Drug intelligence / Profile preview

cyclophosphamide + azathioprine + prednisolone

Development stage
Phase 3
Lead developer
Tehran University of Medical Sciences
Modality
Small Molecules
Administration
Intravenous, Oral
01

Overview

This cytotoxic combination therapy is a multi-drug immunosuppressive regimen used as a standard-of-care or control arm in clinical trials for Behcet's disease, particularly for resistant ocular manifestations such as retinal vasculitis and posterior uveitis. The regimen consists of three primary components: pulse cyclophosphamide (an alkylating agent), azathioprine (a purine synthesis inhibitor), and prednisolone (a corticosteroid). Cyclophosphamide works by cross-linking DNA to inhibit cell division, azathioprine acts as a prodrug that interferes with purine metabolism to suppress T and B cell proliferation, and prednisolone provides broad anti-inflammatory effects by activating glucocorticoid receptors. In a Phase 2 pilot study conducted by the Tehran University of Medical Sciences (NCT00700297), this combination was used as the comparator arm against rituximab in patients with ocular lesions resistant to conventional therapy.

Other names
Cytotoxic Combination
02

Targets

PPAT (Amidophosphoribosyltransferase)DNAGR (Glucocorticoid receptor)

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