#United States #Waltham #AML #Stelexis BioSciences #Eganelisib

Promising Phase 1 Clinical Trial Results for Eganelisib

Stelexis BioSciences, Inc., a Waltham-based biopharmaceutical company, has revealed promising findings from its Phase 1 clinical trial of Eganelisib, an oral treatment targeting myeloid malignancies such as relapsed/refractory acute myeloid leukemia (R/R AML) and higher-risk myelodysplastic syndromes (HR-MDS). The results were shared during an oral session at the 2026 ASCO Annual Meeting by Dr. Mendel Goldfinger of Montefiore Einstein Comprehensive Cancer Center.

The trial focused on a difficult-to-treat patient population, primarily featuring those exhibiting adverse prognostic indicators. Notably, about 29% had TP53 genetic alterations, while a striking 90% demonstrated abnormal cytogenetics. Moreover, 76% of participants had undergone more than two prior therapies, indicating the significant challenges faced by physicians and patients alike in managing these conditions.

Key Findings from the Trial

The study highlighted several key outcomes, particularly the efficacy of Eganelisib in patients with high PI3K-gamma expression. The modified objective response rate showed that 50% of these patients achieved complete remissions or stable disease with associated hematologic improvement, contrasting sharply with the 0% response in patients expressing lower levels of PI3K-gamma. This finding was statistically significant, underscoring the potential relevance of PI3K-gamma as a treatment biomarker.

Among those who responded, improvements in neutrophil counts were rapidly observed, with four out of six participants experiencing normalization of their counts. This response aligns well with the drug’s aim of re-engaging myeloid differentiation without the intrinsic blood count suppression seen with other treatments.

Overall survival rates also underscored the drug's potential benefit. Patients with high PI3K-gamma expression reported a median overall survival of 27 weeks compared to just 9.9 weeks for those with lower expression levels, further validating the findings in light of historical data.

Safety and Tolerability Profile

Importantly, Eganelisib was reported to be well tolerated across both test doses—45 mg and 60 mg—without significant dose-limiting toxicities or intrinsic hematologic side effects. Adverse events reported were mostly disease-related rather than medication-related, with no severe adverse events attributed to Eganelisib itself.

This favorable safety profile is essential in improving treatment options for AML and MDS patients, who often exhaust therapeutic avenues without significant success.

Continuing Efforts in Research

Dr. Konstantinos N. Aprilakis, a board member at Stelexis, remarked on the encouraging nature of these early data, highlighting not only the safety and efficacy of Eganelisib but also the identification of a biomarker that could aid in selecting patients who would most likely benefit from this targeted therapy.

The research team at Stelexis anticipates moving forward with additional clinical trials and plans to further explore combinations of Eganelisib with hypomethylating agents among treatment-naïve patients characterized by high levels of PI3K-gamma. This initiative aims to enhance survival outcomes for those diagnosed with AML and MDS, which are notorious for poor prognoses under existing treatments.

Conclusion

Stelexis BioSciences is paving the way for new and exciting possibilities in the treatment landscape of myeloid malignancies. Eganelisib represents a significant step forward as a targeted therapy, showcasing the game's potential to redefine how clinicians approach AML and HR-MDS treatment. Patients who previously faced limited options may soon have hope for better outcomes, a testament to the innovative spirit and scientific rigor at Stelexis.