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Also known as Carrot Cells Expressing Recombinant Human Glucocerebrosidase, plant expressed recombinant human glucocerebrosidase
PRX-112 · 2 trials · 1 indication
| Name | Type | Description |
|---|---|---|
| PRX-112 | DRUG | - |
PRX-112 is an investigational oral therapy being developed for Gaucher disease, a rare inherited lysosomal storage disorder. It is a small molecule designed to target the enzyme glucocerebrosidase. Protalix BioTherapeutics is developing PRX-112, which has been studied in Phase 1 and Phase 2 clinical trials in patients with Gaucher disease.
PRX-112 targets glucocerebrosidase, the enzyme deficient in Gaucher disease. By acting on this enzyme target, the small molecule is intended to address the underlying enzyme deficiency associated with the condition. Glucocerebrosidase is a lysosomal enzyme responsible for breaking down glucocerebroside, and its dysfunction leads to the accumulation of this substrate in Gaucher disease.
PRX-112 is being developed by Protalix BioTherapeutics, Inc., which trades under the ticker PLX. The company is advancing the oral small molecule for Gaucher disease. Protalix BioTherapeutics has sponsored the clinical trials evaluating PRX-112 in patients with this rare lysosomal storage disorder.
PRX-112 is in Phase 2 clinical development for Gaucher disease. It has completed two clinical trials: a Phase 1 study and a Phase 2 study. PRX-112 is investigational and has not been approved by the FDA. Both completed trials enrolled a total of 26 patients and were conducted in Gaucher disease.
PRX-112 has been evaluated in two completed clinical trials. The Phase 2 trial, NCT02107846, was an open-label dose escalation study of oral PRX-112 with 10 patients. The Phase 1 trial, NCT01747980, assessed safety and pharmacokinetics of oral PRX-112 in 16 Gaucher disease patients. Both studies are completed.