Belinostat pivotal BELIEF trial meets primary endpoint
9/21/2012 2:30 AM EST
Topotarget
Company Announcement
Belinostat pivotal BELIEF trial meets primary endpoint
To NASDAQ OMX Copenhagen A/S
Announcement no. 16-12 / Copenhagen, Denmark
September 21, 2012
Topotarget A/S today announced that the primary endpoint has been met for the
belinostat pivotal trial for patients with peripheral T-cell lymphoma (PTCL).
The pivotal BELIEF trial (PXD101-CLN-19) evaluating the efficacy and safety of
belinostat for the treatment of patients with relapsed/refractory PTCL met its
primary endpoint. A Special Protocol Assessment (SPA) agreement with the U.S.
Food and Drug Administration (FDA) requires the BELIEF trial to reach an
objective response rate (ORR) of at least 20% and this objective was met. Data
from the trial are being further analyzed and are expected to be communicated
during Q4 2012.
Under the terms of a 2010 license and collaboration agreement, belinostat is
currently being developed jointly by Spectrum Pharmaceuticals and Topotarget.
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 H1 2013.
Should Spectrum receive FDA’s acceptance to file the belinostat NDA, Topotarget
is entitled to receive one million shares of common stock in Spectrum and a
double-digit million USD cash payment. If belinostat is approved by the FDA,
Topotarget will further receive a double-digit million USD cash payment.
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.
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 five largest EU countries.
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.
Topotarget A/S
For further information, please contact:
Anders Vadsholt, CEO: Direct: +45 39 17 83 45; Cell: +45 28 98 90 55
Axel Mescheder, CMDO: Direct: +45 39 17 83 14; Cell: +45 51 55 71 66
Background information
About Topotarget
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.
Forward-looking 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.
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