Glycomine has concluded subject enrolment in the Phase IIb POLAR study of GLM101 to treat phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
PMM2-CDG is a rare genetic condition resulting in neurological and multisystem impairments, with more than 90% of patients experiencing ataxia. There are currently no approved treatments for PMM2-CDG.
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The placebo-controlled, double-blind, global randomised, 24-week trial has dosed 43 children and adults, aged four to 47 years, with PMM2-CDG. These participants have been recruited throughout 15 sites in Europe, the UK, and the US.
Its primary aim is to evaluate improvement in ataxia using the International Cooperative Ataxia Rating Scale (ICARS) after 24 weeks.
Secondary measures include the Neuromuscular Gross Motor Outcome (GRO), Scale for the Assessment and Rating of Ataxia (SARA), as well as clinician and patient global impressions of change.
Topline results from the POLAR study are anticipated in the fourth quarter of 2026.
Glycomine chief medical officer Rose Marino said: “The Phase IIb POLAR study will build on the encouraging results observed in our open-label Phase IIa study of GLM101, which demonstrated meaningful improvements in ataxia and other clinical endpoints with a favourable safety profile. We are grateful to the patients, families, caregivers and investigators who have made these trials possible.”
GLM101 holds orphan drug designation in both the US and European Union, as well as rare paediatric disease and fast track designations in the US. Previous evaluations include a Phase I study in healthy volunteers and an open-label Phase IIa trial in PMM2-CDG.
In April 2025, Glycomine raised $115m in Series C financing to support the progression of its lead investigational therapy, GLM101, into a Phase IIb clinical trial for an ultra-rare disorder.
