Ionis Pharmaceuticals Advances Neurological and Cardiovascular Therapies with New Clinical Trials and FDA Approvals

Ionis Pharmaceuticals is making significant strides in the development of treatments for serious neurological diseases and cardiovascular conditions. The company has announced plans to initiate Phase 3 trials for ION582 targeting Angelman syndrome in the first half of 2025, alongside Phase 3 data for zilganersen in Alexander disease and Phase 2 data for ION464 in multiple system atrophy.

In the realm of cardiovascular health, Ionis is continuing its momentum with key partnered programs, including Phase 3 data for pelacarsen, a promising medicine for Lp(a)-driven cardiovascular disease, in collaboration with Novartis.

The U.S. Food and Drug Administration has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). However, TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to the drug or its excipients, with warnings and precautions including hypersensitivity reactions and adverse reactions such as injection site reactions, decreased platelet count, and arthralgia.

Additionally, WAINUA™ (eplontersen) has been approved for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Important safety information for WAINUA includes warnings about reduced serum vitamin A levels, recommending supplementation and referral to an ophthalmologist if symptoms of vitamin A deficiency occur. The most common adverse reactions include decreased vitamin A levels and vomiting.

Ionis Pharmaceuticals, with over three decades of experience in inventing medicines for serious diseases, continues to lead in RNA-targeted medicines and gene editing technologies. The company's forward-looking statements reflect its commitment to advancing life-changing therapies for patients, despite the inherent risks and uncertainties in drug development and commercialization.