To NASDAQ OMX Copenhagen A/S Announcement no. 26-13 / Copenhagen, November 20, 2013
Topotarget announces the final results from the first stage of the phase II part of the phase I/II clinical trial of belinostat in combination with doxorubicin in patients with soft tissue sarcomas (STS) (PXD101-CLN-14).
Trial results The phase I dose escalation part of this study showed that belinostat at a recommended dose of 1000 mg/m2 days 1-5 in combination with 75 mg/m2 doxorubicin on day 5 in a three-week schedule is well-tolerated. Therefore, this dose was used for 20 patients with STS in the phase II part of the study. The study has been closed as an evaluation of the role of belinostat would require a randomized study.
Eighteen out of 25 patients (72%) in the dose-escalation phase of the CLN-14 study achieved disease control and 12 patients remained in disease control at the three-month time point. In this group, the median time to progression was 3.7 months. Eleven out of 16 patients (69%) in the maximum tolerated dose-expansion phase achieved disease control and 8 of these patients remained in disease control at the three-month time point. In the expansion phase, the median time to progression was six months. Four objective responses were seen, including one complete response, and 25 patients achieved disease stabilization. The duration of response was from 93-345 days.
Study design The CLN-14 study was conducted by Topotarget and was an open-label, multicenter, dose-escalation phase I/II study to evaluate safety and efficacy of the combination of belinostat with doxorubicin in patients with advanced solid tumors (the phase I part of the study) and STS (the phase II part of the study).
About soft tissue sarcoma Sarcomas are a group of solid tumors in the connective tissue of the body that are treated with surgery, chemotherapy, and/or radiation. Reported objective response rates are very low with complete responses being rare or absent in the trials that have led to registration of anticancer treatments in this indication over the past five years. Doxorubicin as a single agent or in combination with ifosfamide is the most commonly used chemotherapeutic regimen in patients with advanced STS.
Next step Further development of belinostat in this indication would require a randomized trial. The final results from CLN-14 will be a part of the strategic evaluation of the further development of belinostat.
Outlook for the year This announcement does not change the previously announced outlook statement published on November 7, 2013 and does not affect the filing of an NDA for belinostat in PTCL, which is still expected to take place in Q4 2013.
Topotarget A/S
For further information, please contact: Anders Vadsholt, CEO – direct: +45 39178345
Background information
About Topotarget Topotarget (NASDAQ OMX: TOPO) is a Scandinavian-based biopharmaceutical company headquartered in Copenhagen, Denmark, dedicated to the clinical development and registration of oncology products. In collaboration with Spectrum Pharmaceuticals, Inc., Topotarget focuses on the development of its lead drug candidate, belinostat, which has shown positive results in the treatment of hematological malignancies and solid tumors, obtained by both mono- and combination therapy. For more information, please refer to www.topotarget.com.
Topotarget Safe Harbor Statement This announcement may contain forward-looking statements, including statements about Topotarget A/S’ expectations to the progression of Topotarget A/S’ clinical pipeline and with respect to cash burn guidance. Such statements are subject to risks and uncertainties of which many are outside the control of Topotarget A/S, and which could cause actual results to differ materially from those described. Topotarget A/S disclaims 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 Danish law.
