Pharvaris' genetic disorder drug meets main goal in late-stage trial

The 24-week study enrolled 85 adolescents and adults across 21 countries. Most side effects were mild or moderate, with no treatment-related serious side effects reported.

Sept. 8 (Reuters) - Pharvaris said on Tuesday its experimental oral drug helped prevent swelling attacks in patients with hereditary angioedema, meeting the main goal of a late-stage trial.

Shares of the company were up more than 15% in early trading.

• Hereditary angioedema is a rare genetic disorder that causes repeated swelling in the face, hands, feet, abdomen and throat, which can be life-threatening if it blocks breathing.
• The disorder has three types. Type 1 causes low levels of the C1 inhibitor protein, Type 2 prevents the protein from working properly, while the third occurs despite normal levels and function.
• Pharvaris' once-daily drug, deucrictibant, reduced the average monthly rate of swelling attacks by 83% compared with a placebo in the study. The reduction was 87% among patients with Type 1 or Type 2, the company said.

The biopharma firm is developing two oral formulations of deucrictibant: a once-daily tablet to prevent swelling attacks and a fast-acting capsule to treat them when they occur.

Current preventive options include Takeda's Takhzyro and Ionis Pharmaceuticals' Dawnzera, both injected treatments, as well as BioCryst's once-daily oral drug Orladeyo.

"The vast majority of patients will opt for an oral medicine before an injected medicine," trial investigator Marc Riedl said, adding that newly diagnosed patients, those seeking better control than existing pills provide and those looking to avoid injections could be potential early users.