Announcement • Aug 03
Mezzion Pharma Co., Ltd. Reports Positive In Vivo Results For Udenafil In ADPKD Mezzion Pharma Co. Ltd. announced positive results from a new in vivo study of udenafil, a novel phosphodiesterase type 5 (PDE5) inhibitor, in autosomal dominant polycystic kidney disease (ADPKD). In a preclinical ADPKD model, udenafil reduced kidney cyst burden and blood urea nitrogen (BUN), a blood marker linked to kidney function, compared with controls. Both findings were statistically significant and consistent across the treatment groups evaluated. The study also showed evidence of activity on udenafil's intended cyclic guanosine monophosphate (cGMP) signaling pathway in the kidney. In addition, udenafil produced a statistically significant reduction in a preclinical measure of cardiac hypertrophy, or heart enlargement. The study was conducted at Mayo Clinic in Florida, with Fouad T. Chebib, M.D., serving as principal investigator. The results build on two earlier preclinical studies in which udenafil inhibited cyst growth and reduced cyst burden. Across these complementary ADPKD models, udenafil has shown activity on its intended cGMP pathway, reduced measures of cystic disease, and improved a biochemical measure used in kidney assessment. Together, the data support clinical evaluation in people with ADPKD. Udenafil has been studied in multiple clinical programs over approximately 20 years, including studies with multi-year exposure. The consistency across multiple preclinical models supports evaluating udenafil in a well-designed Phase 2b study using established measures of ADPKD progression. Udenafil is being investigated through modulation of the nitric oxide-cGMP pathway, providing a mechanistically distinct therapeutic approach. ADPKD is a progressive genetic kidney disease affecting approximately 1 in 400 to 1,000 people worldwide. As kidney cysts grow, kidney function can decline and ultimately lead to dialysis or kidney transplantation. ADPKD is also associated with cardiovascular and other complications. Mezzion plans to advance udenafil into a Phase 2b study in adults at risk of rapidly progressing ADPKD, subject to finalization of the protocol and receipt of regulatory and ethics approvals. The planned study is expected to evaluate kidney volume and function, disease-related biomarkers, pharmacokinetics, safety, and tolerability. Udenafil is an investigational product and has not been approved by any regulatory authority. Announcement • Jun 05
Mezzion Pharma Completes FUEL-2 Phase 3 Regulatory Milestone Mezzion Pharma Co. Ltd. announced the results of a planned independent, blinded statistical review of its ongoing global confirmatory Phase 3 FUEL-2 study evaluating udenafil, its investigational therapy for single ventricle heart disease with Fontan circulation. After 50% of the planned study population completed the study's 26-week treatment period, an independent assessment was conducted to determine whether an increase in sample size would be required to maintain the study's planned statistical assumptions. The independent review determined that no increase in sample size was required and recommended that the study continue as planned with the enrollment of 436 patients. Moreover, no changes were recommended to the study design, primary endpoint, statistical analysis plan, or projected study timelines. Mezzion Pharma has now completed all major planned statistical review milestones prior to topline data. Single ventricle heart disease with Fontan circulation is a rare and serious form of congenital heart disease associated with significant long-term complications. Despite advances in surgical care, there are currently no approved therapies specifically indicated for this rare disease with unmet medical need. Udenafil is an investigational product and has not been approved by any regulatory authority. Announcement • May 14
Mezzion Pharma Co.,Ltd. announced that it has received KRW 36.5 billion in funding On May 12, 2026, Mezzion Pharma Co.,Ltd. has closed the transaction. 
