US-based biotechnology company Glycomine has secured $115m in Series C funding to progress the mannose-1-phosphate replacement therapy GLM101 into a randomised Phase IIb trial.

PMM2-CDG stems from a genetic mutation in the PMM2. Credit: Braňo on Unsplash.
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The funding round was spearheaded by CTI Life Sciences Fund, Advent Life Sciences, and abrdn-managed funds.
Current investors, including Sanofi Ventures, Novo Holdings, RiverVest Venture Partners, Sanderling Ventures, Abingworth, Chiesi Ventures, Asahi Kasei Ventures, and Remiges Ventures, also participated in the round.
Glycomine noted that the therapy is being developed to treat the rare and potentially fatal phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).
The ongoing Phase II trial of the therapy has enrolled over 20 subjects in the US and Europe, with the recent commencement of dosing in children.
Glycomine’s open-label Phase II trial data have shown improvements in ataxia symptoms among nine adolescent and adult subjects.
Over a 24-week period, these subjects experienced an average 11.9-point improvement on the International Cooperative Ataxia Rating Scale (ICARS) when treated with the therapy.
In light of this funding round, the company welcomed Advent Life Sciences general partner Dominic Schmidt and CTI Ventures managing partner Youssef Bennani to its board of directors.
Glycomine CEO Steve Axon said: “We are excited to partner with our new investors who have strong track records in rare diseases, and for the continued support from our existing investors.
“This financing will enable us to advance GLM101 into a randomised, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG.”
PMM2-CDG, previously referred to as CDG-1a, is said to be the most common congenital condition of glycosylation, stated the company.
The disorder stems from the phosphomannomutase 2 (PMM2) genetic mutation, leading to the protein’s decreased activity.
The therapy secured orphan drug designation in the European Union and the US. It has also obtained the fast track and rare paediatric disease designations in the US.