New HAE Treatments on the Horizon
Treatments currently undergoing trials in Canada
CSL-312 (Garadacimab): is a novel Factor XIIa-inhibitory monoclonal antibody (FXIIa mAb) currently in Phase 2 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema.
Sebetralstat (KVD900): a potential oral, on-demand therapy for hereditary angioedema attacks that is designed to inhibit kallikrein.
PHVS 416: is a softgel capsule formulation containing PHA121, a highly potent, specific, and orally bioavailable competitive antagonist of the bradykinin B2 receptor being developed to provide rapid exposure of attack-mitigating medicine in a small oral dosage form.
PHVS 416: is an extended-release tablet formulation containing PHA121, a highly potent, specific, and orally bioavailable competitive antagonist of the bradykinin B2 receptor being developed to provide sustained exposure of attack-preventing oral medicine.
Further treatments in development:
Donidalorsen (formerly known as IONIS-PKK-LRx) is an investigational antisense medicine designed to reduce the production of prekallikrein, or PKK, which plays a key role in the activation of inflammatory mediators associated with acute attacks of HAE.