[Edaily Reporter KIM SAE-MI ] SillaJen, Inc.(215600)is expanding the development scope of its next-generation anticancer candidate “BAL0891” from solid tumors to blood cancers. Given that early signs of efficacy have been confirmed in solid tumor clinical trials, all eyes are on whether follow-up development covering both solid and blood cancers will gain momentum.

'BAL0891'

Granted

Orphan Drug Designation

by

U.S. FDA… Secures Benefits for AML Development

According to the biotech industry on the 26th, SillaJen, Inc.’s BAL0891 was recently designated as

an orphan drug

by

the U.S. Food and Drug Administration (FDA

) for the treatment of acute myeloid leukemia (AML).

SillaJen, Inc. is currently conducting a global Phase 1 clinical trial of BAL0891 in patients with solid tumors and blood cancers. BAL0891 is a mitotic checkpoint inhibitor (MCI) designed to simultaneously inhibit threonine/tyrosine kinase (TTK) and polo-like kinase 1 (PLK1), both of which are involved in the cancer cell division process. SillaJen, Inc. is developing the drug with the goal of creating a first-in-class new drug that simultaneously inhibits these two targets.

This Orphan Drug designation expands the support available during the AML clinical development process. SillaJen, Inc. can now take advantage of benefits such as eligibility for clinical research funding programs, tax credits for eligible clinical trial costs in the U.S., and waivers of new drug application fees. Furthermore, if the drug receives FDA marketing approval as an AML treatment, SillaJen, Inc. will secure seven years of market exclusivity in the U.S. as an orphan drug.

Early Signs of Efficacy in Solid Tumors… Original Patent Secured for 3.5 Billion Won

Early clinical data is accumulating for solid tumors. According to the Phase 1 interim results presented by SillaJen, Inc. at the American Society of Clinical Oncology (ASCO 2026) last May, a total of 61 patients with advanced or metastatic solid tumors had received BAL0891 as of the end of last year. Of these, 88% had previously undergone at least two courses of treatment for metastatic cancer.

In the 4-week cycle BAL0891 monotherapy group, one complete remission (CR) and one partial remission (PR) were confirmed. In the paclitaxel combination group, 3 out of 8 evaluable patients showed PR, and 4 showed stable disease (SD), resulting in a disease control rate (DCR) of 88%. Given that this early-phase clinical trial demonstrated signs of tumor reduction and disease control, attention is now focused on the results of subsequent clinical trials.

Safety data necessary for dose determination were also obtained. Treatment-related adverse events of Grade 3 or higher occurred in 62.3% of all patients, with the main adverse events including neutropenia and leukopenia. In one dosing regimen for monotherapy, the maximum tolerated dose (MTD) was set at 240 mg and the recommended Phase 2 dose (RP2D) at 160 mg, thereby specifying the dosages to be used in subsequent clinical trials.

BAL0891 is a pipeline asset that SillaJen, Inc. acquired from the Swiss company Basilia in 2022. At that time, the company secured development assets and rights related to BAL0891, including patents, know-how, and an FDA Investigational New Drug (IND) application.

Last year, the company additionally secured foundational patents for BAL0891—including substance patents and biomarker patents—from the original developer, Crossfire Oncology Holding B.V. of the Netherlands, for 2 million Swiss francs (approximately 3.5 billion won). This also resolved the obligation to pay Crossfire up to 172 million Swiss francs (approximately 296.5 billion won) in milestone payments based on the development stage.

SillaJen, Inc. plans to confirm the potential of BAL0891 in acute myeloid leukemia (AML) following its success in solid tumors. The company is currently conducting a Phase 1 clinical trial in AML patients in the U.S., South Korea, and other countries, and plans to solidify its development strategy by sequentially gathering data for both indications. If efficacy is confirmed in both solid tumors and blood cancers, the scope of application for BAL0891 could expand significantly.

Continuing R&D with Support from M2N Co.,Ltd… Expanding Revenue Base Through Merger with WOOSUNG Pharmaceutical

A key factor enabling SillaJen, Inc. to continue its global clinical trials is

the support

of its largest shareholder,

#M2N Co.

,Ltd. M2N Co.,Ltd became the largest shareholder in June 2021 by participating in a 60 billion won third-party private placement while SillaJen, Inc.’s stock was under a prolonged trading suspension. Since then, SillaJen, Inc. has rebuilt its research and development (R&D) framework by introducing new pipeline candidates and resuming global clinical trials. Currently, SillaJen, Inc. belongs to a corporate group that includes M2N Co.,Ltd., The LEADCORP, Inc., and MASON CAPITAL CORPORATION.

SillaJen, Inc. is also expanding its own business foundation to support new drug development. Last year, the company invested 12.5 billion won to acquire a 100% stake in WOOSUNG Pharmaceutical, a company specializing in intravenous solutions, and subsequently merged with it in July of the same year. Through this merger, SillaJen, Inc. added a specialty pharmaceutical sales and development business to its existing research and development of new anticancer drugs. The company currently sells antipyretic analgesics, antiviral drugs, and mineral injections, and is also developing generics and improved new drugs. This is interpreted as a strategy to secure a stable revenue base separate from new drug development, thereby enhancing the sustainability of its R&D efforts.

Additional clinical data on BAL0891 is also scheduled to be released in the future. SillaJen, Inc. plans to present Phase 1 clinical data on BAL0891 for solid tumors as a poster at the European Society for Medical Oncology (ESMO 2026) conference, to be held in Madrid, Spain, this October. Updated results on the safety and efficacy of BAL0891 as a monotherapy and in combination with paclitaxel are expected to be disclosed.

A biotech industry official stated, “Orphan drug designation signifies that the commercial viability and efficacy of the pipeline have been officially validated,” adding, “The data releases scheduled to begin at the end of this year will serve as a turning point for the revaluation of the company’s market value.”