To NASDAQ OMX Copenhagen A/S
Announcement no. 03-13 / Copenhagen, March 5, 2013
Final top-line data confirm that the primary endpoint was met for the belinostat pivotal trial for patients with relapsed or refractory peripheral T-cell lymphoma (PTCL) with an encouraging objective response rate.
Top-line data show an objective response rate (ORR) in the efficacy analysis set which is above the protocol criterion for a positive outcome of the trial. Belinostat thus has an ORR that is on par with the accelerated approved drugs Folotyn™ and Istodax®. A Special Protocol Assessment agreement with the US Food and Drug Administration (FDA) required the CLN-19 BELIEF trial to reach an ORR of at least 20%. The FDA has granted belinostat Orphan Drug and Fast Track designation for the treatment of PTCL. All patients who received at least one dose of belinostat and had a confirmed PTCL diagnosis by the central pathology review were included in the efficacy analysis set. The responses have been confirmed by central independent radiology review. Final data have been submitted for presentation to the 2013 ASCO annual meeting.
“Our belief in belinostat has never wavered and the encouraging objective response rate together with the earlier announced favorable safety profile further supports the potential of our product. We strive to improve the lives of cancer patients and this is a large step in the right direction" said Anders Vadsholt, CEO of Topotarget.
Favorable safety profile
On January 24, 2013, Topotarget announced that preliminary safety data from the BELIEF trial in PTCL, presented at the T-Cell Lymphoma Forum 2013 in San Francisco, USA, showed that belinostat has a favorable safety profile and that the compound has the potential to become a well-tolerated alternative for the treatment of PTCL. The favorable safety outcome from this trial, as well as experience from earlier belinostat trials, shows that full doses of belinostat can be combined with other cytotoxic regimens making combination therapy for cancer patients feasible. The low incidence of myelosuppression is of special notice as this offers the opportunity to combine belinostat with other cytostatic agents for the treatment of patients with PTCL.
New Drug Application
Belinostat is currently being developed jointly by Topotarget and Spectrum Pharmaceuticals. Spectrum possesses the commercial rights to market belinostat in North America and India. A New Drug Application (NDA) for belinostat in PTCL is expected to be filed with the FDA by Spectrum in mid-2013.
A conference call will be held today, March 5, 2013, at 1:00 pm CET.
CEO Anders Vadsholt will make a presentation of the BELIEF study and answer questions. The teleconference will be conducted in English. A presentation will be available on Topotarget’s website, www.topotarget.com, before the start of the conference call.
To participate in the conference call please dial:
Conference ID: 19438536
You will be able to stream the telephone conference via the following link:
After the call, a playback will be available via the same link.
For further information, please contact:
Anders Vadsholt, CEO: Direct: +45 39178345; Cell +45 28989055
Topotarget (NASDAQ OMX: TOPO) is an international biopharmaceutical company headquartered in Copenhagen, Denmark, dedicated to 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.
Belinostat is a novel pan-HDAC inhibitor in late-stage clinical development with more than 1,000 patients treated. Belinostat has a promising safety profile which allows combination with traditional chemotherapy. Preclinical experiments demonstrated belinostat to be effective against multiple cancers by inhibiting cell proliferation and inducing programed cell death (apoptosis) in tumor cells. Belinostat has been tested in a number of phase I/II clinical trials in hematological cancers and solid tumors both in mono- and combination therapy. Data from these trials have provided evidence of the anti-tumor effect of belinostat, including as monotherapy in PTCL and cutaneous T-cell lymphoma (CTCL), liver cancer, and thymoma.
PTCL is a hematological disease including a heterogeneous group of malignancies of T-cell origin that represents approximately 10-15% of all cases of non-Hodgkin’s lymphoma. PTCL is an aggressive, high-grade type of cancer with a poor prognosis of expected average survival of approximately two years from diagnosis without treatment. The projections for annual cancer incidences point to 15,500 new cases of PTCL in the US, Japan, and Europe.
About the BELIEF trial
BELIEF is a pivotal, open-label, multi-center, single-arm efficacy and safety trial of i.v. belinostat in patients with relapsed or refractory PTCL. FDA has granted belinostat Orphan Drug and Fast Track designation for the treatment of PTCL. The trial was initiated in December 2008 and recruitment was completed with 129 patients in September 2011. In total, the study included approximately 100 clinical centers globally.
FOLOTYN™ (pralatrexate), a folate analogue metabolic inhibitor, was discovered by the Sloan-Kettering Institute for Cancer Research, SRI International and Southern Research Institute and developed by Allos Therapeutics. In September 2009, the FDA granted accelerated approval for FOLOTYN™ for use as a single agent for the treatment of patients with relapsed or refractory PTCL. The indication is based on an overall response rate of 27%1. FOLOTYN™ has been available to patients in the US since October 2009. Allos Therapeutics was acquired by Spectrum Pharmaceutical in September 2012.
ISTODAX® (romidepsin), an HDAC inhibitor, was developed by Gloucester Pharmaceuticals. In June 2011, the FDA granted accelerated approval for a Supplemental New Drug Application (sNDA) for an additional indication for ISTODAX® for injection for the treatment of peripheral T-cell lymphoma (PTCL) in patients who have received at least one prior therapy. ISTODAX® is also approved for the treatment of cutaneous T-cell lymphoma (CTCL) in patients who have received at least one prior systemic therapy. These indications are based on response rates. The PTCL response rate was 25%2. Gloucester Pharmaceuticals was acquired by Celgene in January 2010.
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.
2. Coiffier et al, Journal of Clinical Oncology, February 20, 2012