Pipeline

CU01

CU01

Diabetic Nephropathy

Sortation content
CU01 Dimethyl fumarate (DMF) which is being developed for Diabetic Nephropathy
Indication Diabetic Nephropathy
Unmet Needs

• Only applicable for early symptom relief

• Not applicable for ESRD (End stage Renal Disease) patients

• High unmet medical needs for cardiovascular protection agents

Mechanism of Action

• CU01 inhibits TGF-β/SMAD signaling by activating Nrf2 and has an excellent therapeutic effect in reducing renal fibrosis by suppressing the expression of extracellular matrix.

Efficacy & Safety

• CU01 started to show a significant improvement in the amount of change in eGFR from week 6 after administration, and the consistently increasing change in GFR continued until week 12.

• CU01 group suppressed the progression of diabetic nephropathy by gradually increasing eGFR during the administration period, so it is presumed to have an improvement effect on diabetic nephropathy.

• In the CU01 group, the ACR value at week 12 compared to the baseline was statistically significantly decreased (p=0.0441)

Market

• Average annual growth of 5.9% since 2015

• Market size is expected to grow to 3.8 billion US$ in 2024

Indication

Currently, hypertension and diabetes drugs are mainly used for the treatment of diabetic nephropathy, and there is no drug that can fundamentally improve renal functions or treat kidney fibrosis, a complication of diabetic nephropathy. CU01 is the first-in-class drug with an Nrf2 activity/TGF-β/SMAD3 inhibitory mechanism. It improves renal functions by preventing or treating of renal fibrosis, chronic kidney disease, and diabetic nephropathy.

Unmet Needs

  • Only applicable for early symptom relief​
  • Not applicable for ESRD (End-stage Renal Disease) patients​
  • High unmet medical needs for cardiovascular protection agent

Mechanism of Action

CU01 inhibits TGF-β/SMAD signaling by activating Nrf2 and has an excellent therapeutic effect in reducing renal fibrosis by suppressing the expression of extracellular matrix

Efficacy

Change in eGFR

The change in eGFR from baseline at week 12 was 3.80±8.72 mL/min/1.73 m2 in the CU01 group and -4.63±11.14 mL/min/1.73 m2 in the placebo group. The difference in eGFR change between the groups was 8.43 mL/min. /1.73 m2, which was a statistically significant result (p=0.0121). CU01 started to show a significant improvement in the amount of change in eGFR from week 6 after administration, and the consistently increasing change in GFR continued until week 12. The eGFR of the placebo group gradually decreased over the administration period, and diabetic nephropathy worsened despite taking ARB as a salvage drug. On the other hand, CU01 group suppressed the progression of diabetic nephropathy by gradually increasing eGFR during the administration period, so it is presumed to have an improvement effect on diabetic nephropathy.

Change in Albumin Creatine Ratio(ACR)

In the CU01 group, the ACR value at week 12 compared to the baseline was statistically significantly decreased (p=0.0441).

Safety

Safety results in phase 2a clinical trial:

  • There were no new adverse events other than known adverse reactions (redness, gastrointestinal side effects) of CU01.
  • There were no serious adverse drug reactions related to the study drug. 
  • No deaths