New clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of HAE patients in the APeX-2 trial over 48 weeks is being presented by BioCryst Pharmaceuticals, Inc.

“The data continue to demonstrate the potential of berotralstat – an investigational treatment for the prevention of attacks in patients with HAE – as a prophylactic medication, if approved by the FDA, with sustained reduction in attacks and meaningful improvements in quality of life seen over 48 weeks of treatment. With its oral, once-daily administration, berotralstat would offer patients a therapeutic alternative for managing this chronic disease,” says H. James Wedner, M.D., the Dr. Phillip and Arleen Korenblat Professor of Medicine at Washington University School of Medicine in St. Louis.

Berotralstat Reduces Attacks in Patients with HAE: 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.

Berotralstat Positively Impacts Patient-Reported Satisfaction: Results from the Phase 3 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.

Berotralstat Improves Patient-Reported Quality of Life Through 48 Weeks in the Phase 3 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.
(Source: BioCryst)