Drug intelligence / Profile preview

ornithine phenylacetate

Development stage
Phase 3
Lead developer
Mallinckrodt
Modality
Small Molecules
Administration
Intravenous, Oral
01

Overview

**Ornithine phenylacetate** is an investigational small-molecule ammonia-scavenging agent developed for hyperammonemia associated with advanced liver disease, particularly overt hepatic encephalopathy. It is a single crystalline salt composed of L-ornithine and phenylacetate. L-ornithine promotes peripheral ammonia incorporation into glutamine through glutamine synthetase, while phenylacetate conjugates glutamine to form phenylacetylglutamine, which is eliminated in urine; together these actions provide a non-urea-cycle route for net nitrogen removal. The program has been developed as intravenous and oral formulations, including OCR-002, MNK6105, MNK6106, YAQ006, and YAQ007. Yaqrit and its subsidiary Amalive are advancing intravenous and oral formulations for acute treatment and recurrence prevention of hepatic encephalopathy.

Other names
L-ornithine phenylacetateL-OPAOPAL-ornithine salt of phenylacetic acid
02

Targets

GLS1 (Glutaminase 1)GLUL (Glutamine synthetase)

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