Amsterdam Molecular Therapeutics Reports Full Year Results 2010

By Amsterdam Molecular Therapeutics B.v, PRNE
Tuesday, February 15, 2011

AMSTERDAM, February 16, 2011 - Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field
of human gene therapy, today reported its results for the year to December
31, 2010

Key Highlights

    - Glybera(R) Marketing Authorisation Application remains on track with
      European Medicines Agency (EMA);
    - Responses to Day 120 questions submitted in November 2010;
    - Day 180 list of Outstanding Issues now received;
    - Raised EUR14.3 million through private placement;
    - Novel biomarker for Glybera(R) activity identified;
    - Diagnostic gene chip for LPLD developed by AMT's collaborator,
      Progenika, obtained CE Mark;
    - Hemophilia Phase I/II study underway, being led by St Jude Children's
      Hospital and UCL Hospital; data from initial patients showed safe,
      Stable expression;
    - DMD program progressing with US Orphan Drug Status granted and up to
      EUR 4 million Senter Novem funding;
    - Porphyria program funding through EU grant now finalized;
    - GDNF program progressing following renegotiation of collaboration
      agreement with Amgen; and
    - New collaboration initiated with consortium led by Institut Pasteur to
      develop Sanfilippo gene therapy.

Jorn Aldag, Chief Executive Officer of AMT, commented: "We are excited by
the key milestones achieved in 2010: our lead product Glybera(R) is
progressing towards market approval and we are looking forward, with
confidence, to the Regulatory Agency's decision. We have continued, also, to
make good progress in our other pipeline programs, on both the R&D and
partnering fronts."


Lipoprotein Lipase Deficiency (LPLD)

The advance of AMT's lead product, Glybera(R) towards market approval was
the key focus of the Group's activities during 2010. The regulatory process
continues to progress on schedule. Following the filing of Glybera(R) with
the European Medicines Agency (EMA) as a treatment for lipoprotein lipase
deficiency (LPLD), the agency conducted its initial review of the Glybera(R)
registration dossier in early 2010 and sent the Day 120 List of Questions to
AMT in May. AMT submitted its response to the EMA in November 2010, based in
part on additional data and analyses from patients previously treated with
Glybera(R), including new data available from the last clinical trial and its
one-year extension. The EMA restarted evaluation on November 26 (Day 121),
and following the normal centralized review procedure, AMT has received, at
Day 180, a significantly reduced list of outstanding items. We are now in the
process of compiling the responses and expect to submit answers to the EMA by
end of the first quarter and believe to be on track for a mid 2011 response
on marketing approval.

Because AMT's technology can be applied equally to a wide range of other
genetic diseases, the success of Glybera(R) would validate AMT's approach for
its other pipeline products, targeting a range of orphan diseases and
diseases with large patient populations, in each case where there is high
unmet medical need, including Parkinson's disease, hemophilia, Duchenne
muscular dystrophy (DMD) and acute intermittent porphyria (AIP).

Hemophilia B

The hemophilia B program has entered a Phase I/II clinical study, led by
our partners St Jude Children's Research Hospital, Memphis, Tennessee, and
University College London (UCL) Hospital, London, UK. Initial data presented
at the American Society of Hematology (ASH) conference in Florida in December
showed that dose-dependent, safe, stable, and persistent expression in
patients has been observed.


Our collaboration with FIMA in Pamplona, Spain, to develop a gene therapy
for acute intermittent porphyria (AIP) is progressing very well, and has
generated positive pre-clinical data. The program is now entering toxicology
studies and a Phase I/II study is scheduled to begin in early 2012. Total
grant funding of EUR 3.3 million for this project has been secured from the
EU, with EUR1.1 million assigned to AMT, supporting our financial commitment
to this project.

Duchenne Muscular Dystrophy

The DMD program continues to progress, with up to EUR 4 million of
financial support from Senter Novem, which covers 35% of development costs
through to completion of the Phase I/II study. In September 2010, the US FDA
granted our gene therapy for DMD Orphan Drug status. This complements the
Orphan Drug status granted to the same program by the EMA covering Europe in


AMT has successfully renegotiated the terms of its collaborative
agreement with Amgen covering the use of GDNF in gene therapies. AMT now has
the ability to explore development of additional indications, including
orphan diseases, alongside the development of Parkinson's disease. The
University of Lund, Sweden, is performing preclinical studies in Parkinson's
disease models for this program, the results of which are encouraging.


Immediately after the year-end, AMT announced a collaboration with
Institut Pasteur and the Association Francaise contre les Myopathies (AFM) to
develop a gene therapy for the rare lysosomal disorder, Sanfilippo B for
which Institut Pasteur has achieved preclinical proof of concept. Under the
agreement, AMT will receive funding to manufacture the product using its AAV
vector technology to progress the program through a Phase I/II study.
Thereafter, AMT will have an option to acquire full commercial rights to the


In October 2010, AMT successfully raised EUR 14.3 million of new funds,
before expenses (EUR13.3 million net of costs) via a private placement of
ordinary shares. These funds together with our existing cash resources take
AMT comfortably through the assessment process of Glybera. The Company also
expects to generate significant additional revenue from commercializing
Glybera and from developing its collaborations with other parties. Taking
these additional funding sources into account, AMT believes it will have
sufficient cash resources to meet its operational requirements.



The total net income for the year ended December 31, 2010 amounted to EUR
1.4 million
, a EUR 1.0 million increase compared to the total net income for
the year ended December 31, 2009, which amounted to EUR 0.4 million. These
revenues represent grant income from the Dutch government and the European

Operating costs

Research and development expenditure totaled EUR 16.4 million, compared
to EUR 13.2 million in 2009, an increase of EUR 3.2 million reflecting the
ongoing level of activity to support the filing of Glybera, as well as
ongoing development on AMT's other projects, including the Duchenne program
(which is partly funded by the investment credit from Senter Novem) and
reflecting the activities financed by the grants described above. In
addition, research and development expenditure in 2010 included the EUR 0.5
of impairment charges and the largest part of the EUR 0.5 million
increase in charges relating to share-based incentive schemes, both of which
represent non-cash items.

General and administrative costs decreased to EUR 4.1 million, from EUR
4.9 million
in 2009. This decrease reflected the lower level of advisory
costs in 2010, compared to 2009, as well as the fact that 2009 contained
certain reorganization costs.

Operating result

AMT's operating loss rose to EUR 19.1 million for 2010, from EUR 17.8
for 2009, an increase of EUR 1.3 million. This increase can be
largely accounted for by certain non-cash items amounting to EUR 1.0 million,
comprising an increase of EUR 0.5 million in charges relating to share-based
incentive schemes, a EUR 0.3 million charge relating to impairment charges on
certain intangible assets following the termination of a 2008 collaboration
agreement, and a EUR 0.2 million charge in respect of the impairment of
certain leasehold improvements. After excluding for these non-cash,
non-recurring items, the operating loss in 2010 would have been broadly
equivalent to the operating loss for 2009.

Finance income and costs

Net finance income fell to EUR 0.5 million compared to EUR 0.6 million in
2009, reflecting the lower average cash balances of the Group during 2010
during a period when interest rates available on cash deposits remained low.
In addition, finance costs increased to EUR 0.5 million compared to EUR 0.0
, reflecting the interest charge payable on the 2009 convertible bond,
together with foreign exchange movements on currency accounts. Of these
finance costs, EUR 0.2 million relates to non-cash items.

Result for the Year and Loss per Share

Total net loss for the year ended December 31, 2010 amounted to EUR 19.1
, compared to the net loss for the year ended December 31, 2009, which
amounted to EUR 17.2 million, an increase of EUR 1.9 million. The increase in
the net loss includes an increase in non-cash charges of EUR 1.2 million
described within the 'Operating loss' and 'Finance income and costs'
paragraphs above. The loss per share amounted to EUR 1.13 for 2010 compared
to EUR 1.17 for 2009. The basic and diluted loss per share are the same
because the company is loss-making in both periods.

Cash Flow and Cash Position

Cash and cash equivalents amounted to EUR 17.9 million at December 31,
, a decrease of EUR 4.8 million or 21% compared to EUR 22.6 million at
December 31, 2009. The decrease in cash and cash equivalents is mainly the
result of cash used in operating activities amounting to EUR 17.7 million in
2010 (2009: EUR16.5 million), offset by net cash generated from financing
activities of EUR 13.4 million.

The cash used in operating activities represents our operational loss
adjusted for non-cash items such as share-based payment expenses and changes
in working capital.

The cash flow from financing activities amounted to EUR 13.4 million
reflecting the issue of new shares in October 2010, compared to EUR 4.8
in 2009, which principally represented the drawdown of the
convertible loan.


Shareholders' equity amounted to EUR 13.5 million at December 31, 2010
compared to EUR 18.4 million at December 31, 2009. A total number of
23,512,225 shares were issued and outstanding at December 31, 2010.


The company expects its existing cash resources, together with the net
cash inflows that it expects to generate during 2011 from partnering
activities and from the commercialization of Glybera(R) that would follow a
successful MAA submission, to be sufficient to fund its operations for at
least the next 12 months from the date of publication of the audited
consolidated annual accounts, which is expected to be on March 4, 2011. In
reaching this conclusion, the Management of AMT has considered the
uncertainty inherent in forecasting future net cash inflows, and believes
that its expectations, as described above, are reasonable.

To discuss these results, AMT's management will conduct a conference call
at 3:30 PM CET, which will also be webcast.

To participate in the conference call, please call one of the following
telephone numbers 15 minutes prior to the event, using access code 3705648:
+44(0)20-7806-1956 for the UK; +1-212-444-0413 for the US; and
+31(0)20-707-5510 for the Netherlands. Following the results presentation,
the lines will be opened for a question and answer session. A replay of the
call will be available following the event.

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: Jorn Aldag, CEO, AMT, Tel : +31(0)20-566-7394, j.aldag at; Mike Sinclair, Partner, Halsin Partners, Tel : +44(0)20-7318-2955 msinclair at

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