CU01 – Diabetic Nephropathy
An oral drug candidate targeting kidney disease through NRF2 activation and TGF-β inhibition
MOA of CU01: NRF2 activation & inhibition of TGF-β/Smad signaling
CU01 Phase 2b Results · UACR
UACR improvement was observed versus control in the overall population. In the subgroup receiving both RAAS and SGLT-2 inhibitors, adding CU01 was associated with an additional reduction in UACR compared with control receiving the same standard-of-care therapies.
CU01 Phase 2b Results · eGFR
eGFR remained stable over 24 weeks in the CU01 treatment groups.
Modality
Oral small molecule
Mechanism of Action
Antioxidant, anti-inflammatory, and antifibrotic activity through NRF2 activation and TGF-β inhibition
(Active ingredient: Dimethyl fumarate, DMF)
Indication
Diabetic nephropathy
Development Status
Korea Phase 2b clinical study report (CSR) received (2026.04)
Key Differentiators
Improvement in urinary albumin-to-creatinine ratio (UACR), the primary endpoint, and safety observed in a 240-patient Phase 2b trialMaintenance of estimated glomerular filtration rate (eGFR), suggesting potential to preserve kidney functionPhase 2b subgroup analyses showed additional UACR improvement when combined with standard-of-care therapies, including SGLT-2 inhibitors and RAAS inhibitors, compared with standard of care alone
Market Opportunity
CU01 aims to offer patients a new treatment option by preserving kidney function and slowing the progression of kidney disease. UACR improvement, maintenance of eGFR, and additional UACR improvement in patients receiving standard of care in Phase 2b support this development potential. Building on its favorable safety and tolerability profile, CU01 is being developed with the long-term goal of helping delay progression to kidney failure requiring dialysis or transplantation.A new-use patent application based on the Phase 2b results provides a potential basis for patent protection covering the claimed use through 2046, subject to patent grant and maintenance.
Development & Partnerships
Pursuing domestic out-licensing opportunities
Pursuing global out-licensing opportunities based on the new-use patent application