SillaJen's Acute Myeloid Leukemia Treatment Candidate Designated as FDA Orphan Drug

Next-Generation Anticancer Agent Targeting Both TTK and PLK1

Currently Undergoing Global Phase 1 Clinical Trials

On August 25, SillaJen announced that its next-generation anticancer pipeline candidate, "BAL0891," currently under development, has been designated as an orphan drug for acute myeloid leukemia (AML) by the U.S. Food and Drug Administration (FDA).

BAL0891 is a first-in-class anticancer candidate that simultaneously targets the essential proteins TTK and PLK1, both of which play key roles in cancer cell division. The compound is now undergoing a global Phase 1 clinical trial for both solid tumors and hematologic malignancies.

The FDA’s orphan drug designation is granted to medicines being developed for rare diseases affecting fewer than 200,000 people in the United States. With this designation, SillaJen is now eligible to apply for FDA grants to support the clinical development of BAL0891. Additionally, the company will be able to receive benefits such as a 25% tax credit on clinical trial expenses incurred in the U.S., FDA advisory support for clinical trial design and expedited review, and a waiver of new drug application (NDA) fees.

If ultimately approved for marketing authorization, SillaJen would, in principle, secure a seven-year period of market exclusivity for the same drug and indication in the United States.

AML is a type of blood cancer characterized by the rapid proliferation of hematopoietic stem cells in the bone marrow that have turned into cancer cells. In its early stages, it is often asymptomatic, making early detection difficult, and the disease progresses rapidly.

Existing treatments for AML mainly rely on strong chemotherapeutic agents and hematopoietic stem cell transplantation, both of which have been criticized for causing significant side effects and posing a risk of disease relapse. While targeted therapies aimed at specific genes or proteins have recently emerged, there remains a limitation in that resistance may develop during the course of treatment.

According to the company, BAL0891’s dual inhibitory mechanism on TTK and PLK1—which are involved in cancer cell division—suggests that it could serve as a new treatment option for AML patients who have developed resistance to current therapies.

A SillaJen official stated, "The fact that BAL0891 received orphan drug designation at such an early clinical stage is seen as a degree of recognition of its medical importance and potential. Building on this achievement, we will do our utmost to deliver even more positive results in the remaining clinical milestones."

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