Takeda Pharmaceutical Company Limited announces the publication of the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE) designed to evaluate the long-term safety (primary endpoint) and efficacy of Takhzyro (lanadelumab) 300mg every two weeks for up to 2.5 years. The study results show that preventative treatment with Takhzyro markedly reduced the frequency of HAE attacks in patients 12 years of age and older who received treatment for a mean duration of almost 2.5 years (29.6 months; 8.2 standard deviation). The data were published online this month in the journal Allergy.
Secondary endpoints of the study showed the mean (min;max) HAE attack rate observed in the study population (N=209) was reduced by 87.4 percent (-100; 852.8) overall versus baseline, and attacks requiring acute treatment (N=106) were reduced by 93.4 percent (-100; -52.8). Reductions were also shown (N=209) in the rate of moderate or severe attacks (84.3 percent). Patients treated with Takhzyro 300 mg every two weeks reduced the HAE disease burden by being attack-free for a mean (SD) of 97.7 percent (6.0 percent) of days during treatment, and the average duration of the attack-free period was 14.8 months. Nearly 7 out of 10 patients (68.9 percent) experienced an attack-free period of more than 12 months (n=209). Treatment-related treatment-emergent adverse events (TEAEs) were reported by 54.7 percent of patients (N=116), most commonly injection site reactions. There were no reports of serious treatment-related TEAEs.
The validated Angioedema Quality of Life Questionnaire (AE-QoL) was among the patient-reported outcome tools used to evaluate patients’ quality of life (QoL). Both rollovers and non-rollovers achieved the minimal clinically important difference for the AE-QoL total score: the mean (SD) change in total score from baseline was –10.2 (17.9) and –19.5 (21.3) for rollovers and non-rollovers, respectively. Most of the improvements in AE-QoL scores were observed during the early follow-up period (from day 0 to 56), before reaching a plateau, and scores were generally maintained during subsequent visits.
“Effective prevention backed by clinical evidence is critical for healthcare professionals who treat patients with HAE,” says Aleena Banerji, M.D., Division of Rheumatology, Allergy and Immunology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School and principal investigator for the HELP Study. “The potential to be attack-free for periods of time can help to provide an additional sense of assurance for those living with this chronic and unpredictable disease.”
The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it one of the largest randomized, controlled prevention studies in HAE, with the longest active treatment duration, to date. The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years. The complete results were based on data collected between May 2016 and October 2019 and included 109 rollover patients who were originally evaluated in the HELP Study, and 103 eligible non-rollover patients who did not participate in the initial study but had experienced at least one HAE attack in 12 weeks. The complete results from the HELP Study OLE showed that the safety profile of TAKHZYRO was consistent with the original findings from the HELP Study, with treatment-related treatment-emergent adverse events (TEAEs) occurring in 54.7 percent of patients (n=116) and the most common being injection-site pain, upper respiratory tract infection, or headache.
“This study supports the use of Takhzyro as a long-term preventative treatment option for those 12 years of age and older living with HAE who are seeking a preventative treatment option that is proven to reduce HAE attacks,” says Neil Inhaber, M.D., Vice President, Global Medical Heald, HAE and Transplant at Takeda. “Takeda has more than 10 years supporting people with this rare disease across the HAE portfolio and we are committed to providing these patients with effective treatment options that may help them experience periods of time without attacks. Our legacy and dedication to HAE hopefully empowers patients to confidently navigate their HAE journey.”