Shanghai-based biotechnology company GenFleet Therapeutics has announced that its CDK9 inhibitor, SLS009 (GFH009), has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML). This designation is a significant milestone, recognizing the drug’s potential to address a rare and serious medical condition.
Phase I Study Results
The orphan drug designation is supported by the results of a Sino-US multi-center Phase I study, which has successfully met all key study objectives. The study evaluated GFH009 as a monotherapy in patients with recurrent/refractory hematological tumors, demonstrating superior safety and tolerability. Notably, there were no dose-limiting toxicity events that exceeded expectations or posed evaluation challenges.
Therapeutic Effects and Patient Outcomes
In terms of therapeutic efficacy, GFH009 has shown significant anti-tumor activity. Post-treatment, a maximum reduction rate of 77.3% was observed in medulloblasts. A remarkable 97% of participants exhibited a significant decrease in the expression of MCL1 and MYC in their peripheral blood samples to the expected levels. Additionally, multiple patients were observed to achieve complete and partial remission, indicating the drug’s potential in providing meaningful responses in AML patients.
Conclusion
The orphan drug designation for SLS009 (GFH009) underscores GenFleet Therapeutics’ commitment to developing innovative treatments for acute myeloid leukemia. With a successful Phase I study and the potential for improved patient outcomes, GFH009 holds promise as a new therapeutic option for AML patients in need of effective treatment.-Fineline Info & Tech
