Ionis Pharmaceuticals

Biogen's salanersen (BIIB115/ION306) demonstrated substantial slowing of neurodegeneration and clinically meaningful motor function improvements in children with spinal muscular atrophy previously treated with gene therapy.

Columbia University researchers report that ulefnersen, an experimental drug, demonstrated remarkable efficacy in treating FUS-ALS, a rare genetic form of ALS affecting young people.

Olezarsen demonstrated significant triglyceride reductions of 61% and 58% at 6 months with 80mg and 50mg monthly doses respectively in patients with moderate hypertriglyceridemia.

Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront.

The European Commission has approved WAINZUA (eplontersen), developed by Ionis Pharmaceuticals and AstraZeneca, for treating hereditary transthyretin-mediated amyloidosis with polyneuropathy (ATTRv-PN) in adult patients with stage 1 or 2 disease.

Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.

Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.

• Novartis announced during its Q4 2024 financial results that the timeline for Phase 3 trial data of its cardiac therapy, developed in partnership with Ionis Pharmaceuticals, will be delayed. • The strategic collaboration between Novartis and Ionis Pharmaceuticals faces a setback in their cardiovascular drug development program, impacting their clinical trial readout schedule.

• Novartis announces Phase 3 data for pelacarsen, an Lp(a)-lowering antisense therapy, is now expected in first half of 2026, with regulatory submissions planned for second half. • The Lp(a)HORIZON trial, involving over 8,000 patients, aims to be the first cardiovascular outcomes study evaluating Lp(a) reduction's impact on cardiovascular risk. • Developed by Ionis and licensed to Novartis in 2019, pelacarsen targets a significant unmet need affecting over 8 million people worldwide with elevated Lp(a) and cardiovascular disease.

• Donidalorsen demonstrates a sustained reduction in HAE attack rates and improved quality of life in patients, according to Phase 3 and Phase 2 clinical program analyses. • The investigational RNA-targeted medicine is under FDA review, with a PDUFA date set for August 21, 2025, positioning it for potential commercial launch. • Data from the OASISplus study show that donidalorsen effectively reduces HAE attack rates and improves quality of life in patients switching from other prophylactic treatments. • Long-term analysis of the Phase 2 open-label extension study reveals that donidalorsen maintains a 96% reduction in HAE attack rates from baseline up to three years.