Glycomine’s GLM101 Receives FDA Breakthrough Therapy Designation

Glycomine has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for GLM101, an investigational treatment for phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), a rare multisystem disorder with no approved therapies.

GLM101 is a liposomal mannose-1-phosphate substrate replacement therapy designed to address the underlying deficiency associated with PMM2-CDG and restore disrupted glycosylation processes. 

The designation is supported by clinical findings from an open-label Phase 2a study, which reported improvements in ataxia and other clinical measures following 24 weeks of treatment.

The therapy is currently being evaluated in POLAR, a global, randomised, double-blind, placebo-controlled Phase 2b study involving 43 paediatric and adult patients across 15 sites in the US, UK and Europe. 

Topline data from the randomised portion of the study are expected in the fourth quarter of 2026.

Following the randomised phase, patients may continue receiving GLM101 through Week 48 to assess longer-term safety, durability of response and outcomes following crossover from placebo.

PMM2-CDG is caused by mutations in the PMM2 gene, which reduce enzyme activity and disrupt N-glycosylation, leading to a broad range of clinical manifestations that can be life-threatening.

Source: www.glycomine.com