At the EAACI Hybrid Congress 2021, BioCryst Pharmaceuticals, Inc. announced that HAE patients who were randomized to receive 150 mg of oral, once-daily Orladeyo® (berotralstat) at the start of the APeX-2 trial had an 80 percent average reduction in their mean attack rate per month during weeks 25-96 of the trial, compared to baseline. Median attack rates also decreased from 2.7 attacks/month at baseline to 0.0 attacks per month in 16 of 17 months through the same period.
Orladeyo was generally well-tolerated during the treatment period with fewer drug-related adverse events reported in part 3 (weeks 49-96) as compared to part 1 (weeks 0-24) and part 2 (weeks 25-48). Eighty-one percent of the patients who entered part 3 completed the trial.
“The long-term data we now see from two years of therapy in the clinical program reinforces the substantial, sustained reduction in HAE attacks patients experienced with Orladeyo. These results are consistent with the experience many patients are having in the real world since we launched Orladeyo, which is a key driver for the strong patient demand we are seeing as patients switch from injectable prophylactic agents and injectable acute-only therapies to oral, once-daily Orladeyo for control of their HAE attacks,” says Dr. William Sheridan, Chief Medical Officer of BioCryst.
Additional Presentations of New Orladeyo Data at EAACI
On-demand medication use was reduced in HAE patients treated with Orladeyo (150 mg) in APeX-2: In HAE patients taking oral, once-daily Orladeyo 150 mg who had a ≥50 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was a 78 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.1 fewer doses of on-demand medication per month. In patients who had a ≥70 percent reduction in their rate of investigator-confirmed attacks relative to their baseline attack rate, there was an 85 percent reduction in the use of on-demand medication (doses/month) from baseline to week 24, leading to 2.2 fewer doses of on-demand medication per month.
Orladeyo demonstrated consistently low attack rates in adolescent patients in APeX-S: In an analysis of adolescent patients (ages 12-17) treated with oral, once-daily Orladeyo 150 mg in the open-label safety study, APeX-S, the mean (SEM) attack rate at week 4 was 0.4 attacks/month, which was generally sustained through week 48. Median attack rates in these adolescents were 0.0 attacks/month throughout the 48 weeks of treatment.
Greater than 70 percent of patients were attack-free in weeks 4 to 48. Orladeyo was generally well-tolerated in APeX-S.
Orladeyo demonstrated consistently low HAE attack rates during COVID-19: Stress is a documented trigger for HAE attacks and recently published physician and patient survey data shows an increase in patient-reported HAE attack rates due to an increase in stress related to the COVID-19 pandemic (pre-COVID: 1.5 attacks/3-months vs during COVID: 4.4 attacks/3-months). In an analysis of HAE patients taking oral, once-daily Orladeyo 150 mg in the APeX-S trial, monthly HAE attack rates, pre-COVID and during COVID, remained consistently low, <1 attack/month, for patients treated with Orladeyo. Patients receiving Orladeyo maintained low attack rates during this time of high societal stress and disruption.