#USA #Waltham #Syndax Pharmaceuticals #Revuforj #AML Trials

Revuforj® Shows Promise in Treating Relapsed or Refractory Acute Myeloid Leukemia

Syndax Pharmaceuticals, a leading biopharmaceutical company based in Waltham, Massachusetts, has announced additional positive outcomes from its AUGMENT-101 trial for its investigational medication, Revuforj® (revumenib). This oral, first-in-class menin inhibitor is specifically designed for patients suffering from relapsed or refractory (R/R) mutant NPM1 acute myeloid leukemia (AML).

Overview of the Trials

The company reported encouraging subgroup analysis results from their Phase 2 clinical studies involving 64 adult patients. Notably, the trials demonstrated significant therapeutic responses across various patient segments, including those who had undergone multiple prior treatments. The findings revealed a 26% complete remission (CR) plus complete remission with partial hematological recovery (CRh) rate amongst those enrolled in the efficacy-evaluable category.

Moreover, in the BEAT AML clinical trial, exploring a combination therapy of Revuforj with venetoclax and azacitidine in newly diagnosed AML patients, the results were even more striking. Results indicated a 100% overall response rate (ORR) among 37 evaluable patients, solidifying Revuforj's potential as a game-changing treatment in frontline settings.

In-depth Results from AUGMENT-101 Trial

The AUGMENT-101 trial of Revuforj focused on evaluating its effectiveness in R/R mutant NPM1 AML patients. The data revealed a 23% CR + CRh rate, indicating that the drug not only meets but exceeds expectations for treatment outcomes. Further scrutiny into minimal residual disease (MRD) status highlighted that 64% of patients who achieved CR/CRh showed MRD negativity, a promising indicator of the treatment's success in eliminating disease cells.

The analysis indicated that the drug maintained a consistent safety profile, which is a crucial factor when evaluating new cancer therapies. Syndax’s CEO, Michael A. Metzger, expressed confidence in the drug's transformative potential, stating that Revuforj's promising results signal a significant advancement in standard care practices for leukemia treatments.

Updates from the BEAT AML Trial

The BEAT AML trial represents another exciting chapter for Syndax, specifically targeting newly diagnosed patients with mNPM1 or KMT2A-rearranged AML. The trial's interim data suggest that Revuforj, when paired with venetoclax and azacitidine, delivers exceptional efficacy rates. With a 95% CRc rate, these findings further reaffirm Revuforj's potential to revolutionize treatment strategies employed against acute myeloid leukemia.

The patients enrolled in this trial presented a median age of 71, with results indicating a life-changing opportunity for older patients who are typically unfit for more aggressive chemotherapy.

Safety Profile and Future Implications

Syndax continues to monitor the safety profile of Revuforj, noting that reports of differentiation syndrome, a serious side effect, occurred in 15% of study participants. However, the majority of these events were manageable, showcasing the drug's tolerability. Importantly, no new safety signals were observed compared to the standard venetoclax/azacitidine regime.

The company plans to initiate a pivotal trial with the Revuforj combination for frontline treatment later this year, emphasizing their commitment to improving cancer patient outcomes. The anticipation surrounding Syndax Pharmaceuticals and their innovative therapies marks an exciting time for AML treatment advancements.

Conclusion

As reported at the 66th ASH Annual Meeting, the new data released from Syndax Pharmaceuticals concerning Revuforj lays a promising foundation for future treatment innovations in AML. This progress reflects the ongoing commitment to enhancing recovery and survival rates for patients battling this challenging disease, making Revuforj a focal point in current and upcoming oncology therapies.