Data from Glycomine's Ongoing Natural History Study Provides Insight into Clinical Measures and Biomarkers for PMM2-CDG
August 30, 2023
- This study in 139 patients with PMM2-CDG represents the largest prospective natural history dataset with more than 300 patient-years of data collected to date
- Data from this study provide key insights into future clinical trial design
- The data presented at SSIEM 2023 highlights the important role played by coagulation, as well as liver, metabolic and endocrine systems in PMM2-CDG
SAN CARLOS, Calif., August 30, 2023 – Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium taking place August 29 to September 1 in Jerusalem. The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.
“We are grateful for the time and commitment contributed by the patients and their caregivers and appreciative of the investigators and clinical sites conducting the study,” said Steven Axon, CEO of Glycomine. “The data from this study provide key insights to understand our patients and how clinical measures and biomarkers evolve over time and thereby inform the design of our future interventional studies with our lead clinical candidate, GLM101, currently in Phase 2 clinical studies.”
“New treatments for PMM2-CDG represent a critical unmet need and natural history studies can add significantly to our understanding of this disease,” added Eva Morava, M.D., Ph.D., Mayo Clinic, who presented the findings and is the Principal Investigator of the study. “I look forward to continuing to partner with Glycomine and the community to complete this important undertaking.”
The natural history study has enrolled 139 PMM2-CDG patients at 11 sites around the world, with three sites in the US and eight in Europe (ClinicalTrials.gov Identifier: NCT03173300). Every patient currently enrolled has completed more than two years in the study. The effort has resulted in the largest single dataset in patients with PMM2-CDG from a prospective natural history study to date.
GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S. PMM2-CDG is caused by genetic mutations that lead to a deficiency of the enzyme phosphomannomutase 2 (encoded by the PMM2 gene). GLM101 is designed to deliver mannose-1-phosphate directly into cells and thereby bypass the PMM2 enzyme deficiency and address disease-causing PMM2 mutations to restore pathway function.
About Glycomine, Inc.
Glycomine is a clinical-stage biotechnology company developing novel drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. The company is based in San Carlos, California and supported by leading international life sciences investors.
Corporate Contact: Peter McWilliams, Ph.D., firstname.lastname@example.org