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Pevonedistat Novel Therapy Developed for the Treatment of Patients with HR-MDS

Takeda Pharmaceutical has developed Pevonedistat, a NEDD8-activating enzyme (NAE) inhibitor,a novel treatment for Higher-Risk Myelodysplastic Syndromes (HR-MDS) patients.

Pevonedistat is a first-in-class NEDD8-Activating Enzyme (NAE) inhibitor which blocks the modification of select proteins, resulting in disruption of cell cycle progression and cell survival, leading to cancer cell death.

HR-MDS is associated with poor prognosis, diminished quality of life and a higher chance of transformation to acute myeloid leukemia, another aggressive cancerresulting in people. Hence, combination of pevonedistat and azacitidine shows a promising therapeutic result with the potential to be the first novel treatment advancement for higher-risk MDS.

Pevonedistat is currently being evaluated in Phase 3 studies as a first-line treatment for patients with HR-MDS, HR-CMML and AML, who are ineligible (unfit) for transplant or intensive induction chemotherapy, and in a Phase 2 study in unfit AML in a triple combination with azacitidine and venetoclax. Pevonedistat is an investigational drug for which safety and efficacy have not been established.

US Food and Drug Administration (FDA) grants Breakthrough Therapy Designation for Pevonedistat.The Breakthrough Therapy Designation is based on the final analysis of the Pevonedistat-2001 Phase 2 study, which evaluated pevonedistat plus azacitidine versus azacitidine alone in patients with rare leukemias, including HR-MDS.

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