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Positive SPA Reply From the FDA for TopoTarget's Pivotal Trial With Belinostat in PTCL

Update on the Regulatory Strategy for Belinostat in PTCL

COPENHAGEN, Denmark, September 5 /PRNewswire-FirstCall/ -- TopoTarget A/S (OMX: TOPO) announced that a positive reply from the FDA was received on a Special Protocol Assessment (SPA) for a phase III trial for belinostat in PTCL (Peripheral T-Celle Lymphoma). This pivotal trial is to enrol approximately 120 patients and is to begin in Q4 2008. In June Fast Track designation was granted for the development of belinostat in this indication which supports TopoTarget's rapid market entry strategy. There is currently no standard therapy approved for PTCL.

"This is very good news. We can now move belinostat into a pivotal phase III. With the SPA in place we have agreed on a design for our pivotal phase III trial with the FDA which will begin in Q4 of this year. In addition with the FDA's Fast Track designation received in June for the PTCL indication we can continue with our strategy of rapid market entry for belinostat in an area with a high unmet medical need." Said Professor, Peter Buhl Jensen, MD, CEO of TopoTarget.


The phase III trial is an open-label, multicenter, single arm efficacy and safety trial in patients with relapsed or refractory peripheral T-cell lymphoma who have failed at least one prior systemic therapy. Approximately 120 patients (100 evaluable patients) will be enrolled. Patients will be treated with 1000 mg/m2 belinostat administered as a 30 minute IV infusion on days 1-5 of every 3-week cycle until there is disease progression or unmanageable treatment-related toxicities.


Belinostat is a small molecule class I and II histone deacetylase inhibitor (HDACi). HDACi's modulate gene expression within tumor cells, including tumor suppressor genes, leading to G1 and G2/M cell cycle arrest, induction of apoptosis (programmed cell death), inhibition of angiogenesis, immune modulation, and promotion of cellular differentiation. Clinical activity of belinostat has been observed in an ongoing Phase II trial of belinostat monotherapy in patients with recurrent or refractory cutaneous (CTCL) or peripheral T-cell lymphoma (PTCL). Efficacy is being analyzed separately in PTCL and CTCL patients. In 11 patients evaluable for response in the PTCL arm, 2 durable complete responses (CR) and 4 Stable Diseases (SD) have been observed as announced at "10th International Conference on Malignant Lymphoma" in Lugano in June. In both arms of the study, the objective response rate in the first stage has met the criteria for expansion to the second stage of a Simon 2-stage design.

Objectives and methods of evaluation The primary study endpoint will be objective response rate (ORR). Objective response is complete response (CR) or partial response (PR) based on independent radiology review. Secondary objectives of the study are to determine: safety of belinostat monotherapy, time to response, duration of response, time to progression (TTP), progression-free survival (PFS), one-year progression-free rate, one-year survival rate and overall survival (OS).

Today's news does not change TopoTarget's full-year financial guidance for 2008.

Background information

About Periferal T-Cell Lymphomas (PTCL)

PTCL represent approximately 10% of all non-Hodgkin's lymphomas (NHL) in Western populations and are associated with a poor prognosis. Most patients with PTCL relapse after initial treatment with cytotoxic agents, and 5-year survival is less than 30%. T-cell Non Hodkins Lymphomas, to which PTCL belongs, are associated with a poorer outcome and survival compared to the B-cell lymphomas. The primary response rate for all PTCL subtypes remains at less than 60%, with nearly all patients relapsing. Median survival of all PTCL patients (excluding a few subtypes) is approximately 3-4 years, with a 5-year survival of less than 30%. There are currently no therapies approved specifically for PTCL. Primary treatment for most subtypes of PTCL remains anthracycline-based regimens, predominantly the combination of cyclophosphamide, doxorubicin, vincristine and prednisone (CHOP). With the exception of ALK-positive ALCL, PTCL subtypes respond poorly to these regimens. The use of radiotherapy, with or without chemotherapy, is preferred as front line treatment of extranodal NK/T-cell lymphoma. The majority of patients with PTCL will relapse after primary therapy. A number of chemotherapy regimens are used for salvage therapy. However, there is currently no consensus regarding the optimal treatment approach for PTCL salvage therapy.

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, cis-retinoic acid, azacitidine and Velcade(R) (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 currently being evaluated in multiple clinical trials as a potential treatment for cutaneous and peripheral T-cell lymphomas, B-cell lymphomas, AML, mesothelioma, soft tissue sarcoma, MDS, and liver, colorectal, and ovarian cancers, either alone or in combination with anti- cancer therapies. An oral formulation of belinostat is also being evaluated in a Phase I clinical trial for patients with advanced solid tumors. Several trials in the belinostat program are conducted under a Clinical Trail Agreement (CTA) under which the NCI sponsors clinical trials to investigate belinostat for the treatment of various cancers, both as a single-agent and in combination chemotherapy regimens. In May 2005, TopoTarget announced the signing of 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.

About TopoTarget

TopoTarget (OMX: TOPO) is an international biotech company headquartered in Denmark, dedicated to finding ''Answers for Cancer'' and developing improved cancer therapies. The company is founded and run by clinical cancer specialists and combines years of hands-on clinical experience with in-depth understanding of the molecular mechanisms of cancer. Focus lies on highly predictive cancer models and key cancer targets (including HDACi, NAD+, mTOR, FasLigand and topoisomerase II inhibitors). TopoTarget has a broad cllinical pipeline with 9 products in development, including belinostat which has shown proof of concept as monotherapy in treating haematological malignancies and positive results in solid tumours where it can be used in combination with full doses of chemotherapy. The company's first marketed product Savene(R)/Totect(R) was approved by EMEA in 2006 and the FDA in 2007 and is marketed by TopoTarget's own sales force in Europe and the US. For more information, please refer to

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 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 expo-sure; 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.

SOURCE TopoTarget
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