Pharvaris announced compelling safety and efficacy data for deucrictibant, their investigational oral treatment for Hereditary Angioedema (HAE), at the 2025 American Academy of Allergy, Asthma, & Immunology's Annual Scientific Meeting and World Allergy Organization Joint Congress in San Diego.
The drug, currently under evaluation in two pivotal Phase 3 studies, has demonstrated remarkable effectiveness in both prophylactic and on-demand treatment settings. Long-term prophylactic use resulted in patients experiencing a median of zero days with attack symptoms per month, marking a significant advancement in HAE management.
Clinical Efficacy in Upper Airway Attacks
Particularly noteworthy was deucrictibant's performance in treating upper airway attacks, including laryngeal episodes, which can be life-threatening. Data from seven documented upper airway attacks showed consistent efficacy with the results observed in 328 non-upper airway attacks, demonstrating rapid and complete symptom resolution with just a single dose.
Quality of Life Improvements
The clinical trials revealed substantial improvements in health-related quality of life (HRQoL) measures. Patients reported enhanced outcomes specifically in 'functioning' and 'fear and shame' domains, addressing critical psychological and practical challenges faced by the HAE community.
Expert Perspective
"Topline results of deucrictibant in both prophylactic and on-demand randomized clinical trials substantiate our belief in the mechanism and molecule to provide choice to those living with HAE," stated Peng Lu, M.D., Ph.D., Chief Medical Officer of Pharvaris. He emphasized the significance of the long-term dosing data in supporting deucrictibant's potential to address unmet needs in HAE treatment.
Innovation in HAE Treatment
The development of deucrictibant represents a potential breakthrough in HAE therapy, offering what could be the first oral option for both prevention and acute treatment. This dual functionality, combined with the convenience of oral administration and single-dose efficacy, could significantly improve the standard of care for HAE patients.
The ongoing Phase 3 studies aim to confirm these promising findings and potentially establish deucrictibant as a transformative treatment option in the HAE therapeutic landscape.