- Glybera(R) Clinical Data Presented at Meeting of American HeartAssociation
Amsterdam Molecular Therapeutics (EuroNext Amsterdam: AMT), a leader inthe field of human gene therapy, today provides its non-audited businessupdate in compliance with the EU transparency directive. This reportsummarizes material events and AMT's financial position for the third quarterof 2009.
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Jorn Aldag appointed as Chief Executive Officer
On September 24, 2009 AMT announced that Jorn Aldag, previously Presidentand CEO of Evotec AG, Germany, would join AMT as Chief Executive Officer asof October 05, 2009. Jorn Aldag holds business degrees from the EuropeanBusiness School and the Harvard Business School. He remains Chairman ofMolecular Partners AG, Switzerland, a privately-held biotech company focusedon the development of its proprietary DARPin scaffold technology and itsproprietary pipeline. Mr. Aldag's appointment was approved by AMT'sExtraordinary General Meeting of shareholders on November 4, 2009.
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At the same time, Prof. Sander van Deventer stepped down as interim CEO.He will continue to contribute his expertise and experience to AMT asChairman of the Scientific Advisory Board and Business Consultant.
Business Update
AMT's cash position(*) on September 30, 2009 amounts to EUR21.4 millioncompared to EUR25.0 million on June 30, 2009. The cash outflow in the thirdquarter of 2009, amounting to EUR3.6 million mainly represented operationalcash flow and is well within the guidance for this period. The Company isadjusting its guidance for the year-end cash balance to approximately EUR17million. AMT employed 88 persons as of September 30, 2009. Total expenses inthe third quarter of 2009 were EUR4.4 million compared to EUR4.7 million inthe same period last year.
(*) The Company's cash position is composed of cash and cash equivalents.
Material events after September 30, 2009
On November 11, 2009 AMT announced that it has successfully treatedDuchenne muscular dystrophy (DMD) in an animal model with its proprietarygene therapy. The proof of concept studies were performed in collaborationwith the group of Professor Irene Bozzoni (University of Rome, La Sapienza,Italy) and demonstrated efficacy in the heart as well as in skeletal muscles.In a previous study, AMT's gene therapy approach was shown to be successfulin the treatment of diseased human muscle cells obtained from biopsies of DMDpatients. These data establish a robust basis for AMT's therapeutic approachto DMD.
The ongoing trial with Glybera(R) for lipoprotein lipase deficient (LPLD)patients in Canada was closed for patient recruitment on October 30, 2009with 5 patients dosed. AMT is well on track to file for regulatory approvalfor Glybera(R) within the next three months. The main aim of the ongoingtrial is to gain further insight in the mechanism of action of Glybera(R),and data obtained from this trial will further strengthen the Glybera(R) datapackage that will be filed with the EMEA.
On November 17, 2009, updated safety and efficacy data on Glybera(R) fromthe first two clinical studies was presented at the meeting of the AmericanHeart Association in Orlando. These data confirm the sizeable decrease inpancreatitis incidence after therapy with Glybera(R) and confirm itsexcellent safety profile.
On October 29, 2009, AMT and Progenika Biopharma announced that they haveentered into a development and commercialization agreement for LPLchipTM, adiagnostic tool to rapidly diagnose patients with complete and partiallipoprotein lipase deficiency (LPLD).
On October 8, 2009 AMT received Orphan Drug Designation for its treatmentfor Duchenne Muscular Dystrophy (DMD).
Prospects
The Company remains on track to file for regulatory approval of its leadproduct Glybera(R) within the next three months. This gene therapy is tocontrol LPLD, a serious disease often complicated by potentially lifethreatening pancreatitis incidents.
AMT will continue to develop its own technology platform and exploit itsadvantages in AAV gene therapy by focusing its preclinical development on 4projects: Hemophilia B, Duchenne Muscular Dystrophy (DMD), Acute IntermittentPorphyria (AIP) and Parkinson's Disease. Other projects previously shown inthe Company's pipeline will be postponed or discontinued to control theCompany's cash burn.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that appears to circumvent many ifnot all of the obstacles that have prevented gene therapy from becoming amainstay of clinical medicine. Using adeno-associated viral (AAV) vectors asthe delivery vehicle of choice for therapeutic genes, the company has beenable to design and validate what is probably the first stable and scalableAAV production platform. As such, AMT's proprietary platform holds tremendouspromise for thousands of rare (orphan) diseases, especially diseases that arecaused by one faulty gene. Currently, AMT has a pipeline with severalAAV-based gene therapy products at different stages of research ordevelopment.
Certain statements in this press release are "forward-looking statements"including those that refer to management's plans and expectations for futureoperations, prospects and financial condition. Words such as "strategy,""expects," "plans," "anticipates," "believes," "will," "continues,""estimates," "intends," "projects," "goals," "targets" and other words ofsimilar meaning are intended to identify such forward-looking statements.Such statements are based on the current expectations of the management ofAmsterdam Molecular Therapeutics only. Undue reliance should not be placed onthese statements because, by their nature, they are subject to known andunknown risks and can be affected by factors that are beyond the control ofAMT. Actual results could differ materially from current expectations due toa number of factors and uncertainties affecting AMT's business, including,but not limited to, the timely commencement and success of AMT's clinicaltrials and research endeavors, delays in receiving U.S. Food and DrugAdministration or other regulatory approvals (i.e. EMEA, Health Canada),market acceptance of AMT's products, effectiveness of AMT's marketing andsales efforts, development of competing therapies and/or technologies, theterms of any future strategic alliances, the need for additional capital, theinability to obtain, or meet, conditions imposed for required governmentaland regulatory approvals and consents. AMT expressly disclaims any intent orobligation to update these forward-looking statements except as required bylaw. For a more detailed description of the risk factors and uncertaintiesaffecting AMT, refer to the prospectus of AMT's initial public offering onJune 20, 2007, and AMT's public announcements made from time to time.Q3 2009 Highlights - Jorn Aldag joins AMT as Chief Executive Officer - Glybera(R) remains on track for filing for regulatory approval in next three months
SOURCE Amsterdam Molecular Therapeutics B.V
