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Start of Phase I/II Gene Therapy Clinical Trial for Hemophilia B

Thursday, March 11, 2010 General News J E 4
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AMSTERDAM, March 10, 2010 Amsterdam MolecularTherapeutics (Euronext: AMT), a leader in the field of human gene therapy,announced today that the first patient has been dosed in the Phase I/IIexploratory clinical trial with a gene therapy product for hemophilia B, aseriously debilitating and potentially lethal disease.

The trial is an open label dose-escalation study using a vector-genecombination developed at the renowned St. Jude Children's Research Hospital.Dr. Arthur W. Nienhuis of St. Jude is the principal investigator of theon-going trial. The work was initiated at St. Jude more than a decade ago byDrs. Andrew Davidoff and Amit Nathwani and the collaboration has continuedfollowing Dr. Nathwani's return to London. The collaboration involves St.Jude and University College London and other institutions in the US andBritain. The objective of the trial is to assess the safety and efficacy ofdifferent doses of hemophilia B gene therapy. Hemophilia B is an inheritedcondition in which patients may have repeated and sometimes life threateningbleeds after accidental trauma or medical interventions, because they do nothave sufficient functioning of an essential blood clotting factor, calledFactor IX.

AMT will build on the outcome of this exploratory trial and is preparingfor additional clinical development to establish safety, tolerability andproof-of-concept with Factor IX gene therapy produced using AMT'sproprietary, clinically validated production system. AMT has the exclusivecommercialization rights to the Factor IX gene used in the St. Jude trial andhas the ability to produce gene therapy product for hemophilia B at highquality on a commercial scale. Additional developmental work using AMT'sproduction system is on-going at St. Jude with AMT support.

Jorn Aldag CEO of AMT said: "Dr. Andrew Davidoff and his group at St.Jude, together with Professor Nathwani in London, have done very importantscientific work on hemophilia B. We are really looking forward to the resultsof the trial for continuing our collaboration, aiming for a real cure forpatients with this bleeding disorder. Use of the Factor IX gene fitsperfectly with AMT's proprietary gene therapy platform and our businessstrategy of developing cures for seriously debilitating orphan diseases. "

This hemophilia B gene therapy, administered once, will introduce thefunctional gene for the Factor IX protein into the patient's liver cells withthe goal to restore blood clotting functionality long-term. In pre-clinicalstudies, Factor IX gene therapy resulted in long-term production of Factor IXprotein at a therapeutically significant level after a single administration.If this approach is successful, the long term efficacy of one timeadministered hemophilia B gene therapy is expected to be perceived as asignificant advance over the current regular dosing of recombinant Factor IX.In addition, the efficacy profile of this gene therapy is anticipated toexceed that of current therapy, as the gene therapy should lead to stableFactor IX levels whereas current recombinant protein treatment causes peaksand troughs. It is hoped that hemophilia B gene therapy, therefore, canpotentially replace all recombinant Factor IX products.

The UK Medicines and Healthcare products Regulatory Agency as well as theUS Food and Drug Administration have approved the current trial.

About the Disease

Hemophilia B is characterized by severe episodes of external and internalbleeding, resulting in significant morbidity. The episodes cause long-termdamage, for instance to the joints, and may be fatal if they occur in thebrain. The defect in blood clotting in hemophilia B is caused by the absenceof functional clotting Factor IX as a result of mutations in the geneencoding this protein. The factor IX gene is located on the X chromosome. Itis an X-linked recessive trait, which explains why only males are usuallyaffected.

Hemophilia B is a rare disease, occurring in 1 in 30,000 people, almostalways in males. The total number of patients in Europe and the USA togetheris estimated to be between 35,000 and 40,000.

Currently, frequent intravenous administrations of recombinant Factor IXare required to stop or prevent bleeding. Protein replacement therapy iscostly, cumbersome, and does not completely prevent bleeding.

About Amsterdam Molecular Therapeutics

AMT, founded in 1998 and based in Amsterdam, is a leader in thedevelopment of human gene based therapies. Using AAV as the delivery vehicleof choice for therapeutic genes, the company has been able to design andvalidate what is probably the first stable and scalable AAV productionplatform. This safe and efficacious proprietary platform offers a uniquemanufacturing capability which 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, DMD, Acute Intermittent Porphyria and Parkinson's Disease atdifferent stages of research or development.

For information

AMT will be presenting at Bio Europe Spring 2010 Conference at the CentreConvencions Internacional, Barcelona, at 1200 (CET) on Wednesday March 10,2010.

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.

SOURCE Amsterdam Molecular Therapeutics B.V
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