Regulus Therapeutics

Six companies—Ionis Pharmaceuticals, Isarna Therapeutics, Regulus Therapeutics, Sarepta Therapeutics, Secarna Pharmaceuticals, and Wave Life Sciences—are leading in the development of antisense oligonucleotides, a targeted treatment for various diseases. Ionis, a pioneer with over 40 drugs in its pipeline, recently priced a $500 million IPO. Isarna's ISTH0036 targets TGF-β for ophthalmic conditions. Regulus focuses on microRNA targeting with its lead candidate RGLS8429 for ADPKD. Sarepta has three approved PPMO therapies for DMD and a recent $1 billion deal with Arrowhead Pharmaceuticals. Secarna's SECN-15 targets NRP1 for oncology. Wave Life Sciences' WVE-003, for Huntington's disease, recently reported positive phase 1b/2a trial results. The antisense oligonucleotide market is projected to grow to $5,519 million by 2033.

miRNA therapeutics face challenges like off-target effects and delivery issues, despite promising preclinical results. Unlike siRNA, no miRNA-based drug has reached phase 3 trials or been FDA-approved. miRNA's potential as a diagnostic tool is more immediate, with miRNA panels already in clinical use.

RGLS-8429, targeting miR-17 for ADPKD, expected to reach $941 mn by 2036 in the US. Regulus Therapeutics, developing microRNA-based therapeutics, reported operating and net losses of $31.1 mn and $30 mn in FY2023. Risk-adjusted NPV (rNPV) model considers drug failure risk in clinical development.

Amylyx Pharmaceuticals' shares fell after its ALS treatment failed in a phase III study. Acadia Pharmaceuticals also saw a decline due to a failed schizophrenia treatment study. Regulus Therapeutics reported positive results for a kidney disease treatment, boosting its shares. Moderna initiated a cancer therapy study, increasing its stock value. Regeneron received FDA approval to extend its cholesterol drug's label to pediatric patients.