TOPOTARGET A/S 
By - Registrational trial under a Special Protocol Assessment by FDA
- Granted Fast Track and Orphan Drug Designation by FDA
- On track to file New Drug Application in 2012
Topotarget and Spectrum Pharmaceuticals have now completed the enrollment of a minimum of 100 evaluable relapsed or refractory peripheral T-cell lymphoma (PTCL) patients in the PTCL registration trial. Belinostat, a novel HDAC inhibitor, is being evaluated, under a Special Protocol Assessment (SPA), as monotherapy treatment for relapsed or refractory PTCL, an indication for which it has been granted Orphan Drug and Fast Track Designation by the U.S. Food and Drug Administration (FDA). Central review of pathology for all patients entered into the trial is currently ongoing.
“It is of major importance to us that we successfully achieved this key milestone by completing the enrollment of the pivotal BELIEF trial for the treatment of relapsed or refractory PTCL” says Francois Martelet, MD, CEO of Topotarget. “We are delighted that our lead project is on track for filing in the US”.
“We are pleased to announce that belinostat’s pivotal, registrational trial in relapsed/refractory PTCL has achieved its target enrollment,” says Rajesh C. Shrotriya, MD, Chairman, Chief Executive Officer, and President of Spectrum Pharmaceuticals. “Belinostat is a novel HDAC inhibitor that has the potential to play an important role in therapy for PTCL, an often lethal disease with significant unmet medical need, and in a variety of other indications. It has shown to be active as a single agent and in combination with other chemotherapeutic agents in multiple clinical trials. We plan to file a New Drug Application in 2012. If approved, we believe belinostat will add value to our existing hematology/oncology portfolio, increase our current sales efforts in hematology/oncology, and demonstrate our commitment to developing novel treatments for lymphoma.”
According to the Lymphoma Research Foundation ( www.lymphoma.org), lymphoma is the most common blood cancer. The two main forms of lymphoma are Hodgkin’s lymphoma (HL) and non-Hodgkin’s lymphoma (NHL). Lymphoma occurs when lymphocytes, a type of white blood cell, grow abnormally where they reside in the lymph glands. The body has two main types of lymphocytes that can develop into lymphomas: B-lymphocytes (B-cells) and T-lymphocytes (T-cells). PTCL comprises a group of rare and aggressive NHLs that develop from mature T-cells. PTCL accounts for approximately 10 to 15% of all NHL cases in the US.
About the BELIEF Registrational trial
Belinostat is currently in a registrational, pivotal trial, under a SPA, as monotherapy for relapsed and refractory PTCL, an indication which has been granted Orphan Drug and Fast Track Designation by the FDA. The registrational trial is an open-label, multicenter, single arm efficacy and safety trial in patients with relapsed or refractory PTCL, who have failed at least one prior systemic therapy. The primary endpoint is centrally reviewed objective overall response rate (ORR).
Today’s news does not change Topotarget’s full-year financial guidance for 2011.
Topotarget A/S
For further information, please contact:
Francois Martelet, CEO: Direct: +45 39 17 83 41; Mobile: +45 51 32 83 41
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
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 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.
Belinostat is in pivotal trial in peripheral T-cell lymphoma (PTCL) and is currently being evaluated in multiple clinical trials using IV and oral formulation as a potential treatment for cancer of unknown primary (CUP), a randomized, controlled Ph II trial, 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 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 enrollment 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 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.
