Glycomine has raised $115m in Series C financing to advance its lead investigational therapy, GLM101, into a Phase IIb clinical trial for an ultra-rare disorder.
GLM101 is being developed as a treatment for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare and life-threatening genetic disorder with no approved treatments.
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Glycomine is currently conducting an open-label Phase II clinical trial (NCT06657859) at sites in Europe and the US, with 20 patients enrolled to date. The company recently initiated dosing in paediatric patients. Interim results from nine adult and adolescent patients showed an average 11.9-point improvement over 24 weeks on the international cooperative ataxia rating scale, indicating potential clinical benefit in one of the most debilitating features of the disease.
The candidate is a mannose-1-phosphate replacement therapy aimed at correcting the metabolic deficiency caused by mutations in the PMM2 gene. These mutations impair the synthesis of N-linked glycans, which are critical for the normal function of proteins and lipids in the body. The resulting condition manifests with a wide range of symptoms, like ataxia (poor muscle control), developmental delays, vision loss, and multi-organ involvement. Current management is limited to supportive care such as physiotherapy, assistive equipment, and symptom-targeting interventions.
The latest investment builds on the company’s previous $35m raise in June 2021, which supported its Phase I programme for GLM101.
In the 16 April announcement, Glycomine’s CEO Steve Axon said: “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.”
CTI Life Sciences Fund and Advent Life Sciences co-led the funding round, with participation from Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.
In parallel to Glycomine’s programme, a separate effort is underway to assess the potential of a repurposed therapy for PMM2-CDG. Maggie’s Pearl – a joint venture between the rare disease biotech Perlara, the non-profit organisation Maggie’s Cure, and Mayo Clinic – is sponsoring a Phase III trial of oral epalrestat (NCT04925960) in 40 paediatric patients. The US Food and Drug Administration (FDA) cleared the Phase III study in December 2021, and the study is now fully enrolled and expected to complete by YE. Epalrestat is an aldose reductase inhibitor approved in Japan for the treatment of diabetic neuropathy.
This recent update comes amid growing uncertainty around the future of incentives for rare disease drug development in the US. In particular, the potential discontinuation of the FDA’s paediatric priority review voucher (PRV) programme is raising concerns across the sector.
