Glycomine Completes Enrollment in Phase 2b POLAR Study of GLM101 for Rare Genetic Disorder PMM2-CDG
核心洞察
Glycomine (搜索) has completed enrollment of 43 patients in its Phase 2b POLAR study evaluating GLM101 for PMM2-CDG (搜索), a rare genetic disorder affecting approximately 50,000 patients worldwide.
The randomized, double-blind, placebo-controlled trial spans 15 sites globally and will assess improvement in ataxia (搜索) using the International Cooperative Ataxia Rating Scale over 24 weeks.
PMM2-CDG (搜索) causes serious neurological impairments with ataxia (搜索) affecting more than 90% of patients, and currently has no approved treatments available.
Glycomine (搜索), Inc. announced the completion of enrollment in its Phase 2b POLAR study, a pivotal clinical trial evaluating GLM101 for the treatment of phosphomannomutase 2 (搜索) congenital disorder of glycosylation (搜索) (PMM2-CDG (搜索)). The randomized, double-blind, placebo-controlled study enrolled 43 patients across 15 sites globally, with topline data expected in the fourth quarter of 2026.
PMM2-CDG (搜索) is a rare genetic disorder that causes serious neurological and multisystem impairments, affecting approximately 14,000 people in the United States and Europe and as many as 50,000 patients worldwide. The disorder has an incidence of 1 in 30,000 to 40,000 and is caused by reduced activity of the phosphomannomutase 2 (搜索) enzyme, resulting in a deficiency of mannose-1-phosphate and disruption of N-glycosylation.
Study Design and Endpoints
The POLAR study dosed pediatric and adult patients with PMM2-CDG (搜索) ranging in age from 4 to 47 years across trial sites in the United States, United Kingdom, and Europe. The primary objective is to assess improvement in ataxia (搜索) after 24 weeks of treatment, as measured by the International Cooperative Ataxia Rating Scale (ICARS), a clinical scale used to assess the severity and functional disability resulting from ataxia.
Secondary efficacy endpoints include the Neuromuscular Gross Motor Outcome (GRO), Scale for the Assessment and Rating of Ataxia (搜索) (SARA), and clinician and patient global impressions of change. Ataxia is a core clinical feature of PMM2-CDG (搜索), affecting more than 90% of patients and serving as a key driver of disease burden.
Building on Previous Results
"The Phase 2b POLAR study will build on the encouraging results observed in our open-label Phase 2a study of GLM101, which demonstrated meaningful improvements in ataxia (搜索) and other clinical endpoints with a favorable safety profile," said Rose Marino, M.D., Chief Medical Officer of Glycomine (搜索).
The Phase 2a study was completed in 2025, and data from this earlier trial will be presented at multiple scientific conferences in 2026. Currently, there are no approved treatments for PMM2-CDG (搜索), highlighting the significant unmet medical need in this patient population.
About GLM101
GLM101 is Glycomine (搜索)'s lead investigational drug candidate, designed as a liposomal mannose-1-phosphate substrate replacement therapy. The treatment is administered via weekly IV infusion to address deficient glycosylation in both the central nervous system and periphery. GLM101 has received Orphan Drug Designation in the U.S. and E.U., as well as Rare Pediatric Disease Designation and Fast Track Designation in the U.S.
PMM2-CDG (搜索), previously known as CDG-1a, is the most common congenital disorder of glycosylation (搜索) and results in a wide array of neurological and peripheral clinical symptoms that can be severe and life-threatening. The completion of enrollment in the POLAR study represents a significant milestone for Glycomine (搜索) as it advances toward providing a potential treatment option for this underserved patient population.