Patient Voice Redefines HAE Drug Trials, Bolstering Pharvaris's Strategy

ZUG, SWITZERLAND – March 11, 2026 – A pivotal study published today in Clinical Reviews in Allergy & Immunology is set to reshape how new treatments for hereditary angioedema (HAE) are evaluated, placing the patient's own experience at the core of clinical research. The real-world study, sponsored by late-stage biopharmaceutical company Pharvaris, provides rigorous validation for patient-reported outcome (PRO) measures, a move that promises to standardize trial endpoints and strengthen the development path for a new generation of therapies, including the company’s own oral candidate, deucrictibant.

For years, the HAE community has navigated a complex treatment landscape while grappling with a fundamental challenge in drug development: how to accurately measure the success of a treatment for a disease characterized by unpredictable, subjective, and often terrifying attacks of swelling. The new publication addresses this head-on, offering a scientifically validated framework to ensure that clinical trial results truly reflect benefits that are meaningful to people living with HAE.

The Challenge of Measuring a Subjective Disease

Hereditary angioedema is a rare genetic disorder that causes severe, painful, and potentially life-threatening swelling attacks. These episodes can affect the limbs, face, abdomen, and, most dangerously, the airway. While the HAE treatment market has seen significant innovation, comparing the effectiveness of different on-demand therapies has been notoriously difficult. Clinical trials have historically used a wide variety of endpoints, making direct, head-to-head comparisons nearly impossible for clinicians and patients.

This lack of uniformity has been a major hurdle. The new Pharvaris-sponsored study sought to solve this by validating specific PRO instruments—tools that capture the patient’s perspective directly. The non-interventional study used a mixed-methods approach, combining real-time electronic diaries during HAE attacks with post-attack interviews. This allowed researchers to assess the reliability and sensitivity of tools like the Patient Global Impression of Change (PGI-C) and the Angioedema syMptom Rating scAle (AMRA) in a real-world setting.

The findings confirmed that these instruments are robust and valid. More importantly, the qualitative interviews revealed what truly constitutes a meaningful improvement for a patient during an attack. The study found that even seemingly small changes, such as the moment an attack stops worsening—a concept captured by the endpoint of “End of Progression™”—are considered highly significant by patients. This is often the first sign of relief and a critical turning point in their experience of an attack.

“This study and its findings explore important insights into the care of those living with HAE; by validating patient-reported outcome measures with rigorous evidence, we have supported the definition of clinical study endpoints and reinforced trust in the results of those studies,” commented Danny M. Cohn, M.D., Ph.D., an investigator for the study from Amsterdam UMC. He noted that this understanding “will not only support the development of novel treatments but provide additional evidence to clinicians and people living with HAE to inform decisions on the most appropriate treatment options for them.”

Aligning with a New Global Consensus

The timing of this publication is critical, as it aligns with a broader international effort to standardize HAE research. The study’s patient-centric approach dovetails with the recently established AURORA Consensus, which developed a Core Outcome Set (COS) for all acute HAE treatment trials. This consensus, forged by patients, clinicians, researchers, and regulators, identifies five key outcomes—including change in symptom severity and time to end of progression—that should be reported in every study to ensure consistency and comparability.

Pharvaris has been at the forefront of adopting these new standards. The company has explicitly stated that the findings from this validation study informed the design of its ongoing Phase 3 RAPIDe-3 trial for the on-demand use of deucrictibant. The trial is one of the first to be designed with prespecified endpoints fully compliant with the AURORA recommendations.

“By validating patient-reported outcome measures in a real-world setting, we are ensuring that the experience and voices of people living with HAE directly inform how we assess new therapies in development,” said Peng Lu, M.D., Ph.D., Chief Medical Officer of Pharvaris. “This study gives us greater confidence that clinical outcomes truly reflect meaningful benefits for patients.”

This alignment with global standards not only enhances the scientific credibility of Pharvaris's clinical program but also provides a clearer path for regulatory bodies like the FDA and EMA, which increasingly prioritize patient-focused drug development. By demonstrating that its endpoints are both scientifically valid and deeply relevant to patients, Pharvaris is building a more robust and compelling case for its lead candidate.

Strategic Edge in a Competitive Oral Market

This scientific validation carries significant strategic weight in the highly competitive HAE market, which is projected to reach over $9 billion by 2032. While injectable therapies from companies like CSL, Takeda, and Ionis have long been the standard, the market is rapidly shifting towards the convenience of oral medications. With KalVista Pharmaceuticals’ oral on-demand therapy Ekterly approved in 2025 and BioCryst’s oral prophylactic Orladeyo already established, the demand for effective, non-injectable options is clear.

Pharvaris aims to compete in this space with deucrictibant, a novel oral bradykinin B2 receptor antagonist being developed in two formulations: an immediate-release capsule for on-demand treatment of attacks and an extended-release tablet for long-term prevention (prophylaxis). The RAPIDe-3 study is evaluating the on-demand capsule, while the CHAPTER-3 study is assessing the prophylactic tablet, with topline data from the latter expected in the third quarter of 2026.

By pioneering the validation and implementation of patient-centric endpoints, Pharvaris is not just contributing to better science; it is creating a strategic advantage. The data from the RAPIDe-3 trial will not only be measured against a placebo but will also be framed in a language that is inherently meaningful to patients, payers, and prescribers. This robust, patient-validated data package could differentiate deucrictibant in a crowded field and support a strong value proposition upon its potential market entry, offering a powerful narrative that goes beyond simple efficacy numbers to capture the true impact on a patient’s quality of life.