Amsterdam Molecular Therapeutics completes advance distribution of uniQure depositary receipts to AMT shareholders

 Amsterdam Molecular Therapeutics (AMT) Holding N.V. in liquidation (in liquidatie) (Euronext: AMT), today announces that the advance distribution of uniQure depositary receipts to AMT shareholders was completed today. It is not expected that will be any further (final) distributions made to AMT shareholders.

 More information on the advance distribution and the further upcoming events and steps in the AMT’s 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 and in AMT’s press releases of 6 and 17 April 2012.

Amsterdam Molecular Therapeutics Receives Further Opinion on Glybera Marketing Authorisation Application

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 geneand 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.

Amsterdam Molecular Therapeutics announces completion of disposal of its operations to uniQure B.V. and its dissolution becoming effective

Amsterdam Molecular Therapeutics (AMT) Holding N.V. in liquidation (in liquidatie) (Euronext: AMT), announces that following the approval of the corporate restructuring and financing transaction by the Extraordinary General Meeting ("EGM") of shareholders that was held on 30 March 2012, completion of the disposal of AMT's entire business and operations to uniQure B.V. has taken place on 5 April 2012. Immediately following the completion of the disposal and consequently as per 5 April 2012, the dissolution of the company as resolved by the EGM has become effective, with Mr. Jörn Aldag and Mr. Piers Morgan being the liquidators that shall liquidate the company's dissolved property.

As consideration for the disposed business and operation, AMT received 31,101,065 depositary receipts (certificaten van aandelen) for (class B) ordinary shares in uniQure B.V. ("uniQure DRs"). Because AMT’s dissolved property consists of the uniQure DRs without it having any known financial liabilities, and the financial condition is expected to justify the same, the liquidators of AMT intend to make an advance liquidation distribution (the "Advance Distribution") to the AMT shareholders by distributing and allocating to each such shareholder one uniQure DR for each AMT share held on the Distribution Record Date.  The Distribution Record Date is expected to be 23 April 2012 and the date on which the Advance Distribution of the uniQure DRs is expected to take place is 26 April 2012.

The Advance Distribution of the uniQure DRs to the AMT shareholders will be settled through the book entry system operated by Euroclear Netherlands, and will result in one uniQure DR being credited in the securities account of each AMT shareholder for each AMT share held on the Distribution Record Date. Consequently, to receive uniQure DRs, a person must hold AMT shares on the Distribution Record Date. To hold AMT shares on the Distribution Record Date, a shareholder must have bought such shares before the Ex Date, which shall be the second business day prior to the Distribution Record Date. Conversely, shareholders that do not want to receive uniQure DRs, must sell their AMT shares before the Ex Date. Shareholders having questions or requiring additional information on this settlement though Euroclear Netherlands are recommended to contact their bank or intermediary or other adviser.

Each AMT shareholder holding at least 5% of the shares in the capital of AMT on the Distribution Record Date shall be entitled to exchange its uniQure DRs for an equal number of ordinary shares (class A) in uniQure, subject to such shareholder becoming party to the uniQure Shareholder Agreement (the "Exchange Offer"). An AMT shareholder that wishes to exchange its uniQure DRs for ordinary shares (class A) in uniQure pursuant to the Exchange Offer must give written notice thereof to AMT within ten business days after the Distribution Record Date. The written notice should be accompanied with evidence of their AMT shareholding. More information on the Exchange Offer can also be found in the trust conditions (administratievoorwaarden) of Stichting Administratiekantoor uniQure B.V., the trust foundation that has issued the uniQure DRs. 

For completeness sake it is noted that the AMT shares will not extinguish or otherwise cease to exist upon the Advance Distribution of the DRs. The AMT shares will continue to exist until the liquidation is finalised and AMT ceases to exist, which is expected to occur in the summer of 2012. AMT and its shares ceasing to exist upon the ending of the liquidation shall effectively also result in AMT's delisting, to the extent the delisting cannot be achieved at an earlier date in consultation with Euronext Amsterdam and subject to such conditions as Euronext Amsterdam may propose.

Amsterdam Molecular Therapeutics Publishes Positive Data from Glybera® 14 Week Study

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera® (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal. This results in a much reduced level of newly-formed chylomicrons in the bloodstream, which are considered to be the cause of the acute and recurring bouts of pancreatitis seen in lipoprotein lipase deficiency (LPLD) subjects. LPLD is a very rare inherited condition that is associated with increased levels of chylomicrons. These particles carry certain types of fat in the blood, which because they are not removed from the body can cause recurrent pancreatitis. Data were published online in the Journal of Clinical Endocrinology & Metabolism (JCEM, Mar 2012).

“These data show that Glybera has a profound impact on chylomicron metabolism 14 weeks after a single administration. Although the patient cohort is small, due to the rare nature of LPLD, these results are very encouraging,” explained Dr. André Carpentier, Division of Endocrinology at the Université de Sherbrooke, Quebec, Candada. “LPLD patients often suffer from extremely painful bouts of pancreatitis, which is believed to be caused by the accumulation of chylomicron particles in the blood.”

“This publication provides additional, independent support on the ability of Glybera to restore chylomicron metabolism in LPLD patients. We believe by restoring the body’s ability to metabolize these particles in LPLD patients, Glybera treatment results in fewer pancreatitis attacks,” stated Carlos Camozzi, Chief Medical Officer at AMT. “LPLD patients are under constant risk of these attacks and the associated excruciating pain.”

Study Details

In an open label clinical trial (CT-AMT-011-02), 5 LPLD subjects in Quebec, Canada, were administered alipogene tiparvovec at a dose of 1 x 1012 genome copies per kg. Two weeks before and 14 weeks after administration, chylomicron metabolism, and plasma palmitate (fatty acid) and glycerol appearance rates were determined following ingestion of a low fat meal. Following administration of alipogene tiparvovec, the triglyceride (TG) content of the chylomicron fraction and the chylomicron-triglyceride (TG)/total plasma TG ratio were reduced throughout the postprandial period. The postprandial peak chylomicron level and chylomicron AUC were greatly reduced (by 79% and 93%, 6- and 24 hours after the test meal, respectively). There were no significant changes in plasma fatty acid and glycerol appearance rates. Plasma glucose, insulin and C-peptide also did not change. The data was obtained from AMT’s study in patients treated with Glybera in 2009.