Company intelligence / Profile preview
Regulus Therapeutics
San Diego, California, United Statesir.regulusrx.com ↗
- Ownership
- Public · RGLS
- Development stage
- Phase 1
01
Overview
Farabursen (RGLS8429) successfully completed Phase 1b multiple-ascending dose clinical trial for ADPKD in March 2025, showing promising clinical efficacy and safety, including consistent impact on urinary polycystin (PC) and height-adjusted total kidney volume (htTKV). The company has received FDA alignment on key elements of a Phase 3 trial design as a single pivotal study, with a 12-month htTKV endpoint planned to support Accelerated Approval and a 24-month eGFR endpoint to support Full Approval[9].
- Therapeutic areas
- Nephrology
- Modalities
- Oligonucleotide therapeuticsmicroRNA inhibitors
- Industry
- Biotech
02
Drug pipeline
13 assets12 more assets in the full pipeline.
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Partnerships
Sanofi (2010)AstraZeneca (2014)
04
Financials
- Market cap
- $565M
- Total revenue
- $0
- Enterprise value
- $501.6M
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