US-based biotechnology company Glycomine has dosed the first paediatric subject in a Phase II clinical trial of GLM101 to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
The multicentre, open-label trial will analyse GLM101’s safety, clinical efficacy, tolerability, pharmacodynamic activity and pharmacokinetics in paediatric patients.
Subjects in the trial will be given the treatment for 24 weeks.
Glycomine initially dosed the first adult subject in a multicentre, open-label Phase II trial of the therapy for PMM2-CDG in January this year.
The company subsequently began the current trial in paediatric patients on observing promising findings from the study in ten adult patients with PMM2-CDG.
GLM101 is a mannose-1-phosphate replacement therapy that has obtained orphan drug designation in the US and Europe.
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The treatment has also received a rare paediatric disease designation in the US.
It works by delivering mannose-1-phosphate into cells directly to allow PMM2 enzyme deficiency to be bypassed.
This tackles the PMM2 mutations that cause PMM2-CDG so that pathway function can be restored.
PMM2-CDG is caused by genetic mutations that lead to phosphomannomutase 2 enzyme deficiency.
Glycomine CEO Steven Axon said: “The initiation of dosing in paediatrics is an important milestone for the company.
“We are encouraged by the biomarker and clinical improvements we have observed in the dose-range finding portion of the trial in adult patients, and we are excited to move forward into paediatrics with GLM101, the first potential disease-modifying therapy to be evaluated in PMM2-CDG.”
Glycomine is a clinical-stage biotechnology company focused on the development of new drugs to treat serious rare metabolism and protein misfolding associated ailments.
The company utilises replacement therapies such as substrates, enzymes and proteins and acts on molecules that are clinically significant to cellular compartments.
In 2021, it secured $68m in a Series B funding round to progress the development of GLM101 to the clinic for treating PMM2-CDG.