Written by John Carroll

When Lexington, MA based Shire Ltd. (SHPG) bought out Dyax for up to $6 billion (with an additional...CVR worth $656 million if the drug is approved) in 2015, the big prize was a new drug for hereditary agioedema 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. Shares of the $55 billion biotech were up almost 4% on the news by mid-morning Thursday.

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...

Many analysts believe that Shire...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.

Said Professor Marcus Maurer, M.D., Charité –Universitätsmedizin Berlin, Germany and clinical trial investigator:

“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. Patients with HAE want to live independently and without fear of an angioedema attack.”

...The Phase III results mark another big win for CEO Flemming Ornskov...[who said in a statement]:

“We are extremely encouraged by these topline Phase III results.

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.”