Copenhagen, Denmark - 19 May 2011 - Topotarget A/S (NASDAQ OMX: TOPO.CO)
announced today that clinical data on belinostat will be presented at the 2011
Annual Meeting of the American Society of Clinical Oncology (ASCO) to be held
June 3-7, 2011 at the McCormick Place Convention Center in Chicago.

Shown below are the summaries of the abstracts that are now available for
viewing on the ASCO.org website (www.asco.org).


A phase I and pharmacodynamic (PD) study of the histone deacetylase (HDAC)
inhibitor belinostat (BEL) plus azacitidine (AZC) in advanced myeloid
malignancies.


Abstract No: 6521


Session: Leukemia, Myelodysplasia, and Transplantation. Type: Poster Discussion
Session, Time: Friday June 3, 2:00 PM to 6:00 PM. Location: McCormick Place
E450b

Discussion Time: Friday June 3, 5:00 PM to 6:00 PM. Location: McCormick Place
Arie Crown Theater.


Author(s): O. Odenike, L. A. Godley, J. Madzo, T. Karrison, M. Green, A. S.
Artz, R. J. Mattison, K. W. L. Yee, M. Bennett, N. Fulton, G. Koval, G.
Malnassy, R. A. Larson, M. J. Ratain, W. Stock; The University of Chicago,
Chicago, IL; University of Wisconsin, Madison, WI; Princess Margaret Hospital,
Toronto, ON, Canada.


Abstract:


Background: We hypothesized that targeting two mechanisms of epigenetic
silencing with a potent HDAC inhibitor BEL plus the DNA methyltransferase
inhibitor AZC would be additive or synergistic with regard to transcriptional
de-repression and up-regulation of specific target genes.


Methods: Part 1 of the study was a dose escalation phase to establish the MTD
of BEL in combination with a fixed dose of AZC. 3 to 6 pts were enrolled per
dose level in the absence of a DLT or until the 1000mg/m2 dose level of BEL was
attained. AZC was given at a dose of 75mg/m2/d SC on days 1-5, with escalating
doses of BEL given IV over 30 minutes on the same days in a 28 day cycle. In
part 2, pts were randomized during cycle 1 to AZC alone, or BEL/AZC at the
established MTD of BEL. Part 2 was designed to evaluate the relative
contribution of BEL to the combination, based on change in PD variables
analyzed at day 5 of therapy in comparison to baseline, in cycle 1. In
subsequent cycles all pts could receive BEL/AZC.

Results: 56 pts were enrolled, part 1 (n=24), part 2 (n=32). Median age 68,
range (42-83). AML de novo=53% t- AML/MDS=18%, MDS/CMML=27%, PMF=2%; 68% had
primary refractory or relapsed disease, 43% had poor risk cytogenetics. In part
1, dose escalation was feasible up to the 1000mg/m2 dose level of BEL, without
first course DLT. In part 2, 18 pts (9 in each arm) had marrow samples at
baseline and at day 5 for q-RT-PCR analysis for p21, MDR1 and HIST1H3H
transcript levels. At day 5, MDR1 was significantly upregulated in the BEL/AZC
arm when compared with AZC alone arm (p=0.0089), in contrast to p21 and
HIST1H3H which were not significantly different between the 2 arms. There
were18 responses (CR=6, marrow CR =2, PR= 1, HI-P=7, HI-N=2); 13 of these
occurred at the 1000mg/m2 dose level of BEL.


Conclusions: The combination of BEL and AZC is feasible, responses have been
observed in several pts at the RP2D of 1000mg/m2 of BEL plus 75mg/m2 AZC. A
significant upregulation in MDR1 was observed in the BEL/AZC arm at day 5
compared with the AZC alone arm, suggesting a relative biologic contribution of
BEL to the combination. The clinical correlates of these observations require
evaluation in the context of a larger trial.



Management of thymic epithelial tumors (TETs) at the National Cancer Institute
(NCI).


Abstract No: e17511


Publication-only abstracts (abstract number preceded by an "e"), published in
conjunction with the 2011 Annual Meeting but not presented at the Meeting, can
be found online only. The publication-only abstracts are not included in the
print or USB versions of the ASCO Annual Meeting Proceedings Part I, but they
are citable to the Journal of Clinical Oncology.


Author(s): A. Rajan, R. J. Kelly, A. Lopez-Chavez, C. A. Carter, E. Szabo, B.
Scepura, M. J. Manu, A. W. Berman, G. Giaccone; National Cancer Institute,
Bethesda, MD.


Abstract:


Background: TETs are very rare anterior mediastinal tumors.


Methods: We performed a retrospective review of patients (pts.) with TETs
presenting to the NCI for enrollment in ongoing clinical trials,
standard-of-care therapy or a second opinion.


Results: Seventy-three pts. were seen between 12/2007 and 12/2010; 43 males
(41%), median age at diagnosis: 48 years (range, 17 - 81). Fifty-four pts. were
Caucasian (74%), 7 (10%) African-American, 8 (11%) Asian and 4 (6%) Hispanic.
Histology: B1 5 (7%), B2 13 (18%), B2/B3 1 (1%), B3 9 (12%), thymic carcinoma
(TC) 35 (48%), atypical carcinoid 2 (3%) and unclassified thymoma 8 (11%).
Fifty pts. were enrolled in clinical trials: 28 on a phase II study of the HDAC
inhibitor, belinostat (B), 27 on a phase II study of the IGF -1R inhibitor
cixutumumab (C) (including 10 previously on B), 3 on a phase I study of
chemotherapy (PAC) plus B and 2 on a phase II study of the cyclin-dependant
kinase inhibitor PHA-848125AC. Median number of previous systemic therapy
regimen that had been administered was 2 (range, 0-10). Forty-three pts. (59%)
had extra-thoracic metastases; liver, bone and lymph nodes were the most common
sites. Fourteen pts. (19%) had at least one autoimmune disease; myasthenia
gravis (10%) was the most common. Three out of six pts. with TC with
neuroendocrine features had Cushing's syndrome. Eleven pts. (15%) had
opportunistic infections including shingles (8%) and candidiasis (4%).
Forty-eight pts. On clinical trials were evaluable for response: B: 2PR, 18SD,
8PD; C: 1PR, 16SD, 8PD; PACB: 1PR, 2SD; PHA: 2SD. Median survival for all pts.
was 117 months (T - not reached; TC - 56 months). Among 28 pts. enrolled on B,
the only clinical predictor of response and survival was presence of
intra-thoracic disease only. Of 12 tumors (B1-1, B2-2, B3-1, C-7, atypical
carcinoid-1) evaluated for overexpression of HER2 by IHC, none was positive.
One patient with TC had a c-KIT mutation in exon 11.


Conclusions: Histologic diagnosis of thymoma and the presence of intra-thoracic
disease only predicts for longer survival in pts. with TETs. Targeted drugs can
induce prolonged disease stabilization and few responses in heavily pretreated
patients. Molecular profiling of tumors will be necessary in order to advance
the field.


This announcement has no impact on Topotarget's financial 2011 expectations.



Topotarget A/S


For further information, please contact:

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

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

Axel Mescheder, CDMO Direct: +45 39 17 83 14; Mobile: +45 51 55
71 66

Annette Lykke, IR Direct: +45 39 17 83 44; Mobile: +45
23 28 98 14



Background information


About belinostat

Topotarget 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 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 tumours 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

Topotarget (NASDAQ OMX: TOPO.CO) is an 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 other chemotherapeutic agents, and is currently in a pivotal trial
within PTCL (peripheral T-cell lymphoma) and phase II in cancer of other
unknown primary site (CUP). Topotarget's cancer drug targets are HDAC, NAD+,
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. For more information, please refer to
www.topotarget.com.



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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
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there can be no assurance that actual results or business conditions will not
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following: The risk that any one or more of the drug development programs of
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reasons or due to patient enrollment issues or based on new information from
non-clinical or clinical studies or from other sources; the success of
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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.

Announcement no. 11-11 Belinostat abstracts at ASCO 2011.pdf