=Shire (SHPG): positive topline Phase 3 results for the SAHARA study

Shire plc announces positive topline Phase 3 results for the SAHARA study

Co announces positive topline Phase 3 results for the SAHARA(TM) study, a global, multi-center, randomized, double-blind, placebo- controlled, partial crossover trial that evaluated the efficacy and safety of subcutaneously administered C1 esterase inhibitor [human] Liquid for Injection, also referred to as SHP616 Liquid, versus placebo over two 14-week treatment periods in patients 12 years of age or older with symptomatic Hereditary Angioedema (HAE). 

• This study met its primary endpoint and all key secondary endpoints. The fixed 2000 IU dose, administered every three to four days as a single 4mL subcutaneous injection, led to a statistically significant and clinically meaningful reduction of 2.32 (95% CI: 1.74 - 2.89, p < 0.0001) attacks/month in the mean HAE attack rate (primary endpoint) compared to placebo. In a commonly reported measure of effectiveness, SHP616 Liquid yielded a median HAE attack rate reduction of 79% from Day 0 (entire treatment period) or 85% from Day 14 (after reaching steady state) compared to placebo. A total of 78% of patients' experienced 50% or greater reduction in HAE attack rate (key secondary) compared to placebo, and 38% of patients were attack free during their SHP616 Liquid treatment period, compared to 9% during the placebo period.
• The 75 patients randomized in this study were required to have at least two HAE attacks per month in the three consecutive months prior to screening, and were representative of the full HAE disease spectrum (88% Type 1 HAE; 12% Type 2 HAE; mean of 11.9 attacks three months prior to screening; 51% had a history of prior use of long term prophylaxis). The study was completed by 77% of patients in the crossover sequences and 87% in the active-only sequence.