[Edaily Reporter SONG YOUNG-DOO ] While the domestic pharmaceutical and biotech sector showed a generally weak performance, SillaJen drew attention by hitting the daily price limit. In contrast, Hanmi Pharmaceutical and Hanmi Science, despite posting sharp gains the previous day following the announcement of a technology transfer deal worth trillions of won, failed to sustain their momentum the following day and plummeted.

BAL0891, Which Targets Both TTK and PLK1, Receives FDA Orphan Drug Designation

According to KG Zeroin MP Doctor (formerly Marketpoint) on the 25th, SillaJen closed at 2,960 won, up 29.82% (680 won) from the previous trading day. This is believed to be due to strong buying pressure following the company’s official announcement that its core pipeline candidate, “BAL0891,” had received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML). In fact, the stock price surged to the daily upper price limit in the market immediately after the press release was issued.

A SillaJen official stated, “The stock price rose to the daily upper limit after the press release regarding the FDA’s Orphan Drug Designation was issued.”

BAL0891 is an anticancer candidate that simultaneously targets TTK and PLK1, key proteins involved in the process of cancer cell division. SillaJen is developing it as a first-in-class anticancer drug that inhibits both targets simultaneously and is currently conducting a global Phase 1 clinical trial for solid tumors and blood cancers.

This FDA Orphan Drug Designation has also bolstered the development strategy for BAL0891 in the treatment of blood cancers. The target indication, AML, is a blood cancer characterized by the rapid proliferation of abnormal myeloid cells in the bone marrow. In addition to the disease’s rapid progression, recurrence after treatment and drug resistance are considered major challenges.

SillaJen’s expectations for BAL0891 lie in its differentiated mechanism of action, which simultaneously inhibits TTK and PLK1. Both proteins are involved in the cell division process. The strategy for BAL0891 is to simultaneously target these two molecules involved in cancer cell division, thereby opening up new treatment possibilities for AML patients who have developed resistance to existing therapies.

There are also tangible development benefits associated with the orphan drug designation. SillaJen has secured eligibility for the FDA’s clinical research grant program and is eligible for benefits such as a 25% tax credit on clinical trial costs in the U.S., FDA advisory support for clinical trial plans and expedited review, and a waiver of new drug application fees.

If BAL0891 ultimately receives full marketing approval as an AML treatment, the company could, in principle, secure seven years of market exclusivity in the U.S. for that specific drug and indication. Analysts attribute the significant surge in investor sentiment to the company securing favorable conditions for future development and commercialization at this early stage of clinical trials.

However, FDA Orphan Drug designation does not guarantee the clinical success of BAL0891 or its likelihood of receiving marketing approval. As the drug is currently in Phase 1 clinical trials, its safety and efficacy must be demonstrated in patients. Following today’s sharp rise in the stock price, actual clinical data is expected to be the key factor in determining the company’s market value.

A SillaJen official stated, “Since BAL0891 was designated as an orphan drug from the early clinical stages, we view this as a degree of official recognition of its medical significance and potential,” adding, “Based on this achievement, we will do our utmost to produce even more positive results in the remaining clinical trials.”

Sharp Reversal Just One Day After Technology Transfer; Hanmi Group Stock Weakens

The sentiment surrounding Hanmi Group stocks, which had surged the previous day on the back of major technology transfer news, took a sharp turn just one day later. Hanmi Science closed at 46,300 won, down 18.05% (10,200 won) from the previous trading day. Hanmi Pharmaceutical also fell 7.22% (39,000 won) to close at 501,000 won.

Hanmi Pharmaceutical announced the previous day that it had transferred the core pipeline of its new obesity drug project, “H.O.P.” (Hanmi Obesity Pipeline), to Genentech, a subsidiary of Roche. The deal is valued at up to $2.3 billion, with an upfront payment of $190 million. Although shares of both Hanmi Pharmaceutical and Hanmi Science surged in tandem the previous day on the news of this major technology transfer, they gave up a significant portion of those gains in just one day.

This is believed to be due to a flood of short-term profit-taking after the major positive news of the technology transfer was quickly reflected in the stock price immediately following the announcement. However, market analysts suggest that Hanmi Pharmaceutical’s momentum may continue regardless of the stock price.

In fact, the H.O.P. project—which has yielded this technology transfer success—is Hanmi Pharmaceutical’s next-generation obesity drug project consisting of six candidate compounds. Since its launch in September 2023, the H.O.P. project is on the verge of its first commercialization success in approximately three years. Epfeglenatide (Epe), scheduled for launch in the second half of this year, is a drug that Hanmi Pharmaceutical licensed to Sanofi in 2015 as a new diabetes treatment.

Despite the subsequent return of rights, Hanmi Pharmaceutical did not halt development but continued its independent research, expanding the drug’s development scope to an obesity treatment and positioning it as the first commercialized product of the H.O.P Project.

The launch of Epe is significant as it marks the first new obesity drug to be introduced to the market since the launch of the H.O.P. Project. In particular, given that the drug has progressed from a past technology export and the return of rights to commercialization through in-house development, it serves as a testament to Hanmi Pharmaceutical’s long-term R&D capabilities and commitment to new drug development.

Specifically, HM17321 is a non-incretin UCN2 (Urocortin-2) analog. It addresses the limitation of existing GLP-1-based obesity treatments, which can reduce lean body mass—including muscle—in addition to significant weight loss. The drug aims to be the first-in-class treatment in its class that selectively reduces body fat while preserving or improving muscle mass and function.

The industry is highlighting the significance of Vice Chairman Lim Ju-hyun’s role in the design and development of the H.O.P. project. A Hanmi Pharmaceutical official stated, “Although there were various challenges, including a management dispute, following the passing of Chairman Lim Seong-ki, this is the result of continuing research and development to the end without losing sight of Hanmi Pharmaceutical’s identity as a new drug developer,” adding, “Even when the H.O.P. project faced potential setbacks, Vice Chairman Lim Ju-hyun maintained a steady course and provided the support necessary to see the project through to completion.”