Amsterdam Molecular Therapeutics Reports Half-Year Results 2010

Tuesday, August 31, 2010 Corporate News
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AMSTERDAM, The Netherlands, August 31, 2010 Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field ofhuman gene therapy, today reported its results for the first half year of2010.

"We are very pleased with the key milestones we have achieved in thefirst half year of 2010 in the Glybera(R) approval process which seems firmlyon track. Our dialogue with the EMA is very encouraging and we look forwardto the Agency's decision with confidence. We also continue to make progresswith our pipeline, not only on the partnership front for programs such asHemophilia B, but also in our early stage efforts," said Jorn Aldag, chiefexecutive officer of AMT.


Glybera(R) for Lipoprotein Lipase Deficiency (LPLD)

In January 2010, the European Medicines Agency (EMA) commenced review ofAMT's Marketing Authorization Application. In May 2010 AMT received questions(Day 120 questions) regarding the application. In July we met with the EMA,to clarify the questions they raised, enabling AMT to align its responsestrategy. We are now working towards an official response to the EMA Day 120questions, due by the end of 2010.

As of today we remain confident in the approvability of Glybera(R). Ourassessment is based on the following:

Hemophilia B

Further to their 2009 agreement to co-develop a vector-gene combinationfor the treatment of Hemophilia B, AMT and St. Jude Children's ResearchHospital in the USA have successfully transferred Factor IX to AMT'smanufacturing platform and have demonstrated proof of concept in animals in2010. The multicenter, dose escalation study with this vector-genecombination began in March, 2010 at University College London Hospital in theUnited Kingdom guided by Prof. Amit Nathwani. The first patient has beendosed successfully and demonstrated good results both in terms of clinicalbenefit and side effects. Further enrolment of patients is expected in thesecond half of 2010.

Duchenne Muscular Dystrophy

In support of its program to treat Duchenne Muscular Dystrophy, AMTreceived an investment credit from SenterNovem (now, the Dutchgovernment innovation agency, in January 2010. The credit comprises a loancovering 35% of the costs of the project through to 2013 with a maximum ofEUR 4 million. The loan is repayable only if AMT successfully commercializesthe program. AMT has shown proof of concept in a pre-clinical model with itsoptimized construct for exon skipping using its proprietary AAV technology.

Parkinson's Disease

Together with the University of Lund, Sweden, AMT is diligently workingon the preclinical development of a gene therapy for delivery of the GDNFgene to the brain. Efficacy data in an animal model of PD is anticipated tobe available by the end of the current year


Elevated levels of cholesterol are a major risk factor and contributor tothe development of atherosclerosis and cardiovascular disease (CVD). Earlyresearch at AMT demonstrates that after a single intravenous injection of asilencing gene therapy in animal models, the serum cholesterol levels werereduced by 80% with no signs of toxicity. It is therefore reasonable toexpect a similar effect in patients, resulting in reduced risk foratherosclerosis or CVD. Such a long-term, perhaps life-long active genetherapy could eliminate the need for maintenance statin therapy.

Supervisory Board changes

During the period ended June 30, 2010, Alexander Ribbink and GeorgeMorstyn retired from the Supervisory Board and AMT thanks them for theirsubstantial contributions. On April 28, 2010, AMT's co-founder Sander vanDeventer was appointed to the Supervisory Board, and in addition Joseph M.Feczko, Steven H. Holtzman and Francois Meyer were nominated to theSupervisory Board for consideration at the Extraordinary General Meeting tobe held on September 20, 2010.


Results comparison

Total net loss for the period ended June 30, 2010 amounted to EUR 9.4million, in line with the net loss for the period ended June 30, 2009 whichalso amounted to EUR 9.4 million.

The main item within operating costs reflects the investment inGlybera(R) to support the registration process. Development of our DuchenneMuscular Dystrophy program, which is 35% funded by a research credit fromSenterNovem through to completion of a Phase I clinical study continues.Expenditure on our other development projects has been reduced as we areconstrained by our current resources and are focusing on the successfulcompletion of the Glybera registration process. Research and developmentcosts increased to EUR 8.1 million for the period ended June 30, 2010 fromEUR 7.1 million in the same period of 2009. At the same time, general andadministrative costs decreased to EUR 1.8 million in the period ended June30, 2010 from EUR 2.9 million in the same period of 2009.

Net interest income/(cost) decreased to EUR (0.0) million for the periodended June 30, 2010 from EUR 0.5 million in the same period in 2009 as aresult of the Company's decreasing cash balance combined with continuing lowmarket interest rates for deposits.

Cash and cash equivalents amounted to EUR 13.5 million at June 30, 2010,a decrease of EUR 9.1 million compared to EUR 22.6 million at December 31,2009. The decrease in cash and cash equivalents mainly stems from theoperational cash outflow which amounted to EUR 8.9 million for the periodended June 30, 2010 (compared to an operating cash outflow of EUR 9.5 millionfor the period ended June 30, 2009).


The Company's expenditure continues in line with budget. However, as AMThas not yet reached the point of generating significant revenues that couldfund operations we continue to explore additional opportunities for funding,including non-dilutive sources such as grants and/or collaborations withpartners. In addition, AMT is also tracking opportunities for raisingadditional capital in conjunction with its bankers. The outlook for the yearremains unchanged.

Conference call and webcast presentation

AMT will conduct a conference call open to the public today at 3.30 p.m.CET, which will also be webcast. Netherlands dial in: +31(0)20-712-1304; USdial in: +1-718-354-1361; UK dial in: +44(0)20-7138-0821. Confirmation Code:1128246

To listen to the conference call live via the internet, visit theinvestor relations portion of the AMT website at Pleasego to the website 15 minutes prior to the call to register, download andinstall the necessary audio software.

The archived webcast also will be available for replay shortly after theclose of the call.

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based therapies. Usingadeno-associated viral (AAV) derived vectors as the delivery vehicle ofchoice for therapeutic genes, the company has been able to design andvalidate what is probably the first stable and scalable AAV productionplatform. This proprietary platform can be applied to a large number of rare(orphan) diseases that are caused by one faulty gene. Currently, AMT has aproduct pipeline with several AAV-based gene therapy products in LPLD,Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, andParkinson's Disease at different stages of research or development. AMT wasfounded in 1998 and is based in Amsterdam.


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. EMA, 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.Highlights - Glybera(R): - EMA initiated MAA review in 01/2010 - Approval progressing on schedule for decision mid 2011 - Novel biomarker for Glybera(R) activity identified - Hemophilia B: Phase I/II started - Duchenne Muscular Dystrophy: to benefit from EUR 4 million innovation credit - sRNA: silencing gene therapy technology achieves 80% cholesterol reduction - Supervisory Board nominations: 3 new industry professionals slated to join board - Key financial figures in line with guidance - Cash & cash equivalents of EUR 13.5 million at June 30, 2010

SOURCE Amsterdam Molecular Therapeutics B.V

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