ABOUT GLM101

GLM101: a potential PMM2-CDG treatment

GLM101: a potential PMM2-CDG treatment

Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding, where no other therapeutic options exist and replacing a missing component would restore function. The company’s replacement therapy approach aims to deliver substrates, enzymes, or proteins and to target these molecules to clinically relevant cellular compartments. GLM101 is Glycomine’s first drug from its platform and is designed to intracellularly deliver mannose-1-phosphate as a potential treatment for PMM2-CDG (also known as CDG-1a). GLM101 is currently in clinical trials in the U.S. and Europe and has received Orphan Drug Designation (ODD) in the U.S. and Europe and Rare Pediatric Disease Designation (RPDD) in the U.S.

GLM101 is a mannose-1-phosphate replacement therapy

GLM101 is a mannose-1-phosphate replacement therapy

PMM2-CDG is caused by a deficiency of the enzyme phosphomannomutase 2 (PMM2), which converts mannose-6-phosphate to mannose-1-phosphate. Mannose-1-phosphate is an essential sugar molecule in the N-glycosylation pathway and is crucially important for proper protein structure and function. GLM101 is a mannose-1-phosphate replacement therapy that is designed to supply the missing mannose-1-phosphate into cells. Once inside, it can then be incorporated into the N-glycosylation pathway to restore disrupted protein function. Treatment with GLM101 is intended to bypass the genetic block in the pathway, regardless of mutation type, and provide continuous exposure over long periods of time.

Clinical trial of GLM101 for the treatment of PMM2-CDG

Clinical trial of GLM101 for the treatment of PMM2-CDG

Glycomine has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG. The company is currently dosing healthy volunteers in an open-label Phase 1 clinical study to evaluate safety and tolerability. With successful completion, Glycomine plans to open enrollment for patients in the second half of 2022.

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A natural history study of PMM2-CDG symptoms

A natural history study of PMM2-CDG symptoms

Since glycosylation is such an important and ubiquitous process essential to protein function, the body’s failure to correctly produce glycans causes a wide array of symptoms. Individuals with PMM2-CDG typically develop signs and symptoms of the condition during infancy. Although the symptoms can vary, many infant patients will display hypotonia (muscle weakness), feeding problems, and failure to thrive. Since almost any organ system can be affected, multiple other symptoms may emerge including liver disease, cardiomyopathy, coagulopathies, stroke-like episodes, and developmental delays. 

To learn more about how PMM2-CDG affects patients, Glycomine has initiated a natural history study, which is the largest longitudinal study ever undertaken for this disease. Initial data have uncovered that secondary adrenal insufficiency is a significant risk factor for this disease that is often masked by other comorbidities. Based on this finding, it is recommended that morning cortisol and ACTH levels should be evaluated at least annually for all patients with PMM2-CDG.

Learn more about the initial data from the natural history study here. For more information about this ongoing study (GLY-000) please visit clinicaltrials.gov and search for NCT03173300.

LEARN MORE ABOUT THE NATURAL HISTORY STUDY