Piperidine is an azacycloalkane that is cyclohexane in which one of the carbons is replaced by a nitrogen. Although piperidine is a common organic compound, it is an immensely important class of compounds medicinally: the piperidine ring is the most common heterocyclic subunit among FDA approved drugs.
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Aberrant MYC pathway activation is found in cancer cells, while MYC-induced protein translation depends on GSPT1. Molecular glues targeting GSPT1 is identified as a potential treatment method in oncology. Monte Rosa’s MRT-2359 is an oral and selective molecular glue degrader of the translation termination factor GSPT1. The targeted GSPT1 degradation results in disrupting MYC-driven protein translation and reducing MYC-oncogenic signaling.
MRT-2359 is in an ongoing phase 1/2 study in MYC-driven solid tumors with a current assessment of 0.75 mg dose level. The final phase 2 dose determination and updated clinical results are anticipated in the second half of 2024. MRT-2359 previously received Fast Track Designation in patients with previously treated, metastatic NSCLC with L-MYC or N-MYC expression, Orphan Drug Designation in small cell lung cancer (SCLC), and Fast Track Designation in previously treated, metastatic SCLC with L-MYC or N-MYC expression.