Navigation Links
AMT Starts Preregistration Trial for Glybera(TM)
Date:5/6/2009

AMSTERDAM, May 7 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today the treatment of the first patient in a preregistration clinical trial with Glybera(TM). This gene therapy product targets lipoprotein lipase deficiency (LPLD), a seriously debilitating and potentially lethal disease.

The randomized controlled trial has been designed to gather additional data on the effects of Glybera on lipid metabolism and the mechanisms underlying the prevention of pancreatitis attacks. The trial is being performed under a Clinical Trial Application approved by Health Canada.

The new clinical trial builds on positive data obtained from two previous clinical trials in which a total of 22 LPLD patients were treated. These data indicate that a single treatment with Glybera results in a long-term, statistically significant and clinically important reduction in the incidence of acute pancreatitis in LPLD patients. The longest follow-up of individual patients is well over three years, and the cumulative follow-up of all patients is more than 45 years. The therapy was well tolerated and no drug-related severe adverse events or unexpected side-effects have been observed.

AMT will include the data from the new trial in the Marketing Authorization Application for Glybera. The submission of the dossier to the European Medicines Agency is planned for the second half of 2009.

About the Disease

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. This results in recurrent and severe acute inflammation of the pancreas, called pancreatitis, the most debilitating complication of LPLD. The disease can result in difficult-to-treat diabetes and is associated with significant morbidity and mortality.

About Amsterdam Molecular Therapeutics

AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases, especially those that are caused by one faulty gene. Currently, AMT has a product pipeline with nine products at different stages of development.

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 Amsterdam Molecular Therapeutics 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. Actual results could differ materially from current expectations due to a number of factors and uncertainties affecting AMT's business, including, but not limited to, the timely commencement and success of AMT's clinical trials and research endeavors, delays in receiving U.S. Food and Drug Administration or other regulatory approvals (i.e. EMEA, Health Canada), market acceptance of AMT's products, effectiveness of AMT's marketing and sales efforts, development of competing therapies and/or technologies, the terms of any future strategic alliances, the need for additional capital, the inability to obtain, or meet, conditions imposed for required governmental and regulatory approvals and consents. AMT expressly disclaims any intent or obligation to update these forward-looking statements except as required by law. For a more detailed description of the risk factors and uncertainties affecting AMT, refer to the prospectus of AMT's initial public offering on June 20, 2007, and AMT's public announcements made from time to time.


'/>"/>
SOURCE Amsterdam Molecular Therapeutics B.V
Copyright©2009 PR Newswire.
All rights reserved

Related biology technology :

1. Pierre Fabre Dermatologie Starts Clinical Trials on a Beta-Blocker to Treat Severe Infantile Haemangioma
2. BioCis Pharma Starts Phase IIa Trial in Psoriasis
3. The Pittsburgh Life Sciences Greenhouse Starts 2009 With Investment Momentum
4. China Sky One Medical, Inc. Starts Production at Newly Acquired Facility and Reaffirms Guidance
5. Oncolytics Biotech Inc. Starts Patient Enrolment in U.S. Phase 2 Clinical Trial Investigating REOLYSIN(R) in Combination with Paclitaxel and Carboplatin
6. Gerresheimer Starts Process to Sell off its Business Segment of Technical Plastics
7. Roche starts acquisition process of ARIUS to gain access to new screening platform for antibody therapeutics
8. AMT Starts Collaboration With St. Jude Childrens Research Hospital on Gene Therapy for Hemophilia B. Company Accesses Exclusive Commercial Rights to Final Therapy
9. Anesiva Launches Needle-Free Zingo(TM) to Reduce Pain from IV Starts and Blood Draws in Children
10. Oncolytics Biotech Inc. Starts Patient Enrolment in U.K. Phase II Clinical Trial Investigating REOLYSIN(R) in Combination with Paclitaxel and Carboplatin
11. Oncolytics Biotech Inc. Starts Patient Enrolment in U.K. Combination REOLYSIN(R)/Cyclophosphamide Trial
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/18/2017)... Research and Markets has announced the addition of the "Nanobiotechnology ... ... separate comprehensive analytics for the US, Japan , ... and forecasts are provided for the period 2015 through 2022. Also, ... and analytics are derived from primary and secondary research. This report ...
(Date:2/17/2017)... Feb. 17, 2017  BioGenex, a global leader ... of a novel system for quantitative immunohistochemistry (IHC). ... University of Rochester (NY, USA) and Konica-Minolta Inc. ... system is able to accurately quantify the expression ... epidermal growth factor receptor-2) in clinical samples. Quantitative ...
(Date:2/17/2017)... ... ... Academy of Model Aeronautics (AMA), the nation’s leading voice ... systems (UAS), are launching a joint program to promote safe and responsible drone ... efforts. , AMA and DJI will collaborate on other potential opportunities to mutually ...
(Date:2/16/2017)... Brussels, Belgium (PRWEB) , ... February 16, 2017 , ... ... Development (SIOTAD) framework primarily aimed at the agricultural industry. Pilot studies are about to ... phytosanitary products through IoT, Big Data and 5G innovations. The concept is expected to ...
Breaking Biology Technology:
(Date:2/2/2017)... Fla. , Feb. 2, 2017   ... a clinical-stage immuno-oncology company specializing in the development ... for the treatment of cancer and metastatic disease, ... scale-up and GMP manufacturing of a second clinical ... vaccine targeting folate receptor alpha. The manufactured vaccine ...
(Date:1/30/2017)... Jan. 30, 2017   Invitae Corporation (NYSE: ... genetic information companies, today announced that it will report ... provide 2017 guidance on Monday, February 13, 2017, and ... day at 4:45 p.m. Eastern / 1:45 p.m. Pacific. ... will briefly review financial results, guidance, and recent developments ...
(Date:1/24/2017)... -- It sounds simple and harmless—an electronic sensor attached ... and alerts parents on their smart phones if, ... But pediatric experts argue that such devices may ... of medical benefits, especially to healthy babies. ... of healthy babies, promising peace of mind about ...
Breaking Biology News(10 mins):