Ionis Pharmaceuticals, Inc. initiates OASIS-HAE, the registrational study in the donidalorsen (formerly IONIS-PKK-LRx) Phase 3 clinical program. Donidalorsen is an investigational antisense medicine designed to reduce the production of prekallikrein, which plays a key role in the activation of inflammatory mediators associated with acute attacks of HAE. Donidalorsen uses Ionis’ LIgand-Conjugated Antisense, or LICA, technology.
“Initiating the Phase 3 program for donidalorsen moves us one step closer to bringing a potential best-in-class prophylactic treatment to market for people with HAE globally experiencing recurrent painful and severe HAE attacks,” says Kenneth Newman, M.D., M.B.A., Ionis’ Vice President of Clinical Development and leader of the immunology and pulmonology franchise. “Advancing this program underscores our commitment to the HAE patient community to deliver transformative treatments.”
OASIS-HAE is a double-blind, randomized, placebo-controlled registrational study in up to 84 patients with hereditary angioedema, Types 1 and 2. Patients will be randomized to receive monthly or bi-monthly subcutaneous donidalorsen for 25 weeks. Following the placebo-controlled portion of the study, patients may enter the 52-week open-label extension study.
New Phase 2 data recently presented at ACAAI highlighted an overall reduction in moderate to severe attacks, and a reduction in these attacks starting with the second dose. For the final month of the study, all donidalorsen treated patients were attack-free. In this study, donidalorsen was safe and well tolerated.