Pharvaris drug to treat rare swelling disease reaches Phase 3 targets; FDA submission planned in 2026
Preliminary Phase 3 clinical trial results for a drug from Pharvaris that can quickly relieve symptoms of sudden bouts of swelling caused by a rare genetic disease pave the way for a filing the company plans to submit to the FDA next year. If approved, Pharvaris capsules could become a second oral option for patients requiring urgent treatment for the disease hereditary angioedema (HAE).
Pharvaris is developing two formulations of its drug deucrictibant. The results reported Wednesday were immediate-release capsules as an on-demand treatment for HAE attacks. A separate Phase 3 trial is ongoing to evaluate an extended-release version of the drug for preventive HAE treatment.
HAE is a genetic disorder caused by a deficiency or dysfunction of C1 esterase, a protein that regulates other proteins involved in swelling and inflammation. People with this disease experience sudden, painful episodes of swelling throughout their body. Attacks that affect the airway can be fatal. Drugs that can be used to treat acute HAE attacks include injectable C1 esterase inhibitors marketed by Pharming Group and CSL Behring.
Deucrictibant is an oral, small molecule inhibitor of the bradykinin B2 receptor designed to prevent HAE attack originating from signaling at this receptor. The results released Wednesday came from a placebo-controlled Phase 3 trial of the drug as an as-needed treatment in 134 HAE patients aged 12 years and older. The primary objective was to measure time to symptom relief based on a seven-point scale used to assess symptom change in HAE episodes.
Based on the readings, the median time to symptom relief was 1.28 hours. This measure was not estimable for the placebo group. The study drug also met its secondary goal, with a median time of 17.47 minutes compared with 228.67 minutes in the placebo group. The median time to complete symptom relief with Pharvaris was 11.95 hours. Deucrictibant is a single tablet taken at the first sign of an HAE attack and is well tolerated. No discontinuations due to treatment-emergent adverse events were reported; no safety signals were identified.
With the FDA approval of KalVista Pharmaceuticals' Ekterly, patients will have an oral option to treat acute HAE attacks this summer. Ekterly is an oral, small molecule inhibitor of kallikrein, a different protein involved in HAE swelling, that is taken as two tablets during an HAE attack.
Leerink Partners analyst Joseph Schwartz said in a note to investors that the firm believes the deucrictibant data is very strong. Comparisons across trials are always tricky, but the Pharvaris pill provided much faster symptom relief than Ekterly, which had a median of 1.61 hours for the 300 mg dose and 1.79 hours for the 600 mg dose. But Schwartz noted that there was a stronger difference in the time it took deucrictibant to complete symptom relief: The median time for Pharvaris was 11.95 hours, while the median time for Ekterly was more than 24 hours. Schwartz said the strength of Ekterly's launch validates the opportunity for Pharvaris' drug.
“Overall, we continue to believe the market is large enough to accommodate multiple players, especially as the market continues to expand as new options are approved,” he said.
Pharvaris said it expects to submit a new drug application for deucrictibant to the FDA in the first half of 2026. Preliminary results from Phase 3 testing of an extended-release version of the drug are expected in the second half of next year. Another phase 3 study is underway testing the drug to treat acquired angioedema caused by C1 inhibitor deficiency.
Photo by Flickr user Jernej Furman via Creative Commons license
