Drug intelligence / Profile preview

eflornithine + lomustine

Development stage
Unknown
Lead developer
Orbus Therapeutics
Modality
Small Molecules
Administration
Oral
01

Overview

**eflornithine + lomustine** is a combination therapy evaluated for the treatment of recurrent grade 3 IDH-mutant astrocytoma, a rare malignant brain tumor. Eflornithine is a novel cytostatic agent that irreversibly inhibits *ornithine decarboxylase* (ODC), a key enzyme in polyamine biosynthesis which is upregulated in various cancers, thereby slowing tumor growth and cell proliferation. Lomustine is an alkylating agent from the nitrosourea class, which causes DNA cross-linking and damage, leading to apoptosis in cancer cells. The combination therapy, investigated in the Phase 3 STELLAR trial, displayed clinically meaningful improvements in median overall survival (34.9 months vs. 23.5 months) and progression-free survival (15.8 months vs. 7.2 months) over lomustine monotherapy in the specific patient subpopulation, although the primary endpoint of the study was not met in the entire trial population. The regimen was generally well tolerated, with myelosuppression and hearing impairment as notable grade 3+ adverse events[1][2][3][4][6][7].

02

Targets

ODC1 (Ornithine decarboxylase)DNA

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