Pharma Focus Europe

Glycomine Marks a Significant Milestone as the First Pediatric Patient Receives Dosing in Phase 2 of the Clinical Study for GLM101, a Prospective Treatment for PMM2-CDG

Tuesday, December 12, 2023

Glycomine, Inc., a biotechnology company in the clinical stage, has recently announced the initiation of dosing in the first pediatric patient for a multi-center open-label Phase 2 study of GLM101. GLM101 is a mannose-1-phosphate replacement therapy designed to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Steven Axon, CEO of Glycomine, emphasized the importance of this development, stating, "The commencement of dosing in pediatrics is a significant milestone for the company." Axon expressed optimism regarding the observed biomarker and clinical improvements in the dose-range finding portion of the trial involving adult patients. He further highlighted GLM101 as the first potential disease-modifying therapy under evaluation for PMM2-CDG.

Dr. Mercedes Serrano of Sant Joan de Déu Hospital in Barcelona conveyed enthusiasm about the ongoing collaboration with Glycomine, stating, "We are delighted to continue our collaboration with Glycomine and move forward with GLM101 to initiate dosing in pediatrics." She emphasized the potential positive impact that GLM101 could have on PMM2-CDG patients and the broader community.

The Phase 2 study, following a dose range finding study involving 10 adult PMM2-CDG patients, will focus on pediatric patients with a confirmed PMM2-CDG diagnosis. This open-label study spans 24 weeks and aims to assess potential pharmacodynamic activity, clinical efficacy, safety, tolerability, and pharmacokinetics of GLM101 ( Identifier: NCT05549219).

GLM101 is a therapy designed as a mannose-1-phosphate replacement for PMM2-CDG, formerly known as CDG Type Ia. It has received Orphan Drug Designation in the U.S. and Europe, as well as Rare Pediatric Disease Designation in the U.S. PMM2-CDG is caused by genetic mutations leading to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). GLM101 is engineered to deliver mannose-1-phosphate directly into cells, bypassing the PMM2 enzyme deficiency and addressing disease-causing PMM2 mutations to restore pathway function.



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