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Ablynx buoyant as lead antibody aces phase III trial

Caplacizumab met its primary endpoint and reduced aTTP-related death by 74%

AblynxBelgian biotech Ablynx has chalked up a big win with its lead candidate caplacizumab for a rare blood disorder, reporting positive phase III data to help set up a US filing next year.

The new data will also supplement Ablynx’ already-filed accelerated marketing application for caplacizumab in the EU as a treatment for acquired thrombotic thrombocytopenic purpura (aTTP), a life-threatening, relapsing disease in which patients suffer serious blood clots.

The new data from the 145-patient HERCULES phase III trial shows that it reduced the time to restoring normal platelet activity - the primary endpoint - and also cut the combined outcome measure of aTTP-releated death, recurrence of aTTP and/or major thromboembolic events by 74% compared to placebo.

Another secondary measure showed that the drug also reduced the chances of aTTP recurrence by 67%, and the results also backed up the safety profile of the drug, with the only major difference from placebo a greater tendency to cause bleeding-related side effects.

Analysts at Jefferies said the data were as good as could be hoped for, setting up sales of up to $400m with the drug assuming a launch in mid-2018 in Europe and a year later in the US.

Shares in Ablynx were up around 26% at the time of writing, reflecting the positive data and the fact that the Belgian biotech has retained full-ownership of the programme, self-developing it right the way through from discovery to regulatory filings.

If it gets approval - which is looking increasingly assured given the new trial - it will also commercialise the drug on its own and has started setting up its own sales force in anticipation, which is a rare thing for a European biotech company.

“Patients with aTTP are at risk for significant morbidity and early death,” said Robert Zeldin, Ablynx’ chief medical officer. “We believe these positive phase III study results confirm the potential for caplacizumab to address the high unmet medical need in the treatment of aTTP and to have a meaningful impact on the lives of affected patients.”

Ablynx filed for approval of caplacizumab in Europe on the strength of the phase II TITAN trial and if approved will be the first treatment specifically indicated for aTTP, which is currently managed using procedures such as plasma exchange and high-dose corticosteroids.

The trial is also another endorsement of Ablynx’ ‘nanobody’ technology, which come in at around one tenth the size of regular antibodies. The platform has attracted the interest of big pharma partners including Sanofi, which agreed a €2.4bn deal with Ablynx in July.

Article by
Phil Taylor

2nd October 2017

From: Research



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