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Recruitment into the first randomized, controlled Phase 2 belinostat trial for patients with the solid tumor indication, cancer of unknown primary (CUP) has been completed

23-12-10 kl. 23/12 2010 11:00 | TopoTarget 0,00 (0,00%)

Copenhagen, Denmark - 23 December, 2010 - Topotarget A/S (NASDAQ OMX: TOPO)
announced today that the last patient has been recruited into the Topotarget
sponsored Phase 2 CUP trial with belinostat, carboplatin and paclitaxel versus
carboplatin and paclitaxel.

The study is an open-label randomized, controlled Phase 2 trial with belinostat
in combination with carboplatin and paclitaxel (BelCaP) compared to carboplatin
and paclitaxel (CaP) in patients with previously untreated cancer of unknown
primary (CUP). The study is a part of the collaboration between Topotarget and
its US partner Spectrum Pharmaceuticals, Inc. (NasdaqGM: SPPI).

Belinostat is administered as a 30 minute daily IV infusion on days 1-3 while
belinostat is administered orally on days 4 and 5. Chemotherapy in both groups
is administered on day 3. Treatment is given every third week and repeated for
up to 6 cycles. Patients who have been randomized to the belinostat group may
continue on oral belinostat on days 1-14 every third week until disease
progression. The Phase 2 CUP trial includes a total of 88 patients and is
conducted at centres in Denmark, France, Germany, and the US and is a fully
sponsored Topotarget trial.

The CUP trial conducted by Topotarget is the first randomized, controlled
clinical trial in CUP with a HDAC inhibitor, as well as the first randomized,
controlled clinical trial ever performed to seek to prove the possible benefit
of adding a third drug to the already established combination of carboplatin
and paclitaxel.

The primary objective of the study is to provide an estimate of the hazard
ratio of treatment effect of BelCaP compared to CaP in terms of
progression-free survival, response rate and overall survival in patients with
CUP.

Top-line results of progression-free survival and response rate are expected by
Q3 2011. Final evaluation of overall survival is expected in Q1 2012. The data
from this 88 patient randomized, controlled proof-of concept study will
delineate the role of belinostat in combination with chemotherapy in the
treatment of patients with CUP. It is Topotarget's objective, subject to
positive results from the Phase 2 belinostat trial in CUP, and from a
subsequent pivotal trial, to file a MAA (Market Authorisation Application) with
the European Medicines Agency (EMA) as well as Spectrum Pharmaceuticals Inc.
(Topotarget's US partner) to file a NDA (New Drug Application) for belinostat
with the US Food and Drug Administration (FDA).

“We are both extremely proud and pleased to announce that the recruitment of
this Topotarget-sponsored Phase 2 CUP trial has been completed on time. This is
a significant milestone for Topotarget as this is our first randomized trial in
patients with solid tumors. Further, the trial is in line with our commitment
to develop belinostat for the treatment of patients with solid tumors as well
as to maximize the value of this promising drug candidate”,said Francois
Martelet, MD, CEO of Topotarget. He continued, “It is our belief that
belinostat in combination with other agents has the potential to play a major
role in solid tumor indications for the treatment of cancer.”

Today's news does not change Topotarget's full-year financial guidance for 2010.


Topotarget A/S


For further information, please contact:

Francois Martelet, CEO: Direct: +45 39 17 83 43; Mobile: +45 51 32 83 41

Anders Vadsholt, CFO: Direct: +45 39 17 83 45; Mobile: +45 28 98 90 55


Background information

About CUP

Cancer of unknown primary site (CUP) represents a group of solid tumors that
are defined by the presence of metastases at first presentation for which a
primary site remains unknown at final diagnosis. It accounts for approximately
2-4% of all cancer diagnoses, and it is estimated that 31,000 new cases will be
diagnosed in the US for 2010. European cancer registries indicate similar
incidence rates for CUP as in the US. Diagnosis of CUP is made after a workup
that includes a careful medical history, a thorough physical examination (with
pelvic and rectal examinations), full blood count and biochemistry, urinalysis,
histopathologic analysis of biopsies for organ or tissue specific markers,
serum tumor marker analyses, computed tomography scans of the chest, abdomen
and pelvis, and mammography for women as well as other image procedures and
endoscopies. Histological analyses of tumor biopsies classify CUP in four major
categories:

· 60% adenocarcinomas

· 30% poorly differentiated carcinomas/adenocarcinomas

· 5% poorly differentiated neoplasms

· 5% squamous cell carcinomas

Patients with CUP represent a diverse group, but in the majority of patients,
empiric chemotherapy is the treatment of choice. At present, the combination of
carboplatin and paclitaxel is a common choice for first-line therapy. This
regimen produces a response rate of 25-40%, median progression-free survival of
4-6 months, overall survival of 9-11 months, and a 2-year survival of
approximately 20%.

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 2 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 1 clinical trial for patients with advanced solid tumors and lymphomas.
NCI-sponsored clinical trials are being conducted under a clinical trials
agreement between NCI and Topotarget. 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.
The NCI is an agency of the US government that oversees the US National Cancer
Program, conducts cancer research, and provides funding for research and
training.

About Spectrum Pharmaceuticals, Inc.

Spectrum Pharmaceuticals is a commercial-stage biotechnology company with focus
in oncology. The Company's strategy is comprised of acquiring and developing a
broad and diverse pipeline of late-stage clinical and commercial products. For
more information, please visit the Company's website at www.sppirx.com.

About Topotarget

Topotarget (NASDAQ OMX: TOPO) is a Scandinavian based international biotech
company headquartered in Denmark, dedicated to improve cancer therapies. In
collaboration with Spectrum Pharmaceuticals, Inc. Topotarget currently focuses
on the development in pivotal studies of its lead drug candidate, belinostat,
which has demonstrated a clear anti-neoplastic effect in both hematological
malignancies and solid tumors. Belinostat can be used in combination with full
doses of chemotherapy, and is currently in a pivotal trial within PTCL
(peripheral T-cell lymphoma) and Phase 2 in cancer of unknown primary site
(CUP). Topotarget's key cancer drug targets are HDAC, NAD+, mTOR, Fas ligand
and topoisomerase II. Totect® is a product on the market developed from
Topotarget's drug discovery technology. Totect® is marketed by the company's
own sales specialists in the US. The European rights to Savene® were divested
in March 2010 as a consequence of the focus to develop and commercialise
belinostat. 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, as well as about 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.




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