SAN CARLOS, Calif.--(BUSINESS WIRE)--Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, today announced that the first patient has been dosed in a multi-center open-label Phase 2 study (ClinicalTrials.gov Identifier: NCT05549219) of GLM101, a targeted mannose-1-phosphate replacement therapy, in adult patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
“The initiation of this Phase 2 study for our lead clinical program is an important milestone for the company. GLM101 is the first potential disease-modifying therapy to be evaluated in PMM2-CDG,” said Steven Axon, CEO of Glycomine. “With GLM101, we aim to restore glycosylation in patients, and correct the underlying cause of the disease.”
“We are delighted to be the first site to dose a PMM2-CDG patient in Glycomine’s GLM101 Phase 2 trial,” added Mercedes Serrano, M.D., Ph.D., Sant Joan de Déu Hospital in Barcelona. “This is an exciting time for the PMM2-CDG community, as GLM101 has the potential to make a significant impact on the lives of individuals and families impacted by this disorder.”
The Phase 2 trial is a randomized, open-label, 12-week treatment study to evaluate the potential pharmacodynamic activity, safety, tolerability, and pharmacokinetics of GLM101 in adult patients with a confirmed diagnosis of PMM2-CDG. Eva Morava-Kozicz, M.D., Ph.D., Mayo Clinic, is the Principal Investigator of the Phase 2 study.
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. For more info, visit www.glycomine.com.