Ionis (IONS) Completes Enrolment for Rare Disease Drug Study

Core Insights - Ionis Pharmaceuticals (IONS) has completed full enrollment in a pivotal phase III study for its investigational RNA-targeted therapy, zilganersen, aimed at treating Alexander disease (AxD), a rare neurological condition [11] - The study targets nearly 73 participants aged between 2 to 65 across eight countries [1] - Zilganersen is the first investigational medicine in clinical development specifically for AxD patients, addressing the underlying cause of the disease [6] Group 1: Clinical Development - The pivotal study will randomize participants in a 2:1 ratio to receive zilganersen or placebo over a 60-week double-blind treatment period, followed by a 180-week open-label treatment period [12] - Top-line data from the pivotal study is expected in the second half of 2025 [5] - The primary endpoint of the study is the percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT) at the end of the 60-week treatment period [19] Group 2: Pipeline and Partnerships - Ionis is preparing to file a New Drug Application (NDA) for donidalorsen with the FDA later this year, which is being evaluated for hereditary angioedema (HAE) [9] - The company has partnerships with major pharmaceutical companies like AstraZeneca, Biogen, and Novartis, which provide funding for internal pipeline development through license fees and milestone payments [14] - Ionis has also developed other treatments, including olezarsen, which is under FDA review for familial chylomicronemia syndrome (FCS), with a decision expected before December 19, 2024 [8][17] Group 3: Market Performance - Year to date, Ionis's stock has decreased by 6.4%, compared to a 4.0% decline in the industry [7] - The company’s strategy includes launching wholly-owned medicines independently, with zilganersen being a key candidate in this approach [13]