Glycomine Announces First Pediatric Patient Dosed in Phase 2 Clinical Study of GLM101, a Potential Treatment for PMM2-CDG
11 Dec 2023
Phase 2Orphan Drug
Promising data in adult patients supports advancement into pediatric patients
Data from this portion of the clinical program will inform pivotal study
SAN CARLOS, Calif.--(BUSINESS WIRE)-- Glycomine Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).
“The initiation of dosing in pediatrics is an important milestone for the company,” said Steven Axon, CEO of Glycomine. “We are encouraged by the biomarker and clinical improvements we have observed in the dose-range finding portion of the trial in adult patients, and we are excited to move forward into pediatrics with GLM101, the first potential disease-modifying therapy to be evaluated in PMM2-CDG.”
“We are delighted to continue our collaboration with Glycomine and move forward with GLM101 to initiate dosing in pediatrics,” added Mercedes Serrano, M.D., Ph.D., Sant Joan de Déu Hospital in Barcelona. “We are happy to be a part of an effort that has the potential to make a positive impact on PMM2-CDG patients and the community.”
Following a dose range finding study that enrolled 10 adult PMM2-CDG patients, this Phase 2 study in pediatric patients with a confirmed diagnosis of PMM2-CDG will be an open-label, 24-week treatment with GLM101 (ClinicalTrials.gov Identifier: NCT05549219). The purpose of the study is to evaluate potential pharmacodynamic activity and clinical efficacy, as well as safety, tolerability, and pharmacokinetics of GLM101.
About GLM101
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 .
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