København, 2012-01-18 11:01 CET (GLOBE NEWSWIRE) --



København, Danmark – 18. januar 2012 – Topotarget A/S (NASDAQ OMX: TOPO.CO) har
i dag meddelt, at kliniske data (videnskabeligt resumé) vil blive præsenteret
på The 2012 Gastrointestinal Cancers Symposium den 19.-21. januar 2012.


Nedenfor er det videnskabelige kliniske resumé, der nu er tilgængeligt på
ASCO’s hjemmeside (www.asco.org).


Efficacy of belinostat in advanced hepatocellular carcinoma (HCC): Phase I and
II multicentered study of the Mayo Phase 2 Consortium (P2C) and the Cancer
Therapeutics Research Group (CTRG).


Abstract No.: 259

Session: General Poster Session B: Cancers of the Pancreas, Small Bowel, and
Hepatobiliary Tract

Background:
Belinostat is a novel histone deactylase inhibitor which demonstrates
preclinical activity in HCC. We report the results of a phase I/II study in
belinostat in patients (pts) with unresectable HCC.

Methods:
Major eligibility criteria included histologically confirmed HCC not amenable
to curative treatment; PS ≤ 2; adequate organ function; prior systemic therapy
was allowed. In the phase I portion, belinostat was given i.v. on D1-5 every 3
weeks with dose levels of 600, 900, 1200, and 1400 mg/m 2 /day. In the phase II
portion, belinostat was tested at the MTD. Primary endpoint was PFS and
secondary endpoints were RR according to RECIST and OS. CT assessment was done
every 8 weeks.

Results:
Phase I portion: a total of 18 pts were accrued; no DLTs were observed at
1400mg/m 2 /day for 5 days, and this dose was selected for phase II
development. Phase II portion: 42 pts were accrued; Median age = 57.5 years; 41
had Child’s A function, and 24 pt had ECOG 0. Sixteen (38%) had previous
systemic therapy, and 21 (50%) had previous transarterial therapy. Median
follow-up was 20.0 months. The PR and SD rate was 2.4% (1/42) and 45.2%
(19/42). Median PFS was 2.64 months (95%C.I. 1.55-3.17) and OS was 6.60 months
(95%C.I. 4.53-11.60). Grade 3 or higher toxicities (>5% rate) were abdominal
pain (9.5%), (9.5%) hyperbilirubinemia (9.5%), raised ALT (9.5%); anemia (7.1%)
and vomiting (7.1%).

Conclusions:
Belinostat demonstrates disease stabilization in a predominantly pretreated
population of pts with unresectable HCC with an acceptable safety profile.
Further randomized studies are warranted. Supported in part by N01-CM-62205.


Topotarget A/S

For further information, please contact:

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

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



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, 5-FU, etoposide 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, or
programmed cell death; promote differentiation; inhibit angiogenesis; and
sensitize cancer cells to overcome drug resistance when used in combination
with other anti-cancer agents.

Intravenous belinostat (IV) is in pivotal trial in peripheral T-cell lymphoma
(PTCL) and is currently being evaluated in multiple clinical trials as a
potential treatment for cancer of unknown primary (CUP), ovarian cancer, small
cell lung cancer, thymoma, liver, soft tissue sarcoma, lymphoma, AML, and
Myelodysplastic Syndrome (MDS), either alone or in combination with other
anti-cancer therapies. Continuous intravenous administration (CIV) is being
evaluated in clinical trials in solid tumors as well as in AML. Topotarget has
a Clinical Trial Agreement (CTA) with the NCI to clinical studies on belinostat
in order to better understand its anti-tumor activity.

About Topotarget A/S
Topotarget (NASDAQ-OMX: TOPO.CO) is an international biopharmaceutical company
headquartered in Copenhagen, Denmark, dedicated to clinical development and
registration of oncology products. Topotarget focuses, in collaboration with
Spectrum Pharmaceuticals, Inc., on the development in pivotal studies of its
lead drug candidate, belinostat, which has shown positive results as a
monotherapy treating hematological malignancies and positive results in solid
tumors. Belinostat may be used in combination with full doses of chemotherapy,
and is in a pivotal trial within PTCL (peripheral T-cell lymphoma). For more
information, please refer to www.topotarget.com.

Topotarget A/S Safe Harbor 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 A/S 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 A/S 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 A/S'
history of incurring losses and the uncertainty of achieving profitability;
Topotarget A/S' stage of development as a biopharmaceutical company; government
regulation; patent infringement claims against Topotarget A/S' products,
processes and technologies; the ability to protect Topotarget A/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.

Meddelelse nr. 02-12 Belinostat resume på ASCO Gastrointestinal Cancers Symposium 2012.pdf