Pharvaris (PHVS) Study result summary

Study design and population

• CHAPTER-3 was a global, randomized, double-blind, placebo-controlled phase III study evaluating deucrictibant XR 40 mg once daily for prophylaxis of hereditary angioedema (HAE) attacks in patients aged 12 and older, including all HAE types (type 1, type 2, and normal C1 inhibitor), with 85 participants from 21 countries randomized 2:1 to deucrictibant XR or placebo for 24 weeks.

• 55 participants received deucrictibant XR and 30 received placebo, with balanced baseline demographics and disease characteristics.

• The mean age was 39 years, with most participants having HAE type 1 or 2; five had HAE with normal C1 inhibitor.

• Key inclusion required at least two investigator-confirmed attacks during run-in; the primary endpoint was time-normalized number of HAE attacks.

Efficacy results

• Deucrictibant XR achieved an 83% reduction in mean monthly HAE attack rate versus placebo (0.35 vs 2.06 attacks/month, p<0.0001), with an 87% reduction in HAE type 1 or 2 subgroup.

• Early onset of protection was observed within the first week and sustained throughout the 24-week study.

• All secondary efficacy endpoints were met with statistical significance, including ≥50%, ≥70%, and ≥90% attack rate reductions and increased proportion of attack-free participants.

• Nearly half of deucrictibant XR-treated participants remained attack-free over 24 weeks.

• Robust reductions were observed in moderate to severe attacks and attacks requiring on-demand medication.

Quality of life and patient-reported outcomes

• Deucrictibant XR led to substantial improvements across all domains of the Angioedema Quality of Life Questionnaire, with consistently greater improvements versus placebo.

• Meaningful improvements in angioedema quality of life (AE-QoL) scores were observed, with a significant difference versus placebo (p<0.0001).