RAPIDe-1 (NCT04618211) will evaluate the investigational therapy’s safety, efficacy, and pharmacokinetics, or how the compound moves throughout the body.
The trial is recruiting approximately 54 adults, ages 18-75, with HAE type 1 or 2, with enrollment already started at a few sites across Canada. Additional study locations, including in Europe, are expected to be added. More information about trial contacts and recruiting sites can be found here.
“The initiation of this trial signifies another step towards developing an oral treatment for hereditary angioedema patients experiencing acute attacks,” Berndt Modig, Pharvaris’ CEO and co-founder, said in a press release.
Following enrollment, participants will be randomly assigned to receive a low, medium, or high dose of PHVS416, or a matched placebo, as a treatment for their acute HAE attacks.
The trial’s main goal is to compare the degree of symptom relief — particularly pain and swelling in the skin and abdomen — provided by each dose of the medication during an attack, relative to the placebo. The treatment’s safety also will be assessed.
The study will take place in two parts. In Part 1, patients will receive a single dose of the experimental compound while not experiencing an attack, to allow investigators to study its pharmacokinetics and safety. Part 2 will take place at the patients’ homes. They will have to treat three attacks by self-administering the therapy or the placebo.
“This study will help to determine if our small-dosage oral softgel capsule provides safe, rapid, and convenient on-demand treatment of HAE attacks,” Modig said.
PHVS416 is a soft gel capsule formulation of PHA121 (also known as PHA-022121), a small molecule compound that is able to bind and block the bradykinin B2 receptor. It works by preventing the cascade of biological reactions that would lead to an HAE attack.
Unlike most HAE medications that are administered through injections, PHA121 is an oral and potentially more convenient treatment alternative that addresses an unmet medical need.
“The importance of providing patients with treatment alternatives to injection cannot be overstated,” Modig said. “We hope to confirm in HAE patients the compelling findings of our previous studies.”
Data from two recent studies in healthy volunteers showed that a single dose of PHA121 was safe and well-tolerated, and able to quickly and sustainably block bradykinin-related blood changes.
Additionally, PHA121 remained in circulation in the body at a level above the concentration needed for a therapeutic effect for at least 12 hours, lasting much longer than Firazyr (icatibant acetate) — an approved HAE treatment marketed by Takeda — that remained in circulation for approximately six hours.
Pharvaris also is developing a tablet formulation of PHA121, known as PHVS719, as a preventive treatment for HAE attacks.
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