Announcement • Aug 06
Pharvaris Publishes Results from First In-Depth Qualitative Study Assessing Experiences of People with Aae-C1inh and Validates Relevant Patient-Reported Outcome Measures Pharvaris 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. 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. 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. The full publication can be found here: Angioedema due to acquired C1 inhibitor deficiency: patient experience, conceptual disease model, and assessment of patient-reported outcome measures. Announcement • Jul 06
Pharvaris Announces FDA Acceptance of New Drug Application for Deucrictibant IR for On-Demand Treatment of Hereditary Angioedema Attacks Pharvaris announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for deucrictibant immediate-release (IR) capsule (20 mg) for the on-demand treatment (ODT) of Hereditary Angioedema (HAE) attacks. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of April 23, 2027. Pharvaris’ NDA details a comprehensive clinical development program for deucrictibant IR, including data from the treatment of over 1,300 HAE attacks. RAPIDe-3 (NCT06343779), a global, pivotal, placebo-controlled Phase 3 study of deucrictibant IR for the on-demand treatment of attacks in participants 12 years and older with HAE, including those with HAE with normal C1 inhibitor, met the primary and all 11 secondary efficacy endpoints with statistical significance. Results from RAPIDe-3 demonstrated the rapid and sustained efficacy of deucrictibant IR in treating HAE attacks; the median time to onset of symptom relief was 1.28 hours, to End of Progression™ (EoP) was 17.48 minutes, and to complete resolution of attack symptoms was 11.95 hours. Deucrictibant IR demonstrated a well-tolerated safety profile. Deucrictibant was granted orphan drug designation by the FDA in 2022. Deucrictibant is a novel, potent, orally bioavailable small-molecule bradykinin B2 receptor antagonist currently in clinical development. Deucrictibant is being investigated for its potential to prevent the occurrence of bradykinin-mediated angioedema attacks and to treat the manifestations of attacks if/when they occur by inhibiting bradykinin signaling through the bradykinin B2 receptor. Pharvaris is developing two formulations of deucrictibant for oral administration: an extended-release tablet to enable sustained absorption and efficacy as prophylactic treatment, and an immediate-release capsule to enable rapid onset of activity for on-demand treatment. Deucrictibant has been granted orphan drug designation for the treatment of bradykinin-mediated angioedema by the U.S. Food and Drug Administration, the European Commission, and Swissmedic. 
