additional collaborations and strategic alliancesand expand current collaborations and obtain milestone payments, thesuitability of internally discovered genes for drug development , the abilityof the company to meet its financial requirements, the ability of the companyto protect its proprietary technology, potential limitations on the company'stechnology, the market for the company's products, government regulation inAustralia and the United States, changes in tax and other laws, changes incompetition and the loss of key personnel. These statements are based on ourmanagement's current expectations and are subject to a number of uncertaintiesthat could change the results described in the forward-looking statements.Investors should be aware that there are no assurances that results will notdiffer from those projected.
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Orphan Drug Designation Granted by the U.S. FDA for Ceflatonin(R)