Glycomine Secures $115M Series C Funding to Advance Rare Disease Treatment

Glycomine, a California-based biopharmaceutical company, has successfully raised $115 million in a Series C funding round to further develop its lead candidate for a rare genetic disorder. The financing will support the advancement of GLM101, a potential first-in-class treatment for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), into a pivotal Phase IIb clinical trial.

Breakthrough in Rare Disease Treatment

GLM101 is a mannose-1-phosphate replacement therapy designed to address PMM2-CDG, a rare inherited condition affecting approximately 15,000 to 20,000 patients in the United States and Europe. The disorder, which results from errors in glycosylation, can lead to a wide range of symptoms, including developmental delays, impaired motor function, and a 20% mortality rate within the first five years of life.

Glycomine CEO Steve Axon emphasized the critical need for effective treatments, stating, "There's no other therapies for these patients. There's only supportive care." The company's approach involves delivering mannose-1-phosphate, a component essential for glycosylation that is deficient in PMM2-CDG patients, using a lipid nanoparticle delivery system that extends its half-life in the bloodstream from a few minutes to up to 80 hours.

Promising Clinical Results and Future Plans

Early clinical data from a Phase II trial involving nine adult and adolescent patients showed encouraging results, with an 11.9-point improvement in ataxia scores over 24 weeks. Ataxia, characterized by a lack of muscle control, is a hallmark symptom of PMM2-CDG and serves as the primary clinical endpoint for GLM101's efficacy studies.

The newly secured funding will enable Glycomine to initiate a randomized, placebo-controlled Phase IIb trial by mid-2025. This study is expected to enroll 40 to 50 patients, ranging from two years old to adults, with data anticipated in mid-2026. Axon expressed optimism about potential regulatory discussions, noting the absence of FDA-approved treatments for PMM2-CDG.

Investment Details and Company Growth

The $115 million Series C round was led by CTI Life Sciences Fund, with participation from funds managed by abrdn and Advent Life Sciences. Existing investors, including Novo Holdings, Sanofi Ventures, and Abingworth, also contributed to the financing. As part of the deal, Youssef Bennani, Ph.D., Managing Partner at CTI Ventures, and Dominic Schmidt, Ph.D., General Partner at Advent Life Sciences, will join Glycomine's board of directors.

This latest funding round follows Glycomine's previous raises, including a $12 million Series A in 2016 and a $68 million Series B spread across 2019 and 2021. The company's steady progression through increasingly larger funding rounds reflects growing investor confidence in its innovative approach to treating rare genetic disorders.