Shire’s multibillion-dollar gamble on lanadelumab pays off with stellar PhIII data, quick marketing application

Flemming Ornskov

When Shire bought out Dyax for up to $6.5 billion in 2015, the big prize was a new drug for hereditary angioedema dubbed DX-2930 which posed a direct threat to Cinryze, a franchise drug for the rare disease player.

Thursday morning, Shire CEO Flemming Ornskov says their multi-billion dollar bet paid off with stellar Phase III data for the drug, now called lanadelumab (SHP643).

A 300 mg dose of the drug twice a month delivered an 87% reduction in mean HAE attack frequency, compared to 0% in the placebo group. It hit the primary endpoint with “highly” statistically significant results as well as all the secondaries.

Already designated a breakthrough therapy with orphan drug status, Shire says it will now race to the FDA later this year or early next with a biologics application, with plans to capitalize on the results as soon as possible.

Shares of the $55 billion biotech $SHPG surged 6% on the news in pre-market trading.

Many analysts believe that Shire, which swelled in size after it completed the Baxalta and Dyax acquisitions, is on a short path to blockbuster revenue. Shire itself set a goal of $2 billion in peak sales for this drug. And EvaluatePharma recently picked it as a top-10 orphan drug bound for a booming market with 6-figure price tags.

“The possibility of a new way to address the underlying cause of HAE to prevent attacks could transform how we treat the disease in the future,” said Professor Marcus Maurer, M.D., Charité –Universitätsmedizin Berlin, Germany and clinical trial investigator. “Patients with HAE want to live independently and without fear of an angioedema attack.”

Shire paid close to $6 billion for Dyax, largely on the promise of the next-gen HAE therapy. And Ornskov added a $4 CVR worth $656 million if the drug is approved. That payoff could be looming in 2018 if the data passes muster with regulators.

The Phase III results mark another big win for CEO Flemming Ornskov, who was determined to transform the company, based in Lexington, MA., after he took over. Shire went on to experience some bumps along the road, but in the longterm his strategic plan and willingness to bet big on new drugs has been paying off.

“We are extremely encouraged by these topline Phase III results,” said Ornskov in a statement, “We have nearly a decade of experience and a strong portfolio and pipeline in HAE and believe these data demonstrate high potential for transforming the way patients living with this condition are treated.”

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