Shire: JAMA Publishes Complete Results of Phase 3 HELP Study

Shire (LSE:SHP,NASDAQ:SHPG), the leading global biotechnology company focused on rare diseases, today announced the Journal of the American Medical Association (JAMA) publication of complete results from the Phase 3 HELP Study™, a randomised, placebo-controlled trial evaluating the efficacy and safety of subcutaneously administered lanadelumab versus placebo over 26 weeks in 125 patients 12 years of age or older with hereditary angioedema (HAE).

As quoted in the press release:

Lanadelumab, which is approved under the brand name TAKHZYRO™ in the U.S. and Canada, is a first-of-its-kind monoclonal antibody that inhibits the activity of plasma kallikrein, an enzyme which is uncontrolled in people with HAE, to help prevent attacks. HAE is a rare, genetic and potentially life-threatening disorder that can result in recurrent attacks of oedema (swelling) in various parts of the body.

The study met all primary and secondary endpoints, with all three lanadelumab treatment regimens demonstrating statistically significant reductions in the mean monthly HAE attack rate compared to placebo. At 300 mg every two weeks, lanadelumab reduced the number of mean monthly HAE attacks by 87% relative to placebo (adjusted P<0.001). Patients receiving lanadelumab 300 mg every two weeks had 83% fewer moderate to severe attacks (vs. placebo), 87% fewer attacks that needed on-demand treatment (vs. placebo) and an 89% attack rate reduction (vs. placebo) from day 14 to 182.

Click here to read the full press release.