[Edaily Reporter Kim Jinsoo ] Aptabio Therapeutics Inc.(293780)is making a bold move with a “package deal” as it pursues the technology export of “APX-115” (Isuzinacxib), a pan-NADPH oxidase (Pan-NOX) inhibitor currently under development.

Aptabio Therapeutics Inc. is developing APX-115 for two indications: contrast-induced acute kidney injury (CI-AKI) and diabetic kidney disease (DKD). If the Phase 2 clinical trial for CI-AKI—results of which will be released next month—confirms the drug’s actual efficacy in preventing kidney damage, the deal size for APX-115 is expected to increase.

According to Aptabio Therapeutics Inc. on the 5th, APX-115 is currently undergoing clinical trials for two kidney diseases—contrast-induced acute kidney injury and diabetic kidney disease—and data from the contrast-induced acute kidney injury study is scheduled to be released next month. APX-115 is a small-molecule compound that works by broadly inhibiting NADPH oxidase, which generates reactive oxygen species (ROS), thereby reducing oxidative stress, inflammation, and tissue damage.

Among these, contrast-induced acute kidney injury is a condition in which kidney function suddenly deteriorates due to the contrast agents used during percutaneous coronary intervention (PCI) or coronary angiography. Currently, there is no established standard treatment involving the administration of specific drugs to prevent acute kidney injury; therefore, management focuses on risk mitigation measures such as fluid administration, minimizing contrast agent use, and monitoring kidney function. Consequently, there is a high demand for the development of therapeutic agents.

Acute Kidney Injury: Topline Data to Be Released in October

Aptabio Therapeutics Inc. conducted a clinical trial in which patients undergoing PCI in South Korea and the United States were administered APX-115 for a total of five days—before and after contrast agent administration. The trial concluded last July, and topline data is scheduled to be presented at the American Society of Nephrology (ASN) meeting this October.

The most notable aspect of this data is its “preventive effect.” While the primary endpoint is “tolerability,” it is the secondary endpoints that enhance the value of APX-115. As secondary endpoints, Aptabio Therapeutics Inc. is comparing the incidence of acute kidney injury between the APX-115 treatment group and the placebo group, while also monitoring changes in renal function indicators such as serum creatinine and estimated glomerular filtration rate (eGFR). The study’s core objective is to determine whether the incidence of acute kidney injury is actually reduced in patients treated with APX-115.

If the study not only confirms safety but also detects signs that APX-115 prevents kidney damage, the value of APX-115 is expected to increase significantly, as these findings would be combined with the clinical data on diabetic kidney disease that the company has already established.

Aptabio Therapeutics Inc. previously conducted a Phase 2a clinical trial of APX-115 in Europe involving 140 patients with diabetic kidney disease. While the reduction in urinary albumin-to-creatinine ratio (uACR) did not reach statistical significance in the overall patient population, a statistically significant reduction of approximately 47% in the rate of change in uACR compared to placebo was observed in the subgroup of patients with markedly impaired kidney function. A significant reduction in uACR was also observed in the group with high medication adherence.

Based on these results, Aptabio Therapeutics Inc. is currently conducting a Phase 2b trial in South Korea involving 186 patients with diabetic kidney disease. The company has extended the treatment period to 24 weeks and is assessing whether the efficacy observed in the previous trial can be replicated, with a focus on severe patients who showed signs of efficacy in the earlier study.

Ultimately, if APX-115 demonstrates the potential to improve kidney damage in diabetic kidney disease and prevent kidney damage in acute kidney injury simultaneously, it could serve as a single candidate compound capable of targeting both “treatment and prevention.”

An Aptabio Therapeutics Inc. official stated, “If APX-115 demonstrates efficacy in the two indications it targets, it will be evaluated not merely as a candidate drug for specific kidney diseases, but as a universal kidney disease treatment that can be expanded to various kidney conditions through the same universal NOX inhibition mechanism.”

Targeting New Markets as Well as Existing Large Markets

In particular, next month’s data is expected to be crucial for Aptabio Therapeutics Inc. to secure the “package deal” it is currently pursuing. Aptabio Therapeutics Inc. is currently discussing the potential for technology transfer and joint development of APX-115 with multiple global pharmaceutical companies.

Contrast-induced acute kidney injury is an area with high unmet medical need, as there are currently no approved treatments. If APX-115 demonstrates preventive efficacy, it could become the first-in-class treatment. Diabetic kidney disease (DKD) represents an overwhelmingly large market. According to market research firm Fortune Business Insights, the global DKD treatment market reached approximately $25 billion (about 34 trillion won) in 2023.

From the perspective of a company looking to acquire APX-115, the deal size is bound to be substantial, as it would allow them to simultaneously tap into a new market and an already massive existing one. From Aptabio Therapeutics Inc.’s perspective, this also enables more favorable negotiations regarding upfront payments and milestone terms.

Furthermore, if additional efficacy is demonstrated in the upcoming Phase 2b trial for diabetic kidney disease, Aptabio Therapeutics Inc. is expected to gain an advantage in technology licensing negotiations, and the deal size is anticipated to grow even larger.

An Aptabio Therapeutics Inc. official stated, “Contrast-induced acute kidney injury is very common, occurring in about 10% of patients undergoing PCI procedures, yet there is currently no suitable treatment. Once the topline results are released, discussions regarding the technology export agreement will become more concrete, and we expect the process of pursuing a package deal to accelerate.”