[Edaily Reporter Kim Seung-kwon ] The business focus of the artificial intelligence (AI)-driven new drug development industry is evolving toward the clinical stage through technology transfers or joint development. A recent technology transfer agreement worth approximately 4 trillion won signed between Insilico Medicine and Eli Lilly is cited as a prime example. Insilico Medicine has demonstrated that the innovative potential of preclinical candidates alone is sufficient to secure joint clinical trial agreements with global big pharma companies.

Rasmotinib on the Verge of Global Phase 2 Clinical Trials... Overcoming Resistance as the Best-in-Class Drug

Pharos iBio Co., Ltd.(388870)The company is also accelerating its entry into global joint clinical trials with compounds discovered through AI. Pharos iBio Co., Ltd. is pursuing U.S. clinical trials and strategic collaborations with major global pharmaceutical companies, focusing on its two core drug pipelines: Rasmotinib (PHI-101), a treatment for acute myeloid leukemia (AML), and PHI-501, a treatment for solid tumors.

Kim Kyu-tae, President (Chief Business Officer) of Pharos iBio Co., Ltd., emphasized, “AI is merely an excellent tool for drug discovery; the company’s ultimate goal lies in achieving solid clinical results and securing drug approvals.” He added, “Our core strategy is to go beyond simply showcasing platform technology and instead generate tangible global business development results based on assets that have actually entered the clinical phase.”

Among Pharos iBio Co., Ltd.’s current pipeline, rasmotinib—a treatment for acute myeloid leukemia—is considered the frontrunner with the fastest development pace. Rasmotinib recently successfully completed a Phase 1b clinical trial in patients. It demonstrated efficacy by achieving complete remission (CR)—where cancer cells partially disappeared—in approximately 60% of evaluable patients.

President Kim explained, “We have secured highly significant data not only regarding safety but also efficacy in humans during the Phase 1 clinical trial,” adding, “We are developing lasmotinib not as a ‘First-in-Class’ drug that merely captures market share, but as a ‘Best-in-Class’ drug that surpasses the limitations of existing treatments.”

He continued, “Existing treatments currently on the market have critical limitations, including low patient response rates and a recurrence rate of approximately 75% within two years.” He expressed confidence, adding, “In contrast, Rasmotinib is an innovative new drug that precisely targets unmet medical needs in the global blood cancer market, as it was designed from the outset with the primary goal of overcoming drug resistance and tolerance.”

Based on these Phase 1 data, Pharos iBio Co., Ltd. will actively pursue an application for a global Phase 2 clinical trial (IND) in the second half of this year. The Phase 2 trial will include trials in the United States, the world’s largest market, and the total clinical costs are estimated to range from approximately 20 billion to 30 billion won.

Pharos iBio Co., Ltd. has already proactively established a funding plan to support this effort. According to Pharos iBio Co., Ltd.’s separate financial statements, its cash and cash equivalents are estimated to be around 230억 won. The company explains that this provides more than enough liquidity to cover research and development (R&D) and general operations for the foreseeable future.

The roadmap for conditional approval and early commercialization through Orphan Drug Designation (ODD) is also taking shape. He stated, “We have already successfully completed the first preliminary consultation with the Ministry of Food and Drug Safety,” adding, “We are engaged in in-depth discussions regarding whether we can obtain conditional approval immediately upon completion of the Phase 2 clinical trial, provided it is meticulously designed according to the specific direction we currently envision.”

In fact, negotiations for the early technology transfer of Rasmotinib are also gaining momentum. President Kim emphasized, “We are currently conducting a thorough review with multiple multinational pharmaceutical companies during the due diligence phase,” adding, “Our principle is to finalize the technology transfer at the optimal time when the drug’s valuation is maximized based on clinical data.”

Regarding the core value that has drawn the attention of global Big Pharma companies to Rasmotinib, he said, “Its efficacy in overcoming mutations and resistance is the most important intrinsic value,” adding, “However, from the perspective of actual drug commercialization, the overwhelming safety data demonstrating the absence of cardiac toxicity compared to competing drugs is also a very powerful asset.”

He further analyzed, “The scale and value of the technology transfer can be maximized when both conditions—outstanding efficacy and safety—are met.”

However, CEO Kim took a cautious stance regarding the specific timing of revenue generation. He explained, “While early revenue generation is possible upon obtaining conditional approval, we anticipate full-scale and meaningful product sales only after Phase 2 clinical trials are fully completed,” adding, “Until then, the large upfront payment received through the technology transfer will effectively serve as the company’s core revenue source.”

Pharos iBio Co., Ltd. Holds Multiple Meetings with Big Pharma... Key Details of Solid Tumor Combination Clinical Trial

: In addition to blood cancers, the development of PHI-501 (a Pan-RAF/DDR dual inhibitor)—a next-generation solid tumor therapeutic candidate—is also progressing rapidly. This compound, which has entered global Phase 1 clinical trials, recently attracted the attention of cancer researchers worldwide at the American Association for Cancer Research (AACR) conference by presenting three preclinical data sets demonstrating strong potential for synergistic effects when combined with Merck (MSD)’s world-renowned immunotherapy Keytruda (generic name: pembrolizumab).

CEO Kim stated, “At this year’s AACR, we officially presented animal study data showing that the anticancer effect is maximized when PHI-501 is administered in combination with Keytruda.”

The reason Pharos iBio Co., Ltd. is actively pursuing a combination strategy with immunotherapy lies in the inherent limitations of monotherapy.

He explained, “In patient groups with specific genetic mutations—such as those in colorectal cancer and melanoma (e.g., RAF)—the tumor response rate drops significantly even when the most effective immunotherapy is administered as a monotherapy,” adding, “For these intractable patients, a combination of an immunotherapy and our RAF inhibitor, PHI-501, is essential to achieve therapeutic efficacy. “Since a standard of care has not yet been established in this field, the market potential is vast,” he explained.

PHI-501 is currently in a global Phase 1 clinical trial as a monotherapy to verify its safety (tolerability) and initial efficacy in humans.

CEO Kim stated, “Once we have accumulated sufficient positive data from the ongoing Phase 1 monotherapy trial, we plan to discuss with big pharma a scenario where we naturally expand the indications to combination therapy based on these results.” In particular, he emphasized that if a joint development partnership is established with a global big pharma company, “there is a significant advantage in being able to receive the astronomically expensive Keytruda drug free of charge, thereby dramatically reducing the burden of clinical trial costs, while simultaneously expanding the scope of the project through large-scale, multinational combination trials.”

The company also has a clear differentiation strategy targeting the entire solid tumor sector, including ovarian cancer. He explained, “Ovarian cancer has a very high treatment success rate if diagnosed early, such as in Stage 1 or early Stage 2. However, once the disease progresses to stages 3 or 4 and the window of opportunity is missed, it becomes a fatal disease with virtually no viable treatment options, such as targeted therapies,” he said, adding, “In the past, several major global pharmaceutical companies have attempted to develop targeted therapies for ovarian cancer but have repeatedly abandoned development due to systemic toxicity issues.”

He continued, “By utilizing our internal AI platform to design precise targets based on three-dimensional protein structures and conducting ongoing translational research to identify the optimal dosage and administration regimen, Pharos iBio Co., Ltd. is directly overcoming the toxicity issues that have long been a chronic limitation of competing drugs.”