JAMA publishes results of HELP trial of Takhzyro for hereditary angioedema. Shire/Takeda

Shire plc has announced the Journal of the American Medical Association (JAMA) publication of complete results from the Phase III 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). The HELP Study is the largest randomised controlled prevention study ever conducted to date in HAE. 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.>

A clinically meaningful improvement was also observed in 81% of patients treated with lanadelumab 300 mg every two weeks based on the Angioedema Quality of Life Questionnaire (AE-QoL) compared to 37% of patients in the placebo group. The AE-QoL measures the impact of angioedema over a four-week period across four domains: fear/shame, functioning, fatigue/mood, and nutrition..

See- "Effect of Lanadelumab Compared With Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial"- leena Banerji, MD; Marc A. Riedl, MD, MS; Jonathan A. Bernstein, MD; Marcus Maurer, MD; for the HELP Investigators. et al. JAMA. 2018; 320(20):2108-2121. doi: 10.1001/jama.2018.16773.