Shire’s experimental HAE drug hits phase 3 marks

Shire has posted positive results for its late-stage hereditary angioedema candidate SHP616 against a dummy treatment.

The biopharma’s subcutaneous C1 esterase inhibitor significantly lowered hereditary angioedema (HAE) monthly attack rate against placebo in the pivotal phase 3.

Data from the Sahara test show that median HAE attack reduction hit 79% from day 0, and 85% from day 14. Shire also found that 38% of patients were HAE attack free while on its med.

Its target is a rare genetic disease characterized by recurrent swelling of extremities, gastrointestinal tract and upper airways.

“Patients want and deserve options when it comes to their treatment for HAE,” said Dr. William Lumry, clinical professor of Internal Medicine at Southwestern Medical School, Dallas, Texas. “These results are clinically significant, meaningful and relevant to HAE patients whose needs are currently not met today.”

Howard Mayer, M.D., interim head of R&D at Shire, added: “We are very pleased with the strong results of this study, which demonstrated efficacy with a low volume dosing regimen, and what it potentially could mean for the global HAE community, if approved.”

Shire already sells HAE meds, but the patent expiry on one, Firazyr—used to treat acute HAE attacks—is due next year, and will put almost $600 million in sales at risk of generic erosion.

Shire is now looking to its pipeline for the next generation, and on top of SHP616, is also working on lanadelumab (a.k.a. SHP643), the kallikrein-blocking antibody it got after buying Dyax for $5.9 billion two years ago.

A few months back, that candidate cut the rate of monthly attack in patients with HAE by an impressive 87% compared to placebo in the phase 3 Help trial, setting it on track for a filing in the U.S. later this year, or in early 2018.