DE MONITOR schreef op 22 maart 2023 00:25:
PHARVARIS PRESENTS POSITIVE STUDY DATAAt the 2023 HAEi Regional Conference APAC in Bangkok, Thailand, Pharvaris presents positive data from its Phase 2 RAPIDe-1 study of PHVS416 for the on-demand treatment of HAE attacks.
Marc A. Riedl, M.D., M.S., Professor of Medicine, Clinical Director of the US Hereditary Angioedema Association (HAEA) Angioedema Center at the University of California San Diego (UCSD), and Clinical Service Chief for Allergy/Immunology at UCSD, comments:
“Currently approved on-demand therapies for HAE attacks are administered intravenously or subcutaneously and can be associated with treatment burden. The time required for preparation and administration, as well as potential occurrence of pain, discomfort, or other injection site reactions can lead to treatment delays or untreated HAE symptoms. An unmet need exists for on-demand oral therapies that are effective and well-tolerated, and that may reduce the treatment burden, enabling prompt administration as recommended by clinical guidelines. The consistent results across all endpoints in the RAPIDe-1 trial provide evidence supporting the efficacy and well-tolerated profile of PHVS416 in treating HAE attacks and provide a foundation for its further development as a potential on-demand therapy.”
RAPIDe-1 is a Phase 2, double-blind, placebo-controlled, randomized, cross-over, dose-ranging trial of PHVS416, the oral softgel capsule formulation of PHA121, for the treatment of HAE type 1 and type 2 (HAE-1/2) attacks. The trial enrolled participants in Canada, Europe, Israel, the United Kingdom, and the United States. Eligible participants were between the ages of 18 and 75 years, diagnosed with HAE type I or II and experienced three or more attacks in the last four months or two or more attacks in the last two months prior to screening.
74 participants were enrolled and 62 of them experienced 147 qualifying HAE attacks that were treated with double-blinded study drug (either placebo or PHVS416 10, 20, or 30 mg doses). Analysis of the primary endpoint demonstrated that PHVS416 significantly (p<0.0001; nominal p value for 10 mg dose) reduced attack symptoms measured as change in the mean 3-symptom composite (skin pain, skin swelling, abdominal pain) visual analogue scale (VAS-3) score during HAE attacks, at four hours compared with placebo (LS mean difference of change in VAS-3: -16.75, -15.02, and -16.28 for PHVS416 10, 20 and 30 mg, respectively, vs. placebo). All key secondary efficacy endpoints were also met. Participants on PHVS416 also used substantially less rescue medication compared to placebo (10 mg=18.9%, 20 mg=10.7%, 30 mg=6.5%, placebo=60.8%). PHVS416 was generally well tolerated with three treatment-related adverse events (TRAEs) reported for one PHVS416 30-mg-treated attack (2.8%) and one TRAE reported for one placebo-treated attack (1.9%).