Copenhagen, Denmark - 9 November 2011 - Today Topotarget A/S (NASDAQ OMX:
TOPO.CO) announced that clinical data on belinostat will be presented at The
American Society of Hematology (ASH) 53rd annual, San Diego 10-13 December 2011


Below is a list of the two clinical abstracts accepted at ASH. The full program
is to be found on http://ash.confex.com/ash/2011/webprogram/


”We are pleased to see the positive outcome and especially the favorable safety
profile of both oral belinostat (abstract 3710) and as an IV infusion in
combination with bortezomib (abstract 2598)” said Axel Mescheder, MD, CMDO. He
continued “Having 4 abstracts accepted for presentation at this prestigious
scientific meeting is very exciting and confirms our dedication and efforts in
further developing belinostat in hematological cancer indications”.


Today's news does not change Topotarget's full-year financial guidance for 2011.


Abstract 3710: Session: 624. Lymphoma - Therapy with Biologic Agents, excluding
Pre-Clinical Models: Poster III, Monday, December 12, 2011, 6:00 PM-8:00 PM,
Hall GH (San Diego Convention Center)


Preliminary Results of An Ongoing Phase I Trial of Oral Belinostat a Novel
Histone Deacetylase Inhibitor in Patients with Lymphoid Malignancies


Jasmine M. Zain, MD1, Francine M. Foss, MD2, Catherine S. Diefenbach, MD3,
Daniel Petrylak, MD4*, Ameet Narwal1*, Ellen Neylon, NP, RN, BSN, OCN5*, Poul
Knoblauch6* and Owen A. O'Connor, MD, PhD7

1Hematology/Oncology, NYU Langone Medical Center, New York, NY
2Yale Medical Oncology, New Haven, CT
3New York University Cancer Institute, New York University Langone School of
Medicine, New York, NY
4Oncology, Columbia University, New York, NY
5Nursing, New York University Langone Medical Center, New York, NY
6TopoTarget, Copenhagen, Denmark
7Department of Medicine, NYU Cancer Institute, NYU Langone Medical Center, New
York, NY


Background: Belinostat (Bel) is a pan class I/II histone deacetylase inhibitor
with broad preclinical activity. A phase I study of oral Bel in patients (pts)
with solid tumors identified a maximum tolerated dose (MTD) of 750 mg orally
(PO) daily on days (d) 1-14, of an every 21 day cycle. An allowance for
intra-patient dose escalation was permitted as long as the higher dose level
was deemed safe and not the maximum administrable dose.. The current study was
initiated to assess the safety and dosing of Bel in patients with relapsed or
refractory Hodgkin and non-Hodgkin Lymphoma. Methods: 3-6 pts per dose cohort
were enrolled on study, at the following doses: A 750; B 1000; C 1250; D1500;E
1750; F 2000mg/d. Pts who met eligibility criteria (ANC = ; plts = ) with
evaluable disease were eligible. Definition of dose limiting toxicity (DLT)
included: related non-hem grade (gr) 3/4 tox; gr 4 neutropenia > 5 d or with
fever > 100.5 °F; gr 4 thrombocytopenia > 7 d.
Results: 28 pts, median age 48 (range 21-82),prior regimens: median 5,5 (range
0-13) , (17 had BM transplants, including 5 pts with allogeneic) have been
enrolled. Diagnoses include: HD (12 pts), mantle cell lymphoma (MCL;5 pts),
other NHL (11 pts). Most frequent adverse events seen in > 50% of patients
(regardless of attribution or gr) in 28 pts fully evaluable for toxicity:
diarrhea (25 pts), anorexia/decreased appetite (24 pts), fatigue (23 pts),
nausea (18 pts), vomiting (18 pts), cough (17 pts) and fever (15 pts). Non-hem
gr 3 events included diarrhea in 9 pts (evenly distributed over the co-horts),
grade 3 diarrhea at 1500 and 2000 mg dose were among the 4 DLT's . 5 pts with
gr 3/4 thrombocytopenia (shift from baseline) were seen in cohort C, D, E. 16
pts are evaluable to date, and include r, 1 CR (duration: 2+cycles)in NHL
patient, 1 PR (duration: 8 cycles) in HD patient, and stable disease have been
noted in 12 patients (duration: 1-24 cycles, median 1,5). Aside from the 1 CR
and 1 PR, tumor shrinkage between 25-50% was noted in 8 pts. Conclusions: Oral
Bel can be delivered safely with a d 1-14, q3w schedule in pts with lymphoma at
a daily dose higher than what has been established for pts with solid tumors.
MTD for lymphoma pts was established at 1500 mg/d 1-14, q3w. The safety profile
and early tumor shrinkage noted in both HD, MCL and other NHL warrants
continued evaluation of Bel, especially in combination with other active
compounds.


Abstract 2598: Session: 615. Acute Myeloid Leukemia - Therapy, excluding
Transplantation: Poster II, Sunday, December 11, 2011, 6:00 PM-8:00 PM, Hall GH
(San Diego Convention Center)


Phase I Trial of Belinostat and Bortezomib in Patients with Relapsed or
Refractory Acute Leukemia, Myelodysplastic Syndrome, or Chronic Myelogenous
Leukemia in Blast Crisis


Beata Holkova, MD1, Mary Beth Tombes, RN1*, Ellen Shrader1*, Sheryl S. Cooke,
RN2*, Wen Wan, PhD1*, Heidi Sankala, PhD1*, Maciej Kmieciak, PhD1*, John D.
Roberts, MD1, Guillermo Garcia-Manero2 and Steven Grant, MD1


1Virginia Commonwealth University, Massey Cancer Center, Richmond, VA
2University of Texas, MD Anderson Cancer Center, Houston, TX


Numerous preclinical studies have demonstrated synergistic interactions between
proteasome and histone deacetylase (HDAC) inhibitors, particularly in B-cell
malignancies (e.g., myeloma and lymphoma). However, investigation of this
strategy in acute leukemias has been limited. Very recent preclinical findings
have shown marked synergism between the HDAC inhibitor belinostat and the
proteasome inhibitor bortezomib administered at very low (sub-micromolar)
concentrations, in various cultured and primary acute myelogenous leukemia and
acute lymphocytic leukemia specimens (Dai Y et al. Br J Haematol. 2011). These
interactions were associated with multiple perturbations in survival signaling
proteins, including inactivation of NF-kappa B, down-regulation of Bcl-xL and
XIAP, and up-regulation of the pro-apoptotic protein Bim. These findings
prompted initiation of a phase I trial with the primary objective of
determining the recommended phase II doses (RPTDs) for the combination of
bortezomib and belinostat in patients with relapsed or refractory acute
leukemia, myelodysplastic syndrome (MDS), or chronic myelogenous leukemia in
blast crisis (CML-BC).

To date, 13 patients have been enrolled. Patients with the following disease
types have been treated: acute leukemia (n=9), MDS (n=3), and CML-BC (n=1).
Patient characteristics include male/female ratio n = 6 (46%)/7 (54%), with a
median age of 59 years [range 27‑75]. ECOG performance score 0-2. The median
number of prior therapies was 2 [range 2-5]. The schedule of administration was
belinostat 30 minutes intravenous (IV) infusion on days 1-5 and 8-12; and
bortezomib IV bolus preceding belinostat on days 1, 4, 8, 11; on a 21 day
cycle. Dose level enrollment was: Level 1 = bortezomib 1.0 mg/m2, belinostat
500 mg/m2 (n=6); Level 2 = bortezomib 1.3 mg/m2, belinostat 500 mg/m2 (n=6);
and Level 3 = bortezomib 1.3 mg/m2, belinostat 650 mg/m2 (n=1). The study is
currently enrolling to dose level 3.

No dose-limiting toxicities (DLTs) have been observed to date. Non-DLTs (CTCAE
v4) include: leukopenia (grade 4, 23%), thrombocytopenia (grade 3, 15%), and
peripheral sensory neuropathy (grade 2, 23%). No serious adverse events have
occurred at unexpected frequency or severity. Two deaths have occurred due to
disease progression.

Of the 13 patients treated, 12 have been evaluable for response. There has been
1 complete response in this heavily pretreated population. This response was
achieved in a patient with biphenotypic acute leukemia, refractory to 7+3 and
Flag-Ida. The patient proceeded to allogeneic hematopoietic stem cell
transplantation. Four patients had stable disease, and 7 patients had
progressive disease. Correlative studies examining leukemic blast expression of
nuclear RelA, Bim, Bcl-xL, and XIAP pre- and post-treatment are ongoing.

Collectively, these findings indicate that a regimen combining belinostat and
bortezomib is well tolerated in patients with relapsed or refractory acute
leukemia, MDS, or CML-BC. The maximum tolerated dose (MTD) has not been
reached. Pending identification of the RPTDs, phase II evaluation of this
therapeutic strategy, if warranted, should define its activity more
definitively.


Preclinical abstracts

Two additional abstracts relating to preclinical evaluations will also be
presented: Marci et al, abstract number 2727, Amengual et al, abstract 3733
both to be presented during Sunday December 11 poster session from 6:00-8:00
PM.


Topotarget A/S


For further information, please contact:


Francois Martelet, CEO Direct: +45 39 17 83 41

Axel Mescheder, CMDO Direct: +45 39 17 83 14

Annette Lykke, IR Direct: +45 39 17 83 44


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 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 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 II in cancer of unknown primary site
(CUP). Topotarget's cancer drug target is HDAC. 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 commercialize 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 including the timing for commencement and completion of clinical
trials and with respect to cash burn guidance. Such statements are based on
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Announcement no. 20-11 Belinostat abstracts at ASH 2011.pdf