Positive SPA Reply From the FDA for TopoTarget's Pivotal Trial With Belinost...( Update on the Regulatory St...)

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.

Design

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.

Rational

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. Clinic
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