FDA grants Orphan Drug status for belinostat for the treatment of Peripheral T-cell lymphoma (PTCL)

Symbion
Fruebjergvej 3
DK 2100 Copenhagen
Denmark
Tel: +45 39 17 83 92
Fax: +45 39 17 94 92
CVR-nr: 25695771
www.topotarget.com

Copenhagen, Denmark - September 9, 2009 - TopoTarget A/S (OMX: TOPO) announced
today that FDA (the US health authorities) has granted belinostat Orphan Drug
designation for the treatment of relapsed or refractory peripheral T-cell
lymphoma, a disease with a large unmet medical need where there is no specific
standard treatment approved. The FDA Orphan Drug designation offers incentives
like a 7 year market exclusivity on drug sales upon market approval. TopoTarget
also has a Special protocol Assessment (SPA) agreement for the pivotal PTCL
study and a Fast Track agreement with the FDA. TopoTarget's pivotal study (the
BELIEF study) is to recruit approximately 120 PTCL patients and is aimed for
filing in December 2010. 

The Orphan Drug possibility is meant to encourage pharmaceutical companies to
develop drugs for diseases like PTCL: under the US law, companies that develop
a drug for a disorder affecting fewer than 200,000 people in the United States
may sell it without competition for seven years. In addition to providing a
7-year term of markets exclusivity upon final FDA approval, Orphan Drug
designation also provides advantages through a wide range of financial and
regulatory benefits. 

“We are very happy for the strong continued support for the strategy of the
development of belinostat for cancer patients” said Professor Peter Buhl
Jensen, CEO of TopoTarget. 

“There is a big unmet medical need in this disease and if our pivotal study
reproduces the data from our initial study with response rates of 25% for PTCL
and with complete remissions, belinostat monotherapy can become an important
treatment alone or be part of an effective combination treatment as the benign
safety profile of belinostat allows it to be combined in full dose with
conventional therapies”. Peter Buhl Jensen further comments “The PTCL
indication is the first step in accordance with our clinical development plan
to bring belinostat to the market as fast as possible. At the same time we are
pursuing the steps necessary to bring belinostat to the market in larger
indications like solid tumors in combination therapy to exploit the clinical
and commercial potential of belinostat.” 

Combination therapy is used in order to attack the cancer cell from different
angels and thereby increase response rates. In addition to the benefit obtained
with the drug used as a single agent, belinostat has the big advantage that it
exhibits no dose limiting bone marrow toxicity which is the main cause for dose
reduction in the most successful chemotherapy combinations. 

Today's news does not change TopoTarget's full-year financial guidance.


TopoTarget A/S

For further information, please contact:

Peter Buhl Jensen	  Telephone	+45 39 17 94 99
CEO		  Mobile	   +45 21 60 89 22
	
Background information

About belinostat
Belinostat is a promising small molecule HDAC inhibitor being investigated for
its role in the treatment of a wide range of solid tumors and hematologic
malignancies either as a single-agent, or in combination with other active
anti-cancer agents, including carboplatin, paclitaxel, doxorubicin, idarubicin,
cis-retinoic acid, azacytidine and Velcade® (bortezomib) for injection. HDAC
inhibitors represent a new mechanistic class of anti-cancer therapeutics that
target HDAC enzymes, and have been shown to: arrest growth of cancer cells
(including drug resistant subtypes); induce apoptosis, (programmed cell death);
promote differentiation; inhibit angiogenesis; and sensitize cancer cells to
overcome drug resistance when used in combination with other anti-cancer
agents. Company-sponsored trials of IV-administered belinostat include a
pivotal trial in peripheral T-cell lymphoma (PTCL), a randomized controlled
Phase II trial in cancer of unknown primary (CUP), and studies in ovarian,
colorectal and soft tissue sarcoma patients. NCI-sponsored trials (single agent
and in combination with anti-cancer therapeutics) with IV-administered
belinostat include studies in hepatocellular, thymoma, Myelodysplastic Syndrome
(MDS), and other solid and hematologic cancers. Continuous intravenous
administration (CIV) is being evaluated in clinical trials in solid tumours as
well as in AML.  An oral formulation of belinostat is also being evaluated in a
Phase I clinical trial for patients with advanced solid tumors and lymphomas.
Furthermore TopoTarget has a Cooperative Research and Development Agreement
(CRADA) with the NCI to conduct preclinical and nonclinical studies on
belinostat in order to better understand its anti-tumor activity and to provide
supporting information for clinical trials. 


About Peripheral T-Cell Lymphomas (PTCL)
PTCL represent approximately 10% of all non-Hodgkin's lymphomas (NHL) in
Western populations and are associated with a poor prognosis. Most patients
with PTCL relapse after initial treatment with cytotoxic agents, and 5-year
survival is less than 30%. 
T-cell Non Hodgkins Lymphomas, to which PTCL belongs, are associated with a
poorer outcome and survival compared to the B-cell lymphomas. The primary
response rate for all PTCL subtypes remains at less than 60%, with nearly all
patients relapsing. Median survival of all PTCL patients (excluding a few
subtypes) is approximately 3-4 years, with a 5-year survival of less than 30%. 
There are currently no therapies approved specifically for PTCL. Primary
treatment for most subtypes of PTCL remains anthracycline-based regimens,
predominantly the combination of cyclophosphamide, doxorubicin, vincristine and
prednisone (CHOP). With the exception of ALK-positive ALCL, PTCL subtypes
respond poorly to these regimens. The use of radiotherapy, with or without
chemotherapy, is preferred as front line treatment of extranodal NK/T-cell
lymphoma. The majority of patients with PTCL will relapse after primary
therapy. A number of chemotherapy regimens are used for salvage therapy.
However, there is currently no consensus regarding the optimal treatment
approach for PTCL salvage therapy. 

About TopoTarget 
TopoTarget (OMX: TOPO) is an international biotech company headquartered in
Denmark, dedicated to finding ''Answers for Cancer'' and developing improved
cancer therapies. The company was founded and is run by clinical cancer
specialists and combines years of hands-on clinical experience with in-depth
understanding of the molecular mechanisms of cancer. 
TopoTarget has a broad clinical pipeline but is currently focusing on the
development of belinostat, which has shown proof of concept as monotherapy in
treating haematological malignancies and positive results in solid tumours
where it can be used in combination with full doses of chemotherapy, and is in
a pivotal trial in PTCL. TopoTarget's expertise in translational research is
utilizing its highly predictive in vivo and in vitro cancer models. TopoTarget
is directing its efforts on key cancer targets including HDACi, NAD+, mTOR,
FasLigand and topoisomerase II inhibitors. The company's first marketed product
Savene®/Totect® was approved by EMEA in 2006 and the FDA in 2007 and is
marketed by TopoTarget's own sales force in Europe and the US. For more
information, please refer to www.topotarget.com. 

TopoTarget Safe Harbour Statement
This announcement may contain forward-looking statements, including statements
about our expectations of the progression of our preclinical and clinical
pipeline including the timing for commencement and completion of clinical
trials and with respect to cash burn guidance. Such statements are based on
management's current expectations and are subject to a number of risks and
uncertainties that could cause actual results to differ materially from those
described in the forward-looking statements. TopoTarget cautions investors that
there can be no assurance that actual results or business conditions will not
differ materially from those projected or suggested in such forward-looking
statements as a result of various factors, including, but not limited to, the
following: The risk that any one or more of the drug development programs of
TopoTarget will not proceed as planned for technical, scientific or commercial
reasons or due to patient enrolment issues or based on new information from
non-clinical or clinical studies or from other sources; the success of
competing products and technologies; technological uncertainty and product
development risks;  uncertainty of additional funding; TopoTarget's history of
incurring losses and the uncertainty of achieving profitability; TopoTarget's
stage of development as a biopharmaceutical company; government regulation;
patent infringement claims against TopoTarget's products, processes and
technologies; the ability to protect TopoTarget's patents and proprietary
rights; uncertainties relating to commercialization rights; and product
liability expo-sure; We disclaim any intention or obligation to update or
revise any forward-looking statements, whether as a result of new information,
future events, or otherwise, unless required by law.