The world’s first Endothelial Dysfunction Blocker

Sortation content
CU06 The world’s first Endothelial Dysfunction Blocker(Endothelial Dysfunction Blocker)

• CU06 is a small molecular compound derived from SOLVADYS™ platform technology and is the world’s first endothelial dysfunction blocker that strongly inhibit damage, increased permeability and inflammation of endothelial cells.

• CU06 is a treatment for intractable vascular-related diseases derived from various endothelial cell dysfunction, and completed technology export to Thea, a French ophthalmology company, in October 2021, and clinical trials phase 1 in the United States in June 2022.

Indication Diabetic macular edema, Wet macular degeneration
Unmet Needs

• Non-response rate to Anti-VEGF treatment: 40%

• Risk of inflammation, pain, and fear due to intraocular injection

Mechanism of Action

• Fundamentally blocks hyper-permeability by stabilizing endothelial cell junctions through modification of vascular endothelial cell actin structure.

• Blocks NF-kB inflammatory signaling system in vascular endothelial cells

• Enhances of survival rate and protective effect of vascular endothelial cells

Efficacy & Safety

• In vivo anti-hyperpermeability efficacy of CU06 was determined in EC (Endothelial Cell) dysfunction-related disease models.

• Oral administration of CU06 inhibited retinal vascular leakage in VEGF or Angiopoietin-2 (Ang2) induced vascular leakage mouse model, Streptozotocin (STZ)-induced diabetic retinopathy mouse model and CNV in beagle.


• 3.5 billion US$ in 2016

• 6.66 billion US$ by 2026


Diabetic macular edema

Diabetic macular edema is a disease in which liquid penetrates and swells into the macular, the light sensory layer, and it can cause blindness if symptoms worsen. Although anti-VEGF injections are used, treatment is clearly limited due to frequent unresponsiveness and concerns about side effects such as increased intraocular pressure, cataract, and inflammation. So, it is urgent to develop a new concept of drugs that is convenient to use.

wet Age-related macular degeneration

It's a disease that causes degeneration in the macular, a nerve tissue that allows you to see objects clearly in the eye (the center of the retina inside the eye) and causes symptoms that reduce vision or appear to be crushed. There are 2 types, one is non-exudative (dry) and the other one is exudative (wet). Wet macular degeneration greatly affects vision loss, and if left unattended, it can lead to blindness.

Unmet Needs

  • Non-response rate to Anti-VEGF treatment: 40%
  • Risk of inflammation, pain, and fear due to intraocular injection

Mechanism of Action

Stable vessel (barrier)
Unstable vessel(hyperpermeability)

Signals of reduced permeability

• Generally, 3 types of small GTPase(Rap1, Rac1, Cdc42) are related
• Barrier strengthened by cAMP increasing stimulation
• cAMP directly activate RapGEF, RapGEF activate Rap1

Signals of increased permeability

• RhoA/Rock activation induces the formation of  stress fiber
• Reactive oxygen is produced by endothelial cells, macrophages, polymorphonuclear leukocytes and cytoplasmic calcium
• VE-cadherin phosphorylation and internal introduction, claudin-5 and occludin phosphorylation


CU06 oral-administered animal experiment results: strong inhibition of vascular leakage and angiogenesis

Inhibiting vascular leakage inducer(VEGF, Ang2)

Eylea effects only on single targets(VEGF), but CU06 effectively blocks vascular leakage by targeting multiple factors(VEGF, Ang2)
→ Administration: Eylea(Intravitreal) , CU06 (Oral)

Ang2: Angiopoietin 2

Inhibiting abnormal angiogenesis(OIR model)

Inhibition of abnormal retinal angiogenesis

(OIR: oxygen-induced retinopathy)

Inhibiting vascular leakage(STZ model for diabetic retinopathy)

Similar efficacy compared to Eylea

STZ: streptozotocin

Global Sales Forecast for Macular Edema by Country, 2016 and 2026



  • US
  • France
  • Germany
  • Italy
  • Spain
  • UK
  • Japan

Source : GlobalData