Antisense’s Moment: How Rational Design Is Rewriting Drug Development Economics
Industry data shows small molecules achieve 5–10% approval rates over 15–20 years; antisense oligonucleotides (“ASOs”) are shifting the odds through rational design. Six new antisense drugs gained FDA approval in 2023–2024, bringing total approvals above 20; evidence of accelerating regulatory momentum. Oncotelic Therapeutics’ OT-101 (Trabedersen) is the only TGF-β2-specific antisense in Phase 3 trials, targeting pancreatic cancer and other resistant malignancies. The pharmaceutical industry has long faced sobering odds: about 90% of drug candidates fail before reaching market. For small molecules, approval rates hover at very low odds, often requiring up to two decades of development. In oncology, success rates…
