Quantcast

Amsterdam Molecular Therapeutics Reports Half-Year Results 2010

August 31, 2010

AMSTERDAM, The Netherlands, August 31, 2010 /PRNewswire-FirstCall/ –
Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of
human gene therapy, today reported its results for the first half year of
2010.

    Highlights

    - Glybera(R):
    - EMA initiated MAA review in 01/2010
    - Approval progressing on schedule for decision mid 2011
    - Novel biomarker for Glybera(R) activity identified
    - Hemophilia B: Phase I/II started
    - Duchenne Muscular Dystrophy: to benefit from EUR 4 million
      innovation credit
    - sRNA: silencing gene therapy technology achieves 80% cholesterol
      reduction
    - Supervisory Board nominations: 3 new industry professionals
      slated to join board
    - Key financial figures in line with guidance
    - Cash & cash equivalents of EUR 13.5 million at June 30, 2010

“We are very pleased with the key milestones we have achieved in the
first half year of 2010 in the Glybera(R) approval process which seems firmly
on track. Our dialogue with the EMA is very encouraging and we look forward
to the Agency’s decision with confidence. We also continue to make progress
with our pipeline, not only on the partnership front for programs such as
Hemophilia B, but also in our early stage efforts,” said Jorn Aldag, chief
executive officer of AMT.

Operations

Glybera(R) for Lipoprotein Lipase Deficiency (LPLD)

In January 2010, the European Medicines Agency (EMA) commenced review of
AMT’s Marketing Authorization Application. In May 2010 AMT received questions
(Day 120 questions) regarding the application. In July we met with the EMA,
to clarify the questions they raised, enabling AMT to align its response
strategy. We are now working towards an official response to the EMA Day 120
questions, due by the end of 2010.

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.

Hemophilia B

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 a maximum of
EUR 4 million. The loan is repayable only if AMT successfully commercializes
the program. AMT has shown proof of concept in a pre-clinical model with its
optimized construct for exon skipping using its proprietary AAV technology.

Parkinson’s Disease

Together with the University of Lund, Sweden, AMT is diligently working
on the preclinical development of a gene therapy for delivery of the GDNF
gene to the brain. Efficacy data in an animal model of PD is anticipated to
be available by the end of the current year

sRNA

Elevated levels of cholesterol are a major risk factor and contributor to
the development of atherosclerosis and cardiovascular disease (CVD). Early
research at AMT demonstrates that after a single intravenous injection of a
silencing gene therapy in animal models, the serum cholesterol levels were
reduced by 80% with no signs of toxicity. It is therefore reasonable to
expect a similar effect in patients, resulting in reduced risk for
atherosclerosis or CVD. Such a long-term, perhaps life-long active gene
therapy could eliminate the need for maintenance statin therapy.

Supervisory Board changes

During the period ended June 30, 2010, Alexander Ribbink and George
Morstyn retired from the Supervisory Board and AMT thanks them for their
substantial contributions. On April 28, 2010, AMT’s co-founder Sander van
Deventer
was appointed to the Supervisory Board, and in addition Joseph M.
Feczko
, Steven H. Holtzman and Francois Meyer were nominated to the
Supervisory Board for consideration at the Extraordinary General Meeting to
be held on September 20, 2010.

Financials

Results comparison

Total net loss for the period ended June 30, 2010 amounted to EUR 9.4
million
, in line with the net loss for the period ended June 30, 2009 which
also amounted to EUR 9.4 million.

The main item within operating costs reflects the investment in
Glybera(R) to support the registration process. Development of our Duchenne
Muscular Dystrophy program, which is 35% funded by a research credit from
SenterNovem through to completion of a Phase I clinical study continues.
Expenditure on our other development projects has been reduced as we are
constrained by our current resources and are focusing on the successful
completion of the Glybera registration process. Research and development
costs increased to EUR 8.1 million for the period ended June 30, 2010 from
EUR 7.1 million in the same period of 2009. At the same time, general and
administrative costs decreased to EUR 1.8 million in the period ended June
30, 2010
from EUR 2.9 million in the same period of 2009.

Net interest income/(cost) decreased to EUR (0.0) million for the period
ended June 30, 2010 from EUR 0.5 million in the same period in 2009 as a
result of the Company’s decreasing cash balance combined with continuing low
market interest rates for deposits.

Cash and cash equivalents amounted to EUR 13.5 million at June 30, 2010,
a decrease of EUR 9.1 million compared to EUR 22.6 million at December 31,
2009
. The decrease in cash and cash equivalents mainly stems from the
operational cash outflow which amounted to EUR 8.9 million for the period
ended June 30, 2010 (compared to an operating cash outflow of EUR 9.5 million
for the period ended June 30, 2009).

Outlook

The Company’s expenditure continues in line with budget. However, as AMT
has not yet reached the point of generating significant revenues that could
fund operations we continue to explore additional opportunities for funding,
including non-dilutive sources such as grants and/or collaborations with
partners. In addition, AMT is also tracking opportunities for raising
additional capital in conjunction with its bankers. The outlook for the year
remains unchanged.

Conference call and webcast presentation

AMT will conduct a conference call open to the public today at 3.30 p.m.
CET
, which will also be webcast. Netherlands dial in: +31(0)20-712-1304; US
dial in: +1-718-354-1361; UK dial in: +44(0)20-7138-0821. Confirmation Code:
1128246

To listen to the conference call live via the internet, visit the
investor relations portion of the AMT website at www.amtbiopharma.com. Please
go to the website 15 minutes prior to the call to register, download and
install the necessary audio software.

The archived webcast also will be available for replay shortly after the
close of the call.

About Amsterdam Molecular Therapeutics

AMT is a leader in the development of human gene based therapies. 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 what is probably the first stable and scalable AAV production
platform. This proprietary platform can be applied to a large number of rare
(orphan) diseases that are caused by one faulty gene. Currently, AMT has a
product pipeline with several AAV-based gene therapy products in LPLD,
Hemophilia B, Duchenne Muscular Dystrophy, Acute Intermittent Porphyria, and
Parkinson’s Disease at different stages of research or development. AMT was
founded in 1998 and is based in Amsterdam.

————————————————————————-

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. EMA, 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


Source: newswire



comments powered by Disqus