POLAR is a randomized, double-blind, placebo-controlled study that enrolled 43 patients with PMM2-CDG across 15 sites globally with topline data expected in the fourth quarter of 2026
Primary endpoint evaluates change in ataxia at 24 weeks, as measured by the International Cooperative Ataxia Rating Scale (ICARS)
SAN CARLOS, Calif.--(BUSINESS WIRE)--#CDG--Glycomine, Inc. announced today that enrollment has been completed in the Phase 2b POLAR study, a global, randomized, double-blind, placebo-controlled clinical trial evaluating GLM101 for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG). PMM2-CDG is a rare genetic disorder that causes serious neurological and multisystem impairments. More than 90% of patients with PMM2-CDG have ataxia, a key neurological manifestation and driver of disease burden. There are currently no approved treatments for PMM2-CDG.
“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 and other clinical endpoints with a favorable safety profile,” said Rose Marino, M.D., Chief Medical Officer of Glycomine. “We are grateful to the patients, families, caregivers and investigators who have made these trials possible.”
The POLAR study has dosed 43 pediatric and adult patients with PMM2-CDG, ranging in age from 4 to 47 years across 15 trial sites in the United States, United Kingdom, and Europe (ClinicalTrials.gov Identifier: NCT06892288). The primary objective of the study is to assess improvement in ataxia, 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 (SARA), and clinician & patient global impressions of change.
Topline data from the POLAR study is expected in the fourth quarter of 2026. Data from the Phase 2a study that was completed in 2025 will be presented at multiple scientific conferences in 2026.
About PMM2-CDG
Phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG-1a, is the most common congenital disorder of glycosylation. PMM2-CDG has an incidence of 1 in 30,000 to 40,000, affecting approximately 14,000 people in the United States and Europe and as many as 50,000 patients world-wide. PMM2-CDG is caused by reduced activity of the phosphomannomutase 2 enzyme, resulting in a deficiency of mannose-1-phosphate and disruption of N-glycosylation. PMM2-CDG results in a wide array of neurological and peripheral clinical symptoms, which can be severe and life-threatening. Ataxia is a core clinical feature of the disorder, affecting more than 90% of patients and is a key driver of disease burden.
About GLM101
Glycomine’s lead investigational drug candidate GLM101 is a liposomal mannose-1-phosphate substrate replacement therapy in development to treat PMM2-CDG. GLM101 is administered via a 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. and Rare Pediatric Disease Designation and Fast Track Designation in the U.S. GLM101 has previously been tested in a Phase 1 healthy volunteers study and a Phase 2a open-label study in PMM2-CDG (ClinicalTrials.gov Identifier: NCT05549219).
About Glycomine, Inc.
Glycomine is a clinical-stage biotechnology company that is advancing treatments for serious rare diseases with no other therapeutic options. The company is based in San Carlos, California, and supported by leading international life sciences investors. For more information, visit www.glycomine.com.
Contacts
Corporate Contact: Peter McWilliams, Ph.D., info@glycomine.com
Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com
