Shire plc announced new Phase 3 results from the HELP Study™, a global, multi-center, randomized, double-blind placebo-controlled parallel group trial that evaluated 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). Lanadelumab is an investigational treatment being evaluated for the prevention of angioedema attacks in patients with HAE, a rare genetic disease characterized by recurrent swelling of extremities, gastrointestinal tract, and upper airways.
“The data presented today further support the overall clinical profile of lanadelumab, which has been shown to significantly reduce the number of monthly HAE attacks when administered subcutaneously every two or four weeks,” said Jennifer Schranz, Global Development Lead, HAE, Shire. “HAE places a large burden on those living with the disease. We believe that this investigational treatment, if approved, has the potential to transform the treatment landscape for people with HAE.”
The HELP Study is the largest prevention study in HAE conducted to date. Data will be highlighted in three poster presentation discussions at the 2018 American Academy of Allergy, Asthma & Immunology (AAAAI) and World Allergy Organization (WAO) Joint Congress. Patients in the study with ≥ 1 attacks per month were randomized into one of four treatment arms to receive repeated subcutaneous administrations of lanadelumab 300 mg every two weeks, 300 mg every four weeks, 150 mg every four weeks or placebo in a 2:1 (lanadelumab to placebo) ratio.
“The data on lanadelumab continue to demonstrate the importance of controlling plasma kallikrein activity, which is chronically uncontrolled in those living with HAE – even between attacks,” said Marc Riedl, M.D., Professor of Medicine and Clinical Director at the US HAEA Angioedema Center at The University of California San Diego and clinical trial investigator. “With its targeted mechanism of action, lanadelumab, if approved, has the potential to significantly reduce the number of monthly attacks for people with HAE.”
About the HELP Study™
The HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ is an international, multi-center, randomized, double-blind placebo-controlled parallel group, Phase 3 trial that evaluated the efficacy and safety of subcutaneously administered lanadelumab versus placebo over the course of 26 weeks.
The study population comprised 125 patients age 12 or older with HAE Type I or II who were randomized to one of four groups: 150 mg of lanadelumab every four weeks (150 mg Q4W), 300 mg every four weeks (300 mg Q4W), 300 mg every two weeks (300 mg Q2W), or placebo.
The most commonly reported treatment-related adverse events in patients treated with lanadelumab during the treatment period were injection site pain, viral upper respiratory tract infection, headache, injection site erythema, injection site bruising, and dizziness. Most adverse events were mild to moderate in severity.
No treatment related serious adverse events (SAEs) or deaths were reported. One subject experienced two related severe TEAEs leading to discontinuation. There were no safety signals identified in terms of any clinical laboratory test abnormalities, vital signs, physical examinations, or ECGs.