Glycomine Raises $115 Million to Advance Therapy for Metabolic Disorder

Rare Daily Staff

Glycomine said it completed a $115 million series C financing to advance its lead experimental therapy GLM101, a treatment for a form of congenital disorders of glycosylation.

CTI Life Sciences Fund, funds managed by abrdn Inc., and Advent Life Sciences led the round, which included participation from existing investors Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.

PMM2-CDG, also known as CDG Type Ia, is the most prevalent of the more than 100 different congenital disorders of glycosylation (CDG). CDGs result in defective formation of glycan chains, which are essential for the structure and function of glycosylated proteins that represent as many as half of all proteins in the body. PMM2-CDG is caused by a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). PMM2 converts mannose-6-phosphate to mannose-1-phosphate, an essential sugar molecule in the N-glycosylation pathway and is crucial for proper glycoprotein structure and function. There is no approved treatment for the condition today.

GLM101 is designed to deliver mannose-1-phosphate directly into cells, bypassing the PMM2 enzyme deficiency and addressing all disease-causing PMM2 mutations to restore pathway function. It is a first-in-class mannose-1-phosphate replacement therapy in development for PMM2-CDG. GLM101 has received Orphan Drug designation in the United States and Europe, and Rare Pediatric Disease designation in the United States.

Glycomine has enrolled more than 20 patients across Europe and the U.S. in its ongoing phase 2 study and recently initiated dosing in pediatric patients. Data from the study have demonstrated promising improvements in ataxia, a hallmark debilitating manifestation of PMM2-CDG.

“This financing will enable us to advance GLM101 into a randomized, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG,” said Steve Axon, Glycomine’s CEO.

Photo: Steve Axon, Glycomine’s CEO