APeX-2 phase III trial data abstracts of BCX 7353 show efficacy in hereditary angioedema.- BioCryst Pharma

BioCryst Pharmaceuticals presented new clinical data of BCX 7353 (berotralstat) that further evaluates the attack rate reductions, patient satisfaction and quality of life of hereditary angioedema (HAE) patients in the APeX-2 trial over 48 weeks. In the APeX-2 Trial 48 Week Results, patients treated with oral, once-daily berotralstat 150 mg for 48 weeks experienced a sustained reduction in mean investigator confirmed HAE attack rates through month 12. In patients re-randomized to berotralstat 150 mg after 24 weeks on placebo, there was a marked reduction in investigator-confirmed HAE attack rates over 24 weeks of treatment. These patients had a mean attack rate per month of 2.5 at baseline, 1.7 at month six (while on placebo), 0.6 at month seven (one month after starting berotralstat 150 mg) and 0.6 at month 12 (six months after starting berotralstat 150 mg). Berotralstat was generally well-tolerated in APeX-2 through 48 weeks. The safety profile observed from weeks 24 to 48 was consistent with the data observed through the first 24 weeks. The most commonly reported treatment-related adverse events were upper respiratory tract infection, abdominal pain, diarrhea and vomiting. In results from the Phase III APeX-2 trial, patient satisfaction with treatment was assessed using the validated Treatment Satisfaction Questionnaire for Medicine (TSQM), which is comprised of three specific scales (side effects, effectiveness and convenience) and is scored on a global satisfaction scale from 0-100. HAE patients who transitioned from placebo to berotralstat 150 mg at week 24 reported improved overall treatment satisfaction and effectiveness. These patients experienced statistically significant improvements from weeks 24 to 48, with a mean global satisfaction increase of 26 points (p=0.005) and a mean effectiveness increase of 29.6 points (p<0.001). convenience scores remained high through week 48 reflecting the positive experiences patients had taking an oral medication. finally in quality of life through 48 weeks in the phase iii apex-2 trial quality of life was assessed with the angioedema quality-of-life ae-qol questionnaire a validated tool to measure impairment of qol based on a total and domain functioning fatigue mood fear shame and nutrition scores. the minimal clinically important difference mcid is defined as an improvement of six points. clinically meaningful improvements in mean ae-qol total scores were observed as early as week four with a mean improvement from baseline of 15 points at week 24. this improvement was sustained through 48 weeks of treatment with berotralstat 150 mg. improvements were observed in all four domains functioning fatigue mood fear shame nutrition through week 48. notably 77 percent of patients exceeded the mcid in total ae-qol total scores at 48 weeks indicating the reduction in attacks following berotralstat therapy appears to have a positive impact on patients quality of life. the data from three abstracts including a distinguished industry oral abstract are being presented at the 2020 annual scientific meeting of the american college of allergy asthma immunology acaai which is being conducted virtually from november 13-15.>