Initiation of phase 1 study of belinostat in combination with Bortezomib for Relapsed or Refractory Acute Leukemia or Myelodysplastic Syndromes (MDS)

Topotarget A/S
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 - 11 June, 2010 - Topotarget A/S (NASDAQ OMX: TOPO)
announced today that the first patient has been dosed in the
investigator-sponsored phase 1 dose escalating study with belinostat in
combination with bortezomid (Velcade®) for the treatment of Acute Leukemia or
Myelodysplastic Syndromes (MDS) patients. The primary study objective is to
determine the recommended phase 2 dose for the combination of belinostat and
bortezomib. The study is expected to recruit approximately 24 patients. 

“We are very happy to be working with Principal Investigators, Drs. Steven
Grant and Beata Holkava, at VCU Massey Cancer Center, Richmond, VA on this
study designed to improve on existing therapies to treat patients with relapsed
or refractory leukemia or MDS". 

“There is abundant preclinical data indicating that HDAC inhibitors such as
belinostat and proteasome inhibitors such as bortezomib act synergistically to
induce cell death in cancer cells”,  said Dr. Steven Grant, VCU Massey Cancer
Center, Richmond, Virginia, USA. ”Even though there are treatments approved for
acute leukemia and MDS there is still a significant unmet medical  need for new
therapeutic strategies as these patients are rarely cured. We are pleased to be
collaborating with Dr. Guillermo Garcia-Manero at MD Anderson on this
interesting clinical trial.” 

“We are pleased that belinostat continues to be investigated in combination
with common chemotherapeutic regimens,” said Francois Martelet, MD, Chief
Executive Officer of Topotarget. “We believe belinostat has the potential to be
an effective treatment in combination with existing chemotherapeutic agents for
the treatment of a variety of cancers, including leukemia and MDS.” 

More information on this phase 1 study can be found at www.clinicaltrials.gov,
identifier NCT01075425. 

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

Topotarget A/S

For further information, please contact:

Francois Martelet, CEO: Direct: +45 39 17 94 99; Mobile: +45 31 36 83 41
Anders Vadsholt, CFO: Direct: +45 39 17 83 45; Mobile: +45 28 98 90 55
Background information

About belinostat 
Belinostat (PXD 101) is a Class I and II HDAC inhibitor that is being studied
in multiple clinical trials as a single agent or in combination with
chemotherapeutic agents for the treatment of various hematological and solid
cancers.  Its anticancer effect is thought to be mediated through multiple
mechanisms of action, including the inhibition of cell proliferation, induction
of apoptosis (programmed cell death), inhibition of angiogenesis, induction of
differentiation, and the resensitization of cells that have overcome drug
resistance to anticancer agents such as platinums, taxanes and topoisomerase II
inhibitors.  Belinostat is the only HDAC inhibitor in clinical development with
multiple potential routes of administration, including intravenous
administration, continuous intravenous infusion and oral administration. 

Belinostat is currently in a registrational trial, under a Special Protocol
Assessment (SPA), as a monotherapy for relapsed or refractory Peripheral T-Cell
Lymphoma (PTCL), an indication for which it has been granted Orphan Drug and
Fast Track designation by the U.S. Food and Drug Administration.  The Company
currently plans to file a New Drug Application (NDA) in 2011.  Belinostat is
also under investigation in a randomized phase 2 trial, as a combination
therapy with carboplatin and paclitaxel, for cancer of unknown primary (CUP). 
Additionally, the National Cancer Institute is currently conducting several
clinical trials of Belinostat in a variety of hematological and solid tumors,
both as monotherapy as well as combination therapy. 

About Topotarget
Topotarget (NASDAQ OMX: TOPO) is an international biotech company headquartered
in Denmark, dedicated to improve cancer therapies. Topotarget currently
focuses, in collaboration with Spectrum Pharmaceuticals, Inc., on the
development in pivotal studies of its lead drug candidate, belinostat, which
has shown proof-of-concept as monotherapy in treating haematological
malignancies and positive results in solid tumours. Belinostat can be used in
combination with full doses of chemotherapy, and is currently in a pivotal
trial within PTCL (peripheral T-cell lymphoma). Topotarget's key cancer drugs
target HDAC, NAD+, mTOR, Fas ligand and topoisomerase II. 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 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 exposure; 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.