Ionis Pharmaceuticals, Inc. Initiates Phase 3 Clinical Program of Donidalorsen in Patients with Hereditary Angioedema
November 18, 2021 at 01:30 pm EST
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Ionis Pharmaceuticals, Inc. announced initiation of 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 hereditary angioedema (HAE). Donidalorsen uses Ionis' LIgand-Conjugated Antisense, or LICA, technology. 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.
Ionis Pharmaceuticals, Inc. is engaged in ribonucleic acid (RNA)-targeted therapeutics. The Company has five marketed medicines and a pipeline in neurology, cardiology, and other areas of high patient need. It has nine medicines in Phase III development and multiple additional medicines in early and mid-stage development. The Companyâs products include SPINRAZA, QALSODY, WAINUA, TEGSEDI and WAYLIVRA. SPINRAZA is used for the treatment of patients with spinal muscular atrophy (SMA), a progressive, debilitating and often fatal genetic disease. QALSODY is an antisense medicine that is used for the treatment of adult patients with superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS), a rare, neurodegenerative disorder that causes progressive loss of motor neurons leading to death. WAINUA is a self-administered subcutaneous LIgand-Conjugated Antisense (LICA) medicine used for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN).