Ionis Pharmaceuticals (NASDAQ:IONS) has become a focal point in biotech after the November 2025 FDA approval of REDEMPLO. This marks the first siRNA therapy for familial chylomicronemia syndrome (FCS), a major milestone for its RNA-targeted platform.
Q3 results exceeded expectations, prompting a ~$50M revenue guidance raise. Revenue surged 47.5% over 12 months to $1.06B, and the stock returned 129% in the past year.
REDEMPLO offers quarterly at-home dosing with a clean safety profile. Phase 3 trials showed a median 80% triglyceride reduction, positioning it as a potential standard of care.
The pipeline is rich: Phase 3 data from SHASTA-3, SHASTA-4, SHASTA-5, and MUIR-3 are due in Q3 2026. Partner programs target hepatitis B, Lp(a)-CVD, and transthyretin amyloid cardiomyopathy.
Financially, Ionis has a current ratio of 4.1 and a market cap of $12.49B, though EPS losses are projected at -$2.47 for FY2026.
Expansion into severe hypertriglyceridemia (sHTG) offers significant upside, as the broader market is much larger than FCS alone.
