Amsterdam Molecular Therapeutics Receives Further Opinion on Glybera® Marketing Authorisation Application

AMSTERDAM, The Netherlands, April 23, 2012 /PRNewswire/ --

Amsterdam Molecular Therapeutics (AMT) Holding N.V. (in liquidatie) (Euronext: AMT), a leader in the field of human gene therapy, has received notice that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has maintained its earlier opinion that Glybera (alipogene tiparvovec) is not approvable at this time.

The decision was reached contrary to the majority of votes cast by the CHMP itself, which voted 16 - 15 in favor of approving Glybera under exceptional circumstances. It is also contrary to the earlier positive advice of the Committee for Advanced Therapies (CAT), a committee specifically implemented to provide expert guidance to the CHMP on advanced therapeutics such as gene and cell therapies. In addition, the Scientific Advisory Group (SAG), an expert panel specifically selected to evaluate clinical results and the science of the product, also recommended that Glybera should be approved under exceptional circumstances.

The SAG and the CAT, after extensive review and analysis of data (including interviews with the investigator, the leading clinicians and patients), concluded that Glybera demonstrated meaningful evidence of clinical efficacy, without any major safety concerns. However, members of the CHMP are not bound to follow the advice of the experts from CAT and SAG.

For a drug to receive a positive opinion the rules require an absolute majority from all CHMP members, including those who do not participate in the meeting and the vote. The CHMP is composed of 32 voting members, and at least 17 positive votes are required. Accordingly, this vote of 16 - 15 in favor of Glybera represents a negative opinion. Members absent at the time of the vote cannot vote by proxy or retrospectively.

The final step in the procedure is now with the European Commission to confirm, or not, the CHMP's opinion.

"We find it hard to explain to LPLD patients who suffer from extremely painful, recurring, potentially lethal pancreatitis attacks, and for whom Glybera is the only therapeutic option, that they will now be deprived of a treatment when a great majority of the scientific experts involved recommended Glybera for approval and the majority of the CHMP voting members shared the experts' view," said Jörn Aldag, liquidator of AMT (and CEO of uniQure B.V., its successor company). "While we are deeply disappointed with the current outcome on Glybera, we are encouraged by the validation provided by the majority verdict of the CHMP's voting members, the Scientific Advisory Group and the Committee for Advanced Therapies regarding our gene therapy platform, which indicates that neither safety nor GMP quality are an issue."

AMT has recently transferred its gene therapy assets to a new company, uniQure B.V., which will continue its focus on strategic advancement of the promising projects in its development pipeline.

About Glybera^®

AMT has developed Glybera as a treatment for patients with the genetic disorder lipoprotein lipase deficiency. LPLD is an orphan disease for which no treatment exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent activity of LPL protein in patients. This protein is needed in order to break down large fat-carrying particles that circulate in the blood after each meal. When such particles, called chylomicrons, accumulate in the blood, they may obstruct small blood vessels. Excess chylomicrons result in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating and life-threatening clinical complication of LPLD. Glybera^® has orphan drug status in the EU and US. The EMA was evaluating Glybera for treatment of patients with a history of severe or recurring pancreatitis.

About AMT

More information on AMT's dissolution and liquidation process and delisting and the timing thereof can be found in the overview of the key transaction elements and the transaction timetable that is available via AMT's website - http://www.amtbiopharma.com

About uniQure B.V.

uniQure develops human gene based therapies. In addition to Glybera^®, uniQure has a product pipeline of gene therapy products in development for hemophilia B, Parkinson's disease, acute intermittent porphyria and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has the ability to design, validate and manufacture AAV vectors. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene. uniQure acquired all its gene therapy assets from Amsterdam Molecular Therapeutics and was founded in 2012 in Amsterdam by Forbion Capital Partners. (http://www.uniQure.nl)

Certain statements in this press release are "forward-looking statements" including those that refer to management's plans and expectations for future operations, prospects and financial condition. Words such as "strategy," "expects," "plans," "anticipates," "believes," "will," "continues," "estimates," "intends," "projects," "goals," "targets" and other words of similar meaning are intended to identify such forward-looking statements. Such statements are based on the current expectations of the management of AMT only. Undue reliance should not be placed on these statements because, by their nature, they are subject to known and unknown risks and can be affected by factors that are beyond the control of AMT.

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