[Edaily Reporter KIM JI-WAN ] “We’ve been preparing for the post-Fontan era.” Following its treatment for a rare heart disease— Mezzion Pharma Co., Ltd.(140410)—is now positioning autosomal dominant polycystic kidney disease (ADPKD), a rare kidney disorder, as its next-generation growth engine.

Having recently secured an opinion from the U.S. Food and Drug Administration (FDA) allowing it to skip Phase 1 clinical trials and proceed directly to Phase 2, the company is further establishing its presence as a new pipeline player by attracting interest from global pharmaceutical companies and investors at Bio USA, the largest event in the U.S. biotech industry.

12 million people worldwide suffer from the disease, but there is effectively only one treatment available

. According to Mezion Pharma Co., Ltd. on the 28th, the company is currently developing an ADPKD treatment using udenafil. Mezion Pharma Co., Ltd. aims to complete preclinical trials this year and then move into the clinical trial phase.

In particular, during a Type C meeting with the FDA, the company received confirmation that it could utilize clinical and safety data accumulated through its existing Fontan program, laying the groundwork for shortening the development timeline. An FDA Type C meeting is an official consultation process conducted to obtain the regulatory agency’s input in advance regarding clinical trial design and development direction.

ADPKD is a representative rare genetic disease characterized by the formation of numerous cysts in the kidneys, causing them to enlarge and lose function over time. If one parent has the disease, there is a 50% chance that their child will inherit it.

According to international guidelines, the global patient population is estimated at approximately 12 million. However, since there are no noticeable symptoms in the early stages, a significant number of people live their lives unaware that they even have the disease. The company estimates that only about 30% of all patients are actually diagnosed and receive treatment.

The situation in South Korea is not much different. While the number of currently diagnosed patients is estimated at around 10,000, the medical community believes the actual number is much higher when potential patients are included. Above all, the most significant characteristic of this market is that while there are many patients, there are almost no available treatments.

Currently, Otsuka’s tolvaptan is the only commercially available treatment. However, due to the risk of liver damage, patients must undergo regular blood tests, and after taking the medication, they must drink large amounts of water and endure frequent urination. As a result, the number of patients actually prescribed the drug is limited, and it is reported that there are quite a few cases where patients discontinue treatment.

Mezzion Pharma Co., Ltd. explained, “Current treatments are mostly used only for some patients whose disease progresses rapidly,” adding, “There are still far more patients who are not receiving appropriate treatment, making this a market with significant unmet need.”

Udenafil: Focusing on “Renal Blood Flow” Rather Than “Cysts”

The reason Mezion Pharma Co., Ltd. has high hopes for this drug is that its approach differs from existing treatments. Many new drug candidates currently in development focus on inhibiting cyst growth or specific genes. In contrast, Udenafil adopts a new strategy to slow kidney damage by improving blood circulation to the kidneys and vascular function.

Recent studies have also shown that ADPKD is not merely a disease characterized by the enlargement of cysts; rather, impaired renal microvascular function, reduced blood flow, and ischemic damage play significant roles in the progression of the disease. Mezion Pharma Co., Ltd. believes that, in this regard, Udenafil possesses a competitive edge over existing drugs.

Mezzion Pharma Co., Ltd. stated, “Udenafil has the potential to be applied to a relatively broad range of patients, as it is not limited to specific genetic mutations or patient subgroups,” adding, “We particularly anticipate its potential use in early-stage and mild cases where there are currently no treatment options.”

The official added, “Rather than replacing existing drugs, it can also be developed as a combination therapy, making it suitable for a variety of treatment strategies.”

FDA Also Positive on Development Strategy… “Clinical Trials Will Accelerate”

During recent consultations with the FDA, Mezion Pharma Co., Ltd. also secured positive feedback regarding the clinical trial design. In particular, the FDA indicated it would consider the company’s proposal to use imaging markers—specifically, changes in kidney volume measured via MRI—to assess kidney enlargement as a key endpoint in early-phase clinical trials. This approach takes into account the nature of rare diseases, where it takes a long time for patients’ kidney function to actually deteriorate, and is viewed as a marker that can confirm treatment efficacy more quickly.

In addition, the FDA recommended that the company also assess improvements in estimated glomerular filtration rate (eGFR), a key metric indicating how effectively the kidneys filter blood. Based on these discussions, the company expects to be able to skip Phase 1 clinical trials and proceed directly to Phase 2, thereby gaining a competitive edge in terms of development speed and cost.

The company plans to begin Phase 2 trials after completing preclinical studies in August or September of this year. Its strategy is to minimize financial burdens by pursuing development in phases rather than conducting a single large-scale clinical trial. At Bio USA, Mezion Pharma Co., Ltd.’s ADPKD project was confirmed to be attracting attention from the global industry. The company reportedly held discussions with numerous global firms and investors while presenting its preclinical research results and market strategy at the event.

In particular, the fact that it is an oral medication with a relatively well-established safety profile and that it can be used in combination with existing treatments reportedly received positive evaluations.

Mezzion Pharma Co., Ltd. stated, “ADPKD is still a market with a significant treatment gap, and there is high demand for new approaches,” adding, “If Udenafil is successfully developed, it has the potential to expand the treatment scope to include not only patients with rapidly progressing disease but also those in the early stages.”

The official continued, “Given its potential for combination use with gene therapies and RNA therapies currently in development, it holds high strategic value,” and emphasized, “Our goal is to grow it into Mezion Pharma Co., Ltd.’s second core pipeline—following the Fontan therapy—that will elevate the company’s enterprise value to the next level.”