Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year
Summary
California-based biopharma Glycomine announced a $115 million Series C raise Wednesday to push its lead rare disease molecule into Phase IIb trials.Were working on glycosylation disorders, putting sugar chains on proteins. The disorder that Glycomine is targeting is, according to Axon, the largest of that group: phosphomannomutase-2 congenital disorder of glycosylation, or PMM2-CDG, for which there is no FDA-approved treatment.We think its maybe up to 15- 20 thousand patients in [the] U.S. and Europe, which is a relatively large population for a rare disease, Axon said, likening the demographic size to Pompe disease.Glycomine is using the new funds to push its lead molecule, GLM101, into a randomized, placebo-controlled Phase IIb trial. In early 2024, the company encouraging data for a Phase II trial for the drug, showing improvements for nine adult and adolescent patients in ataxia, neurological symptoms characterized by loss of muscle control. Symptoms and severity and most patients experience some sort of neurological dysfunction, as well as language and motor issues from childhood. The lipid nanoparticle delivery vehicle is critical, Axon said, because it extends M1Ps half life in the bloodstream from a few minutes to upwards of 80 hours.Glycomine is also investigating mRNA treatments to produce enzyme replacements for PMM2-CDG, but so far the company is sticking with simply replacing the missing molecule, an approach Axon called simple and elegant.What weve settled on is something that has really wide biodistribution, he said.The company has gone through multiple raises of increasing sizes on its journey to the clinic.