Results

Ablynx spikes as PhIII aTTP study comes through with promising data, setting up an FDA application

Ablynx shares {$ABLX BB} surged 25% this morning after the Belgian biotech reported that its lead drug for an ultra-rare blood clotting disease hit the primary and two key secondary endpoints in the pivotal Phase III study. And with the European application already under review with the mid-stage results, Ablynx says it will line up a US filing planned for next year.

Edwin Moses

The drug caplacizumab targets acquired thrombotic thrombocytopenic purpura (aTTP), a disorder that triggers low platelet counts. In the Phase III study, researchers reported top-line results that included a significant (p=0.01) reduction in the time it took for the platelet counts to return to normal for the drug arm — a likely indicator for the prevention of microvascular thrombosis.

There was also a 74% drop in the percentage of patients to die, see disease recurrence or at least one major thromboembolic event — a secondary driven largely by a drop in recurrence. And in the overall study period there was a 67% drop in recurrence.

Two other secondaries — patients refractory to treatment or the trend to faster normalization of the organ damage markers — failed, though researchers say they saw some encouraging trends in their favor.

Jefferies’ Peter Welford says he’s encouraged, noting:

Commercializing capla itself we assume launches mid-2018E in EU and 1H19E in US for highly profitable $400m WW peak sales from 60% penetration, for a €12/share NPV at 60% probability.

Ablynx CEO Edwin Moses had this to say:

I am delighted by this outcome as it reinforces all our beliefs in the potential for caplacizumab to change the lives of patients affected by aTTP. This is a very important milestone for the Company as it further validates our Nanobody platform and demonstrates our ability to discover and develop medicines that make a real difference for society. These results strengthen our resolve to obtain marketing approval as quickly as possible so that caplacizumab rapidly becomes available to patients suffering from this severe disease for which there is currently no approved drug available.


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