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-Manero2and 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