As of today we remain confident in the approvability of Glybera(R). Our assessment is based on the following:
- Our response to the EMA does not require further clinical trials with additional new to be treated patients. We expect to be able to formulate our response satisfactorily by submitting data and further analyses from already treated patients. - More, highly relevant, data from our last clinical trial CT-AMT-011-02 AMT strongly suggest that Glybera's effects are lasting (one year) via a mechanism that causes clearance of chylomicrons, the fat carrying particles which are responsible for pancreatitis in LPLD patients. - Overall we have developed a clear response strategy, which, if executed with no unforeseen adverse events or delays, should allow us to remain on track for a positive EMA decision in the middle of 2011.
Further to their 2009 agreement to co-develop a vector-gene combination for the treatment of Hemophilia B, AMT and St. Jude Children's Research Hospital in the USA have successfully transferred Factor IX to AMT's manufacturing platform and have demonstrated proof of concept in animals in 2010. The multicenter, dose escalation study with this vector-gene combination began in March, 2010 at University College London Hospital in the United Kingdom guided by Prof. Amit Nathwani. The first patient has been dosed successfully and demonstrated good results both in terms of clinical benefit and side effects. Further enrolment of patients is expected in the second half of 2010.
Duchenne Muscular Dystrophy
In support of its program to treat Duchenne Muscular Dystrophy, AMT
received an investment credit from SenterNovem (now Agentschap.nl), the Dutch
government innovation agency, in January 2010. The credit comprises a loan
covering 35% of the costs of the project through to 2013 with
|SOURCE Amsterdam Molecular Therapeutics B.V|
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