Drug intelligence / Profile preview

AM001

Development stage
Unknown
Lead developer
AmDerma Pharmaceuticals
Modality
Small Molecules
Administration
Topical
01

Overview

AM001 is a topical cream formulation of sodium phenylbutyrate, a small molecule histone deacetylase (HDAC) inhibitor, developed by AmDerma for the treatment of plaque psoriasis. Sodium phenylbutyrate is traditionally known as an ammonia scavenger used systemically for urea cycle disorders; however, in AM001, it is utilized for its epigenetic modulating properties. By inhibiting HDACs, the drug aims to normalize keratinocyte differentiation, reduce excessive cell proliferation, and suppress the production of pro-inflammatory cytokines within psoriatic lesions. The drug was evaluated in Phase 2 clinical trials to assess its safety and efficacy in patients with moderate to severe plaque psoriasis.

Other names
sodium phenylbutyrate creamAM001 Cream, 7.5%
02

Targets

FGFR3 (Fibroblast growth factor receptor 3)EPAC1 (Rap guanine nucleotide exchange factor 3)

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