FDA Awards Fast Track Designation to LBS-007 for Acute Myeloid Leukemia

The FDA has granted fast track designation to LBS-007, a non-ATP competitive CDC7 inhibitor, for use as a potential therapeutic option in patients with acute myeloid leukemia (AML).¹

The agent inhibits CDC7 in cell cycle regulation; it targets S phase progression, inhibits the role of CDC7 in DNA replication, and prevents cell division.2 In preclinical studies, LBS-007, which also obtained orphan drug designation from the agency in March 2018, has showcased activity against leukemia and solid tumors, particularly in chemotherapy-resistant and other cancer cell lines.^1,3

The safety and tolerability of LBS-007 are under examination in patients with relapsed or resistant acute leukemias as part of a phase 1/2 trial (NCT05756322), which is currently recruiting.⁴

“We are thrilled to see LBS-007 showing signs of early treatment response and potential efficacy in addressing this critical unmet medical need,” Tom Lin, PhD, chairman of Lin BioScience, stated in a news release.¹ “Receiving FDA fast track designation is a significant milestone, offering an expedited pathway to advance the development of this promising therapy. We remain deeply committed to delivering transformative solutions for patients in need.”

The open-label, dose-escalation and -expansion study is enrolling patients at least 18 years of age who have a pathologically confirmed diagnosis of relapsed or resistant AML or acute lymphoblastic leukemia.⁴ Patients cannot be eligible for standard treatment anticipated to lead to durable remission or cure or who do not have known options with documented benefit. They also need to have an ECOG performance status up to 2.

If they are receiving concomitant chemotherapy, radiation, or immunotherapy, they will be excluded. Those receiving any other investigational agents concurrently or within 30 days before screening, those with acute promyelocytic leukemia or leukemia with active central nervous system involvement, or those with a history of another active malignancy within 2 years before entering the study, will not be included. Other exclusion criteria include mental deficits and/or psychiatric history.

In the first phase of the research, LBS-007 will be evaluated both as a single agent and paired with venetoclax (Venclexta) and azacitidine to identify optimal dosage. The phase 2 dose-expansion portion will further evaluate the monotherapy and combinations at the dose determined in the first phase.

The key objectives of the trial are to identify the number, severity, and duration of adverse effects (AEs) and treatment-related AEs by CTCAE v.5 criteria and to determine the recommended phase 2 dose of LBS-007 in this patient population. Secondary objectives will look at the drug's pharmacokinetic profile, including the maximum plasma concentration, time to maximum plasma concentration, and the area under the drug concentration-time curve of the drug in plasma. The drug's efficacy will also be examined.

“During the phase 1 dose escalation, we have not observed any Definitely or Probably Related Adverse Effects in all patients who have received low to high doses, demonstrating the safety profile is very tolerable and promising,” Irene Wang, PhD, president & chief scientific officer of Lin BioScience, added in the news release.¹

The study is estimated to be completed by December 2025.⁴

References

1. Lin BioScience receives US FDA fast track designation for LBS-007. News release. Lin Bioscience. November 27, 2024. Accessed December 2, 2024. https://www.linbioscience.com/IM/NewsDetail/75
2. Bringing hope to incurable disease. Lin Bioscience. November 2023. Accessed December 2, 2024. https://www.linbioscience.com/_Files/Company/0B7CBD08A2EABD484A1A5DBF3C6BC63C/Lin%20Bioscience%20-%20Company%20Presentation%20EN%20202311.pdf
3. Pipeline. Lin Bioscience website. Accessed December 2, 2024. https://www.linbioscience.com/Pipeline/LBS007
4. The safety and tolerability of LBS-007 in patients with relapsed or resistant acute leukaemias. ClinicalTrials.gov. Updated October 15, 2024. Accessed December 2, 2024. https://clinicaltrials.gov/study/NCT05756322