Pharvaris Drug for Rare Swelling Diseases Achieves Phase 3 Targets; FDA filing expected in 2026

A Pharvaris drug provided rapid relief and resolution of symptoms of sudden attacks of swelling caused by a rare genetic disorder, preliminary results from a Phase 3 clinical trial that pave the way for a submission to the FDA that the company plans to file next year. If approved, the Pharvaris capsule could become the second oral option for patients requiring acute treatment of attacks caused by the disease, hereditary angioedema (HAE).

Pharvaris is developing two formulations of its drug, deucrictibant. The results reported Wednesday involve the immediate-release capsule as an on-demand treatment for HAE attacks. A separate ongoing Phase 3 trial is evaluating an extended-release version of the drug for the prophylactic treatment of HAE.

HAE is an inherited disease caused by a deficiency or dysfunction of C1 esterase, a protein that regulates other proteins involved in swelling and inflammation. Patients with this condition may develop sudden, painful attacks of swelling throughout the body. Attacks that affect the respiratory tract can become fatal. Medications available to treat acute attacks of HAE include injectable C1 esterase inhibitors marketed by Pharming Group and CSL Behring.

Deucrictibant is an oral small molecule that inhibits the bradykinin B2 receptor, an approach intended to prevent HAE attacks that arise from signaling through this receptor. The results reported Wednesday come from a placebo-controlled phase 3 trial of the drug as an on-demand treatment in 134 HAE patients ages 12 and older. The primary objective was to measure the time taken for symptom relief according to a seven-point scale used to assess the progression of HAE attack symptoms.

According to the data read, the median time to onset of symptom relief was 1.28 hours. This measure was not estimable for the placebo arm. The study drug also met secondary endpoints with cessation of progression of attack symptoms achieved in a median time of 17.47 minutes, compared to 228.67 minutes for the placebo group. Complete resolution of symptoms for the Pharvaris drug averaged 11.95 hours. Deucrictibant, a single tablet taken at the first signs of an HAE attack, was well tolerated. No discontinuations were reported due to adverse events occurring during treatment; no safety signals have been identified.

Patients were given the opportunity to benefit from oral treatment for acute attacks of HAE over the summer thanks to the FDA approval of Ekterly from KalVista Pharmaceuticals. Taken as two tablets at the sign of an HAE attack, Ekterly is an oral small molecule that inhibits kallikrein, another protein involved in HAE swelling.

In a note sent to investors, Joseph Schwartz, an analyst at Leerink Partners, said the company views the supporting data as very strong. Comparisons between trials are always tricky, but the speed of symptom relief for Pharvaris’ pill is progressively faster than that of Ekterly, which showed symptom relief in a median of 1.61 hours for the 300 mg dose and a median of 1.79 hours for the 600 mg dose. But Schwartz noted a stronger differentiation for deucrictibant on the measure of time needed to complete symptom relief: a median of 11.95 hours for the drug Pharvaris, compared to more than 24 hours for Ekterly. Schwartz said the strength of Ekterly’s launch validates the opportunity for the Pharvaris drug.

“Overall, we continue to believe the market is large enough to accommodate multiple players, especially as it continues to expand with new options being approved,” he said.

Pharvaris said it plans to submit a new drug application for deucrictibant to the FDA in the first half of 2026. Preliminary results from Phase 3 testing of the extended-release version of the drug are expected in the second half of next year. Another phase 3 study is underway to test the drug in acquired angioedema due to C1 inhibitor deficiency.

Photo by Flickr user Jernej Furman via Creative Commons license