Pharvaris (Nasdaq: PHVS), a late-stage biopharmaceutical company developing novel, oral bradykinin B2 receptor antagonists to help address unmet needs of those living with bradykinin-mediated angioedema, such as hereditary angioedema (HAE) and acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH), today announced that results from the first in-depth qualitative study assessing the experiences of people with AAE-C1INH and validating relevant patient-reported outcome (PRO) measures have been published in Frontiers in Immunology. The study findings directly informed the design of and endpoint selection for the ongoing Phase 3 CREAATE study (NCT07266805) investigating deucrictibant for the prophylaxis and on-demand treatment of AAE-C1INH attacks.

“People living with AAE-C1INH face a profoundly challenging disease, including struggling through misdiagnoses, often due to lack of awareness of AAE-C1INH. Attacks are often painful, cause significant discomfort, and can limit functioning—disrupting daily life and creating a considerable burden,” said Danny M. Cohn, M.D., Ph.D., Department of Vascular Medicine, Amsterdam UMC (an accredited center of ACARE) and principal investigator in CREAATE. “Despite this, there are currently no therapies approved for the prevention or treatment of AAE-C1INH attacks. This study is an important step forward, capturing the patient experience in a rigorous way and helping to ensure that clinical studies evaluate outcomes that are truly meaningful to patients.”

The study represents the first in-depth qualitative assessment in AAE-C1INH, an ultra-rare and serious disease with no approved therapies for the prevention or treatment of bradykinin-mediated angioedema attacks. Findings provide foundational insights into disease burden and establish a framework to support the selection of clinical endpoints for clinical trials in this condition.

Interviews with people diagnosed with AAE-C1INH characterized disease manifestations, the impact on daily life, and perspectives on treatment benefit. Study results demonstrated that AAE-C1INH imposes a significant and multifaceted burden on patients, with participants reporting frequent, painful swelling attacks that disrupt daily functioning, often following prolonged initial periods of misdiagnosis and emergency care. Interviews also revealed broad impact across physical, emotional, social, and work-related domains, with individuals commonly unable to carry out routine activities, travel, or maintain employment during attacks. All participants relied on off-label therapies, underscoring the unmet needs associated with the absence of approved treatment options.

The study evaluated the relevance and interpretability of established PRO instruments, including the Patient Global Impression of Change (PGI-C), Patient Global Impression of Severity (PGI-S), and Patient Global Assessment (PGA) measures, in the AAE-C1INH population. Results demonstrated that these tools are meaningful and applicable for assessing treatment benefits in this disease context, helping to define clinically relevant thresholds for symptom improvement and resolution. Importantly, the study showed that a PGI-C rating of “better” was most consistently deemed meaningful across all participants at time points of up to 4 hours post treatment.

Peng Lu, M.D., Ph.D., President of Pharvaris, added, “these findings represent an important step forward in building a rigorous, patient-centered evidence base in AAE-C1INH. Aligned with guidance from the U.S. Food and Drug Administration (FDA), capturing the patient experience and defining meaningful changes in patient-centered outcomes strengthens the scientific foundation for the evaluation of treatment benefit in this underserved population. These insights directly informed our clinical trial design, ensuring our programs measure outcomes that truly matter to patients and enable us to advance our broader goal of developing effective, well-tolerated therapies that address unmet needs for people living with bradykinin-mediated angioedema.”