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

Glycomine, a San Carlos, California-based biotechnology startup, has successfully raised $115 million in a Series C funding round to further develop its leading drug candidate for a rare genetic disorder. The company's focus on addressing unmet needs in the treatment of congenital disorders of glycosylation (CDG) has attracted significant investor interest, positioning Glycomine as a potential game-changer in the rare disease therapeutics landscape.

Pioneering Treatment for PMM2-CDG

Glycomine's lead program, GLM101, targets phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), the most common form of CDG. These inherited disorders disrupt glycosylation, a crucial biological process that attaches sugar chains to proteins, affecting cellular function throughout the body. With an estimated 15,000 patients in the U.S. and Europe, PMM2-CDG currently lacks approved treatments, leaving patients with only supportive care options.

GLM101 aims to address this unmet need by delivering mannose-1-phosphate, a component essential for glycosylation that is deficient in PMM2-CDG patients. This approach bears similarities to enzyme replacement therapies used in other rare diseases but focuses on replacing a missing sugar molecule rather than an enzyme.

Steven Axon, Glycomine's CEO, emphasized the critical nature of their work, stating, "That need is extremely high. There's no other therapies for these patients. There's only supportive care."

Promising Clinical Progress and Future Plans

Glycomine's Series C funding follows encouraging Phase 2 data released last year, which indicated GLM101's potential impact on ataxia, a common symptom of PMM2-CDG. The new capital will support a more comprehensive Phase 2b study, set to commence in mid-2025. This randomized, placebo-controlled trial aims to enroll 40 to 50 participants, with results expected in 2026.

Axon expressed optimism about the upcoming study, noting that it "will really tell us whether the effect we were seeing in the earlier clinical program has been confirmed." Positive outcomes from this trial could pave the way for potential partnerships with pharmaceutical companies, as Axon suggested that such data would be "highly derisking for a pharma partner."

Investment Details and Company Outlook

The $115 million Series C round was led by CTI Life Sciences Fund, funds managed by Abrdn, and Advent Life Sciences. Other notable investors include Novo Holdings, Sanofi Ventures, and Abingworth. This latest funding builds upon Glycomine's previous $80 million raised across Series A and B rounds.

While Glycomine's primary focus remains on advancing GLM101 for PMM2-CDG, the company has hinted at a broader pipeline. Axon revealed that Glycomine is developing drug candidates for other rare diseases beyond PMM2-CDG, though specific details remain undisclosed.

As Glycomine progresses its lead candidate and expands its rare disease portfolio, the substantial Series C funding underscores growing investor confidence in the company's innovative approach to addressing critical unmet needs in the rare disease space.