'Aribio’s 7 Trillion Won Export Deal: Reinforcing Phase 3 Trial for Robust FDA Approval'

Doubling Patient Enrollment and Strengthening Validation to Ensure Data Reliability
"Statistical Analysis Methods Finalized in Agreement with FDA"
"Expecting Market Expansion for PDE5 Mechanism Novel Drugs as Competitors Join"

Aribio, which is developing the oral Alzheimer's disease treatment candidate 'AR1001,' announced that it has nearly doubled the patient enrollment for its Phase 3 clinical trial to secure U.S. Food and Drug Administration (FDA) approval, and has also enhanced its biomarker validation methods. The company expressed confidence, stating that even if clinical costs increase, it aims to enhance data reliability and obtain simultaneous approvals in both the United States and Europe with a single Phase 3 trial.

At a press conference held on the 18th at Fairmont Ambassador Seoul in Yeongdeungpo-gu, Seoul, executives of Aribio are speaking. From the left: Sunho Kim, Head of the U.S. Branch; Byunggeon Lee, Advisor; Jaejun Jung, Chairman (CEO); Suhyun Sung, CEO. Photo by Jeongyeon Park

Following its recent technology transfer agreement with China’s Fuxing Pharmaceutical worth up to 7 trillion won, Aribio held a press conference on the 18th at Fairmont Ambassador Seoul in Yeongdeungpo-gu, Seoul, to share updates on the global Phase 3 trial of AR1001, known as 'POLARIS-AD,' and to unveil its approval strategy.

During the press conference, Aribio executives emphasized that they have thoroughly prepared to ensure data reliability in the upcoming approval process, with topline results from the Phase 3 trial expected in the third quarter. Sunho Kim, Head of Aribio’s U.S. branch, stated, “The trial, initially designed for around 800 patients in the U.S. alone, has been expanded to 1,535 patients. While this was not a requirement from the FDA, we decided to increase the number of participants at our own expense because it raises the probability of achieving statistical significance.”

The company also strengthened its biomarker validation process. Kim explained, “At first, we considered conducting the validation using only blood-based methods, but based on discussions with the FDA, we added cerebrospinal fluid and positron emission tomography (PET) imaging, which incurred additional costs.” By incorporating validation methods that are much more expensive than blood tests, Aribio aimed to ensure the objectivity of its data.

The clinical trial design and statistical analysis methods were also thoroughly prepared through formal consultations with the FDA. Jaejun Jung, Chairman of Aribio, said, “We reached an agreement on the Phase 3 design at the End-of-Phase 2 meeting, and have already finalized the last outstanding issue, which was the statistical analysis method.” Kim added, “Even during the trial, we proactively communicated with the FDA by holding more than four Type C meetings to preemptively address any potential issues that might arise at the point of data submission.”

The company further stated that it expects to be able to apply for approval based on the single Phase 3 trial. Kim noted, “Recently approved drugs in the Alzheimer’s field have all been based on single Phase 3 trials, and the FDA’s approval trend is shifting in that direction. If the results are favorable, a single Phase 3 trial should be sufficient for approval.”

Earlier, in May, Aribio signed a technology transfer agreement with Fuxing Pharmaceutical for AR1001, with a total contract value in the 7 trillion won range. Under the agreement, Fuxing Pharmaceutical is granted development and commercialization rights in the United States, China, and the ASEAN region, with an upfront payment alone amounting to about 210 billion won. According to Aribio, the driving force behind the agreement was Fuxing Pharmaceutical’s ambition to globalize. Kim noted, “The capital Fuxing is committing to U.S. commercialization of AR1001 exceeds the contract payment to us, reflecting Fuxing Group’s strong intent to become a major pharmaceutical company.”

AR1001 is a PDE5 inhibitor, belonging to the same class as erectile dysfunction drugs. It works as a multi-target drug by dilating cerebral blood vessels, increasing blood flow, enhancing the memory and learning signals of nerve cells, and reducing the accumulation of amyloid-beta, the protein responsible for Alzheimer’s disease.

Some in the industry have questioned why, despite longstanding academic interest in the potential Alzheimer’s benefits of PDE5 inhibitors, global pharmaceutical companies have not pursued full-scale clinical development. Addressing this at the press conference, Jung explained, “Although AR1001 is in the same class as drugs like Viagra, its selectivity for PDE5 is about 10 times higher, and its blood-brain barrier (BBB) penetration is superior. Achieving similar effects with Viagra or Cialis would require doses about five times higher, but Viagra already carries an FDA black box warning for potential visual disturbances with long-term high-dose use, which likely discouraged major pharmaceutical companies from further development.”

Jung also emphasized the need for greater interest from both domestic and international biotech companies in the PDE5 mechanism. “You can’t expand the market with just one drug,” he said. “Aribio aims to be first in the market, and as more competitors enter the field, the market itself will grow.” CEO Suhyun Sung added, “For Korea to establish itself as a powerhouse in new drug development, more companies must take on the challenge of developing innovative mechanisms.”

This content was produced with the assistance of AI translation services.