AMT Receives EUR 1.1 Million Funding for Acute Intermittent Porphyria Gene Therapy as part of EU Consortium GrantBy Amsterdam Molecular Therapeutics B.v, PRNE
Sunday, January 30, 2011
AMSTERDAM, The Netherlands, January 31, 2011 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in
the field of human gene therapy, announced today that the European Union (EU)
has finalized a EUR 3.3 million grant to the AIPGENE consortium, of which AMT
is a member, for the development of a gene therapy product for Acute
Intermittent Porphyria (AIP). AIP is a severe and progressive disease caused
by the inability of the body to produce the heme protein, a component of
hemoglobin, as well as other important proteins.
From the grant, made under the EU's FP7 program, AMT will
receive EUR 1.1 million. The grant will cover approximately 75% of AMT's
overall development costs to bring this product forward to completion of a
Phase I/II study in humans. AMT holds the commercialization rights to the AIP
gene therapy (AMT-021). The AIPGENE consortium is led by Fundación para la
Investigatión Médica Aplicada (FIMA, Spain), and in addition to AMT, it
includes Clínica Universidad de Navarra (Spain), Stockholms Läns Landsting
(Sweden), Deutsches Krebsforschungszentrum (Germany), Digna Biotech (Spain)
and Servicio Madrileño de Salud (Spain).
"This grant allows us to prioritize the development of our AIP
gene therapy program for this progressive and devastating disease within
AMT's pipeline, as well as initiating the next step in advancing a
potentially more effective, long-term treatment for patients," said Jörn
Aldag, Chief Executive Officer of AMT.
AIP results from mutations in the PBDG gene, which encodes for
the enzyme porphobilinogen deaminase, a liver protein necessary for the
production of heme. AMT-021 is intended to provide long-term normalization of
the PBGD protein in order to prevent acute attacks and their complications.
AMT has demonstrated that AMT-021 produces this normalization of the PBGD
protein in an animal model of AIP. In addition, it completely prevented the
occurrence of attacks and significantly ameliorated the neuropathy that
develops in untreated animals. AMT's partner at FIMA has shown persistence of
expression of genes in the liver for more than a year using AAV-mediated
delivery methods similar to AMT-021. With the support of the all the AIPGENE
partners, AMT anticipates AIP patient enrolment in a clinical trial to begin
On 28 May 2008, the European Medicines Agency (EMA) granted
Orphan Drug Designation to AMT's gene therapy product for the treatment of
AIP. This entitles AMT to ten years of market exclusivity to treat AIP in
Europe following marketing approval, provided that this product candidate is
the first such approved new drug with a major medical benefit.
About Acute Intermittent Porphyria
Acute intermittent porphyria is a rare genetic disease where
mutations in the porphobilinogen deaminase (PBGD) gene, results in
insufficient activity of a protein necessary for heme synthesis. This leads
to the accumulation of toxic intermediate metabolites that produce a wide
variety of problems including acute and severe abdominal pains, psychiatric
and neurological illnesses. There is currently no cure for this condition and
the disease is typically progressive.
About Amsterdam Molecular Therapeutics
AMT is a world leader in the development of human gene based
therapies. The company's lead product Glybera(R), a gene therapy for the
treatment of lipoprotein lipase deficiency (LPLD), is currently under review
by the European Medicines Agency (EMA). If approved, Glybera will be the
first gene therapy product to be marketed in Europe. AMT also has a product
pipeline of several gene therapy products in development for hemophilia B,
Duchenne muscular dystrophy, acute intermittent porphyria, and Parkinson's
disease. Using adeno-associated viral (AAV) derived vectors as the delivery
vehicle of choice for therapeutic genes, the company has been able to design
and validate probably the world's first stable and scalable AAV manufacturing
platform. This proprietary platform can be applied to a large number of rare
(orphan) diseases caused by one faulty gene and allows AMT to pursue its
strategy of focusing on this sector of the industry. AMT was founded in 1998
and is based in Amsterdam. Further information can be found at
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.
Actual results could differ materially from current expectations due to a
number of factors and uncertainties affecting AMT's business. AMT expressly
disclaims any intent or obligation to update any forward-looking statements
herein except as required by law.
For further enquiries: Jörn Aldag, CEO, AMT, Tel : +31-20-566-7394, j.aldag at amtbiopharma.com ; Mike Sinclair, Partner, Halsin Partners, Tel : +44-20-7318-2955, msinclair at halsin.com .
Tags: Amsterdam, Amsterdam Molecular Therapeutics B.V, January 31, Netherlands, The Netherlands